FDA Funding for FY 11: Back to the Future

Not so long ago, FDA's appropriation barely budged from year-to-year. A good year was a 2% to 3% increase. This changed four years ago, after the formation of the Alliance for a Stronger FDA. The agency's case for more resources—always a good one—finally had an independent, multi-stakeholder voice. Champions on the Hill and in the Executive Branch emerged.

The agency appropriation has grown 50% in the last three fiscal years. So far, FY 11 looks more like the past than it does the last few years. FDA Matters believes that the consequences could be severe.

For the new fiscal year (FY 11) starting on October 1, Congress passed a Continuing Resolution (CR) that funds the government until December 3. As a result, the government is open, but no agency received new funding. For the next 2 months, government agencies (including FDA) will only be able to spend the amount they were appropriated in fiscal year 2010.

One of this year's Alliance themes has been the need for FDA to be an exception to the overall dismal federal budget situation. The agency must receive funding increases to do its job, which grows larger every day. There is strong support for this in Congress. The Alliance expects FDA to receive serious attention anytime Congress debates funding priorities.

This didn't happen for this first round of the CR for a fairly simple reason: Congress put this CR together without acknowledging any funding priorities. The Alliance believes FDA can win the debate to get increased funding, but only if that debate occurs.

When Congress returns in mid-November and looks at FY 11 funding again, there will be an opportunity to make the case that FDA should receive more funds. However, it is only a guess that this debate will occur before December 3. Congress may decide to pass the same CR to cover the period from December 3 to February 4.  If so, the pain of level funding will only become more intense for FDA.

Funding FDA at last year's levels is a particularly acute problem because over 80% of the agency's budget is people-related costs: salary, benefits, rent, IT, travel, support services. FDA cannot delay spending, as NIH might, by waiting a few extra months to disburse grant funds.

FDA management is reviewing contingency plans to preserve its priorities and maximize output. Retaining the fiscal 2010 spending levels will impact FDA's ability to hire medical product reviewers and food inspectors and personnel for other areas of growing need. The agency must use its CR funding to pay for October 1 raises and rent increases, which obviously are not reflected in the FY 10 appropriations level.

The overall situation, as well as these new costs, restricts FDA's ability to make commitments in hiring, new programming, better IT, and contracts. They will also not want to create obligations now that they may not be able to fund later in the year.

Congress will eventually be ready to address the nation's funding priorities, hopefully in November. Regardless of when this debate occurs, all of FDA's supporters will need to rise up as one to convince Congress that more monies are needed.

Steven

For purposes of disclosure: I am a founder and Deputy Executive Director of the Alliance for a Stronger FDA. It is the only multi-stakeholder group devoted to education and advocacy to increase the appropriated resources available to FDA. Members include patient and consumer groups, professional societies, research advocacy groups, associations, companies, consultants and individuals. For more information about the Alliance, go to www.StrengthenFDA.org or write to me at sgrossman@StrengthenFDA.org.

FDA's Yearly Appropriations Growth Since FY 2003

FY 03 (base year):         $1.390 billion

FY 04 ($11M increase):    $1.401 billion

FY 05 ($51M increase):    $1.452 billion

FY 06 ($41M increase):     $1.493 billion

FY 07 ($77M increase):    $1.569 billion (most domestic programs received no increase)

FY 08 ($145M increase):    $1.714 billion (plus $150M one-time supplemental, non-add)

FY 09 ($325M increase):    $2.039 billion

FY 10 ($306M increase):     $2.345 billion

FDA and Quran Burning: Trouble Can Start With a Tweet

Bravo! Florida Pastor Terry Jones has decided not to burn a copy of the Quran. The next danger is that the Pastor's "success" will be seen narrowly as the unique confluence of 9/11, the Ground Zero mosque, and the readiness of millions to take to the streets at signs of American intolerance toward Muslims.

FDA Matters thinks the lessons are larger and urges FDA to pay attention to how this reflects changes in the way crises develop and decisions are made.

According to the Washington Post, on July 12 the Pastor sent a simple message on Twitter: "9/11/2010 Int Burn a Koran Day." From that beginning, he became the focus of international concern and one of the most watched people on the planet. All news sources—print, television and internet—were filled with stories about his intended actions. The situation most resembled a dramatic, high-stakes hostage negotiation. In a sense, it was, with Pastor Jones'copy of the Quran as hostage.

If we are imaginative, there is much more to concern us than Pastor Jones. We now know that one man's temper tantrum (or cause?)—distributed via Internet, Twitter, YouTube, 24/7 news outlets, etc.—can leave the world's leaders pleading for a favorable outcome. The power to capture the public eye, move public sentiment and galvanize the world...now belongs to all us. As Pastor Jones demonstrated, not everyone has the restraint to use that power responsibly.

In the late 1980's, AIDS activists staged a large demonstration that caused a multi-day lock-down situation at FDA. Patients and their friends were expressing their desperate need for access to drugs in development. It was a scary time at the FDA. No one wanted to create the precedent that FDA "would bend to a mob," but there also was a lot of sympathy for the plight of HIV patients.

Soon thereafter and over the next few years, FDA made a number of significant changes to the drug approval process. For example, the demonstrations eventually led to an accelerated approval process and "fast-tracking" of certain new drug applications. FDA began to treat surrogate endpoints seriously (e.g. CD4 viral load count rather than HIV disease progression). Most of these changes have been judged "good" with the passage of time, but there was a lot of uncertainty at the time.

With all the new avenues for communication, this scenario could easily repeat itself today on behalf of any number of diseases that are life-threatening or crippling. Could a group of parents with dying children try to force FDA to grant compassionate use access to a drug in pre-clinical (animal) testing? To make their point, the parents could create a vigil for the children with countdown clocks, while posting daily video showing their deterioration.

Yet another scenario might blend the skills of the Tylenol poisoner with the long-term persistence of the Unabomber. Every three months, anonymous videos might be posted that show gloved hands adulterating some additional batches of a food, drug or device. This would be a corporate nightmare that would also bring all FDA-regulated products under suspicion. FDA would be at the center of an enormous public health crisis.

I know I have not reflected the depth or breadth of challenges FDA could face. Hopefully, these few make the point that FDA is increasingly vulnerable as an agency. It must prepare for risks well beyond those faced by most federal agencies.

Commissioner Hamburg ought to meet soon with senior staff to review, extend, and refresh FDA's crisis plans. Even if they met two weeks ago, they should do it again because Pastor Jones has proven that the world has changed.

And while senior staff is having this discussion, they should also plan some simulations and practice drills. A crisis plan is only as good as the ability to implement it.

Steven

The Washington Post story describing the escalation after the tweet is at: http://www.washingtonpost.com/wp-dyn/content/article/2010/09/10/AR2010091007033.html?hpid=topnews

Is the New FDA Different than the Old FDA?

Two weeks ago, FDA Matters explored Dr. Hamburg's legacy, focusing on advocacy for resources, prioritizing regulatory science and upgrading enforcement. These will be accomplished before she leaves office. But is she making similar progress in creating "a new FDA?"

Judging by her first year's effort, FDA is becoming "new" in some important ways. Still, there are signs of retrograde attitudes and some ways in which FDA just doesn't seem capable of changing.

