It is even possible to think this has occurred. It has been a long summer of media and Congressional attention to safety: whether drug manufacturing, medical products already on the market or Salmonella contaminated eggs. A closer look suggests to FDA Matters that theoretical safety risks and inappropriate FDA concerns about safety are not the issue.

As laid out in more detail in “Safe”: Many Meanings Complicate FDA Policymaking, there is a strong tendency to think of FDA’s safety mission as if it were one type of activity. There are at least six different meanings of “safe” that are relevant to FDA.

For example, we want our food to be “inherently safe,” “safe from intentional and negligent contamination” and “safe from unintentional contamination.” Much of FDA’s attention to food safety this summer has been about unintentional contamination (seafood in the Gulf and eggs with Salmonella).

The agency needs more resources for food inspections and, in the case of eggs, it should benefit from implementation of new standards that were already being phased in. New food safety legislation should also help, but only if Congress appropriates the funds for it to work properly. Whatever substantive or political barriers exist to passage of this legislation, there does not appear to be widespread disagreement about FDA’s role or the standards it applies in assuring food safety.

We want medical products (drugs, biologics and devices) to be “safe as tested pre-approval,”"safe as used post-approval” and “safe as manufactured and distributed.” None of this summer’s medical product safety issues appear to be about nuances of safety or shifts in FDA’s approach to balancing risk and benefits in these three areas of safety concern.

Concern over potential cardiovascular risks from the diabetes drug Avandia existed before its approval. I do not see this as an issue of FDA standards or focus—but rather conflict over the proper interpretation of studies and data. Policymakers and industry should be wary of drawing any larger meaning from this about FDA’s policies on safety.

Much of the rest of the summer’s medical product safety issues were about “safe as manufactured and distributed.” There seems to be consensus—even among those with problems– that real lapses occurred and were violations of reasonable safety policies. As described in prior columns, this requires CEO’s to take manufacturing and distribution seriously, rather than for FDA to re-think its existing safety policies.

While this may have been FDA’s “summer of safety concerns,” there doesn’t seem to have been any change in how the agency balances competing demands involving safety. As the FY 11 appropriations process comes to a conclusion this Fall, there is good reason to put more funding into safety analysis and enforcement. Drawing any larger conclusions is, at best, premature and probably unwarranted.

Steven

Since before Memorial Day, FDA Matters has blogged extensively about FDA and safety issues, although this was not planned. Here is a sampling of columns:

“Safe”: Many Meanings Complicate FDA Policymaking May 23rd, 2010

FDA Matters is in favor of safe foods and safe medical products. Who isn’t? If you are a consumer, maybe that’s all that matters. However, being in favor of safe foods and safe medical products is not enough if you are FDA, the media, Congressional authorizers and appropriators, OMB, and industry. It sounds good, but what does it really mean? In the FDA context, “safe” means many things, some of which are barely related to each other.
Read the rest of this entry »

Not Too Soon to Consider the Hamburg Legacy May 27th, 2010

May 18 marked one year since Dr. Margaret Hamburg was sworn in as Commissioner of the US Food and Drug Administration. The challenges are great, the torrent of issues is never-ending and most days you can smile but you can’t win. Nonetheless, I think it has been a very good first year for her and for Principal Deputy Commissioner, Dr. Joshua Sharfstein. It may seem premature to be discussing “the Hamburg legacy.” But you know that she is thinking about it (all commissioners do), so why can’t FDA Matters talk about it? Read the rest of this entry »

Quality Control Woes: What’s a CEO to Do? June 2nd, 2010

Medical products companies are struggling to assure FDA and the American people that their products are “safe as manufactured and distributed.” We don’t know whether quality control has become lax, FDA is discovering more problems or industry has just had a run of bad luck.

We do know that quality control relies on a lot of people maintaining tough standards…and that manufacturing is rarely a priority of a drug and device company CEO. Earlier this year, in the wake of Toyota’s problems, FDA Matters asked: “what’s a CEO to do?” Read the rest of this entry »

FDA to Industry: Contractors R U June 17th, 2010

It seems a rather uncontroversial proposition: FDA-regulated companies are responsible for their vendors, including every contracted piece of work that is done on the company’s behalf. If problems develop, it makes no difference whether a company did it…or a contractor did it for them. Two seemingly unrelated items this week suggest that FDA is becoming concerned about whether FDA-regulated companies are overseeing their vendors. Read the rest of this entry »

Hot Town, Summer in the City—2010 July 11th, 2010

For the news media, the only FDA story this coming week will be the two-day advisory committee meeting reviewing the diabetes drug, Avandia. Missing from public dialogue is the extraordinary (perhaps unprecedented) number of large, consequential projects that FDA will be working on this summer. Every part of FDA is involved in some initiative that could become a “game-changer” for the agency. Read the rest of this entry »

Benefit, Risk and the Coming Age of REMS August 8th, 2010

FDA supposedly swings back and forth between emphasizing “expedited approvals of promising therapies” and “extended pre-approval examination of every safety issue.” Current thinking is that FDA is now leaning more toward the safety end of this spectrum.

