Short Takes and Updates -- September 5, 2025

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FDA Matters doesn't cover the news....we provide analysis of what's behind the news

This week:

• My Comment on Secretary Kennedy’s September 4 Appearance Before the Senate Finance Committee

• MAHA Strategy Plan Likely to Be Released Week of September 8.

• MAHA vs. MAGA: Much Ado About the Usual Interest Group Politics?

• HIMS vs. Novo Nordisk Advertising: What I Saw While Watching College Football

• What I Am Listening To: FDA Watch Podcast with Wayne Pines and Paul Kim

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My Comment on Secretary Kennedy’s September 4 Appearance Before the Senate Finance Committee. A reporter asked me. Here is what I told him:

 “As political theatre goes, it was an excellent hearing. It turned heavily on Kennedy’s stubborn self-assurance and unwillingness to depart even an iota from his well-rehearsed script. As a rule, actual shouting matches are rare, but there were several.

As to the consequences of the hearing...you can’t judge the room by what was being said. What matters is what Senators say to each other about Secretary Kennedy when the cameras aren’t rolling and no one else can hear.”

MAHA Strategy Plan Likely to Be Released Week of September 8. According to Helena Bottemiller's Food Fix (subscribe at www.foodfix.co), the MAHA strategy plan is likely to be released next week. I wrote about this topic twice when I thought the launch would be in August:

•  “A Message to MAHA: Every American Should Have Access to a Healthy and Affordable Diet" (https://bit.ly/4mxWUS8), and

• “My specific message to media & advocates” (first question at https://bit.ly/4gdyaMA)

To sum up my thoughts: MAHA is a slowly unfolding train wreck, which is a shame because the MAHA movement raises questions about the American food supply and healthy diets that are worth the time and trouble to resolve.

I have urged the media and lead advocacy organizations to focus on the primary goal: providing better dietary options that Americans can afford, access, and integrate into their lives. The safety of food additives is a real issue, but it should be secondary.

MAHA vs. MAGA: Much Ado About Politics as Usual? Over the last week, several press accounts have played up the differences between traditional MAGA (Make America Great Again) constituencies and the comparatively new MAHA (Make America Healthy Again) constituencies.

Oversimplifying it a bit, the first category includes agricultural interests, particularly farm communities that voted decisively for President Trump and are mostly okay with the status quo. The second category is mainly comprised of conservative moms who support HHS Secretary Kennedy’s view that Americans, particularly children, are being poisoned by our food supply and that an increase in childhood chronic diseases is the result.

The forthcoming battle (and there will be a battle) is going to be as DC-traditional as they come. Elected officials--who want the love--and votes--of everyone will try to placate both interest groups and will likely find that’s “mission impossible.”

There are only two likely outcomes: compromises are made and something happens. Or after months of shouting, the process breaks down completely. Nobody is going to lose their job over the melee.

To be specific, I don’t know who the HHS Secretary will be 24 months from now, but if Kennedy is gone, I don’t think it will be because MAGA and MAHA clashed.

HIMS vs. Novo Nordisk Advertising: What I Learned While Watching College Football.

I thought I was taking a break when I joined a friend to watch Notre Dame vs. Miami last Sunday night. Mostly it worked that way....except there was a HIMS commercial promoting its offering of GLP-1 drugs. About 10 minutes later, there was a Novo Nordisk ad for Ozempic.

The ads provided an instructive comparison. The HIMS product is a compounded, unregulated product. Despite the absence of a market shortage that would permit mass compounding, it continues to be sold. It is neither an originator nor a generic drug. It is not manufactured in a facility that is subject to FDA inspection.

As an unregulated compounded product, HIMS is not required to meet any of the “fair balance” and “disclosure” requirements for drug advertising. The HIMS ad could have been an ad for any consumer product.

In contrast, the Novo Nordisk ad contained disclosures of side effects and presented a fair balance between risks and benefits. There was plenty of information that made clear that it was not just another consumer product.

It doesn’t seem fair that an unapproved product claiming to be “just the same” can advertise without restrictions, while the FDA-approved product has significant limitations on what can be said.

What I Am Listening To: FDA Watch Podcast with Wayne Pines and Paul Kim. I enjoyed listening--and learned a lot--from “Dissecting the Direction of CDER and CBER with host Wayne Pines and his guest, Paul Kim. Well worth your time to listen. here

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Behold: The Incredible Breadth, Depth, and Complexity of FDA’s Responsibilities

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For much of the past 20 years, my primary professional responsibility has been to impress upon lawmakers, the media, and stakeholders that FDA’s value far exceeds its budget.

Sometimes, I would emphasize the economics — FDA oversees goods and services that total nearly $4 trillion per year globally. The US component amounts to about 20% of all consumer spending.

At other times, I would emphasize the history — the FDA was created in response to the dramatic failure of caveat emptor, which resulted in unsafe foods and medicines being a common and constant risk.

Sometimes, I would emphasize the complexity of the FDA’s mission. The Social Security Administration manages trillions of dollars but has only six or seven program activities that are repeated millions of times. In contrast, the FDA has hundreds, if not thousands, of program activities that require separate attention because they do not duplicate each other.

At other times, I would emphasize the global reach and responsibilities of the agency. FDA-regulated products are manufactured or handled at nearly 300,000 registered facilities. More than half of those facilities are located outside the U.S.

Regardless of my approach, I would always emphasize that FDA’s dedicated and diligent staff are the key to the agency’s success in managing this incredible breadth, depth, and complexity of responsibilities. It is crucial to recognize that FDA’s responsibilities are core functions of government. Accordingly, consumers are at risk whenever FDA lacks the necessary funding, workforce, and leadership.

With that perspective, I appreciate Dr. Makary’s efforts to build up staff and find more efficient ways to fulfill the agency’s responsibilities. Even still, I am troubled by how many FDA initiatives start with labor-saving conclusions rather than labor-expanding questions. In the past, FDA has always seen the wisdom of public input, especially consultation with outside experts through advisory committees chosen for balance and breadth of view. We need that back ASAP.

My most widely reprinted column, “ All of Us Need to be Rooting for FDA,” expresses a sentiment that not everyone shares. However, it may read differently to many in light of this week’s multiple resignations among CDC’s leadership (covered by MedPage Today here).  Regardless of any disagreements we may have with current FDA policies and leadership, we must prioritize stability and continuity for the FDA to continue serving the American people effectively.

My role, through FDA Matters, is an extension of what I have done for several decades: perform policy analyses of issues facing FDA and advocate for positions that help the agency fulfill its responsibilities.

I hold the current administration to the same standards that I have applied to its predecessors in the past. That will never change because FDA’s incredible breadth, depth, and complexity require constant attention.

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The goal of FDA Matters is to analyze FDA policy and regulatory issues and advocate for a more effective, efficient, and fair FDA.

FDA Matters doesn't cover the news....we provide analysis of what's behind the news.

If you would like to subscribe (for free), you can do so on our website (www.fdamatters.com) or by sending a note to sgrossman@fdamatters.com. We recognize that you are busy, so we limit our content to 1-2 columns per week and ensure it is never more than a 3 to 5-minute read.

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A Message to MAHA: Every American Should Have Access to a Healthy and Affordable Diet

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(An earlier version of this column appeared in Food Safety News at https://bit.ly/417cLOJ)  

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Preamble: The Make America Healthy Again (MAHA) movement has sparked an overdue examination of how to improve the health of Americans, particularly in terms of safe food and healthy diets. Unfortunately, this effort has devolved into an acrimonious debate among competing interests and ideologies. The rancor is likely to escalate further once the MAHA Commission Action Plan is released by the Administration sometime later this month or in September.

Before the heat builds further, FDA Matters would like to remind all parties of the primary goal and most urgent need: a plan for all Americans to have access to a healthy and affordable diet.

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We have entered into a dynamic and growing dialogue about food in America — what we should be eating, how we should prepare it, what constitutes a nutritious meal, the need for additives in food, and the role of government in regulating the food industry and defining healthy diets.[1]

Stimulating the immediate dialogue are the Make America Healthy Again (MAHA) Action Plan, originally due August 12 but now delayed; the revised dietary guidelines, scheduled for release in September; and the HHS/USDA Request for Information on defining ultra-processed food (here), which will be open for comments through September 23.

Hopefully, the result will be a more cohesive path forward for improving the food Americans eat and creating a consensus around the specifics of a healthier diet. We need government food policies that are sensible, feasible, flexible, and beneficial.

Given that food sustains us and is deeply ingrained in our lives and culture, we must proceed with caution. There is an unlimited potential for downstream consequences that we do not intend and cannot necessarily foresee.

Broad Concepts to Guide the Discussion

1.     Every American should have the ability to afford a healthy diet.

Millions of Americans are food insecure. It is especially rampant among children, affecting approximately one in five American kids.

The number of food-insecure Americans is expected to increase over the next few years due to ongoing and proposed cuts to SNAP, school lunch programs, and support for food banks and other feeding programs.

Hungry people buy whatever sustenance they can afford from whoever can supply it. Quality, safety, and nutritional value become, at best, secondary considerations.

A broad reconsideration of our national food and dietary policies and guidelines is valuable. Still, it should be tempered by an awareness that increasing the cost of food will lead to millions more Americans becoming food insecure.

2.     Every American should have access to a healthy diet.

Affordability is also an access issue. Beyond that, there are food deserts that leave millions of Americans isolated from a range of healthy foods. There is a risk these will expand further because many smaller and rural grocery stores are heavily dependent on serving families with SNAP benefits.

I know that many states and localities are working on encouraging supermarkets to expand into these underserved areas. That’s ideal, although it is uncertain whether this approach will be successful. Another approach is to identify and promote healthy diets that can be accessed through corner markets, bodegas, and other small groceries that are more widely dispersed but typically have a more limited range of foods.

Access is also becoming more difficult due to cuts to the Local Food Purchase Assistance Cooperative Agreement and Local Food for Schools federal programs. Those cuts saved the government about $1 billion but came at the expense of providing schools, child care centers, and food banks with fresh food from local farmers (here).

3.     Every American should have access to foods that are made from safe ingredients.

Healthier diets should reduce the incidence and impact of diseases, including both acute and chronic conditions. However, there is a paucity of good research, especially on causation. A healthy diet should be built around some “better to be safe” changes but also allow for innovation and information derived from new and ongoing research.

 4.     Every American should have access to safe foods with minimal risk of intentional and unintentional adulteration.

A healthy diet should be compatible with maintaining food safety. Preservatives, in particular, are essential to many products and may need to remain even if they have names that people cannot pronounce. Intentional and negligent adulteration is a serious issue (my article on this topic is available here), and reducing the frequency of such incidents should also be a consideration when recommending healthy dietary choices.

5.     Every American should have access to healthy and convenient foods tailored to their life stage, offering a range of choices.

There cannot be one healthy diet to the exclusion of others. Providing multiple ways to eat healthier is beneficial in itself, as it respects the fact that there are lifestyle, cultural, gender, and age differences, in addition to individual taste preferences.

A healthy diet must also allow for multiple convenient options. It is fine that some people want to cook meals made only from single ingredients they have grown or purchased themselves. However, a significant portion of the American population lacks the time, space, and/or necessary equipment to cook at home, especially given their family and work obligations and living conditions.

One size does not fit all. Recommendations for a healthy diet must provide good choices for a wide variety of situations and circumstances.