Although FDA has long called itself a "public health agency," it has been run by individuals who came from academic health centers. Dr. Hamburg and Dr. Sharfstein ran big city health departments. The agency's decisionmaking standard has become "what's best for the public health." I think this is becoming a core part of "a new FDA."

Dr. Hamburg has had new funds to work with, through both appropriations and user fees. Along with normal turnover, this means a substantial part of the agency workforce ten years from now will have been hired and trained during Dr. Hamburg's tenure. All by itself, this contributes to "a new FDA" that will reflect her stamp.

Dr. Hamburg is trying to create "a new FDA culture," a difficult task in any governmental agency. Thus far, it is defined by an intensity of activity and a broad range of initiatives. I see a new spirit within the agency: issues can be addressed if there is a public health impact, regardless of whether they would have been acted upon in the past.

The idea of "a new FDA" may be making less progress elsewhere at the agency. Traditionally, tougher enforcement has been cyclical. It will not become a constant force unless Dr. Hamburg chooses wisely for the new head of Office of Regulatory Affairs. The character of this person--FDA knowledge, superior leadership skills, new ideas about effectiveness and fairness, commitment to standards—will determine whether enforcement becomes a central part of "a new FDA."

And then there are parts of FDA that still look a lot like "the old FDA." Issue and activity silos are still the norm rather than the exception. Dr. Hamburg is setting a good example with her efforts to strengthen science agency-wide. But FDA cannot be considered "new" without substantial progress in making FDA and the American public the first loyalty of employees. It cannot be their branch, division or Center.

Despite sincere efforts by Drs. Hamburg and Sharfstein to clarify and expand upon agency positions and actions, public communications are still "old FDA." The agency is struggling with so-called new media (Twitter, Facebook, blogs, etc), while attacking industry for recognizing and acting upon this new form of communications. FDA cannot be "new" (or even current) until it provides insight, guidance and "leadership by example" in this area. Efforts at improving transparency at FDA need to acknowledge that a broader range of senior leadership needs to be available to the press on a regular basis.

A "new FDA" cannot be achieved without the strongest possible commitment to innovation--in actions and not just words. Critical Path and advocacy for regulatory science don't go far enough. Efforts to develop bio-markers and new statistical methodologies are worthwhile, but have the feel of "one-offs" instead of concerted efforts to systemically modernize the clinical trials system and the standards for FDA approvals.

Like her legacy, Dr. Hamburg's effectiveness in creating "a new FDA" is still unwritten. It is too soon to know if she will succeed. She gets an A for having chosen far-reaching, worthy legacy items. The effort to create "a new FDA" must be considered a B- so far, showing good aptitude but still in need of better application and follow-through.

Steven

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

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 »

Fortuitous Timing and Public Health Leadership at FDA
March 14th, 2010

Commissioner Hamburg and Principal Deputy Commissioner Sharfstein are very good leaders who have also benefitted from their prior public health experiences and the timing of their appointments. Here is FDA Matters' analysis: Read the rest of this entry »

Commissioner Hamburg's Most Important Personnel Decision
February 21st, 2010

With due respect to the many fine individuals that Commissioner Hamburg has recruited, FDA Matters thinks the most important appointment needs to be made soon: choosing the right person to be Associate Commissioner for Regulatory Affairs. Read the rest of this entry »

Not Too Soon to Consider the Hamburg Legacy

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?

Top-line: the agency has a renewed energy and sense of purpose, which I attribute to her leadership. She has been aided by something none of her recent predecessors have had: the flow of new monies. This has allowed her to make choices about priorities and invest time and manpower into them.

Dr. Hamburg's most important legacy will be whether she can sustain this momentum. FDA is still severely under-resourced. The FDA commissioner who can reverse this trend for more than a few years will always be remembered for that.

Improving regulatory sciences is apparently quite high on Dr. Hamburg's list and I have written favorably about it a number of times. I also know that a lot of FDA-watchers are puzzled by it. Here is FDA Matter's explanation:

"Regulatory sciences" means the tools, techniques and knowledge needed by food and medical product regulators to carry out their public responsibilities. Fundamental to the concept is that consumers, patients and regulated industries benefit when regulators have sophisticated, state-of-the art capabilities and use them transparently so that no stakeholder has to guess about the agency's approach.

"Regulatory science" is most often thought of in relation to medical product approvals and food safety, but actually extends to every aspect of the FDA's responsibilities, including manufacturing, product tracking, laboratory procedures, post-market standards, sentinel monitoring, etc.

Accomplishing this—even getting it firmly launched—is a legacy item. We will all benefit if she succeeds…and we will certainly remember her for it.

A third area emerging as a legacy item is a new FDA toughness on enforcement. At one point, I had thought that this was a threshold item: Congress wouldn't let Dr. Hamburg address her other priorities unless she proved that she could assure the safety of medical products and food. That may even be how she thought of it a year ago.

This mission turned out to be more than just eliminating some marginal players and confirming that the mainstream regulated industries were playing by the rules. The past year, we have seen a number of established, name-brand companies held accountable for lapses that should not—by their own admission—have occurred.

One important consequence of inspections and enforcement is to keep everyone on their toes. Maybe CEO's of FDA-regulated companies are not asking tough enough questions or don't appreciate how much the company can be hurt by people many levels below them. If the behavior of mainstream industry is markedly improved and the agency is clearer and more predictable in its standards and enforcement actions, then this would be a powerful legacy for Commissioner Hamburg.

With a year in office, it's not too soon to discuss Dr. Hamburg's potential legacy. What do you think of my list? What would you add? Please post your comments or send them to me at sgrossman@fdamatters.com

Steven

Some previous columns that touch on each of these legacy items:

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 »

CARS: The Vehicle for FDA's Future

October 25th, 2009

Since Labor Day, Commissioner Hamburg has spoken a number of times about the importance of regulatory science. She is right. FDA must have the scientific tools and methodologies to be a 21st century regulatory agency. FDA needs to define regulatory science, develop programs to support it, and package them in a way that will quickly bring recognition and funding. Read the rest of this entry »

Shaken to the Core: What FDA and FDA-Regulated Companies Can Learn from Toyota

February 25th, 2010

I do not believe that Toyota became a global success by cutting corners and ignoring safety concerns. Nonetheless, the company may not survive the investigations, the lawsuits, the civil and criminal fines, the securities litigation, the recalls (8.5 million cars so far), the loss of consumer confidence and the possible criminal indictments.

FDA Matters hopes that the CEO's of FDA-regulated companies are paying attention. They need to understand that their company can be "shaken to the core," as Toyota has. Read the rest of this entry »

“Safe”: Many Meanings Complicate FDA Policymaking

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.

What are FDA's safety goals and their means to achieve them? What programs should they strengthen? What people should they hire? Each of these questions has different answers depending on what kind of "safe" is being considered.

In the food area I can think of at least three non-redundant contexts in which the meaning of "safe" is different.

First, we want our foods to be "inherently safe," a product that is formulated properly and with no negative impact on our health. We do not want to be offered "tomato and arsenic soup." For this FDA needs food scientists and regulators to determine ingredients that are "generally recognized as safe" and to assure that products conform to standards of identify for specific types of foods.