FDA Matters thinks a lot of this is perceptual. Approval decisions reflect FDA’s honest and relatively clear judgment on medical need, quality of the clinical data, and the risks and benefits of a specific product. Mostly, I can understand FDA’s decisions, even when I don’t agree. Still, there is a lot of tension within FDA and with various stakeholders about approvals versus safety risk. Read the rest of this entry »

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The Secretary released the findings and recommendations from a top-to-bottom review of the Department’s efforts with regard to the development of MCM. In the view of FDA Matters, the report thrusts FDA back into its rightful place as a key agency deserving more resources and respect for its national security responsibilities.

MCM are products that will decrease morbidity and mortality from a bioterror attack or from naturally occurring emerging infectious diseases. Think anthrax or radioactivity from an improvised nuclear device for the first, think H1N1 influenza for the second.

Scientifically and medically, these are difficult products to discover and develop. Financially, they won’t ever be developed without:

• federal assistance to promising research; and
  
• a strategic national stockpile and government contracts that will buy proven MCM’s.
  

As with the larger promise of moving medical therapies from “bench to bedside,” there is no progress without FDA. The agency encourages companies by helping them to define appropriate safety and efficacy endpoints for their particular MCM and works with them to resolve questions of animal models, lab standards, statistical plans, quality manufacturing, etc.  

Then, the agency evaluates the testing results and determines whether to approve the product. This work has an additional wrinkle. With most MCM’s (maybe all) it is unethical to do human efficacy trials (e.g. intentionally expose a human being to anthrax to see if the MCM works). Instead, the agency (and the company) must make the difficult evaluation as to whether efficacy in animals is a sufficient surrogate for efficacy in humans.

The Secretary (and the underlying report) found that FDA’s efforts in this area are insufficiently funded. Perhaps for the first time, there was a more global recognition of FDA’s central role in making us safer from bioterrorism and naturally occurring emerging infectious diseases. The Secretary also recognized that FDA needs resources above its current level to do this job well.

The Administration placed a price tag–$170 million in funds to be available until expended–on the size and scope of the monies needed to upgrade FDA’s efforts in this area.  The monies will come from dollars previously appropriated to HHS to combat pandemic flu. HHS and OMB have agreed that the monies can be transferred administratively as long as they retain their original purpose of helping to deal with pandemic flu.

However, the Secretary’s recommendation is for the transferred funds to also be used for non-pandemic medical countermeasures. This requires a budget amendment to be sent to Congress to broaden the permitted uses of these funds. How likely is Congress to approve this? We probably won’t know until after September 13.

What we do know for sure is that FDA can use the additional resources and America will ultimately be safer as a result.

Steven

FDA Matters‘ most recent (and still accurate) assessment of FDA and the FY 2011 appropriations process:

Update on FDA’s Appropriation for FY 11 July 18th, 2010

On July 1, the House Appropriations Agriculture/FDA Subcommittee marked up it FY 11 bill. The bottomline was good for FDA: $2.571 billion, a $214 million increase over FY 10, about 9%. No further details will be released until the full committee marks up.

On July 15, the Senate Appropriations Committee marked up its version of the Agriculture/FDA funding bill. The good news is that the Senate agreed that FDA needs better funding….and provided $2.516 billion, a $158 million in new monies. This is a bit more than 6%. Here is FDA Matters‘ analysis of this critical budget battle. Read the rest of this entry »

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Guest Column: Summer, Camp, Kids, Cancer

By Margaret Anderson, Executive Director, FasterCures

While we focus on improving the efficiencies of the system that discovers treatments and cures for disease, there are untold numbers of people taking a medical treatment journey right now. For the kids partaking in the 28th year of Camp Fantastic in Virginia this week, they get to focus more on the fun, and less on the challenges of coping with a cancer diagnosis and with treatment. Camp Fantastic is a program of a nonprofit called Special Love that gives cancer families support.