AND MOST OF ALL:

Every American — rich and poor — should be able to afford and access a healthy diet. That’s a simple standard that will be incredibly hard to achieve. Nonetheless, policymakers and stakeholders must fully embrace this challenge.

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The goal of FDA Matters is to analyze FDA policy and regulatory issues and advocate for a more effective, efficient, and fair FDA. FDA Matters doesn't cover the news....we provide analysis of what's behind the news. scribe on the website (www.fdamatters.com) or send me a note at sgrossman@fdamatters.com.

[1] This ignores (at our nation's peril) the ever-present food safety risks; we should add this to the conversation without waiting for a crisis-inducing incident (here).

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Six Causes Championed by FDA Matters

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FDA Matters doesn't cover the news....we provide analysis of what's behind the news

The goal of FDA Matters is to analyze FDA policy and regulatory issues and advocate for a more effective, more efficient, and fairer FDA. In support of the agency, I have spent years as an analyst and advocate. I have no intention of stopping now.

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 When I launched FDA Matters on January 10th of this year, I started with some topics to analyze, but no sense of which subjects represented my advocacy priorities. That turned out to be a good thing--since the new Administration brought so many new issues to the fore, ones that would not have been on my list at the start of 2025.

Nearly fifty columns later, I see that patterns have emerged--certain topics on which I have dug deeper and spoken more forcefully. While there is room for additions to the list, here are my top six priorities at this time:

1.     FDA Is Under Multiple Challenges to Its Authority and Must Act to Preserve Its Ability to Protect Consumers. Mass compounding of drug products threatens the public by usurping the FDA’s responsibilities for manufacturing, distribution, and marketing (A Dangerous Idea: Two Manufacturing Pathways, One Regulated, One Not; A Dangerous Idea Revisited).

On two other fronts: a/ Montana is trying to create a haven for doctors to treat patients with unapproved and experimental drugs (here; Short Takes and Updates August 8 2025 ); and b/ the lack of strong central policy direction at the federal level is opening the door for 50 states to create 50 different food regulatory systems. There are more challenges coming soon and I will write about those in future columns.

2.     FDA’s Inspections and Enforcement Responsibilities are Going to Grow Massively in the Near Future and These Programs Need Far More Policy, Programming, and Budgetary Attention Than They Have Been Getting. Among other things, massive budget cuts in Medicaid, SNAP, and other feeding programs will result in more adulterated and counterfeit food and medical products (FDA in a Post-Reconciliation World and here).

This is one of many good reasons that MAHA’s focus on ingredients should not come at the expense of urgent support for food safety inspections and enforcement (see my most recent column on this topic here).

3.     Enhancing Public Trust in FDA’s Integrity. The agency has two primary strengths--its expertise and its credibility. Those qualities are being undermined. When FDA is not transparent; doesn’t welcome public input; and advances positions that are off-point or wrong, credibility is lost and will be difficult to reclaim.

With the upcoming user fee cycle, there will be voices opposing the program because they allege it gives FDA too much control over agency decisions. That isn’t true, but it is just as dangerous if policymakers and the public believe it is true. Various columns have covered these issues, including Alice,  Humpty-Dumpty and the New Administration, Picking Apart the Claim that Industry Controls FDA, and FDA and Politics: An Unhealthy Combination.

4.     FDA Must Support Innovation as Part of Our National Investment in Life Sciences. We  Abandon This At Our Peril. Other nations will jump in to fill the void. FDA Matters believes we have a collective responsibility to invest in policies that will drive growth and prosperity in the United States--now and in the future. FDA needs to carry out its mission-driven and statutorily mandated responsibilities as an investment in that future (Elon Musk and the Future of Government Investment in Our Nation’s Future).

5.     AI will be Transformative for Nearly Every Part of FDA. The Agency Needs to Work Through Step-By-Step Integration Rather than All-At-Once by Fiat. The Skeptical Enthusiasts Guide to AI at FDA explores the incredible upside for the agency and urges more validation of AI systems. This will require significant human oversight at the beginning and expertise in the specific niche that is being validated.

6.     New Agenda Needed for Addressing Chronic Progressive Diseases at FDA. Every day, we are failing patients with chronic progressive neurological, neuromuscular, and genetic diseases, by not investing more in developing better tools to evaluate therapies for their condition and considering alternative pathways for availability of therapies. FDA Matters covered this in Needed: A National Agenda for Advancing Therapies for Chronic Progressive Diseases.

The new Administration has made wholesale changes in the size, philosophy, and goals of government. FDA has not been spared. Some of its former leading advocates have even gone as far as saying that the agency is doomed.

I share their frustration with--and opposition to--many of the new policies and procedures. I doubt the agency will ever be the same---it may be better, worse, or simply different in the future.

In one of my most widely reprinted columns, I laid out the case for why All of Us Need to be Rooting for FDA. It is a realistic analysis of the agency’s situation and offers reasons to be pragmatic. My impression is that a lot of readers approached the column skeptically and came away seeing the good sense in my approach.

Being realistic and pragmatic doesn’t mean giving up on your beliefs. I will continue to write about obstacles to the agency’s success....and I will not hold back on my advocacy for these six causes.

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Short Takes and Updates -- August 8, 2025

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FDA Matters doesn't cover the news....we provide analysis of what's behind the news

This week:

• August in DC—No Longer Slow

• The Difficult Path to An FDA that Is “Just Right,”  Neither Too Hard Nor Too Soft

• Former FDA Commissioner David Kessler Submits Citizen’s Petition Calling for Action on Ultra-Processed Foods (UPF)

August in DC--No Longer Slow. Years ago, when I ran the planning, evaluation, and policy shop for the Assistant Secretary for Health, I observed that we were just as busy in August--but it was because I had half the staff covering about half the workload!

No more. Even with a full staff it is hard to cover everything going on in DC in August. Here are two “hot” topics to match the summer heat:

The Difficult Path to An FDA that Is “Just Right,”  Neither Too Hard Nor Too Soft

Possibly too hard: The writings of CBER Director Dr. Vinay Prasad’s positioned him as generally uncompromising on study protocols and analysis. It did not seem like he was that open to situations where standards might need to be adapted, such as with drugs targeting very small populations. That seemed to put him at odds with Commissioner Makary’s somewhat softer stance on orphan drugs and Makary’s calls for more innovative approaches to drug development.

Dr. Prasad’s initial public comments, including his hour-long presentation at a webinar with the National Organization for Rare Disorders (here), led many stakeholders to believe that he had moved closer to the Commissioner’s views. During his three months at FDA, Dr. Prasad made several decisions that suggested otherwise.

A narrative  developed that Dr. Prasad  was hostile (or at least uncomfortable) with pushing the envelope on innovative therapies and with relying on “reasonable but not completely proven” assumptions. As a result, there is widespread belief that Dr. Prasad’s departure opens the agency to a less rigid view of orphan drugs and gene therapies.

Definitely too soft:   On August 3, a WSJ article (here) exposed Montana's bid to generate medical tourism with an "anything goes" policy toward availability of experimental drug therapies. Specifically, “licensed experimental clinics would be permitted to recommend and administer drugs that have only completed Phase I trials—the earliest stage of human testing, focused on safety rather than effectiveness.” The preceding quote and an incisive explanation of why this is bad policy and bad medicine was written by Alec Ginsberg, the Drug Store Cowboy, and can be found here.

Neither too hard nor too soft? We definitely need a better approach to the challenge of progressive diseases that limit human development (think DMD) or are fatal (think ALS), as I described at https://lnkd.in/eXfJh93j. Dr. Prasad apparently would have required pre-approval endpoints that would have been impossible to meet. On the other hand, the Montana approach is a disservice, maybe providing a glimmer of hope but not much else. It is not a good answer.

Former FDA Commissioner David Kessler Submits Citizen’s Petition Calling for Action on Ultra-Processed Foods (UPF)

Late Wednesday evening, August  6, Dr. Kessler submitted a cover letter (full text here) and a Citizen's Petition to FDA proposing a definition of ultra-processed foods (UPF) (full text here). Media coverage started with the NY Times (here).

In former Commissioner Kessler’s words: I am submitting a petition to the FDA that provides a realistic, science-based, and legally sound path for the Administration to [define ultra-processed foods]. It calls on the Agency to remove processed refined carbohydrates from the food supply on the basis that they should no longer be considered “Generally Recognized as Safe” (GRAS).”

The key questions this raises: Is the definition he proposes operational in practice? Is he correct that there is enough information to withdraw GRAS (permitted to be marketed) status? Does it matter the outcome, if his Citizen's Petition galvanizes debate and pushes forward the possibility of serious action? (CP's have a public relations value apart from the legal issues/outcome requested).

The FDA Matters reaction:   "Secretary Kennedy and Commissioner Makary understood that eliminating ultra-processed foods would require a practical, scientifically-sound definition. By homing in on the definition, Commissioner David Kessler’s Citizen’s Petition will force a serious debate and move the needle much closer to action.”  

The CP appears to be responsive to (but not a result of) the June 16. 2025 FDA Matters (www.fdamatters.com) (at: http://bit.ly/3UdUqM6). Here are a couple of points from that column:  “A key plank of the MAHA (Make America Healthy Again) initiative is to build federal and state policies that result in the elimination of ultra-processed foods (UPF) from the American diet... Is UPF defined by the number of ingredients? By the presence or absence of certain ingredients? By certain processing steps (and which ones)? What, if any, is the level of proof needed to establish a food as “good” or “bad? Something more specific and scientifically-sound is needed."

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Sign up (free) on the website (www.fdamatters.com) or send me a note at sgrossman@fdamatters.com and ask to be added to the subscriber list.

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Needed: A National Agenda for Advancing Therapies for Chronic Progressive Diseases

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Every day, we are failing patients with chronic progressive neurological, neuromuscular, and genetic diseases.

The brouhaha about Sarepta and Elevidys[1][2] is just a reminder that we are not systematically addressing the common plight of these patients: 1/ their disease is progressive and they cannot wait for safer and more effective treatments to be developed and approved, 2/ they are willing to take on a lot more “safety risk” and “efficacy uncertainty” for even the slightest chance of a benefit, and 3/ they are dealing with complex heterogenous diseases that, regardless of patient population size, will make drug discovery and development difficult.

It is past time to help these patients with something responsive to their situation. We need: 1/ a regulatory structure and 2/ scientifically and statistically-based evaluatory standards that address chronic progressive disabling and fatal diseases and

•        responds to patient needs, and

•        encourages research and innovation

While rare disease patients will benefit greatly from such an approach, it attempts to respond to a more personal need. For the parent of a child with Duchenne’s Muscular Dystrophy the most compelling challenge is that the disease is progressive, not whether there are 75 or 3,000 other children.

FDA doesn’t have to give in to chaos; it just needs to provide a more comprehensive and thoughtful framework that is “fit for purpose” and responsive to the plight of these patients.

FDA’s longstanding vehicle--treatment IND’s--is  a necessary and helpful response, but not a sufficient one.

We never seem to get to the central problem with constructing a framework for chronic progressive debilitating and fatal diseases: our lack of knowledge and paucity of tools to develop and evaluate therapies for these diseases.

FDA’s new Rare Disease Innovation Hub[3] and Office of Regulatory and Emerging Diseases[4] are addressing some of the issues, but neither has the resources nor authority to comprehensively approach the unique patient impact of chronic progressive diseases. A good approach would be for FDA or the patient community to seek the help of the Reagan-Udall Foundation for the FDA.