We also want foods to be "safe from intentional and negligent contamination." We do not want melamine in milk nor heedless disregard of procedures to prevent botulism, pesticide residues, etc. FDA requires well-trained inspectors, backed by laboratories to perform chemical and biological analysis of otherwise safe foods. Criminal investigators and prosecutors are also part of assuring foods are safe from intentional and negligent contamination.

We also want foods to be "safe from unintentional contamination" by bacteria, insects, fungi, and naturally-occurring toxins. To provide this protection, FDA needs epidemiologists, biologists and health professionals with public health training, along with laboratories that can do sophisticated analysis of pathogens.

Likewise, in the medical products area I can think of at least three non-redundant contexts in which the meaning of "safe" is different.

First, we want medical products (drugs, biologics and devices) to be "safe for use" before they can be marketed. The FDA's team is composed of scientists and statisticians who can: analyze the chemical and biological foundations of a product; dissect the degree of safety demonstrated in animal and human trials; and work with fellow regulators to determine the balance of risk and benefit.

We also want medical products to be "safe as used" once they are in the marketplace. For this, FDA increasingly needs public health, data and medical analysts who can: evaluate individual case reports and derive usable knowledge from population-based data, such as FDA's new Sentinel System. I think that post-market safety is often considered a mere extension of pre-approval safety. This won't be true in five years.

We also want medical products to be "safe as manufactured and distributed." This requires well-trained inspectors, backed by engineers and manufacturing and supply chain experts. Data systems are needed here, too, to track facilities, shipments, processors, importers, etc. Criminal investigators and prosecutors are also part of assuring safe manufacturing and distribution of medical products.

As can be seen, Commissioner Hamburg's challenge is much more complex than "hiring more safety people" or "investing more of the agency's budget on safety programs." As she defends her priorities, her position would be stronger if it rested on a comprehensive analysis of how the agency is working on all the different meanings of "safe."

Steven

PS: This is a conceptual analysis with strong real-world consequences. There are many situations where the lines I've drawn are not as clear as I've suggested. Also, I do not want to diminish the abilities of many FDA staff who routinely contribute to more than one type of "safety."

Improving safety and improving information technology go together. Two earlier columns reflect on this:

The Science Board's IT Report: Too Technical to Read, Too Important to Ignore October 18th, 2009

Some of FDA's most difficult tasks are: defining the agency's role in nanotechnology, creating a pathway for follow-on biologics, implementing a risk-based food safety system, and establishing the right policy for "new media" communications. All rolled together, they are not as complicated or important as transforming information technology (IT) at FDA. Read the rest of this entry »

Turning Data into Knowledge    June 2nd, 2009

Through statute and directive, FDA has been asked to collect, analyze, interpret and utilize massive amounts of data. This includes biological, clinical, adverse event, production and distribution data, medical and food product tracking, and the Sentinel system for early discovery of potential drug safety problems. The systems are not in place to do any of this, at least not at the required level of sophistication. Even if they were, sifting valuable information from background noise is extraordinarily hard. As a result, FDA needs to manage Congressional and public expectations as to "what is possible and when." Read the rest of this entry »

Clinical Trials: Gold Standard In Need of Improvement

There is an emerging crisis in the development of drugs, biologics and complex new medical devices. Clinical trials take too long, cost too much and often produce imperfect knowledge. Many promising medical products are not developed because of the difficulty and expense of proving safety and efficacy—a loss that is costly to society.

FDA Matters believes that the key lies in developing new approaches to generating rigorous data and analysis. Ultimately, this will require the re-invention of the clinical trial.

Clinical trials produce the knowledge that makes FDA approvals possible. Without them, we would all become test subjects in a dangerous game of medical trial and error. FDA (and patients) want a reasonable level of certainty about safety, efficacy and risk-benefit before medical products are marketed. Except in extraordinary cases, FDA should never be put into a position to accept less.

The clinical trial is, and must remain, the gold standard. To understand why, it is useful to look at another type of medical knowledge that is increasingly in vogue: analysis of real-world data. The Medicare Claims database would be an example. Another would be patient data compiled by large health plans. Analysis of real-world data sets is becoming a cornerstone of reimbursement policy and plays a significant role in comparative effectiveness determinations.

The supposed advantage is the ability to look at hundreds of thousands of patients and discern patterns that might not be seen in clinical trials. However, the association of data points tells us nothing about causality. It only signals where additional analysis is needed. Real-world datasets also lack rigor:

Real-world data sets → post-hoc analysis using uncontrolled variables + inconsistent definitions + incomplete data collection + questionable data accuracy

By comparison, clinical trials produce a wealth of reliable knowledge (albeit far from infallible). This can be expressed as:

Clinical trial data sets → prospectively-defined analysis using controlled variables + randomization of patients + double-blind protocol + placebo controlled + pre-defined standard for data collection and data integrity

"Prospectively planned" means a drug or device sponsor must declare in advance the precise findings that will determine whether the treatment caused a beneficial outcome. Sponsors are limited in their ability to go back afterward to "data dredge" for positive correlations that might be spurious. To some extent, all analysis of real-world data sets is data dredging.

"Controlled variables" means that the outcomes of patients in the clinical trial can be compared with some degree of reliability. In real-world data sets, you can never be sure.

"Randomization" and "double blind" work together to assure there is no bias in patient selection (e.g. putting healthier patients in one arm of the trial) and that neither patients nor medical staff knows who is getting the study drug.

"Placebo controlled" allows a reliable determination of the impact of treatment. Since some patients will improve regardless of whether they are getting treatment or placebo, treatment effectiveness is the differential between those who improve in one study arm over the ones who improve in the other.

"Pre-defined protocols for data collection and data integrity" assures that definitions stay constant and results from different trial sites and different investigators can be combined. In real-world data sets, no one has yet figured out why medicine is practiced differently in Boston compared to Hartford.

Taken together, these features of the clinical trial serve to produce reliable data that support a conclusion (or not) that the treatment caused the benefit. The challenge is to improve upon this gold standard while maintaining confidence in the results.

Future columns will explore how this might be done. Meantime, readers are encouraged to post their thoughts or send me their ideas.

Steven

Here are two earlier columns that partially address this topic:

Long-term Challenges Need Short-term Attention

December 13th, 2009

We are less than 7 months into the new Commissioner's tenure. Three or four years from now, she will be judged by whether she moved the agency forward in these areas. I think she has gotten off to a very good start, but there is immense amount of work still required. Read the rest of this entry »

Turning Data into Knowledge

June 2nd, 2009

Through statute and directive, FDA has been asked to collect, analyze, interpret and utilize massive amounts of data. This includes biological, clinical, adverse event, production and distribution data, medical and food product tracking, and the Sentinel system for early discovery of potential drug safety problems. The systems are not in place to do any of this, at least not at the required level of sophistication. Even if they were, sifting valuable information from background noise is extraordinarily hard. Read the rest of this entry »

About FDA Matters

FDA Matters has grown steadily since I started it less than a year ago. My inaugural column was about the goals of the blog, but I have not written on the topic since or about my background and viewpoint.