I learned of this amazing place from Kathy Russell, who has been involved since its beginning and who also runs the Children’s Inn at NIH. The NIH Children’s Inn helps families with kids in treatment at the NIH Clinical Center to get a bit of normalcy in their lives by providing a warm atmosphere for them to stay in versus an isolating hotel room. The overriding philosophy is that families make a key difference in the lives of their sick children. The work they do represents the full spectrum of NIH investment – from bench to bedside.

By the end of my chat with Kathy, after I dabbed my eyes, I was ready to pack my bags and tell everyone I knew to come with me to help prepare Camp Fantastic and allow kids there to take a break from cancer and be just kids. This year there will be nearly 100 kids at Camp Fantastic between ages 7-17. Usually one-half of them are in active treatment and there are upwards of 60 medical professionals (in addition to countless other folks) who volunteer their time before and during the camp. They literally set up a mini-hospital on-site because it’s in a remote location and far from a hospital with specialized pediatric oncology services. She told me of how kids get their bloodwork done in the am, and a van takes the samples into NIH to do labs and then turns back around with results and medication.

Every year, at least one child is usually transported from the camp in an ambulance or a helicopter to a hospital for further treatment, requiring diligent attention to medical details as well as a carefully thought through psychosocial plan of action to share that news with the other campers. Families are communicated with each day, and as you’d imagine many are nervous and excited about their kids being there. Some children participate while in their final stages of life.

Their families make the ultimate sacrifice by being selfless enough to share their children with others, so that their kids can live out their final hopes and dreams – the same dreams we all have. To do the things we dream of doing, especially those seemingly simple things associated with summertime.

Stories like this remind me of the passion and dedication of the medical research community, of the care providing community, and of the volunteer community. It’s a reality check for me, and for those of us who work on policy-related issues. These kids and their families point out the obvious – that time is of the essence in all we are doing to get to faster cures.

It can be hard to make the FasterCures message personal at times as we deal with mostly macro-level issues, but hearing about Camp Fantastic reminded me why we do what we do. Because it’s summer vacation time, and every kid (and for that matter, every grown-up) deserves a shot at creating their own lazy crazy hazy days of summer memories.

www.fastercures.org

With thanks to Margaret and Faster Cures for allowing me to reprint this from their blog.

Steven

One of my earlier columns for FDA Matters:

November 11th, 2009

Most seriously-ill patients wake each morning wondering how they will surmount the day’s challenges. They are concerned about the health of all Americans, but can’t help being focused upon their own medical situation. Most of the people I know in the medical products industry wake each morning with the hope that their day’s efforts can contribute to improving the health of all Americans, as well as provide specific benefits to seriously-ill patients.

The world-views of patients and industry are not dissimilar. They can share a dream (and an action plan)…if it is built on an unwavering commitment to patients coming first. Read the rest of this entry »

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Indeed, it was the 5-day emergency contraceptive pill, Ella, that was approved. So far, FDA seems to have achieved its goal of less coverage. But I was left wondering if the announcement required that treatment and why it led one advocate to describe the decision as “further evidence that the FDA is committed to restoring scientific integrity in its decisions.”

Abortion is, of course, one of the so-called “third rails” in American politics. For politicians, this means: if you touch it, you will get burned politically. It has never been easy for FDA either. For many years, they were caught in the middle of Congressional and societal fights over the abortifacient, RU-486.

As I have written before (link below), FDA has been a master of little-noticed decisions that create or re-position a disease category. With the new approval, they seem on relatively safe ground in deciding it is an emergency contraceptive. Apparently, there is a nuance as to whether the drug’s mechanism of actions solely delays ovulation or also makes the womb less receptive to implantation. There may be a theological difference, but I don’t see any practical difference.

Ella’s approval doesn’t seem to have been a hard decision and I can’t imagine FDA found it particularly difficult. By announcing the decision late on Friday afternoon, they probably made it seem more consequential and controversial than if they had sent out a more complete press release at a time more conducive to news coverage.

That brings me back to whether this approval is really about “restoring scientific integrity” in FDA decisions. The comparison is being made to the controversy over Plan B, an emergency contraceptive with a much shorter window of efficacy than Ella.

The main issues for FDA regarding Plan B were: should the drug be available over-the-counter (OTC) without the input of a prescribing physician and whether it should be available OTC for women under 18. In my opinion, these were not primarily scientific issues, but social and societal ones. It is completely unlike the current approval of Ella, which will be dispensed only by prescription and doesn’t raise the same societal issues or maybe doesn’t raise any at all.

I am not defending the Bush administration’s handling of Plan B. They could not have done a worse job and they deserve the criticisms they received. But the administration was probably right that the issues were not primarily scientific ones and required input from politically-accountable levels of the government and elected officials—people who have responsibility for public policy that impacts social and societal interests. The Bush Administration should have been more honest about that, rather than pushing the decision down to FDA officials.