In sum, there is a compelling class issue (therapies for progressive debilitating and fatal diseases) that never gets addressed if we continue to insist on it being dealt with exclusively by one-off, product-by-product decisions, as we have with Sarepta.

Sarepta. With limited knowledge and tools, clinical trials for chronic progressive disease therapies are often going to produce less robust efficacy data than other products. What went wrong two weeks ago was an attempt to use unevaluated patient deaths in ongoing clinical trials of an unapproved indication (a safety issue) as an excuse to re-litigate the efficacy component of Sarepta’s approved indication. Ultimately, things worked out the way they should have: a stop-order on the clinical trials…and only a short hold for the approved indication while the company and FDA determined whether the death of a patient on the approved indication was treatment related.

In the interim, there were unattributed reports that FDA, regardless of the outcome of the safety review, was going to insist on a new study of efficacy and safety before allowing the product back on the market.[5] That was not appropriate and maybe wouldn’t have been a possibility if there were a larger framework in place. Good regulatory decisions need to start with an acknowledgment of the gaps in what is known and the lack of good measuring tools for progressive debilitating and fatal diseases….and the recognition that decisions often need to be made on less than perfect data, especially for patients whose time is running out.

 We need to build a future that advances knowledge and develops tools for progressive diseases. At least one commentator posed the Sarepta situation as a dichotomy: either the drug doesn't work, or it does work, and the company/advocates have not worked hard enough at finding the right study design.

There is a third possibility--especially relevant to progressive debilitating neurological, neuromuscular, and genetic diseases---that we don’t yet have the tools and understanding to construct study designs that accurately evaluate efficacy in some of these complex diseases, especially those with very small orphan populations (such as DMD) and/or heterogenous progression (such as ALS).  

A couple of examples from the recent past illustrate the impact of new knowledge and tools. Better and less controversial regulatory decisions are made today because 1/ someone decided 20 years ago to examine whether Bayesian statistics[6] might contribute fresh insights to clinical trial analysis and 2/ someone decided 10 years ago to examine whether umbrella and basket trials[7] could produce more insights with a smaller resource investment.

Drugs for progressive debilitating and fatal diseases will always involve tough decisions because “delaying for more certainty” has such a cruel impact.

But if we see the problem as one of inadequate knowledge and too few tools, maybe we can build a framework and standards that will enable a future where we 1/ treat these patients more compassionately and 2/ make drug development in this area more productive and less controversial.

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[1] Elevidys is Sarepta’s gene therapy for Duchenne’s Muscular Dystrophy.

[2] I have been in and out of this issue over the years. Sometimes, I was helping clients with orphan products targeted at progressive diseases; other times, I have been working on potential policy solutions. I have no current or recent clients interested in this topic and I have not, to my recollection, ever done work for Sarepta.

[3] https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/fda-rare-disease-innovation-hub 

[4] https://www.fda.gov/science-research/science-and-research-special-topics/advancing-regulatory-science

[5] https://endpoints.news/sarepta-would-have-to-conduct-new-studies-to-get-back-on-market-fda-official-says/

[6] “.....with the advent of powerful computers and new algorithms like Markov chain Monte Carlo, Bayesian methods have gained increasing prominence in statistics in the 21st century.”

[7] https://pmc.ncbi.nlm.nih.gov/articles/PMC8220876/

Short Takes and Updates -- July 25, 2025

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FDA Matters doesn't cover the news....we provide analysis of what's behind the news

This week:

• Familiarity Breeds Respect

• Imminent Danger Regarding Food Priorities

• New CDER Leadership

• Re-Exploring Reconciliation Cuts’ Impact on FDA Consumer Protection Responsibilities

• About FDA Matters

There are an overwhelming number of policy and regulatory issues facing the agency and the stakeholder community this week. Here are a few that deserve consideration.

Familiarity Breeds Respect. Getting to know people is often a pathway to overcoming prejudice about them.

Prior to coming on board to lead the agency, Commissioner Makary was outspoken in his criticisms of the agency and its staff. Michael McCaughan  of Pink Sheet/Regulatory sees a different trend emerging: effusive praise for FDA staff by Dr. Makary and admiration for their ability to handle large and difficult workloads (the article is here).

Dr. Makary’s embrace of the FDA workforce gives us hope that he will be their advocate... and will more fully engage them in public meetings and other areas where staff expertise has always been an FDA hallmark.

Imminent Danger that Food Ingredients Will Upstage Food Safety. Given Secretary Kennedy’s and Commissioner Makary’s focus on chronic disease in children and their emphasis on developing healthier eating options, it is no surprise that food ingredients have received a lot of attention. We can all argue about the merits of specific recommendations, but the ongoing discussion and scrutiny is almost certainly a good thing.

That said, I would argue that the safety of our food supply is and should remain FDA’s number one food priority. More emphasis on a safe food supply doesn’t preclude a deep dive into ingredients...we can do both. If one were to go by what is being written and said--it sure feels like the importance of a safe food supply is no longer a priority. I cannot overstate how serious the consequences would be if that turned out to be true.

New CDER leadership Fits in the Mold of the Commissioner. I told the press this week that:

“What is most notable about Dr. Tidmarsh's appointment to head CDER....is how much he is like Commissioner Makary and Dr. Prasad, the new head of CBER."

That is, they are all intellectually elite, extremely articulate, deeply tied to academic medicine, and steeped in clinical trial design topics and questions of epistemology.

Additionally, they are all out-spoken, critical of the so-called scientific establishment, and relish the role of iconoclast. CDER is much larger than CBER and requires more management experience, something Dr. Tidmarsh provides as well."

The FDA announcement of Dr. Tidmarsh’s appointment is here.

Re-Exploring How Cuts to Health Insurance and Feeding Programs Will Result in Increased Consumer Fraud in Health Products and Foods. My recent article, FDA In a Post-Reconciliation World, seems to have been understood generally as an interesting and concerning consequence of reconciliation cuts.

Based on feedback I received, I realize many of my readers have no sense of how and why food and medical products are a sweet spot for consumer fraud. I have been using the example of melamine adulteration of milk products, a worldwide crisis in 2008 that caused many deaths and altogether affected about 300,000 children.[1]  The motivation was crystal clear: when tested, melamine (which is inexpensive) mimics protein whey (which is the most expensive component of milk products).

This example helped, but I am seeking more ways to illustrate the dangers from a weakened FDA role in consumer protection. It is unthinkable that we could return to the days of patent medicines and foods that are routinely adulterated. BUT that is possible...so I will be writing more on the topic and would love examples that make this risk more concrete.

A Little More About FDA Matters for those reading it for the first time:

FDA Matters doesn't cover the news....we provide analysis of what's behind the news.

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[1] https://en.wikipedia.org/wiki/2008_Chinese_milk_scandal

Food User Fees--the Good, the Bad and....the Possibilities

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A little under half of the FDA’s roughly $7 billion budget is funded by user fees. Tobacco user fees pay for the entire tobacco program, while medical product user fees pay for between 20% and 70% of most medical programs.

Food programs are, effectively, “all taxpayer funding, no user fees.”[1]

Are food user fees a desirable option?

When a complex regulatory system is under resourced—as is the case with human food safety at FDA--you get what you get: a haphazard system that is mostly good, but very vulnerable. On any given day, it works fine, but it is hard to be confident it will always get the job done.

Properly resourcing the food safety components of FDA would allow the agency to develop and maintain a system that is intentionally and consistently excellent.

Realistically, the required level of funding is not going to come from taxpayer funding, so food user fees would be an alternative funding source.

How do FDA’s industry user fee programs work?

The medical products user fee programs--which would be the model for food user fees--can be described as: industry voluntarily enters into 5-year agreements with FDA for a specific workplan (metrics of efficiency and timeliness, not metrics of approval). What makes the medical products user fees work:

• Broad industry buy-in;  and

• An equitable fee-able event that makes business sense for companies to support.

Tobacco user fees are mandatory and imposed upon industry, not a model for food user fees.[2]

Why aren’t there food user fees now?

Industry buy-in for user fees has never existed in the food domain, at least not long enough for anything to have been adopted and implemented. There are at least four barriers:

Barrier 1: Agreement on a fee-able event. With drugs and other medical products, market entry is barred by a requirement for product approval and an inspection of the facility in which the product will be made. This creates fee-able events that link with the corporate bottom-line. Although not always described in these terms, medical products user fees usually have a favorable ROI for companies.

There is no obvious fee-able event in food safety, although there is some potential in the narrower area of food additives.

Barrier 2: Different economic realities of food versus medical product industries. Comparatively speaking, medical products are a low volume, high profit margin industry and the food products are a high volume, low profit margin industry. Compared to a $30 billion food company, a $30 billion medical products company: will be far more profitable; will have a large R&D budget; and will be far more dependent on one specific product gaining market access. This results in a greater incentive for a medical products company to participate in a user fee program.

Barrier 3: Congressional gauntlet to enact a user fee program. House and Senate authorizers create FDA user fee programs, but House and Senate appropriators still need to appropriate the monies for it to be collected. If a segment of the food community were to oppose the creation of food user fees, they would only need to block action in one of four committees. That underscores the need for strong industry support if a food user fee program were to be considered.

There is precedent for the four committees not being in agreement. Around 2008, authorizers created a user fee program for medical product sponsors to bear the cost of the FDA office that reviews promotional materials and advertising. House appropriators were not enthused and would not allow funds to be collected. Eventually, the provisions were repealed, and the program never became active.

Barrier 4: Perception that a food user fee to support FDA is a tax on food. Even if we assume that all user fees are eventually paid by the end-users in the form of higher prices, this is not seen as an issue for medical products....and is a welcome feature of tobacco user fees. Food is perceived differently and opposition to food user fees would likely focus on how it is a tax on everyday consumers.

CONCLUSION: What would it take to establish a food user fee program?

The Food Safety Modernization Act, the recent FDA human food safety reorganization, and significant increases in funding have improved the FDA food safety programs over the last 20 years.

Yet, there is a near-universal feeling that we are a long way from an “intentionally and consistently excellent” FDA food program. While there are many potential paths for additional reform and improvement, none of them will work without more resources.

It is time to look at the issue of food user fees again--recognizing the barriers but also reflecting the enormous need for better funding. If some group is already exploring this, I am happy to join their efforts.

Meantime, if you are interested in being part of a new dialogue on food user fees, please contact me at sgrossman@fdamatters.com.  I am not sure where renewed interest in food user fees will take us, but I want to find out.  

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[1] The extremely modest user fees received by FDA food programs come primarily from a reinspection program and a qualified importer program.

[2] Tobacco products user fees are imposed on industry without their consent, to implement a workplan with which they disagree. Permanent in law. It is not a model for food user fees.

FDA in a Post-Reconciliation World

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“The One Big Beautiful Bill Act” (aka OBBBA) was signed into law on July 4, 2025. The immediate and long-term consequences of this bill are the most important story to write about today. Other topics can wait.

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Whether for an individual or a nation, how we spend our money is a concrete statement of our values and priorities. OBBBA reflects President Trump’s priorities. At least until January 3, 2027,[1] it also represents America’s values.

Although the impact of OBBBA on FDA is largely (if not entirely) indirect,[2] there are, nonetheless, consequences for the agency.