I started the blog because of my frustration about how FDA was being described and analyzed during the period from Election Day to Dr. Hamburg's confirmation. In particular, I spent a month telling colleagues: it is not true that there is going to be a power-sharing arrangement where Dr. Hamburg will concentrate on foods, while Dr. Sharfstein will concentrate on drugs and devices.

Even though I had no inside information, it was so clear to me…and yet many people thought otherwise and were impossible to persuade. I wished I had a platform to speak out, so I created one and launched it about 6 weeks later!

My goal is to write a blog that provides fresh insights and new perspectives for the broader community of people "involved in FDA matters and for whom FDA matters." Hopefully, it is achieving that purpose by focusing on what the agency is thinking and how its actions are shaped by Congress, the media, stakeholders and external events. FDA Matters aspires to be a source of understanding about FDA, both directly and by stimulating dialogue.

Since FDA Matters began, one continuing theme has been the need to plan for FDA's future. Another has been how the public health backgrounds of FDA's leadership team make their thought process and actions quite different from any of their predecessors. Some themes I want to explore this year: re-inventing the clinical trial, the roll-out of the new regulatory pathway for follow-on biologics and improving regulatory science.

FDA Matters is not a voice for any interest group. The blog reflects my own analysis and commentary based on 35 years working in DC on health policy and legislative and regulatory issues. Earlier in my career, I was Health Staff Director and Counsel to the Senate Committee on Labor and Human Resources (now the HELP Committee). I had the great fortune to be one of the negotiators on the Drug Price Competition and Patent Term Restoration Act (Hatch-Waxman) and on the Orphan Drug Act.

Subsequently, I was a Deputy Assistant Secretary for Health at HHS, responsible for policy development, planning and evaluation for the Public Health Service agencies. Since then, I have been a public affairs and regulatory consultant for a big firm, then started HPS Group, LLC in 2001 (www.hpsgroup.com).

One of my more recent accomplishments was helping to found the Alliance for a Stronger FDA (www.strengthenFDA.org). I serve (part-time) as the Deputy Executive Director of the organization. I believe strongly that FDA is dramatically underfunded and I write about this a lot. However, anything I write in FDA Matters is my own view and not that of the Alliance.

Over the years, my clients have included patient groups, health professions societies, research advocacy groups and individual companies. Many clients use me for legislative and regulatory analysis and to facilitate the development of policy and regulatory positions. Other clients use me for strategic regulatory counsel to help with development of medical products that are in phase II or phase III.

I welcome reader's comments, either posted on the blog or by e-mail. I would be pleased if readers helped me initiate two features of the blog: point/counterpoint exchanges and "Ask Steven About FDA."

Most of all: I believe that a better FDA is worth the effort to stay involved. I hope you see it that way, too.

Steven

Follow-on Biologics: 1-2-3-GO

The long fight is over for follow-on biologic (FOBs). The Senate-passed version of health reform will become law, even while the larger fight continues over the reconciliation package. Within 10 days, FDA will be busy implementing an approval pathway for FOBs.

The world of biopharmaceuticals will never be the same, but not in the ways that many players expect. Here is FDA Matters' guide to understanding the next phase.

Laws set the rules, but what happens next is remarkably dynamic and unpredictable. I was one of the Senate negotiators on the Patent Term Restoration and Drug Price Competition Act (Hatch-Waxman) and the Orphan Drug Act (ODA). What we thought would happen…and what actually happened…were not the same. Not necessarily better or worse. Just different.

We thought Hatch-Waxman would create an orderly world of patent extensions and generic approvals. We could not imagine the scandal in the generic drug office a few years later and would have been astounded that companies might still be litigating ground rules 25 years later.

The ODA was a triumph of good intentions, but would not have worked without the subsequent amendment redefining orphan drugs as affecting fewer than 200,000 Americans each year. We were not thinking about cancer patients. Yet they have been among those who have benefitted most by the ODA.

What will happen to FOBs will be just as dynamic and unpredictable.

The market was not waiting for the law to pass. Even though a legislatively-created FOB approval process was uncertain, Pfizer, Merck, Novartis, Teva and other major biopharmaceutical companies had already made decisions to be involved. Billions have already been spent or committed by companies before they knew the final FOB ground rules in the US.

More knowledge about the discovery, creation and manufacturing of biologics will be good for innovators, as much as imitators. Some of those biologically-similar products will themselves be innovative. As a result, many will require full approvals rather than being able to take advantage of an abbreviated FOB pathway.

Innovators will benefit from progress on characterizing biologic molecules, new testing methodologies and manufacturing improvements. To take a single example, the FOB market will force new investments in understanding immunogenicity that will benefit the entire industry, as well as patients.

FDA will be remarkably conservative for at least the next 5 years. FDA is ready for FOBs. Passage of the law gives the agency an important new public health mission: assure the safety and efficacy of biological products that will provide better access and greater affordability for life-saving and life-enhancing therapies.

Enthusiasm aside, the FDA is likely to be conservative in its policies and actions. They have not forgotten the problems in managing generic drug applications after Hatch-Waxman. They are fully aware of potentially big consequences in very small differences in biological products. They have lived through contaminated heparin and Genzyme's manufacturing problems.

FDA will want new safety and efficacy data from all applicants, with an emphasis on immunogenicity testing. Knowledge in these matters will increase rapidly and FDA will loosen up over time. But not soon…and in measured steps.

Steven

Here is an earlier column on the likely dynamics of the FOB marketplace.

The Follow-on Biologics Market

Since the debate began several years ago, the policy and politics of follow-on biologics (FOB) have been driven by assumptions and projections of the anticipated market. There has been a lot of fuzzy thinking about what type of companies will be players and how they will position themselves. Read the rest of this entry »

Fortuitous Timing and Public Health Leadership at FDA

At the end of March, Commissioner Hamburg will have been at FDA for 10 months and Dr. Sharfstein for a year. They are very good leaders who have also benefitted from their prior experiences and the timing of their appointments. Here is FDA Matters' analysis:

Public Health vs. Academic Medicine. Since at least the mid-1970's, FDA commissioners have come from academic health centers or government. Not one had run a public health agency at the federal, state or local level. In contrast, Dr. Hamburg spent 6 years leading the New York City Health Department. Dr. Sharfstein was recruited from his job heading the Baltimore City Health Department.

The difference is largely unappreciated. Running an academic health center is inwardly focused, with the goal of making a highly-complex institution run well. The primary outputs are patient care, research, and teaching.

Power in an academic health center is widely shared and interaction based on collegial ties. External forces, particularly the government political process, are often intermittent and remote and crises rarely come from outside the organization. Accountability is a virtue, but often not a requirement. Diplomacy and people management are the primary skills.

In contrast, a city public health agency is externally focused, with the goal of improving the health of a population. The primary outputs are programs of intervention and education, along with regulations. The structure in a public health department is hierarchical at every level.

The commissioner is integral to the political process and must be responsive to the Mayor and the City Council. External forces drive much of the activity. Crises are so frequent as to become routine and accountability is a necessity. Diplomacy and people management are valuable skills, but the premium is on concrete actions occurring in real-time, even if someone's feelings are bruised.

FDA is very much like a big-city health department, only minimally like an academic health center. This gives an enormous advantage to Drs. Hamburg and Sharfstein.