Apart from a couple of decisions like Plan B, which are rare, controversial and were badly mishandled, I don’t see that the agency made scientific decisions during those years that lacked integrity. There are always controversies about application of “safe” and “effective” standards….and accusations that science is being ignored. Ella is science-based decisionmaking and business as usual for FDA, not some restoration of agency integrity.

Steven

The FDA press release and the WP and NYT coverage:

http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm222428.htm

http://www.washingtonpost.com/wp-dyn/content/article/2010/08/13/AR2010081305098.html

http://www.nytimes.com/2010/08/14/health/policy/14pill.html?scp=1&sq=ella%20contraceptive&st=cse

An earlier column that is relevant. It includes a brief comment on FDA and pregnancy.

The nature of disease and its constant changes are pertinent to FDA, which often makes decisions on behalf of society that reshape our understanding of disease. Read the rest of this entry »

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FDA Matters thinks a lot of this is perceptual. Approval decisions reflect FDA’s honest and relatively clear judgment on medical need, quality of the clinical data, and the risks and benefits of a specific product. Mostly, I can understand FDA’s decisions, even when I don’t agree. Still, there is a lot of tension within FDA and with various stakeholders about approvals versus safety risk.

In 2007, Congress thought it was doing FDA a favor by providing new tools to speed approvals while better controlling safety risks. The agency is now required, prior to approval of each drug and biologic, to consider the possible value of a Risk Evaluation and Mitigation Strategy (REMS) plan.

REMS replaced a succession of more limited FDA programs designed to decrease the risk that medical products result in adverse outcomes. Risk reduction might involve more detailed patient materials to be dispensed with a prescription, mandated patient counseling, restricted distribution channels (e.g. only specialty pharmacies) and so on.

Congress also thought it was doing industry a favor. The expectation was that REMS plans would provide a way for FDA to approve more drugs when there were important patient benefits, but also significant safety risks. Control the risks….and the benefits of a medical product will more often outweigh those risks. At least at the beginning, REMS plans helped FDA approve some drugs that had languished at the agency.

As I recollect, industry was, at best, lukewarm toward REMS and found it hard to see what favor Congress was bestowing on them. They feared that approvals would come with REMS plans that were so onerous that patient access to new therapies would be threatened (along with the company’s hoped-for market). Many patient organizations agreed that REMS might inappropriately restrict patient access.

In an ideal world, physicians would prescribe omnisciently, always giving patients the right drug at the right dose to maximize the treatment benefit with little risk of bad outcomes. In turn, patients would diligently absorb and follow all drug information and instructions they receive, thus benefiting from the therapy with a negligible risk. And patients would always know what side effect or symptom meant they should return to the doctor. They would also know how the instructions attached to one drug related to instructions on another drug.

Instead, our current system is far from ideal. REMS remains the immediate best hope of reducing adverse outcomes and therapeutic failures. FDA recognizes this and has devoted substantial effort to making REMS work. For example, FDA is testing whether REMS by drug class and indication (e.g. opioids for long-term pain) can maximize patient benefit, level the playing field for competing products, and reduce the agency’s workload.

Even more importantly, the FDA recently held two days of hearings to receive feedback from patients, industry, physicians, pharmacists and health plans. All have a stake in REMS plans that are effective without being onerous or confusing.

Based on my experience, it will take five years of debate, reaction, and conflicting demands for FDA to work out some fundamental and predictable rules for REMS plans. Gauging by the calendar and substantive progress, FDA is about halfway there.

Everyone in the medical products industries needs to keep an eye on the evolution of REMS. Its long-term success is critical to FDA’s careful weighing of “expedited approvals of promising therapies” and “extended pre-approval examination of every safety issue.”

Steven

Some earlier related columns:

When Abbreviated May Not Mean Faster or Easier
July 25th, 2010

FDA is working on an approval pathway for bio-similars, re-examining the way medical devices are reviewed, trying to upgrade the quality and speed of generic drug reviews and will soon be evaluating its process for granting accelerated approvals to drugs.

These seemingly unconnected activities all have in common that they are supposed to be abbreviated processes to get new products to patients more quickly without risking safety or quality problems. FDA Matters thinks FDA should articulate its philosophy about how these short-cuts should work and what standards apply in all instances. Read the rest of this entry »

“Safe”: Many Meanings Complicate FDA Policymaking
May 23rd, 2010

FDA Matters is in favor of safe foods and safe medical products. Who isn’t? If you are a consumer, maybe that’s all that matters.