Medicaid, SNAP, CHIP, and Affordable Care Act Changes. The OBBBA cuts to feeding and health programs--which are spread over several years--represent a significant challenge to FDA as the primary federal agency responsible for the safety of both the food supply and medical products.

Hungry people buy whatever sustenance they can afford from whomever can supply it. Quality, safety, and nutritional value become, at best, secondary considerations.

Unscrupulous manufacturers, distributors, and marketers can be expected to expand  their efforts to fill the market demand with cheap foods of questionable quality. As an example, in 2008, there was a massive food safety scandal around Chinese manufacturers substituting melamine in milk to give the appearance of higher protein levels. More than 300,000 children were affected worldwide.[3]

The situation for medical products is similar. Alternative and questionable medical providers using gray market, counterfeit, and unapproved products can be expected to expand their efforts to take advantage of the millions who will be losing coverage. Also, mainstream medical providers and hospitals will, in many cases, face significant reductions in Medicaid revenue. They will be looking to find less expensive medical supplies, some of which may unintentionally come from questionable sources.

Squeeze on Appropriators. As I had predicted in a May 28 column,[4]  the last dozen or so House Republican hold-outs for passing OBBBA were all fiscal hawks. In order to garner their votes, they were promised OMB's full cooperation in further cuts in discretionary spending. This will play out in rescissions and through the FY 26 and FY 27 appropriations cycles. In the near-term, it will be harder for appropriators to restore funds where the President has proposed large FY 26 cuts: CDC (minus 50%), NIH (minus 40%), and FDA (minus 8 to 10%).

By the numbers, it looks like FDA is less at risk and probably will be for FY 26. Presumably, appropriators will be loath to 1/ tamper with “maintenance of effort” provisions that mandate certain appropriations levels in order to be able to collect medical products user fees, and 2/ shortchange MAHA and food safety programs given their status as Administration priorities. However, post-reconciliation and over the next few years, the House and Senate Ag/FDA appropriators may have a tight squeeze trying to pay for both agricultural programs and FDA....and there is an ever-present risk of FDA being caught up in across-the-board cuts.

Additionally, it is hard to define specifics, but I have no doubt that FDA programs and responsibilities will be affected if NIH and CDC (especially) are subjected to massive funding decreases.

Lack of Deficit Reduction. As I have written several times,[5] our nation’s debt is a serious problem. From a public health standpoint, we got the worst combination possible: human service programs paying for most of the tax cuts AND the final bill adds yet another $3 trillion (or more[6]) to the national debt.

While speculative on my part, here is a possible scenario where OBBBA’s failure to achieve deficit reduction might impact FDA:

OBBBA and the FY 26 appropriations cycle are not likely to sate demands for large cuts to non-defense programs. At the same time, interest payments on the national debt (determined by the markets, not Congress) will be growing more costly and consume a larger part of the federal budget.

As a result, the President’s FY 27 Budget Request is likely to propose even deeper cuts to domestic and human services programs. Its release in February 2026 will come in the midst of user fee negotiations.

The pressure for cutting government spending will be intense and may--potentially--push FDA to ask for substantially more user fee income, lower maintenance of effort requirements, more flexibility and control over how monies are spent, and a broader range of activities that can be paid for with user fee funds.

It may also force the agency to consider food user fees, even over possible industry objections.

Again, this is speculative on my part.

Conclusion

As a regulatory agency that oversees more than $3 trillion in goods and services and is essential to commerce, FDA has a better chance than most federal agencies to avoid steep budget-driven cuts.

However, OBBBA’s deep cuts in human service programs sets up circumstances where FDA’s consumer protection responsibilities will be more important than ever. It is hard to see how FDA can fully support that role with projected funding and staffing levels.

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[1] The date for the swearing in of Members of Congress following the November 2026 mid-term elections.

[2] Apparently, the change in the handling of multi-indication orphan drugs as part of drug price negotiations  under the Medicare Modernization Act was part of the final bill. However, that impacts CMS, not FDA.

[3] https://en.wikipedia.org/wiki/2008_Chinese_milk_scandal

[4] https://www.fdamatters.com/fdamatters/non-defense-discretionary-spending-threatened-by-budget-reconciliation-shortfalls

[5] Notably at: https://www.fdamatters.com/fdamatters/non-defense-discretionary-spending-threatened-by-budget-reconciliation-shortfalls

[6] Some estimates run as high as $6 trillion. https://www.washingtonpost.com/opinions/2025/07/03/last-gasp-conservatism-obbb-house-vote/

All of Us Need to Be Rooting for FDA

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There is very little support among stakeholders and FDA employees for the 2025 version of the FDA.[1] Some of us are discouraged. Some of us are trying hard to find the good in agency proposals and actions.

Regardless of where any of us stand, all of us need to be rooting for FDA to be successful.

Commissioner Makary is operating in a political environment that is arguably more challenging than any FDA commissioner has faced since the agency’s creation in 1906.

Some of the obstacles that Commissioner Makary faces include:

•       An Administration that prefers the “rule of men” and does not respect the “rule of law.”

•     An Administration that does not acknowledge the importance of expertise, notably but not exclusively scientific expertise.

•     An Administration that wants to thwart China’s ambition for global leadership yet is unable or unwilling to see that slashing NIH’s budget by 40%[2] and cutting upwards of 20% of FDA staff is a wholly inappropriate response to that challenge.

•      An Administration that claims it has a commitment to children’s health, food safety, and nutrition, but relies on experts handpicked for their biases and supports cuts to feeding programs and Medicaid without acknowledging any connection between those actions and their stated goals.[3]  

•      An Administration that claims to want to combat chronic disease but has been strangely silent about the most easily implemented action: further reduction in tobacco consumption.

•      An Administration that thinks FDA is a sock-puppet for industry.

Heavy, indeed, is the head that wears the FDA crown.

I appreciate the enthusiasm, intellect, and articulateness that Commissioner Makary brings to the job. I support (up to a point) his plans for shortening review times. I agree with FDA working cooperatively with patients and industry. I hope he has surrounded himself with people who will tell him “no” when the situation warrants a “no.” I really like his commitment to the adoption of AI. The rehiring’s have been a plus, as well as his sensible decision to support user fees negotiations.[4]

Of course, my “being discouraged” and “looking for the good in agency proposals and actions” reflects the many things that I would wish for him to do differently or better. For example, I urged the agency to be more incremental in its roll-out of AI.[5] I am confused by his use of “gold standard science” and “radical transparency.”[6] I am skeptical of his glossy optimism to FDA’s post-RIF capabilities. I would like him to figure out what the FDA cannot do or cannot complete in a specific timeframe…and publicly address statutory, Congressional, and stakeholder concerns.[7] I worry about the decline of outside consultation through advisory committees, and it scares me that the specter of political interference is ever-present.[8]

Why root for FDA and for Commissioner Makary to be successful?

•      Because a stable and functional FDA, regardless of its flaws, will be better for the American public and stakeholders than a chaotic and dysfunctional agency.

•      Because our nation’s public health and commerce are threatened when $3 trillion of goods and services lack meaningful oversight. None of us benefit from a return to “caveat emptor” in the food and drug marketplaces.

•      Because the CDC vaccine debacle, borne at least somewhat from a vacuum of leadership, shows how important it is to have an FDA commissioner who 1/ will defend good science and 2/ resist the triumph of random (and often untrue) anecdotes. Hopefully, Commissioner Makary will commit to defending science in the face of such threats.[9] He certainly understands the bedrock of good science: findings based on statistics, well-controlled trials, and reproducible results.

•      Because the next two to three appropriations cycles and the negotiation of the next user fee agreements will be on Commissioner Makary’s watch. An underfunded regulatory agency will say “no” far more often because it does not have the time, resources, or focus to reach a nuanced “yes.”

Nobody gets a free pass, as reading FDA Matters columns[10] will continue to demonstrate. I may be optimistically projecting more than Dr. Makary can or wants to deliver.

Nonetheless, it is a complex world where the path to a stronger and more effective FDA often contains twists and turns....and “the perfect is often the enemy of the good.”

We need continued dialogue among all stakeholders and the FDA that reflects that the betterment of FDA is a universal goal.

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[1] https://www.linkedin.com/pulse/inside-changing-fda-winners-losers-uncertainty-steve-usdin-15jbe/?trackingId=0rXpBMQ9l6kQ8R3M9Hbkkg%3D%3D

[2] https://www.fdamatters.com/fdamatters/elon-musk-and-the-future-of-government-investment-in-our-nations-future

[3] For example, in 2023, 41.5% of mothers had Medicaid at the time of birth. https://www.marchofdimes.org/peristats/data?reg=99&top=11&stop=154&slev=1&obj=18

[4] https://www.fdamatters.com/fdamatters/finally-some-encouraging-news-from-fda

[5] https://www.fdamatters.com/fdamatters/the-skeptical-enthusiasts-guide-to-ai-at-fda

[6] https://www.fdamatters.com/fdamatters/alice-humpty-dumpty-and-the-new-administration

[7] https://www.fdamatters.com/fdamatters/truth-telling-fda-user-fee-edition

[8] https://www.fdamatters.com/fdamatters/fda-and-politics-an-unhealthy-combination

[9] https://www.nytimes.com/2025/06/05/health/kennedy-stem-cells-experimental-treatments.html

[10] www.fdamatters.com. Readers can subscribe on the website or send a note to sgrossman@fdamatters.com to ask to be added to the subscription list.

“A Dangerous Idea” Revisited

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This article builds upon my May 22 column, entitled A Dangerous Idea: Two Drug Manufacturing Pathways, One Regulated, One Not.[1][2]

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Recently Novo Nordisk dropped out of its arrangement with Hims and Hers because the latter continued to sell compounded GLP-1 weight-loss products that are no longer legal (except in extremely limited situations).

The resulting commentaries and comments on Linked-In have run hot and heavy. Some of the comments are little more than flaming tirades against the pharmaceutical industry, but others reflect thoughtful concerns about access, price, cost, quality, and safety. It is easy to get lost trying to figure out who’s right and for what reasons. 

However, if you step back and look beyond just GLP-1’s, it turns out to be simple: the narrow exceptions that compounders want to expand would cross critical boundaries that FDA needs to preserve.

There is so much that feels special about GLP-1 products that we tend to forget that, from a regulatory perspective, they are just another class of regulated drug products. There cannot be one set of rules for GLP-1’s and another set of rules for other drug products. 

A short history of GLP-1 compounding

Given the anticipation for any new weight loss drug and the sterling results from the GLP-1 obesity clinical trials, it is no mystery why demand for GLP-1 drugs was so large. GLP-1 drugs were quickly the “go to” drugs for weight loss. As a result, the initial manufacturing capacity of Novo Nordisk and Lilly were insufficient, and FDA declared a shortage until the commercially available supply could catch up to demand. 

In March 2025, FDA declared the shortage ended. Compounding was to cease no later than May 22. As we now know, many compounders stopped, but many didn’t.

Those who continue to compound GLP-1’s have shifted their justification from  “shortage compounding” (no longer legal) to “special needs compounding” (the only other exception recognized by law). 

That is problematic because “special needs” is an extremely limited category that requires a specific physician to establish that a specific patient has “special needs” that cannot be met by commercially available products. Special needs compounding was never intended to be  a landing place for shortage compounders to go once a shortage ended. 

“Special needs” might come into play with a Parkinson’s patient who can’t swallow pills or a child who needs special dosing to take a medicine normally intended for adults. 