FDA in 2010: A Chance to Grow Stronger. In a hostile and uninterested environment, the very best leadership can survive, but not prosper. The new FDA leadership team is fortunate to have the best situation in 30 years for improving FDA.

FDA's budget has grown over the last three fiscal years, providing the new Commissioner with resources to strengthen the agency and prioritize initiatives. Over much of the prior two decades, the agency received annual increases close to, or below, inflation. There is not much new you can do when you are struggling to fund pay raises and rent increases.

Further, it matters when a Commissioner serves in the Presidential life cycle. As a rule, there is a lot of policymaking in the first few years of a Presidency and comparatively little in the later years. If policy improvements are to be made at FDA, 2010 is the time.

In addition, President Obama is alone among the post-war presidents in his interest in public health. Inadvertently, this was reinforced last year when he had to handle the peanut crisis himself because there were no confirmed appointees at DHHS. President Obama knows what FDA does and has a feeling for why it is important.

None of this is to take anything away from Drs. Hamburg and Sharfstein. They deserve credit for progress occurring at FDA. They are fortunate to have experience and timing that are likely to take them further than most of their predecessors.

Steven

FDA Needs at Least a $250 Million Increase in FY 2011

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?

Despite three good years of increases for FDA, we are still fighting decades of neglect. Appropriated staffing levels in 2010 are only back to where they were in 1994. Over a 25-year period, CDC has grown from an agency the size of FDA to one that is three times as large. Meantime, FDA has new responsibilities and an ever more-complex environment in which to function.

A 6% increase doesn't go very far. A tad more than $100 million will be taken up by increases in salaries, benefits and rents. Other costs go up also. On a FY 10 base of $2.35 billion, about $40 million is available for new programming in FY 11. It will probably go to hire more food inspectors, which is an important need.

This suggests that the rest of the agency (apart from food inspection) will be able to operate in FY 11 at their FY 10 staffing/program levels. In reality, this won't be the case unless the Congress provides FDA with an appropriation above the President's request.

Here is why the picture is actually quite grim. According to FDA budget documents, the increased cost of salaries in FY 11 is $66 million. As far as I can tell, only about $3 million is actually part of the President's request. So, what we consider as an increase to cover inflation…..actually does nothing of the sort. The President has told Congress: give FDA a $146 million increase in monies and it will provide a $143 million increase in programming.

So, food inspections, patient safety and advancing regulatory science  (the President's budgetary priorities) will happen….and parts of the rest of the agency will shrink (because they can't cover inflation) OR vital, existing services will continue, but FDA won't be able to deliver on the President's initiatives. It will take an extra $100M (an increase of $250 million total) to cover inflation in salary, rents, etc. and provide the new programming in the President's request.

To bring this down to specifics, the President has requested $25 million for advancing regulatory sciences. This is a priority of Commissioner Hamburg and much lauded by the stakeholder communities. At an IOM meeting last week on regulatory science, there was palpable excitement that the President had made a great first-year commitment. But follow the logic of this analysis and it is unclear whether there really will be monies to get started in FY 2011.

The bottomline: FDA needs more than the increase proposed in the President's request. The President, by his budget justification, agrees....and envisions an FDA that will require at least a $ 250 million increase.  

Steven

Some recent thoughts on the same topic, but with a different focus:

The President's Budget Request: Is 6% Enough for FDA?

February 4th, 2010

The President's FY 11 budget request for FDA includes a $146 million increase in appropriated (non-user fee) funding. This is about 6% of the $2.36 billion appropriation that FDA received in FY 10. With the President's tough talk about deficit reduction, anything above a freeze should be considered good. Why was the Alliance for a Stronger FDA disappointed? Read the rest of this entry »

FDA’s Growing Presence Outside of the Washington Beltway

The federal government is closed for the fourth straight day. However, it would be more accurate to say: closed in the DC area. A significant part of FDA is open and has been all week....making this a good time for FDA Matters to acknowledge and explore the growth of FDA in places far from the Washington Beltway.

Overseas Offices. FDA's commitment to creating overseas offices has been widely praised and deservedly so. The ultimate goal is to have FDA employees and offices in every area of the world where there are significant amounts of commerce in FDA-regulated products or their constituent ingredients. If food and drug problems can be identified and fixed near the beginning of the supply chain, then America becomes less dependent on successful inspections of cargoes entering the US.

FDA has created permanent offices in China (Beijing, Shanghai, and Guangzhou) and India (New Delhi and Mumbai) over the last two years. It has also upgraded and formalized its presence in Latin America (Costa Rica, Chile, and Mexico City) and Europe (Brussels and London).

Other areas, such as the Middle-East and Asia/Africa, are still overseen from FDA headquarters, but with an increasing local FDA presence in those areas. Likewise, headquarters handles various treaty arrangements (e.g. with Canada and Mexico) and relationships with international organizations.

Growth in Field Staff. Since 2006, FDA employees assigned to "field activities" has grown from 3450 to 4200. These individuals work for the Office of Regulatory Affairs (ORA) and are involved in inspections and enforcement. ORA personnel represent about 45% of the total FDA staff paid out of the agency's appropriations (non-user fee) budget.

According to FDA's website, over 85 percent of ORA's staff works in 5 Regional Offices, 20 District Offices, 13 Laboratories, and more than 150 Resident Posts and Border Stations.  This means more than 3500 FDA staffers in ORA are conducting government business away from headquarters.

National Center for Toxicological Research. NCTR is less well-know, but an important part of FDA. Its 210 employees are located in Jefferson, Arkansas, far from snowy DC. Its webpage was updated yesterday, so we know they are at work.

NCTR defines itself as conducting "FDA mission-critical, peer-reviewed, critical path (translational) research targeted to develop a scientifically sound basis for regulatory decisions and reduce risks associated with FDA-regulated products."

In plainer language, they are the applied sciences part of FDA, focused on toxicology and toxic effects of food, drugs, packaging materials and so on. As Commissioner Hamburg's campaign to promote "regulatory science" gains momentum, NCTR will be a key part of the effort.

In total, nearly 4,000 FDA staffers work outside the DC metropolitan area. This is a good thing, since the nation's food and medical products don't stop because Washington, DC had back-to-back blizzards.

Steven

For more about FDA's overseas offices, go to: http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm185769.htm

For more information about the Office of Regulatory Affairs, go to: http://www.fda.gov/AboutFDA/CentersOffices/ORA/default.htm

For more information about NCTR, go to: http://www.fda.gov/AboutFDA/CentersOffices/NCTR/WhatWeDo/default.htm

The President’s Budget Request: Is 6% Enough for FDA?

The President's FY 11 budget request for FDA includes a $146 million increase in appropriated (non-user fee) funding. This is about 6% of the $2.36 billion appropriation that FDA received in FY 10. With the President's tough talk about deficit reduction, anything above a freeze should be considered good. Why was the Alliance for a Stronger FDA disappointed?

Or to be more precise, the Alliance (www.StrengthenFDA.org) appreciated that FDA got more than most agencies, but felt that an inflation level increase was insufficient for FDA. The Alliance pointed out that the increase will not cover the ever-growing demands on the agency or strengthen the agency's capacity to fulfill its mission. They added: three good appropriations cycles for FDA (FY 08 to FY 10) have not undone more than a decade of budgetary neglect.