However, being in favor of safe foods and safe medical products is not enough if you are FDA, the media, Congressional authorizers and appropriators, OMB, and industry. It sounds good, but what does it really mean? In the FDA context, “safe” means many things, some of which are barely related to each other.
Read the rest of this entry »

Has FDA Slipped Back into Anti-industry Mode?
January 13th, 2010

An industry CEO wrote me to observe: FDA is returning to the anti-industry paradigm of the past.  His concern is understandable. Yet, I respectfully disagreed with him. It is natural to fear change. It is easy to confuse activism with ideology.

FDA Matters believes there are two perspectives from which to judge the situation of FDA versus industry. Read the rest of this entry »

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What Congress, FDA, and NIH have learned over the last 30 years is that there are many more medical zebras in the United States than anyone imagined. NIH has catalogued nearly 7,000 rare diseases. More are being discovered all the time. Altogether, it is estimated that 25 to 30 million Americans are affected by rare diseases.

When I first became involved in health policymaking in the mid-1970’s, the “war on cancer” was in its first, high-growth phase and cardiovascular disease was rampant. An “orphan drug” was one that would help third world diseases. During that decade, there were less than a dozen therapies developed for diseases that were rare in the US and elsewhere in the world.

The realization that there could be political strength in unity among those with rare diseases led to the creation of the National Organization for Rare Disorders (NORD) and the passage of the Orphan Drug Act of 1983 (ODA). A rare disease was defined as one that affects fewer than 200,000 Americans. Many rare disease populations are above 100,000, but far more are under a few thousand.

As a result of the ODA, more than 350 orphan products have been approved. The growth of biotechnology and the growth of orphan drugs have been closely aligned. Likewise, knowledge gained through orphan drug development has returned benefits for patients with more prevalent diseases.

More aggressive efforts are being undertaken to stimulate the development of orphan products. Just this year, FDA has been involved in:

• Helping to develop and work with NIH’s Therapeutics for Rare and Neglected Diseases program (TRND), which is intended to move promising orphan drugs forward in the drug development pipeline until they meet FDA requirements for an Investigational New Drug (IND) application.
  
• Creating a new position, Associate Director for Rare Diseases in the Center for Drug Evaluation and Research, who will assist stakeholders and developers of drug and biologic products in navigating the complex regulatory requirements for approval of therapies for rare diseases.
  
• Developing the Rare Disease Repurposing Database that identifies drugs that are deemed promising for rare diseases and are already approved by FDA for another disease.
  
• Stepping up its training of medical product reviewers to devote more time and focus to the construction and analysis of small clinical trials.
  

These efforts have been welcomed by the rare disease community as important steps. Deservedly so.

But will these actions prompt a change in thinking at FDA? FDA claims that orphan drugs are reviewed with the same standards for safety and effectiveness as other drugs. This has become a barrier rather than an advantage.

When reviewing medical product applications for rare diseases, FDA needs to apply a somewhat difference set of rules to account for the special challenges of developing treatments for very small patient populations. Simply put, you can’t expect a 500-person safety database in a disease that only affects 500 people.

With more than 25 million “zebras” roaming the United States, such special considerations seem the least we can do. It is imperative that we advance the scientific and regulatory knowledge that provides these patients with hope and, ultimately, with therapies.

Steven

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These seemingly unconnected activities all have in common that they are supposed to be abbreviated processes to get new products to patients more quickly without risking safety or quality problems. FDA Matters thinks FDA should articulate its philosophy about how these short-cuts should work and what standards apply in all instances.

There is a constant tension between going faster and going slower in making any approval decision. No matter what it does, the agency will be criticized by somebody who thinks they should have waited longer or acted more quickly. The four abbreviated processes seem to bring particular problems because they challenge regulators to balance safety vs. risk and faster vs. slower. In addition, they tend to heighten the distance between companies that are winners and losers.

There is constant tumult around generic drugs. Is proving bio-equivalence really enough to prove two drugs will work the same and thus speed market availability of the generic? Given the relative ease of a generic approval, why does the Office of Generic Drugs have a large and ever-growing backlog?

Likewise, there are always questions about the medical device review process. In particular, the 510(k) approval process is never without skeptics. Many would like all complex devices to meet standards similar to drug approvals. Others point to the quicker pace of innovation and the more incremental nature of new devices as reasons to reserve more elaborate reviews for the most complex and groundbreaking devices.

FDA is currently deciding how to implement the newly created bio-similar pathway. Many (this author included) have suggested that many products for which this abbreviated process was designed will find it advantageous to use the traditional approval route. Surely, this is not why new approval paths are created.