“Special needs” is not, for example, mass-compounding of GLP-1’s with vitamin B-12 added. Both products are commercially available, there is no clinical rationale, and it is not plausible that that all or most of a company’s GLP-1 patients have the exact same special need. 

Compounding lacks important consumer protections under the FDCA. 

Among other things, the Food, Drug, and Cosmetics Act (FDCA) provides for the regulation of biopharmaceutical products--approval, manufacture, distribution, and labeling. While certainly not perfect, it is a comprehensive set of rules that protect consumers from products that are unsafe or ineffective and that guide appropriate use through labeling. 

Compounding sits outside those FDCA consumer protections. FDA does not approve the compounded product, it does not review whether the compounding is done under Current Good Manufacturing Practices (cGMP), it does not inspect compounding facilities, and there is no requirement for labeling. 

A consumer has no way of knowing if the compounded product is the same as the approved one. Maybe it is just as good, but that is unproven and, in most instances, unknowable.

For that reason, compounding is allowed in only two instances--shortages and special needs--where the additional risk is justified by the circumstances. 

If GLP-1 “special needs” compounding is broadly allowed, then it is precedent for any drug that can be compounded. 

If trivial adjustments to a commercially-available product qualifies as “special needs,” then the shift from regulated manufacturers to unregulated compounders will expand to many additional drug products beyond GLP-1’s. Compounding would then be far beyond what the law was intended to permit, and far from the consumer protections we now enjoy. 

FDA urgently needs to address the permissible scope of special needs compounding or risk the creation of an alternative pharmaceutical market that operates outside the safeguards and consumer protections provided by the Food Drug and Cosmetics Act. 

Beyond consumer protection, an alternative unregulated pharmaceutical market will adversely impact drug development. 

Traditional companies will be less interested in investing in research and development of any product that could be readily compounded by others. Likewise, a generic company will have less incentive to gain FDA approval of their product if the target market is already eroded by compounding. 

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1. For a pharmacist’s perspective—along with illustrations of compounder pitches and commentary on the problems they create: From the FDA to Instagram: How Ozempic Got Hijacked by Compounders by Alec Ginsberg.

2. For an overview of the compound-related marketing practices of telehealth companies: https://sherwood.news/business/the-controversial-rise-of-franken-pills-telehealth-companies-push-to-mix/ 

Alice, Humpty Dumpty, and the New Administration

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As children, we are taught that “sticks and stones will break my bones, but words will never hurt me.” That may have helped children develop resilience against verbal bullying, but it totally discounted the power of words. As adults, we know better. Words matter! 

Notably, words have the ability to control as well as inform. 

“When I use a word,’ Humpty Dumpty said in rather a scornful tone, ‘it means just what I choose it to mean — neither more nor less.’

’The question is,’ said Alice, ‘whether you can make words mean so many different things.’

’The question is,’ said Humpty Dumpty, ‘which is to be master — that’s all.”

― Lewis Carroll, Through the Looking Glass

Each political party uses terms and slogans to advance their causes. In doing so, they often use words that imply a clearer definition and greater certainty than the situation, facts, and proof points justify. 

The new administration is fond of: radical transparency, gold standard science, and ultra-processed foods. But what is meant by these catchphrases may not always be clear.

Radical Transparency. The conclusions and the reasoning behind government decisions should be readily available and easy to understand. They should provide tangible evidence that public opinion was sought, received, and considered. 

At least four federal laws are involved: the Administrative Procedures Act,[1] the Federal Advisory Committee Act[2], the Freedom of Information Act[3], and the National Archives and Records Administration statutes[4]. In the face of these four laws, it is fair to wonder what differentiates “radical transparency” from “transparency?” 

Despite the catchy phrase, the new Administration has shown little interest in transparency as traditionally defined and manifested in existing laws. 

As Dr. Jeremy Faust has observed: visibility—widely-distributed fiat announcement of government policy—is different from transparency about and public inclusion in the government decisionmaking process.[5]

Perhaps “radical visibility” should replace “radical transparency?”

Gold standard science. In my nearly 20 years as an advocate for FDA resources, I made hundreds of references to FDA being “the global gold standard for regulation of food and medical products” because of its reliance on science-based decisionmaking. Recently, FDA Commissioner Marty Makary and CBER Director Vinay Prasad made this their center-piece, describing “the two guiding principles of our philosophy—gold-standard science and common sense.”[6]

In a recent executive order[7], gold standard science was defined, enumerating nine characteristics. 

What seems missing from the administration’s definition of “gold standard science” is an appreciation of the complexity and ambiguity of scientific results. Often, science is (or needs to be) actionable, even if it is not absolute or immutable.

There is, necessarily, a strong situational relativity when you factor in the difficulty, cost, and timeliness of achieving complete certainty. As a result, sometimes gold standard science has to be “what we knew at the time, exercising best judgment, and having surveyed all relevant evidence.”  

Over time, new data may refine or refute previously held interpretations of previous studies. Mis-directions from the past are not necessarily blameworthy. They may simply reflect how scientific knowledge evolves. 

Ultra-Processed Food. A key plank of the MAHA (Make America Healthy Again) initiative is to build federal and state policies that result in the elimination of ultra-processed foods (UPF) from the American diet. This term was already in use and gaining momentum before the new administration made it a central tenet of government food policy. 

Before UPF becomes a legal standard by which foods are judged to be good/healthy or bad/unhealthy, there needs to be some honesty about how ill-defined the term is.

Is UPF defined by the number of ingredients? By the presence or absence of certain ingredients? By certain processing steps (and which ones)? What, if any, is the level of proof needed to establish a food as “good” or “bad?” 

Something more specific and scientifically-sound is needed...if Congress is to pass a law, FDA and USDA are to promulgate regulations, or government officials are to threaten industry with withdrawal of foods, ingredients, additives, and processes in common usage today. Consumers, too, ought to have the benefit of clarity when government eliminates and/or demonizes certain foods, ingredients, or processes. 

I am not alone in such concerns. Marion Nestle, the doyenne of nutritional food policy, recently offered the following comment about proposed California legislation that would ban UPF’s from the state’s school lunch program:  “I hope it passes, not least because I can’t wait to find out how its authors think the state will go about identifying the specific foods blocked from schools.”[8]

English is enriched every day by new words and new meanings. As the exchange between Humpty Dumpty and Alice illustrates, we are often the initiators of those changes. When that occurs, we should endeavor to be crystal clear about what we mean. 

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1. APA provides for “notice and comment rulemaking,” which solicits public input prior to finalizing regulations. https://en.wikipedia.org/wiki/Administrative_Procedure_Act  

2. FACA sets standards for federal meetings, assuring that the public has advanced notice and, in many instances, opportunity for public comment. https://www.gsa.gov/policy-regulations/policy/federal-advisory-committee-management 

3. FOIA provides a process and rules for public access to government decisional documents. https://www.law.cornell.edu/wex/freedom_of_information_act_(foia) 

4. NARA is a collection of laws/rules about public records retention so that contact with public officials is tracked  https://www.archives.gov/about/laws 

5. https://insidemedicine.substack.com/p/hhs-visibility-transparency-the-legal?r=5p3cr&selection=b8e4fc9f-7268-4cab-a44c-2cf9f664b353&utm_campaign=post-share-selection&utm_medium=web&triedRedirect=true#:~:text=Let%20me%20state%20this%20clearly

6. https://jamanetwork.com/journals/jama/fullarticle/2835314 

7. https://www.whitehouse.gov/presidential-actions/2025/05/restoring-gold-standard-science/ 

8. https://www.foodpolitics.com/2025/06/california-is-considering-banning-ultra-processed-foods-from-schools-really/?lctg=20327868 

Elon Musk and Government Investment in Our Nation’s Future

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Elon Musk thought cutting government programs was about deficit reduction. When the “One Big Beautiful Bill” reconciliation legislation passed the House with a $3 trillion price tag, he realized he had been misled and reacted accordingly. His falling out with the President--charitably compared to two kindergartners squabbling; uncharitably compared to two scorpions trapped in a bottle--was a fascinating part of the last few days. And a distraction from more important issues.

It would be wonderful (although not likely) if their feud morphed into what we really need: 

A deficit-neutral reconciliation bill that does not gut human services programs or abandon government investment in scientific research and innovation. Hint: more revenue and more judiciously-chosen program cuts will be needed. This more balanced approach can (and should) be justified for what it is: an investment in our nation’s future. 

Collective Responsibility and Barriers to Investment 

FDA Matters believes we have a collective responsibility to invest in policies that will drive growth and prosperity in the United States--now and in the future. FDA needs to carry out its mission-driven and statutorily mandated responsibilities because it is just such an investment. It is inexplicable how the proposed 40% cut in NIH funding could be justified as supporting investment from either the governmental or private sectors.

Beyond that, we would point to the need for continued investment in our nation’s deteriorating infrastructure; our system of higher education and research institutions; and programs that assure that everyone has access to food, healthcare, and education, which are a prerequisite for individual success. 

There are two immediate barriers to continuing and expanding government investments in our nation’s future. First, our nation is $36 trillion in debt, and we are adding another $1.9 trillion in FY 25.[1][2] That cannot go unaddressed any longer[3], even if it takes several years to reach the goal of deficit neutrality in annual expenditures.   

Second, DOGE-driven policies and program cuts are not only destroying  government investment, but creating uncertainty for the companies, investors, institutions, and individuals who we want to encourage to invest in our nation’s future. 

A Call for Renewed Government Investment in Our Nation’s Future

For at least three generations, our country has been driven by the worst possible budget cycle. When in power, Democrats tend to expand government programs (increasing expenditures); when in power, Republicans tend to cut taxes (reducing revenue). 

Each approach has popular appeal to certain constituencies. Combined they have left our nation in a precarious position relative to government and private investment: 

• Financing our $36 trillion debt keeps interest rates higher than they otherwise would be--making investments more difficult and more expensive. Ongoing and future deficit spending will eventually reset bond prices and drive interest rates to higher levels. 

• The administration’s FY 26 proposals would shrink our national investment in science, proposing a 40% cut to NIH, a 50% cut to CDC, and a 60% cut to the National Science Foundation. 

• Major research universities are under attack on multiple fronts, including indirect costs, autonomy, and access to foreign scientists and institutions. 

• The environment for private investment has notably deteriorated.[4] There is a low degree of certainty about the rules and programs that will shape the success or failure of investments. Government agencies are no longer reliable partners. The long-standing cooperative relationship between regulated industries and FDA is under fire. The ever-changing tariff situation makes business planning difficult. 

Oscar Wilde famously defined a cynic as "a man who knows the price of everything and the value of nothing". Unless we can reinvigorate our commitment to investing in our nation and its people, cynicism will prevail, and the future is likely to be bleak. 

It doesn’t have to be that way. Choosing between deficit reduction and investment in our nation’s future is a false dichotomy. We can have both if we are prepared to make tough choices and ask everyone to pitch in. We are capable of rising to the occasion, reaching a shared goal of a prosperous future.

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1. https://budget.house.gov/press-release/congressional-budget-office-updates-baseline-deficit-totals-to-third-highest-in-american-history

2. The best  number I can glean from the charts suggests that the House-passed reconciliation bill would reduce this by about $100 to $200 billion in FY 25. Reconciliation changes would add to the deficit in every subsequent year.