Of the $146 million increase for the agency's appropriated budget, CFSAN would get $60 million, about an 8% increase. This is a little more than an inflationary increase and foods programs will be able to grow marginally in FY 11. This is progress…though not nearly what is needed to meet the continued challenge of keeping foods safe and the increasing complexity and international scope of the food supply chain.

Far less was provided for medical products. Drugs and biologics received a combined $28 million increase in the President's request (about 4%). Devices received an $11 million increase (less than 4%), which does not cover the cost of pay increases. Other items that will increase this year (rent, IT, training, and travel) will need to come from existing program dollars. 

There is near-universal acceptance that the federal budget needs to be trimmed. Few domestic agencies did better in the President's request than FDA.

But there are differences in the needs of federal agencies. FDA's budget is almost entirely people costs. Salaries are more than 60% of the agency's budget. When you add in benefits, rent, IT services, travel, training, etc., 80% of the budget is devoted to people and the support they need to do their jobs.

If the FDA's appropriated budget does not grow by at least inflation each year, then staff levels decrease. This is what happened in most years from 1994 to 2007.

For example, after 9/11 the agency received significant new monies to hire inspectors to decrease the risks of agro- and bio-terror. Within 5 years, this increase in the FDA field force had disappeared, due to inflation, attrition and a lack of supportive funding. There is nothing hypothetical about the consequences of underfunding FDA.

Undoubtedly, FDA will find ways to be more efficient in FY 11, which will relieve a little of the pressure of doing more without new money. However, the amount to be gained in this manner is not large.

In contrast, there are plenty of areas for additional investment. FDA is operating a 1990's IT system, requiring it to work with databases that are incomplete and do not provide sophisticated support for decision making. Training the FDA workforce is another priority for additional funding.

FDA is still very much in the catch-up mode from past underfunding. Six percent is not enough!

Steven

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

Chart provided by the Alliance for a Stronger FDA:

President Obama's FY 11 Request for the FDA

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

Budget Authority Appropriations (does not include user fees)

Function Note: budget authority only, by center	FY 08 Actual (December 2007)	FY 09 Final (March 2009)	FY 10 Final (October 2009)		FY 11 Alliance Request	FY 11 President's Request
Food	$   510 million	$   649 million	$   784 million		$ 955 million	$ 848 million
Human Drugs	$   353 million	$   413 million	$    465 million		$ 580 million	$ 484 million
Biologics	$   155 million	$   183 million	$    206 million		$ 255 million	$ 215 million
Animal Drugs/Feed	$     97 million	$   116 million	$    135 million		$ 165 million	$ 141 million
Devices & Radiological Health	$   238 million	$   280 million	$    315 million		$ 385 million	$ 326 million
Natl. Ctr. For Toxicological Research	$    44 million	$    52 million	$    59 million		$ 72 million	$ 61 million
HQ, Office of Commissioner and Other	$    97 million	$   121 million	$    144 million		$ 183 million	$ 162 million
Rent & Facilities Cost	$  220 million	$   223 million	$    237 million		$ 250 million	$ 259 million
SUBTOTAL, Salaries and Expenses	$ 1.714 billion (+$145M over FY 07)	$ 2.039 billion (+$325M over FY 08)	$ 2.346 billion (+$307M over FY 09)		$ 2.845 billion	$2.496 billion
Building and Facilities Repair	$ 8 million	$ 16 million	$ 16 million		$ 12 million	$ 12 million
TOTAL, ALL Budget Authority Appropriations (no user fees)	$1.722 billion	$2.055 billion	$ 2.362 billion		$ 2.857 billion (Proposes + $495M over FY10)	$2.508 billion (Proposes $146 million over FY 10)

Because OMB includes new and proposed user fees in their totals, these numbers vary considerably from those being discussed by the Administration and reported by many sources. Notably, the President's request includes user fees being collected for the new tobacco center at FDA, monies that exactly match the cost of new responsibilities added this year.

A New Food Agency Has Become Unnecessary (For Now)

FDA Matters applauds the appointment of Mr. Michael Taylor to be the first Deputy Commissioner for Foods at FDA. With more authority, experience and stature than any previous food leader, he has the opportunity to shape and re-shape food regulation and the safety of the food supply.

Because Mr. Taylor will be outstanding in this new post, the campaign for a separate food agency will go away, at least for a couple of years.

People solve problems, reorganizations don't. Whatever problems exist within FDA would still exist within a separate food agency. Sometimes matters get even worse, as they did with the creation of the Department of Homeland Security.

Enter Mike Taylor, the person who can solve the problems. He has been an advisor on foods to the Commissioner for months and has been promoted to line manager and decisionmaker for every food activity within FDA. Most of us think only of the Center for Food Safety and Applied Nutrition (CFSAN), but there are food programs in the Center for Veterinary Medicine, Office of Regulatory Affairs and elsewhere at FDA. He will be responsible for all of them.

Mr. Taylor has all the elements for providing transformational leadership: experience, knowledge, respect, determination and foresight. By bringing leadership to foods, he will eliminate the urgency-- and probably the need-- for a separate food agency.

Intriguingly, Mike Taylor has been an advocate for a single food agency and there is some question about how that might affect his leadership. It won't. Some might think his appointment accelerates the move to a separate food agency. It doesn't.

Circumstances have changed. To continue to advocate for a separate food agency, Mr. Taylor would need to point to activities and policies that would be different and better if done outside FDA. It is hard to imagine what those would be--with him in charge and a supportive Commissioner. Or said another way: neglect, not the organizational structure, has always been the problem.

Things may look different in a couple of years. A new food safety law seems certain to be adopted in 2010. Implementing that law might create new reasons to support a separate food agency.

If so, an additional benefit of Mike Taylor's appointment would be continuity. His leadership and his structure would be carried over into a new organization. And he would have no trouble being confirmed by the Senate if that were required.

Which leaves one remaining question: will increased attention to food come at the expense of attention needed by the medical products function at FDA? I see no reason to think so. The broad, grassroots push of patient groups for safe and effective treatments has no counterpart on the food side.

Steven

FDA Leadership and Challenges: Seven Columns to Re-read for 2010

As a rule, FDA Matters does not cover the day-to-day events at FDA and in Congress. Most readers have multiple sources for news about the agency. I doubt I could do better.

Rather, the goal of this blog is to cover larger themes and provide deeper insights into the world of FDA. I place a premium on exploring FDA's future, as an observer, commentator and instigator. Here are seven columns about FDA leadership and challenges that will help you to better understand the agency in 2010.