Accelerated approval allows drugs for significant unmet medical needs, primarily life-threatening diseases, to gain market access while further clinical testing is underway. It is not often used (nor should it), but in special cases it allows patients and their physicians to make their own judgments about the risk of the drug relative to the potential benefit. This process is likely to be reviewed now that a drug with accelerated approval proved unsuccessful in further testing.

FDA should always be looking to create greater predictability in its actions. Abbreviated processes hold the potential to benefit patients, increase access, lower costs, and promote innovation. The actual mix of FDA actions more often obscures this, leaving abbreviated pathways to look like an industry battleground rather than a reasoned way to maximize public good.

I envision FDA guidelines on abbreviated pathways that will tell agency employees, patients and industry about appropriate expectations, conditions for use of pathways, levels of proof, and avenues for appeals or to provide greater clarification.

Without this larger FDA view, we will have more of the same: abbreviated approval processes that are often not faster or easier….nor in the public interst.

Steven

For those readers still thinking about this month’s Avandia advisory committee, which featured a sharply divided FDA, this recent column may be useful:

Dissent and Efficiency: Difficult Trade-offs for FDA
May 9th, 2010

FDA has a reputation for being tough on dissent, whether it comes from employees or regulated companies. It is often alleged that FDA employees with contrary views are re-assigned, marginalized or ousted. Within the regulated industries, there is a widespread belief that arguing with FDA has adverse consequences for a company.

Whatever the truth has been in the past, FDA is trying to develop an institutional cultural that welcomes and accepts dissent from employees, industry and other stakeholders. It is difficult, even messy, to do this. Yet, FDA’s reputation and authority rests on showing that it listened to all competing views–without unreasonably slowing the decisionmaking process. Read the rest of this entry »

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On July 15, the Senate Appropriations Committee marked up its version of the Agriculture/FDA funding bill. The good news is that the Senate agreed that FDA needs better funding….and provided $2.516 billion, a $158 million in new monies. This is a bit more than 6%. Here is FDA Matters’ analysis of this critical budget battle.

The Senate’s funding level for FDA is the same as the Administration’s request. There was hope that the Senate could find the $55 million more to reach the House level. Even the House’s proposed funding is not nearly enough to meet FDA’s responsibilities in the coming year and the Senate is clearly even further behind with its number. Even so, the Senate’s 6% increase is more than what most domestic federal agencies will receive.

The Senate mark-up proved one important point. Both the Senate and the House agree that FDA needs to be an exception to this year’s tough round of budget-cutting.

There is still a ways to go. The House full committee needs to mark up its bill, and then both bodies will need to consider and approve these appropriations bills. This presumably would occur in September, although it may occur in late July. It looks like the Agriculture/FDA legislation might be one of the first appropriations bills to move through the pipeline. Hopefully it will be enacted into law before the Congress has to pass a Continuing Resolution (September 30 in advance of the new fiscal year, which starts on October 1).

The primary advocacy group for FDA resources, the Alliance for a Stronger FDA, continues to work for the House level or higher.  It is difficult for any agency in this appropriations cycle. Hill deliberations are already sharply skewed by the upcoming 2010 elections. Despite this environment, the FDA’s friends in the Administration, the House and the Senate are still trying hard to get the agency more money for FY 11.  The Alliance is working with them to preserve FDA gains and to raise awareness that FDA is still under-resourced relative to the agency’s responsibilities.

In past years, it would have been safe to assume FDA would receive an increase of $158 million to $214 million. However, none of the current numbers can be relied upon until the House and the Senate reach some understanding about total spending for FY 11.

The Senate is considering proposals that would reduce domestic discretionary spending by either $4 billion or $20 billion. If it is the latter, a large number of domestic programs will face deeper cuts than currently planned. Those who are doing comparatively better may find their gains wiped away. This macro-budgetary risk means the case for FDA must continue to be made and that numbers being discussed now cannot be relied upon.

Below I have provided links to prior columns that discuss why FDA’s needs grow each year and why the agency must be an exception to budget cuts. Also below is a chart comparing FY 10 to the levels proposed by the Alliance, the Administration, the House and the Senate.

For information about adding your voice to those advocating for more FDA resources, go to www.strengthenfda.org or send me a note at sgrossman@strengthenfda.org.

Steven

For purposes of disclosure, I am one of the founders of the Alliance for a Stronger FDA and serve as its deputy executive director. FDA Matters is not affiliated in any way with the Alliance.