3.  Apparently, Elon Musk and I share this view.

4. The House version of the reconciliation bill would restore tax incentives for research and development, business equipment and debt interest through 2029. The Senate is talking about making those permanent.  As proponents assert, these are important pro-growth provisions. However, I consider them “necessary but insufficient.” They reflect no new analysis of what is needed. Notably, they do not offset the impact of budget cuts and disruptive government policies on investment.

The Skeptical Enthusiast’s Guide to “AI at FDA”

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I welcome Commissioner Makary’s embrace of AI[1] and applaud yesterday’s announcement of the next step in agency adoption of AI technology. [2]

I wrote earlier that: “AI can help FDA make better regulatory decisions based on more refined and accurate information…. [although] there are legitimate concerns about relying on AI too quickly and without system refinement and validation.”  

As a result, I characterize myself as a “skeptical enthusiast” on the topic of AI at FDA. Here is why: 

An historical perspective: When the Clinton administration downsized the federal government in the 1990’s,[3] some of the success of that effort was attributable to embracing new technology. The Internet[4] was gaining traction and there was the opportunity to create efficiencies in government around that then-new technology.[5]  

A similar opportunity exists today for AI to play a role in making the US government more efficient. Then, as now, we need to appreciate that we are working with a technology whose capabilities, strengths and weaknesses are formative, not set. 

Fast forward to Current Headlines. HHS Secretary’s “Make America Healthy Again” (MAHA) report was released on May 22, 2025. It contained a number of citations to research that were never undertaken or never published. 

Researchers face frontier justice[6] if they make things up. There need to be consequences for relying on AI output that has not been evaluated for possible computational errors, biased sampling, and invented data, studies, and publications. 

AI is a wonderous tool now and promises to be even better in the future. However, we cannot just dismiss problems because AI is the wave of the future. 

AI 101: Artificial intelligence is the capability of computational systems to perform tasks typically associated with human intelligence, such as learning, reasoning, problem-solving, and decisionmaking.[7] Sometimes AI is faster or has greater bandwidth or possesses more patience than humans; increasingly AI  is capable of producing timely results that involve more inputs and more computations than any human mind could ever handle. 

There are three features that are leading us into broader and more sophisticated uses of AI. These are: 1/high-speed processing that enables AI to take on a broader range of more complex tasks, 2/ expansion of AI engines to include nearly everything ever written (LLM or Large Language Model), and 3/ the transition from predictive AI to generative AI. Predictive AI uses data to predict likely outcomes. Generative AI learns patterns from large datasets and uses that knowledge to generate original, unique outputs.[8] 

The Opportunity and Challenge for AI at FDA. Like most new transformative technologies: the promise of AI is running far ahead of its proven capabilities. 

Nonetheless, FDA has no choice but to move forward. 

It already receives applications for review that incorporate artificial intelligence, be it in patient recruitment, trial design, evaluation of safety and efficacy, or in generating useful post-marketing information.[9] As a result, the agency must have staff that are fully capable of understanding and evaluating what is being proposed. 

Equally interesting is the potential for FDA to incorporate AI into its own processes. FDA already invests in regulatory science,[10] seeing great potential for more efficient, as well as better-informed decisionmaking. AI will accomplish both, with more efficient decisions being very visible, while the better-informed decisionmaking being less visible but profound in its impact. 

FDA’s two greatest strengths are its expertise and the public trust in its decisionmaking. Those are particularly important given the complexity of regulating medical products and the inevitable inexactitude of scientific proof in biomedicine and medtech. 

Before AI can help FDA make better regulatory decisions more rapidly, the agency and its staff need to feel confident that they can trust the output from AI and that there are controls in place to limit misadventure. 

The MAHA report is a reminder that AI is not yet a trustworthy tool for many of its proposed uses--even seemingly simple ones. An error in a policy position paper is a small concern compared to an error in a complicated and important regulatory decision! 

How can FDA use AI effectively while respecting it’s known limitations, including citations to literature that do not exist? 

I asked for some ideas from Shannon Lantzy (shannon@shannonlantzy.com), a regulatory innovation strategist who has led major efforts to assess and improve FDA’s regulatory review performance. She replied: 

Trust in automation is not black and white; it can grow slowly with quality and accuracy checks along the way. Trust in AI needs to grow like trust in a new hire who needs to be trained, monitored, and coached with adequate feedback. 

Yes, GPT hallucinates. Reviewers can check the suggestions of AI review support, just like branch chiefs thoroughly review their brand-new hires’ work. LLMs can be prompted and tailored to reduce hallucination. Use cases can start with humans very much in the loop. It is relatively simple to build an LLM to check another LLM’s work, evaluating the validity of every citation. 

Critics of AI for regulatory work imagine the worst, but they don’t imagine what we already tolerate: drudgery work for experienced and highly skilled reviewers and little scale in review tools despite significant increases in reviewer workloads and medical product complexity. 

Conclusion: Understandably, most of the controversy about AI is focused on the quality of the outputs (useful vs. unvalidated vs. hallucinogenic). We need to move forward to address the  process itself….the difficulty of incorporating fundamental and sophisticated changes into an already complex regulatory decisionmaking system. 

Four things seem most important to me:  

• Acceptance: All parties need to accept that broad application of AI at FDA is appropriate and inevitable. They need to work together to maximize its potential and minimize input and output errors. 

• Transparency: FDA needs to articulate how it uses AI in its work now, what uses are formative, and what uses are aspirational. 

• Validation: If trust in FDA and its decisions is to be maintained, then FDA needs to show how the tools it relies upon, such as AI, have been validated. 

• Control: There needs to be ongoing means by which applications are assessed for bias and error. 

AI changes everything, but not instantly and not without humans assessing the inputs and outputs. 

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1. https://www.fdamatters.com/fdamatters/finally-some-encouraging-news-from-fda 

2. https://www.fda.gov/news-events/press-announcements/fda-launches-agency-wide-ai-tool-optimize-performance-american-people?utm_medium=email&utm_source=govdelivery 

3. https://www.fdamatters.com/fdamatters/what-is-happening-to-federal-workers-at-fda 

4. https://apnews.com/article/trump-musk-doge-clinton-reinventing-government-gore-a95795eb75cacc03734ef0065c1b0a6d.

5. Public access to the Internet and the launch of the Worldwide Web occurred in the early 1990’s. https://en.wikipedia.org/wiki/Internet 

6. Were a researcher to make up a citation or article, colleagues would likely ostracize them; all of their prior research scrutinized; their grants might be cancelled; and their academic affiliations might be suspended. 

7. Once incorporated into common experience, AI is often not perceived as AI per se. AI is already in widespread use in Google Search, Siri, Yelp recommendations, and autonomous vehicles. https://en.wikipedia.org/wiki/Artificial_intelligence

8. That is, a lot of past AI work could, in theory, have been done by a sufficiently large team with a sufficiently large amount of time and infinite patience. For example, how many times does Shakespeare use “thou” in his plays. Generative AI can create outputs that no amount of people or time could accomplish. For example, build an algorithm and demonstrate how a radiologist, in real time,  can compare one X-ray with 100,000 prior ones that are documented in the literature.

9. AI will also play a significant role in food safety regulation, but I have not yet explored the topic.

10. Regulatory Science is the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of some FDA-regulated products. https://www.fda.gov/science-research/focus-areas-regulatory-science-report/focus-areas-regulatory-science-introduction 

Non-Defense Discretionary Spending Threatened By Budget Reconciliation Shortfalls

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“While discretionary spending is not part of the reconciliation process, some policymakers may point to savings from that category as “offsets” for other policies.”[1]

FDA makes significant contributions to public health, public safety, scientific and technological innovation, US investment, and the flow of commerce—producing a positive return-on-investment that far exceeds its budget.[2] Yet, owing largely to our nation’s ongoing and precarious fiscal situation, it gets harder each year to convince the Administration and Congress to provide FDA with robust funding.  

FY 26 (which starts October 1, 2025) may be a year when Congressional appropriators have little or no leeway to “plus-up” funding for  programs and agencies whose appropriations may be targeted for significant cuts (such as FDA and NIH). 

Precarious US Fiscal Situation Worsened Over the Last Two Weeks

Republicans started the budget reconciliation process with the goal of extending tax cuts and enacting other costly Trump priorities…while incorporating enough cuts from mandatory spending so that the total package would be “deficit neutral.” 

Instead, the House bill — re-named “The One Big Beautiful Bill Act” (OBBB) — would increase the national debt by an estimated $3 to $4 trillion over 10 years. 

This has rattled the bond markets. Notably, Moody’s, one of the three major credit rating agencies, has downgraded US debt.[3]

The Squeeze on Non-Defense Discretionary (NDD) Spending

The FY 25 House Budget Resolution (with impacts through FY 34)  drives the current reconciliation process that targets mandatory spending. It also contains assumptions about the amount of discretionary spending in each of the next 10 years — in the aggregate but also by general category. 

As described by the Peter G. Peterson Foundation: 

The tables associated with the House budget resolution include an unlikely assumption that discretionary spending will be reduced by $1.8 trillion between 2025 and 2034 relative to the Congressional Budget Office’s (CBO’s) baseline — all from non-defense programs.[4]

The President’s FY 26 Discretionary Budget Request, released May 2, also targets non-defense discretionary (NDD) spending, albeit some programs will receive increases while others will face deep cuts or elimination.[5] Specifically, NDD spending is reduced by $163 billion or 22.6 percent from the FY 25 enacted level,[6] “while still providing support for our Nation’s veterans, seniors, law enforcement, and other critical priorities for the Federal Government.”

Of specific relevance to FDA: the House and Senate subcommittees handling Agriculture/FDA appropriations would, under the President’s Request, have $5 billion less (-18.3%) to spend in FY 26 compared to the FY 25 enacted levels. 

Spill-over from Reconciliation May Be the Biggest Threat to NDD

As bad as the FY 26 NDD numbers look under the House budget resolution and the FY 26 President’s Budget Request, the greatest threat to NDD funding is the likely failure of reconciliation to achieve deficit reduction. 

Conservative Republican “fiscal hawks” in the House reluctantly voted for OBBB, hoping it comes back from the Senate with deeper cuts and a much smaller increase in the national debt.[7] The Senate bill may be more to their liking (or not), but it is hard to imagine at this point that any reconciliation legislation will meet the original goal of deficit neutrality….or, at least, do so without a larger contribution from discretionary spending.

If reconciliation is not deficit neutral, House and Senate fiscal hawks will be looking for any victories that provide recompense for swallowing the bitter pill of a reconciliation that increases the national debt. They could advocate for a smaller tax cut, argue for increased revenues, or redouble their efforts to cut NDD funding. All three may be needed, but you can be sure that the last option will be on the table.

…. and the Hardest for Appropriators to Overcome. 

NDD funding was always going to be a target for the fiscal hawks. If their votes are needed to pass the final version of the OBBB and that bill increases the deficit (as seems likely), then they will have incredible leverage to force additional NDD cuts to offset the growing deficit. 

There are consequences. Senate Appropriations Chair, Senator Susan Collins, in particular, may find herself in an endgame in which the Appropriations Committees do not have enough discretionary funds for her to patch all the NDD programs she wants to protect (notably, thinking of FDA and NIH). 

