FDA Matters Blog
This week’s FDA Matters column, entitled “FDA is Built on Predictability and Expertise, Not Norm-Breaking,” can be found here.
My position has been “whatever you think of the specific policies and actions that Commissioner Makary is pursuing, and 2/ whatever you think of his leadership and management styles....we must speak our truth AND YET also make clear that we support his leadership of FDA.”
Unfortunately, the situation at FDA has deteriorated over the last few weeks. The downside risk has increased greatly. To understand why, please read my column here.
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FDA Matters analyzes FDA policy and regulatory issues and advocates for a more effective, efficient, and fair FDA.
FDA Matters doesn't cover the news....we provide analysis of what's behind the news.
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FDA Matters subscribers often start their week with my Q&A Monday column. To join them, you can subscribe (free) at www.fdamatters.com.
A substantial amount of new information surfaced during the often quiet Thanksgiving week, so I wanted to share an edited and updated version of yesterday’s (December 1) Q&A column.
This column addresses four issues:
Commissioner Makary’s standing with the White House and HHS and what would likely happen if he quit or was pushed out,
food policy priorities and the relative lack of urgency on hunger and food safety relative to other goals,
Dr. Pazdur disagreements with the Commissioner and why it has resulted in today’s retirement announcement, and
CDC’s implied support of a vaccine/autism connection and how anti-vaccine efforts have now spread to FDA.
Steven’s Traditional Thanksgiving Message to the FDA Stakeholder Community
This week, many of us will be sitting down to a Thanksgiving dinner and talking about the parts of our lives for which we are grateful.
Consider adding thanks to FDA and its more than 15,000 diligent and dedicated staff. Because of them, we have confidence when we sit down for a meal or use a drug or medical device….
HAPPY THANKSGIVING TO ALL
Dr. Pazdur understands that--in this Administration---his becoming CDER director is not an endorsement of the status quo or a mere mandate to stabilize CDER leadership. He will have to deliver on the promises Commissioner Makary has made, including substantially reducing review times, integrating generative and performative AI into all CDER activities, invigorating orphan drug development, and embracing the National Priority Review Vouchers.
None of this is beyond Dr. Pazdur’s experience or prodigious leadership skills. The challenge will be to demonstrate to Commissioner Makary that “continuity and carefully-chosen precedent” are the friend, not the enemy, of the Commissioner’s ambitious agenda.
Since the start of Trump’s second term, the agency has experienced significant losses in staff, capacity, morale, and respected leadership. We do not know whether the situation will improve from here or continue on a downward spiral.
FDA workers need a reason to stay, and job seekers need a reason to want to work at the agency.
When it comes to food and medical products, FDA is our nation’s Department of Homeland Security. FDA needs to be funded and staffed at a level that reflects our vulnerability and the potential consequences. The threat is both domestic and foreign; we need support for systems that are effective in protecting us from both sources....
Very few Americans appreciate the challenge faced by FDA....[As I have discovered] even long-time FDA-watchers don’t understand the strength of the motivation or the size of the profit that is derived from cutting corners on product quality or otherwise evading FDA regulation.
Since the founding of our republic, there has been debate about states’ rights vs federal rights. And in times of stress, the discussion gets more heated and gains more traction. Once again, FDA is moving into the center of such debates.
National uniformity is central to FDA’s mission and responsibilities. In general, food and drug regulation is based on the need for--and value of--comprehensive nationwide coverage of regulated goods and services…
In addition, consumers benefit from the protection offered by one federal agency. Access to safe food and drugs should not vary depending on where a person lives….
FDA appears to be on the brink of fresh engagement where states’ rights and preemption will be central issues….
The announcement that former CBER Director, Dr. Peter Marks, was joining Lilly as SVP for molecule discovery and head of infectious diseases....set the internet ablaze with unjustified outrage about his transition from a government career to industry. My multiple comments and responses on LinkedIn (e.g. here) received nearly 20,000 impressions in total.
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Below are questions from FDA Matters subscribers (www.fdamatters.com). Follow the link to read the full discussion.
Q: Peter Marks would have had many attractive post-FDA opportunities besides pharma. Why choose the one he did? [partial answer]
A: ....His passion — and his most significant contribution to society — has been his commitment to the well-being of patients and his role in facilitating the development of safer and more effective therapies, especially for those with rare diseases. If he cannot be doing that at FDA, what better place than at one of the world’s largest drug discovery organizations, with the budget and mandate to make a difference?...
Other reader questions I address are:
Q: Isn’t Dr. Marks working for a drug company an example of industry controlling FDA?
Q: Why do so many FDA employees work in regulated industries after they leave FDA?
Q: Do you expect more stories about FDA staff moving to industry?
Q: Are FDA employees allowed to own stock in companies that they regulate?
Every day, our country is failing patients with relentlessly progressive chronic neurological, neuromuscular, and genetic diseases. Their plight necessitates a significantly different risk-benefit calculation than that of patients with other types of diseases....
This past summer’s brouhaha about Sarepta’s Elevidys for Duchenne’s Muscular Dystrophy is a stark reminder that we have no systematic way of thinking about these patients and the clinical trials they participate in. FDA’s risk-benefit calculations need to be more context-sensitive to accommodate their situation.
Relentlessly progressive chronic diseases require:
A regulatory framework that reflects the different risk-benefit considerations; and
Scientifically and statistically-based standards for evaluation.
Without those, FDA will not be able to move past its current situation: unable to appropriately respond to the needs of these patients and failing to encourage the types of research and innovation needed to make regulatory decisions for these diseases easier in the future....
Every Monday, FDA Matters subscribers receive “Q&A Monday.” They may recirculate, quote from, or reprint it, etc., at their own discretion, which can be particularly useful for media. I do not publicly post the column until the following Monday. You can find past columns on the Q&A Monday tab (here).
If you would like to receive Q&A Monday columns every week “when-issued,” please subscribe on the site or email me at sgrossman@fdamatters.com.
I have experienced numerous shutdowns and shutdown threats. Sadly, they have become a familiar part of my DC life. Most often, there is a last-minute deal--whether a 48-hour Continuing Resolution (CR), a week-long CR, or a 6-week CR. No one expects a last-minute deal to happen this time….
Regardless of what is being said, Congress feels the pressure and the sense of failure that accompanies a shutdown….
Today (September 30), every affected federal employee was given notice of their status for tomorrow (October 1). As a practical matter, there will be three categories…