FDA: A Bargain at Two Cents Per Day Per American
March 28th, 2010

FDA touches every American many times each day. Today’s investment (2 cents per day per American) is a pittance compared to the benefit of a strong FDA and the risk of an underfunded FDA. There cannot be many agencies in the US government that have such a vast scope of responsibilities and so few dollars to get the job done.

This is the powerful message that the Alliance for a Stronger FDA has been delivering to Capitol Hill. Even still, it will be a difficult year for any federal agency whose mission and responsibilities are growing. Read the rest of this entry »

FDA Needs at Least a $250 Million Increase in FY 2011
March 7th, 2010

The President’s proposal to freeze domestic discretionary programs in FY 2011 (and beyond) will force painful cuts across government and in programs that millions of American rely upon. Even some traditionally-favored agencies, such as NIH, are looking at only small increases. With a proposed 6% increase (about $150 million), FDA would seem to be doing far better than most.

FDA Matters feels strongly that this is not nearly enough. By my calculations, at least a $250 million increase for FDA would be needed, just to achieve the program levels anticipated in the President’s budget request. The Alliance for a Stronger FDA has asked for a $495 million increase, which could be put to good use by the agency. Why is 6% not enough? Read the rest of this entry »

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Status of FY 11 Appropriations for the FDA

Compared to the Alliance for a Stronger FDA’s FY 11 Request

Updated July 16, 2010

Budget Authority Appropriations (does not include user fees)

Some columns may not add due to rounding.

The House has released the total for the FDA, but does not release the allocation until full committee mark-up expected later this month.

For more information about the Alliance for a Stronger FDA:

Ladd Wiley, phone: (202) 887-4083 email:  lwiley@StrengthenFDA.org

Steven Grossman, phone: 1- (301) 539-9660 email:  sgrossman@StrengthenFDA.org

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History tells us that the choice of a new PhRMA President will reflect the Board’s view of the state of the industry and its most pressing needs. The new appointment continues that tradition.

At the outset, let me say that Mr. Castellani appears to be eminently qualified. He has roots in corporate America and has spent years steering a powerful association. He knows how to manage CEO’s. Early news coverage has characterized him as a manager, straight-shooter and non-partisan (although he mostly contributes to Republican campaigns).

However, there is more to his appointment. In a column earlier this year (link below), FDA Matters examined the past five Presidents of PhRMA, going back to the 1970’s. It is hard to imagine that the same association could be led by individuals with such different backgrounds. Yet, on examination, each appointment was appropriate for the industry’s needs at the time they were appointed.

Whether the PhRMA search committees have acted intuitively or consciously, their choices have been spot-on to what the industry needed.

In the earlier column, I stated: When his [Tauzin's] successor is chosen, it will tell us a lot about how the pharmaceutical industry sees itself….and what the big pharmaceutical companies think is their next major challenge. I think there are two such challenges faced by the bio-pharmaceutical industry:

• Strengthen solidarity with non-pharma industries, and
  
• Preserve its status as a major domestic industry, despite the growth and pull of globalization.
  

CEO’s from other US industries are far more concerned about rising health care costs than the well-being of pharma companies. There have been moments when it seemed possible that broad industry groups might support positions affecting pharmaceutical companies that they would never support for their own companies.

For example, PhRMA would have great difficulty winning fights on re-importation and drug price negotiations if the rest of corporate America took the opposite position. To reduce or eliminate this possibility, who better to choose than the man who has represented the larger corporate community for the last 9 years?

With regard to the second challenge, PhRMA has successfully leveraged its status as a major domestic industry with a positive contribution to the American economy. This has helped soften the politically dangerous perception that American taxpayers, insurers and patients are cross-subsidizing the cost of medicines in other countries.

To sustain that leverage in the face of globalization, PhRMA needs to demonstrate that the US is still the home of the bio-pharmaceutical industry and that the growth of the industry is matched (or exceeded) by the economic benefit to the US. Who better to choose than a nationally-recognized advocate for policies to create domestic job growth and investment?

Some readers may say that these are not reasons enough to drive the choice of Castellani. Or they might argue that these are side-benefits rather than the primary point. I acknowledge that he is a “man for all reasons” and an obvious choice now that it has been announced.

I am intentionally looking at a larger perspective on the appointment, trying to see how it relates to the most important determinants of the long-term success or failure of PhRMA. My conclusion:

If John Castellani can maintain solidarity with corporate America, grow the perceived role of the pharmaceutical industry in the US economy, and manage the staff’s communications and lobbying efforts….then everything should turn out fine for PhRMA.

Let me know what you think?

Steven

The press release announcing the appointment is at: http://www.phrma.org/news/news/john_j_castellani_lead_phrma_new_president_ceo.