Conclusion: Advocates for strong NDD funding (including FDA and NIH) have an important stake in the outcome of reconciliation. The larger the amounts that reconciliation adds to the national debt over the next ten years…the greater the risk that Congressional fiscal hawks will press for additional cuts in NDD funding that are greater than those cuts already anticipated under the House budget resolution and the President’s FY 26 request.

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1.  https://www.pgpf.org/article/the-budget-resolution-contains-optimistic-assumptions-about-discretionary-spending/ 

2.  The same can be said about the larger cluster of FDA, NIH, CDC, and USDA food safety programs.

3.  By itself, the Moody’s downgrade is more a tremor (signal), than  an earthquake. The other two agencies downgraded US debt in 2011 and 2023. Moody’s change in May 2025 is still significant because it is a reaction to growth in the deficit that would occur under OBBB. 

4.  https://www.pgpf.org/article/the-budget-resolution-contains-optimistic-assumptions-about-discretionary-spending/

5.  https://www.whitehouse.gov/wp-content/uploads/2025/05/Fiscal-Year-2026-Discretionary-Budget-Request.pdf 

6.  The budget resolution compares NDD funding cuts to the CBO baseline while the President’s budget compares NDD funding cuts to the FY 25 spending levels. I have not researched whether there is a way to make the numbers comparable.

7.  https://jeffdufour1.substack.com/p/debt-bomb. My thanks to Jeff Dufour and National Journal for making this document available.

A Dangerous Idea: Two Drug Manufacturing Pathways–One Regulated, One Not

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Redefining “Special Medical Needs Compounding” to Permit Mass Production Threatens Consumer Protections

Today, May 22, 2025, is the last day that mass compounded GLP-1 obesity medicines can be sold because FDA has declared the shortage over. Yet, some companies intend to continue selling compounded GLP-1 drugs to a large number of their customers by gratuitously adding other ingredients or making similar modest and unnecessary changes in dosing…then saying it is being compounded for the special medical needs of a specific patient. The hypocrisy is clear: a special medical need is not a mass need. 

What happens next is about consumer protections and manufacturing standards under the Food Drug and Cosmetics Act (FDCA). FDA can enforce the law or allow a race to the bottom that ultimately robs consumers of protections they need and deprives companies of the standards that assure a level playing field and quality products. 

If this were just about a class of products, I would probably not write about it in FDA Matters. However, the stakes are much, much higher than that. 

If a compounder can mass produce any drug they want by claiming special medical needs for individuals --with minimal regulatory requirements and oversight--then we have enabled an alternative pathway for the manufacturing and sale of both prescription and generic drug products. Manufacturers would be regulated. Mass production compounders would not. That doesn’t sound good for consumers or innovators…and it really isn’t[1] (see important footnote). 

The Current System Works and It Protects Consumers 

There are over 20,000 pharmaceutical drug products that are FDA-approved in the US[2]. Most pharmacies will carry several thousand of those, depending on store volume. What a pharmacy does not already have in stock, it can usually fill within 2 or 3 days from a central warehouse.

This is a system that works, fulfilling essentially all patient needs in the US through more than 60,000 pharmacies nationwide. FDA-approved drugs dispensed through pharmacies are FDA-evaluated and approved, and have uniform quality and formulation. They are standardized and manufactured by companies that follow FDA guidance on Current Good Manufacturing Procedures (cGMP) in facilities subject to FDA inspection. They are proven to be safe and effective. 

Compounding Addresses Shortages and the Special Medical Needs of a Specific Patient

By its nature, compounding by-passes the entirety of this process and the comprehensive consumer protections built into the Food Drug and Cosmetics Act (FDCA). Not only is the compounded product unregulated, but it can be given to patients without even so much as FDA-approved labeling. For good reason, compounding of commercially available drug products is only permitted under certain well-defined and limited circumstances. 

In addition to shortage situations, compounding is permitted to address the special medical needs of a specific patient. 

Such compounding is triggered by a specific doctor determining that a specific patient’s special medical needs require a compounded product, dosage, or method of delivery that will make a “significant difference” for the patient compared to an available commercial product. A wide variety of circumstances might be involved; examples might be a child who cannot tolerate a bad tasting oral medicine or a person with a swallowing disorder who needs a different dosage form than what is being marketed. 

Special Medical Needs of a Specific Patient Is Inconsistent with Mass Compounding

I am told there is no clinical evidence supporting delivery of GLP-1’s other than the current approved FDA labeling. For example, why mix vitamin B3 or B12 into the GLP-1 compounded product other than to be able to claim it is different? Or why allow oral, sublingual, or patch methods of delivery--except on an individual basis--since no such product has yet been considered or approved by FDA? If and when FDA approves any of those routes of administrations, there will be a commercial-available product that should preclude mass compounding (while leaving the special medical needs exemption in place for legitimate individual patients). 

There may be some limited cases where modifying GLP-1’s makes sense, but only if it truly addresses a specific patient’s special medical needs and not as a stratagem to support mass production. 

You don’t hear much about this type of compounding because it is limited to a specific patient’s special medical needs relative to commercially-available product. Pertinently, special medical needs compounded products cannot be produced beforehand or in quantity--the exemption is justified by its application to an individual patient’s needs[3].  

However, you will start hearing a lot about this type of compounding if FDA allows “special medical needs” and “significant difference” and “mass compounding” to be defined by compounders. Then the race will be on to find other lucrative drugs that can be retrofitted into the GLP-1 compounding model[4][5].,

Conclusions 

It would seem self-evident that an exemption permitting compounding for the special medical needs of an individual patient is incompatible with mass compounding. Special medical needs compounding was never intended to be a place for shortage-driven mass compounding to move after the shortage has concluded. 

It is like trying to pull an elephant through a mousehole. If you make the hole big enough to accommodate the elephant, it is no longer a mousehole. 

FDA urgently needs to address the permissible scope of special needs compounding or risk the creation of an alternative pharmaceutical market that operates outside the safeguards and consumer protections provided by the Food Drug and Cosmetics Act. 

In addition to the risk of FDA losing its ability to protect consumers by setting and enforcing manufacturing standards for drug products, there is a significant market competition risk. Paraphrasing Gresham’s Law that bad money always drives out good money[6]: 

If there are two forms of pharmaceutical products in circulation, both of which are accepted by regulatory authorities as validly marketed, then the more valuable product (the one that is FDA-approved) will gradually disappear from the market.

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[1] Note: there are two very important issues not addressed here: whether the compounded products work as well and are as safe as the manufactured ones, and ownership of the intellectual property. 

[2] FDA Facts at a Glance. November 2020. https://www.fda.gov/media/143704/download

[3] The risk-benefit of going outside the proven and approved labeling is acceptable for a single person with genuine special medical needs that cannot be met by a commercially-available product.

[4] As previously noted, this analysis does not address whether the compounded products are as safe and effective as the manufactured one. FDA must have some inkling that they are not: In May 2025, FDA disclosed plans for an extensive survey of compounding pharmacies, in part because “FDA continues to find concerning quality and safety problems during inspections of outsourcing facilities.” https://public-inspection.federalregister.gov/2025-07557.pdf 

[5] As previously noted, this analysis does not address intellectual property rights, which are a significant issue all by itself.

[6] Application of Gresham’s Law is not always intuitive. If enough readers would like a further explanation, I will do a follow-up column. https://en.wikipedia.org/wiki/Gresham%27s_law.

Finally, Some Encouraging News from FDA

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The most important questions facing FDA and the stakeholder community are: 

After the lay-offs, RIFs, and voluntary departures, does the FDA have enough staff and expertise to fulfill the agency’s responsibilities?

If there are tasks and obligations that will not be met--or not met in a specified timeframe--will the agency address statutory, Congressional, and stakeholder concerns? 

It will take a while for Commissioner Makary to have answers. When he does, I hope he will address them forthrightly and not increase the many uncertainties about the agency’s future and priorities. 

Meantime, FDA Matters is monitoring the situation and can report some encouraging signs of progress. 

Rehiring. We are starting to hear more reports about RIF’ed and laid off employees being asked to return to their old jobs. I have no idea whether this is a few or a lot of people. It is not ideal (actually quite cruel) to have fired a large number of people and only then start reviewing their remit to figure out who should have been retained rather than fired.

That said, callbacks are occurring and that is an encouraging sign for the agency. Among other things, it looks like the commissioner is upholding his promise to Congress that he would review all of the downsizing that occurred before he was confirmed. 

User fees. Along with other commentators, I have had serious concerns about the future of user fees. That’s understandable given Trump/Kennedy/Makary projecting their views along the lines of “FDA is a sock puppet for industry.” 

There are now some encouraging signs we can point to. Regulatory Focus reported[1] on Deputy Commissioner Grace Graham’s speech to FDLI last week in which she discussed some early thinking about restructuring user fees. Also, July dates have been announced for the first PDUFA and GDUFA  public hearings. 

Asked by a reporter to comment on concerns about Graham’s thinking. I replied “1/ you can’t get to the end unless there is a beginning and we weren’t certain there would be a beginning,  2/ no matter the starting position of FDA, it is a long negotiation and there is no reason at this point to believe that the end-product won’t be satisfactory to all parties, and 3/ the PDUFA agreements have evolved over 30 years and it is appropriate that a new administration is looking at how to improve the program.” I added: “All that said: this one is likely to be testier and more public than past negotiations….but the administration engaging the process is all good news.” 

More Insights from the Commissioner About His Plans and Priorities. Commissioner Makary seems to have picked up the pace on talking publicly about his views. As we have all seen, he is smart and articulate. 

He has some interesting ideas, which he delivers in an “I can get it done” breeziness that is refreshing. While we disagree about the likely impact of the RIF’s, he seems to understand the FDA cannot be run without a substantial and expert staff. 

Whether you like what he says or not, having him out in public advocating for the agency is encouraging. 

Staff Travel and Meetings. It’s not unusual for incoming administrations to limit employee participation in stakeholder meetings and  tamp down or even freeze travel until new officials are in place. For a while it looked like the Trump administration might try to maximize the time during which things were halted. 

I am hearing enough reports to believe things are going in the right direction. 

Implementation of AI. AI is the future, especially for an agency like FDA that often receives reports that are thousands of pages long and relies on databases that might contain millions of data points. In particular, AI can help FDA make better regulatory decisions based on more refined and accurate information. 

Commissioner Makary’s embrace of AI is encouraging news. There are legitimate concerns about relying on AI too quickly and without system refinement and validation. Details are scarce at the moment, so I am inclined to believe that the actual uptake will be appropriately spaced over time and not rushed.

Finally, there is some encouraging news from FDA. I am pleased to share it.

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1.  https://www.raps.org/news-and-articles/news-articles/2025/5/fda-deputy-commissioner-hints-at-potential-changes?utm_campaign=regulatory_focus&utm_medium=email&_hsenc=p2ANqtz-_rvGbKXqqfzLXRI-d3eFQauxxlA8mWMNMs_7z5LGbDSTAy7GqADQny6Z8sg6TPlJ6S2He5SoKuLXOWBbkfsd1njgOcFQ&_hsmi=362270950&utm_content=362270950&utm_source=hs_email 

If You Are Not Confused, Then You Are Not Paying Attention: FDA Edition

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“Flooding the zone” has worked as a strategy for the Trump Administration, at least so far. We are all so busy reacting to the newest proposals that we don’t have time to address the previous week’s initiatives from the Administration.

While I can’t seem to get ahead, I usually have a good sense of what is coming next or, at least, the range of possibilities. That’s an important aspect of both my FDA Matters writing and my client work.