Public Health Leadership Comes to FDA

FDA leadership–Dr. Hamburg and Dr. Sharfstein– come from an entirely different mold than their predecessors. They have begun an era of public health leadership at the agency. FDA staff and agency stakeholders will eventually come to appreciate that this difference is good for FDA. Read the rest of this entry »

Turning Data into Knowledge

Through statute and directive, FDA has been asked to collect, analyze, interpret and utilize massive amounts of data. The systems are not in place to do this, at least not at the required level of sophistication. Even if they were in place, sifting valuable information from background noise is extraordinarily hard. As a result, FDA needs to manage Congressional and public expectations as to "what is possible and when." Read the rest of this entry »

FDA and NIH: Natural Allies

Tension between CMS and FDA is a fact of life at HHS. This is not surprising because they have fundamentally different missions and world views. An analysis of the FDA-CMS relationship leads to an interesting conclusion: FDA should be doing a lot more with NIH because they have complementary missions and similar world views. They are natural allies. Read the rest of this entry »

In Praise of Predictability

FDA has always found it challenging to make its actions predictable. This problem will worsen while Dr. Hamburg redefines the agency's mission, policies, actions and working assumptions. Once this has been accomplished, the agency will become dramatically more predictable to stakeholders, including Congress. Read the rest of this entry »

To Whom Much is Given, Much is Expected

FDA has received $306 million (15%) more to spend in FY 10. This is the third good year for FDA, after years of bad ones. The agency is still severely underfunded, but progress is finally being made. Now the hard work begins: spending the new money wisely and showing that it has been used to accomplish important public health missions. Read the rest of this entry »

CARS: The Vehicle for FDA's Future

Commissioner Hamburg has spoken a number of times about the importance of regulatory science. She is right. FDA must have the scientific tools and methodologies to be a 21^st century regulatory agency. FDA needs to define regulatory science, develop programs to support it, and package them in a way that will quickly bring recognition and funding. Read the rest of this entry »

Long-term Challenges Need Short-term Attention

FDA Matters has identified seven long-term challenges for FDA. Some of these challenges may take years to accomplish; all need to be started now. Three or four years from now, Commissioner Hamburg will be judged by whether she moved the agency forward in these areas. Read the rest of this entry »

Steven

Scientific Reductionism and the End of Medicine

"For the last 400 years, science has advanced by reductionism ... The idea is that you could understand the world, all of nature, by examining smaller and smaller pieces of it. When assembled, the small pieces would explain the whole." (John Holland).

Have you ever heard someone accused of "reductionist thinking?" You probably will in 2010 because scientific reductionism is a critical, but rarely articulated, foundation of personalized medicine.

Reductionism is an attempt or tendency to explain a complex set of facts, entities, phenomena or structures by another, simpler set of constituent parts. Historically, scientific reductionism has held that all biology can be explained in terms of chemical reactions. In turn, these chemical reactions can be explained at the atomic level by physics.

An example of scientific reductionism is the belief that a blueprint for understanding and curing all disease will result from mapping genomes (human, bacteria, etc.). In effect, the complexity of biology ultimately yields to a much simpler paradigm based on de-coding the meaning of each component in the human genome and then delivering medical therapy personalized to the individual's genetic make-up. To oversimplify a little, biology then becomes a predictable "machine," subject only to additional reductions that yield smaller pieces and even more insight.

In contrast, many scientists believe that the complexity found in biology is more than just the inability of scientists to simplify the tangle of life and disease. No matter how much we know about genomic causation and associations, we will never have a full picture of life nor unlock the secrets to all diseases. These scientists believe that life is more than the sum of its parts. To them, reductionism is not wrong; it just produces an incomplete vision of biology because it cannot account for systems effects. A new field, systems biology, is trying to develop ways to understand the complex, irreducible biological qualities of life.

FDA Matters views scientific reductionism as a source of actionable knowledge. But just as the book, "The End of History," was more provocative than predictive, there is no "end of medicine" where human biology is reduced to the point of near-total knowledge and flawless cures.

Thus, personalized medicine will not defeat biological complexity. Further, the reductive process will incorporate knowledge from the human genome, but then take us past it into even more difficult and unpredictable challenges to understanding biology and curing disease.

Meantime, public policy is being shaped by the belief that biology and medicine will eventually yield answers that are concrete and totally reliable. But even when personalized medicine provides better targeted therapies, there will still be phase II and phase III clinical trials that inexplicably fail to show patient benefit. After approval, even the most well-documented and logical therapies may prove harmful and require modification or recall. FDA will need to constantly manage the expectations of Congress, the media and the general public to be sure that they understand that no amount of knowledge or evidence renders a medical therapy certain or riskless.

One prominent futurist has said: if we can just live long enough, progress in medicine will allow us to live forever. I say: not so.

The nature of medicine will be quite different 20 years from now, but unpredictability will still be common. As we develop vast amounts of new biological and medical information, old uncertainties about diseases and drug development will be resolved. New uncertainties will emerge.

Steven

Can FDA Withstand the FY 11 Budget-Cutters?

Tradition says nothing happens between Thanksgiving and New Year's Day.  People check out mentally and, as often as possible, physically. Not this December, not in Washington, DC.

Most attention is on the US Senate, which is working this weekend and remains under threat of "no days off" until healthcare reform legislation passes. Just as intensely, but much less visibly, the Executive Branch is working full-tilt to complete the President's Budget Request for FY 11 prior to the State of the Union speech in late January.

Of the two December activities, the President's budget is far more important to FDA.

The President's FY 11 budget request is likely to be deeply constrained. Having advocated successfully for a number of costly initiatives, President Obama will need to show that he can also reduce the deficit. He asked Cabinet secretaries to submit their budget requests at a no-growth level and an alternative budget on the assumption that their Department might be reduced by 5% overall.

With Congressional elections less than a year away, Congress will also feel pressure to dramatically reduce government spending. This will be particularly acute for Democrats, because the President's party usually loses a substantial number of seats in the mid-term elections.

Much of the government is likely to be flat-funded in the President's budget request. Even agencies and programs that advance Democratic policy priorities may not get increases.

There are three other reasons why FY 11 may be a rough year for FDA funding:

• a number of unfunded mandates (FOB, food safety) in upcoming legislation may consume FDA's budget increases ($300M is not an increase if the agency gets $400M in new responsibilities),
  
• FDA could get lost in the funding and implementation of health care reform, and
  
• increases over the last three years may be seen as sufficient to tide the agency over during a difficult budget year.
  

FDA's fate on FY 11 appropriations is not sealed. It will require many good arguments and hard work for the agency to get a meaningful increase in the year ahead.

The Alliance for a Stronger FDA will continue to lead the fight to get the agency more funds. Since the Alliance's founding three years ago, FDA's budget has increased by nearly $800M, a 50% increase. (FY 10 compared with FY 07).

The Alliance has 180 members, representing all FDA stakeholders: consumers, patients, associations, companies and individuals. They all agree that the agency needs to be better funded. Three former Secretaries of Health and Human Services and 6 former FDA commissioners support the Alliance's efforts and are honorary members.

If your organization, company, association, law firm or consulting firm are not members of the Alliance, please contact me at sgrossman@StrengthenFDA.org for details about joining. The Alliance also has individual memberships for those not associated with an organization.

The broader and deeper the Alliance membership, the more successful they can be. If FDA matters to you, please act now to help strengthen the agency through increased appropriations.

Steven

In the interests of disclosure, I am one of the founders of the Alliance and serve as its Deputy Executive Director. FDA Matters represents only my own views and is a product of my health policy and regulatory consulting firm, HPS Group, LLC. The Alliance and HPS Group are not affiliated in any way.