An interesting profile: http://www.whorunsgov.com/Profiles/John_Castellani.

An interesting analysis: http://www.muckety.com/John-J-Castellani/96246.muckety

My earlier column:

Transition at PhRMA
February 14th, 2010

Former Representative Billy Tauzin tendered his resignation this week, ending a 5 ½ year tenure as President of the Pharmaceutical Research and Manufacturing Association (PhRMA). When his successor is chosen, it will tell us a lot about how the pharmaceutical industry sees itself….and what the big pharmaceutical companies think is their next major challenge. Here is FDA Matters’ analysis. Read the rest of this entry »

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Missing from public dialogue is the extraordinary (perhaps unprecedented) number of large, consequential projects that FDA will be working on this summer. Every part of FDA is involved in some initiative that could become a “game-changer” for the agency.

FDA shares at least two summer issues with Congress: comprehensive food safety reform and drug safety reorganization. Food safety legislation has passed the House. A different version is awaiting Senate floor action. Since final legislation is not guaranteed, FDA is working hard to develop an approach that is not dependent on statutory changes.

Although drug safety is not an active legislative item, several senior Members of Congress have been persistently calling for re-organization and other changes in how drug safety is evaluated and tracked. The Avandia advisory committee meeting has providing focus for these critics, but their positions do not depend on the outcome.

FDA’s efforts to stay in control of drug safety are reflected in at least three initiatives that FDA is working on this summer: creating workable risk management plans (REMS) to accompany drug approvals; safety issues that are becoming part of the negotiations on renewal of drug user fees; and continuing efforts to update Sentinel and related tools for tracking adverse events and safety signals in large populations.

FDA continues its efforts to clarify its policies on safety and effectiveness of medical devices. Pre-approval issues include possible changes in the 510(k) pathway. Post-approval efforts include better device tracking.

Follow-on biologics (now re-named bio-similars) are also keeping FDA busy. This is the first new drug approval pathway in 25 years and FDA has already declared itself ready to accept product applications. At the same time, the agency has acknowledged that there are multiple policy issues to be resolved before agency guidance will be available. What FDA decides now (both on applications and policy) will reshape the world of bio-pharmaceuticals.

Some other top-level agency initiatives with potentially large consequences:

• FDA is grappling with its role in comparative effectiveness research.
  
• The FDA’s Transparency Task Force has just reported its findings and recommendations.
  
• Upgrading inspections and enforcement are an immediate and ongoing priority for the agency.
  
• FDA is building a new relationship with NIH through a series of initiatives that will fail without serious attention.
  

Around the agency, here are a few more that could bring significant changes:

• FDA, NIH, patients and industry are trying to upgrade research on rare diseases and increase approvals of orphan drugs.
  
• FDA has promised guidance later this year on medical product communications on the Internet and in social media.
  
• FDA is wrestling with antibiotic use in food animals and kicking up some controversy.
  
• Implementation of the year-old tobacco legislation is ratcheting up after various provisions became effective in June.
  

Even upcoming product reviews may have interesting consequences. Over the next few months, FDA will be looking at three new drugs to treat obesity. This is a difficult product category with a history of safety problems. Yet, millions of Americans are likely to use these products if they are approved.

Despite the number of potential “game-changers” I have identified…no one knows better than Drs. Hamburg and Sharfstein how incomplete my list is. Fortunately, FDA has a great staff. I suspect most of them will be overloaded this summer.

Steven

FDA commissioners need to stay focused on their legacy, while dealing with the mountain of important issues discussed in today’s column:

Not Too Soon to Consider the Hamburg Legacy
May 27th, 2010

May 18 marked one year since Dr. Margaret Hamburg was sworn in as Commissioner of the US Food and Drug Administration. The challenges are great, the torrent of issues is never-ending and most days you can smile but you can’t win. It may seem premature to be discussing “the Hamburg legacy.” But you know that she is thinking about it (all commissioners do), so why can’t FDA Matters talk about it? Read the rest of this entry »

My earlier column that relates to the Avandia advisory committee meeting:

Dissent and Efficiency: Difficult Trade-offs for FDA
May 9th, 2010

FDA has a reputation for being tough on dissent, whether it comes from employees or regulated companies. Whatever the truth has been in the past, FDA is trying to develop an institutional cultural that welcomes and accepts dissent from employees, industry and other stakeholders. It is difficult, even messy, to do this. Yet, FDA’s reputation and authority rests on showing that it listened to all competing views–without unreasonably slowing the decisionmaking process. Read the rest of this entry »

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