However, it has been unusually difficult this year because of another strategy being used by this Administration: pervasive norm-breaking. Norms limit the number of plausible scenarios even in complex situations. Without norms, our predictions have to account for a nearly endless stream of improbable possibilities that cannot be eliminated.

In contrast, FDA’s strength should be in its predictability, the opposite of norm-breaking. The agency reliably sets and enforces standards (norms) for $3 trillion worth of goods and services.

Because of norm-breaking scenarios, my FDA crystal ball has never been more opaque. Here are some of the items with the most impact and the least clarity:

Will the Secretary Try to Control FDA? Secretary Kennedy’s predecessors were chosen for a combination of their management experiences, and political and policy chops.[1] Kennedy has none of that, plus he has no known interest in how his predecessors ran the department.

So, it is unclear whether he will adhere to past norms about when--and how much--the Secretary of HHS intervenes in FDA’s decisions and operations. Here is my interpretation of those norms:  

• Budget: okay for HHS to intervene;

• Regulatory policy: okay for HHS to intervene on a case-by-case basis on major items;

• Product decisions: not okay for HHS  to intervene, but there is some history of HHS involvement in reproductive health products;  and

• scientific and medical evaluations and information: not okay for HHS to intervene.

Nothing prevents the Secretary from breaking those norms and making himself the final clearance for all FDA actions, including product decisions.[2] How can we predict or create scenarios for future FDA actions if intervention by the Secretary--ad hoc, selective, total, or permanent--is an ever-present variable?

Will the Commissioner Defend FDA’s Independent, Science-Based Evaluations? I feel certain that Commissioner Makary intends to stand up for FDA with regard to scientific and medical evaluations and information. He didn’t take the Commissioner’s job so that others above him--who do not have appropriate education or training--could substitute their medical and scientific judgment for his own and that of FDA staff.

However, with an unpredictable and self-certain HHS Secretary who is interested in public health, food, nutrition, and vaccines, a continuing stream of large and small disagreements seems inevitable. Some may be uncomfortable or worse for Dr. Makary. We could see this as soon as next week when the MAHA report comes out…or it could come at any time down the line.

How these conflicts are resolved is all-important to predicting  future FDA actions. Yet, the situation is opaque, at least in part because norm-breaking opens up so many possibilities.  

How Much Can FDA Do With Reduced Staff? While far, far from perfect, FDA mostly fulfills its responsibilities and completes its tasks in credible fashion, and in a reasonably timely manner. That is the norm we are all used to.

Because of the 20-25% staff loss, I have urged FDA and the Commissioner to engage in some truth-telling about what the agency can still do and in what timeframe.[3]  I am doubtful that things are fine. I assume there will be an increasing number of missed deadlines.

Much of FDA’s future over the next couple of years depends on whether there is a staff shortage and how existing staff are deployed. What is the new norm for FDA in this regard?  We do not know and may not know for months. The situation is opaque and makes scenario-building and prediction nearly impossible.

FDA will survive whatever comes next…and the next thing after that. However, our ability to see the agency’s direction and predict its future depends on the resolution of several unknowns. The potential for norm-breaking by the Administration multiplies the scenarios and makes each situation opaque.

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1. https://www.fdamatters.com/fdamatters/is-rfk-jr-better-suited-to-be-an-advisor-than-an-executive

2. From an earlier FDA Matters column: FDA statutory authority is assigned to the Secretary, who has delegated it to the Commissioner, who I believe has delegated certain decisions to the center directors. All of these delegations can be withdrawn, in whole or part, at any time.  https://www.fdamatters.com/fdamatters/fda-and-politics-an-unhealthy-combination.

3. https://www.fdamatters.com/fdamatters/truth-telling-fda-user-fee-edition 

Picking Apart the Destructive Claim That Industry Controls FDA

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I am tired of the continued defamation of FDA as an agency controlled by industry. 

That is not to say that FDA is perfect or that there have never been FDA-industry interactions that looked (and maybe were) bad. However, as a generalization….no, FDA is not controlled by industry. 

Such allegations are usually based on ignorance or a genuine misunderstanding of how FDA works. Sometimes it is hard to tell, as with recent statements that advisory committees: decide drug approvals (not true)[1], are dominated by industry (not true), and are cleansed in some meaningful way by eliminating the single, non-voting industry member of the committee (not true)[2]. 

While untrue…allegations of industry-control have consequences. They weaken public acceptance of FDA’s  independent, science-based decisions. They endanger the upcoming user fee reauthorization negotiations. They demoralize staff—a significant proportion of whom want lengthy public service and public health careers at FDA and are not looking for industry jobs. 

The Resolving Door: An Every Day, Everywhere Phenomena

One of the most often cited examples of industry influence is the so-called “FDA revolving door.”  One of my readers even wrote me a note last week, wondering “Isn’t it interesting  how many ex-FDA officials wind up in prominent roles within the industry they were charged to provide oversight.”  

To which I replied: next jobs are built on current jobs. In both the public and private sectors, employers hire well-qualified people based largely on their career experiences.  

There is nothing nefarious—or even out of the ordinary—that people leaving FDA wind up working in regulated industries. That’s where the bulk of the opportunities will be for them. It’s also where people with their credentials—with or without FDA experience--are most likely to be valued. 

There may be a lot of ways a pharmacologist (or a food chemist, or a biostatistician) can make a living, but a significant percentage of those opportunities will be in industry. Does a PhD chemist have to become a car salesperson because otherwise he or she would be perceived as taking advantage of their education, training, and experience to work in or adjacent to FDA regulated industry? 

A Self-Fulfilling Prophecy of Alleged Industry Influence

The DOGE initiative was responsible for the RIF of 3,500 FDA employees. Incentives and lay-offs were used to drive an unknown number of others to leave FDA. 

The bulk of these people had made their careers at FDA or were planning to do so. They were not looking for industry jobs but are being forced to do so now. 

DOGE has created a self-fulfilling prophecy of FDA employees going to work for industry. By Fall, I envision stories that will feed the defamation of FDA: “John Smith, formerly FDA division director for X, now works at [insert name of company with interests in developing X-type products].”

Why shouldn’t they hire him: no one else will have a better understanding of what FDA will require for approval of their product. The company will still have to prove the product is safe and effective and can be supplied at scale to the market using current good manufacturing practices and after an inspection.

FDA Is Not So Easy to Influence

The supposed revolving door is not the only argument you hear. Each and every medical and food decision that appears to favor industry seems to be fair game for the accusation that influence was involved. 

Let me repeat my caveat here: I am not saying that FDA is perfect or that there have never been FDA-industry interactions that looked (and maybe were) bad. However, as a generalization….no, FDA is not controlled by industry. 

At least on the medical products review side, FDA decisionmaking has three characteristics: it is bottom-up; it is multi-disciplinary; and no one has authority to make non-routine final decisions except the center directors.

As a result, lots of FDA staff have the opportunity to have their say about recommended actions as they make their way up the chain. Plus, if there is continued contention, there is often an internal review that includes senior staff not previously involved. In addition, the decisionmaking process often includes a public advisory committee meeting, with independent experts. 

No one can make a decision at FDA that hasn’t been scrutinized by at least a few people and, in most cases, by many people. There is no secret or unilateral decision making[3]. There is really not much opportunity to influence FDA (no less control it) that dozens of people would not know about. 

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1. FDA cannot delegate drug approvals to a non-governmental entity or person, even if it wanted to.

2. Applying Chesterton’s Fence to Industry Representatives on FDA Advisory Committees. Third item at: https://www.fdamatters.com/fdamatters/how-many-people-still-work-for-fda-amp-other-fda-thoughts 

3. There are a few well-publicized examples of center directors overruling senior staff on efficacy issues (not safety), but the issues involved were well-aired publicly both before and after the decision was made. https://www.fiercebiotech.com/biotech/fdas-marks-i-contradict-fda-reviewers-trepidation 

New CBER Leadership: The FDA Matters Analysis

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The headline, “Dr. Prasad to succeed Peter Marks as CBER director,” spread like wildfire Tuesday night after Endpoints News broke the story. 

Reactions ranged from “Omigod, he’ll destroy the industry and the hopes of patients” to “no big deal, staff do the product reviews regardless of who runs the Center.” Experience tells me his tenure is likely to be a little bit of “omigod” and a little bit of hands-off deference to reviewers….but mostly his time at CBER will be measured by how well he led the center during a time of great promise for products within its jurisdiction. 

My three observations (below) were written before two further stories broke last night, recounting the results of Dr. Prasad’s incredibly positive first meeting with FDA staff. The stories are at Inside Medicine and STAT News. They more than vindicate my belief that Dr. Prasad’s prior and more recent statements should not pre-judge what he will do as the CBER director. 

Observation 1: When outsiders become insiders, they take on responsibilities that did not burden their prior opinions. 

It is easy to have an opinion when someone else will be making the decisions and bearing the consequences. It is quite a different matter when you have the power to decide, and your decision will directly affect the course of many people’s lives. 

It is not so much that Dr. Prasad will change his mind, but rather personal responsibility and public accountability change your perspective. He may come to the same conclusion as when he was a commentator, but often he will see things differently. 

I said this of Dr. Makary—and it is the universal experience of becoming a government official--nobody knows for certain how they will respond to a given situation until they actually have to make the decision that affects people’s lives. 

Observation 2: Advocates would be well-advised to continue focusing on their medical and policy positions and not project a straight-line from Dr. Prasad’s past comments to his future actions.

I received a frantic note from a friend and former colleague asking how to block the appointment because Dr. Prasad was going to kill off research and investment in targeted cancer therapies. I could have gotten similar notes with any of a half-dozen other topics in the subject line.

I responded that there was no way (to my knowledge) to block the appointment. I added that while CBER will be different under Dr. Prasad, it doesn’t mean that all the decisions will be different from his predecessors. Some will be genuinely different, but some might be the same, perhaps with small tweaks and new names.

In light of this, I would advise advocates to focus on tightening the case for the policy they want. Show that the science is good and the unmet need serious and worthy of attention. 

Rebutting past statements by Dr. Prasad just feels off by tying him to the past. Advocates should stick with positive positions that are deeply documented and convincingly presented. 

Observation 3: Drs. Makary and Prasad appear to be aligned on vaccines (cautious support but also skeptical), but do not initially appear to be aligned on cell and gene therapy and orphan drugs. 

Along with Dr. Makary, Dr. Prasad was part of a group of physicians who were loudly critical of much of the US public health response to COVID-19. There is a presumption (but no evidence yet) that the two are aligned on vaccines. The bigger question is likely to be how the two of them will handle Secretary Kennedy’s far more critical view of vaccines. 

On orphan drugs (including cell and gene therapies), there is an initial misalignment that I do not expect to last. Dr. Makary has advocated for a new pathway for drugs for ultra-rare diseases (based on plausible mechanism)[1] and has acknowledged the difficulties of trials with small populations. Dr. Prasad has generally emphasized stronger trials that are designed to produce more definitive information. 

Based on my own sense of the situation—reinforced by the Inside Medicine and STAT News articles on his first remarks to FDA staff—I see an easy transition for him to be more supportive of orphan drugs without backing down overall on seeking greater clinical trial rigor. 

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1.  https://friendsofcancerresearch.org/news/biocentury-makary-new-ultrarare-path-will-provide-approval-based-on-plausible-mechanism/