My last column on appropriations addressed FY 10's successful efforts…and examined one of the key long-term concerns: FDA's need to show that new monies have been well-spent in ways that support and promote the public heath:

To Whom Much is Given, Much is Expected
October 6th, 2009

There is good news to report now that House-Senate conferees have finalized work on FDA's FY 10 budget. FDA received $306 million (15%) more to spend this fiscal year. Every center will have more resources to work with (see table at the bottom of this article).

This is the third good year for FDA, after years of bad ones. The agency is still severely underfunded, but progress is finally being made. Now the hard work begins: spending the new money wisely and showing that it has been used to accomplish important public health missions. Read the rest of this entry »

CARS: The Vehicle for FDA’s Future

Since Labor Day, Commissioner Hamburg has spoken a number of times about the importance of regulatory science. She is right. FDA must have the scientific tools and methodologies to be a 21st century regulatory agency. FDA needs to define regulatory science, develop programs to support it, and package them in a way that will quickly bring recognition and funding.

This is a large task for Commissioner Hamburg to take on while running FDA and overseeing products that account for one-quarter of consumer spending. Yet, the agency will never improve if she doesn't find the time. Given increasing globalization, ever more complex science, and new Congressional mandates, the agency may even lose ground without important advances in regulatory sciences.

FDA is already involved in creating scientific tools and methodologies to support good decisions. The Critical Path Initiative (CPI) has been an important first step in creating a list of worthwhile projects to advance regulatory science. Many have been funded.

The National Center for Toxicological Research, a backwater of sorts at FDA, has been quietly supporting the scientific needs of the rest of FDA by developing new scientific tools, methodologies, and knowledge. Subtract field activities from the budget of the Center for Food Safety and Nutrition (CFSAN) and a large part of the Center's remaining budget is devoted to advancing regulatory science (e.g. creating faster assays to test for bacterial contamination).

The Center for Drug Evaluation and Research (CDER) also plays a role. Developing biomarkers is part of advancing regulatory science, as are new tools for surveillance and bioinformatics. The value is realized when these become part of tomorrow's regulatory decisions.

CPI was once considered the way to pull all of these activities together to advance regulatory science at FDA. It has not developed the vision or support necessary to accomplish this. Efforts to develop public-private funding for CPI activities have also been a distraction. We now know that Congress needs to fund these activities so the regulators lead regulatory science, not the regulated.

Several months ago, FDA Matters proposed the creation of the Center for the Advancement of Regulatory Sciences (CARS) at FDA. The Center was to be a defining enterprise—consolidating existing activities within FDA, providing a separate basis for advocacy and funding, and making clear that advances in regulatory science serve the future needs of the FDA Centers. I believe the CARS concept can be a starting point for discussing how advancement of regulatory science can become integrated into FDA's mission.

By talking about advancing regulatory science, Commissioner Hamburg is onto something important. We need to support her and help her develop broad acceptance of regulatory science among the policymakers who authorize programs and appropriate monies for FDA.

Steven

A definition of "regulatory science" and additional discussion are contained in my earlier columns:

Save the Critical Path—Part 1, June 17th, 2009

The American public and the global marketplace wish to have access to innovation—whether in medical products or foods. Simultaneously, there are strong countervailing concerns about product safety. Both occur within an environment in which FDA's knowledge and tools are inadequate and failing.

The Critical Path program and related initiatives in CFSAN and other centers are designed to meet this challenge. Unfortunately, there has never been a sustained agency-wide commitment to these efforts. Further, most of Congress has not embraced the Critical Path, either conceptually or with substantial funding. Read the rest of this entry »

Save the Critical Path—Part 2, June 28th, 2009

Transforming FDA into a first-class, 21st-century regulatory agency will not be easy. It requires planning, commitment and a broad vision. Science-based decisionmaking is a central part of the transformation, but it doesn't just happen by itself. Regulatory science needs to provide the tools, standards and knowledge for FDA to handle an ever-more complex world of science and commerce. Read the rest of this entry »

The Science Board’s IT Report: Too Technical to Read, Too Important to Ignore

Some of FDA's most difficult tasks are: defining the agency's role in nanotechnology, creating a pathway for follow-on biologics, implementing a risk-based food safety system, and establishing the right policy for "new media" communications.

All rolled together, they are not as complicated or important as transforming information technology (IT) at FDA.

The anecdotes are legion:

• adverse events reports come in electronically, are printed and re-keyed into a database;
  
• hardware and software vendors bring people out of retirement to service FDA's out-dated systems (so-called legacy systems);
  
• a report e-mailed to Capitol Hill for the next day arrives after the meeting.
  

I can only vouch for the last one, but the others are believable.

In August, the external FDA Science Board reviewed a report on information technology submitted by its subcommittee. FDA has put substantial resources and made valuable commitments to creating new systems. The biggest gains have been in infrastructure. Progress is being made in hardware, software, integration, capacity, security, infrastructure, and planning.

Clearly, the subcommittee liked what it saw. Their review also highlighted continued challenges in creating a 21^st century IT system capable of supporting all of FDA's mission-critical activities.

The report made some news in trade publications for a few days, then was quickly gone from public view. The issues are too technical to interest most people. Even with a glossary, I confess to not understanding at least half of the report.

Maybe the lack of visibility is alright if FDA and its contractors can absorb the many suggestions and recommendations in the report. FDA Matters recommends that an external review be undertaken every 3 or 4 months to be sure the effort remains on track.

While one can quibble about specific Congressional directives that depend on information technology systems, the main direction has been appropriate. FDA needs to fix the machines, create the databases, and integrate functions

This initiative is so important that non-IT people need to keep informed. Simply put, the agency's ability to conduct its work, increase effectiveness, and become more sophisticated is more contingent on the IT overhaul then the personnel that are being hired into the various centers to conduct the actual work of the agency.

In 5 years, nobody at FDA will be able to do their job if the IT overhaul isn't a success.

Steven

The FDA Science Board subcommittee report was completed in August and can be found on the FDA website at:

http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/ScienceBoardtotheFoodandDrugAdministration/UCM176824.pdf

In an earlier column, I addressed another facet of the agency's IT challenge:

Turning Data into Knowledge   June 2nd, 2009

Through statute and directive, FDA has been asked to collect, analyze, interpret and utilize massive amounts of data. This includes biological, clinical, adverse event, production and distribution data, medical and food product tracking, and the Sentinel system for early discovery of potential drug safety problems. The systems are not in place to do any of this, at least not at the required level of sophistication. Even if they were, sifting valuable information from background noise is extraordinarily hard. As a result, FDA needs to manage Congressional and public expectations as to "what is possible and when." Read the rest of this entry »

In Praise of Predictability

FDA has always found it challenging to make its actions predictable. This problem will worsen for a number of months while Dr. Hamburg redefines the agency's mission, policies, actions and working assumptions. Once this has been accomplished, the agency will become dramatically more predictable to stakeholders, including Congress.

I have written how Drs. Hamburg and Sharfstein's public health backgrounds make them different from their predecessor over the last 35 years--who were mostly researchers, teachers and clinicians from academic health centers. "Public health" as practiced in a big city health department has a gritty immediacy that shapes every activity. In contrast, academic health centers are devoted to providing clinical services to patients and educating students and house staff. Public health is a valuable by-product, but rarely a primary mission.

"Predictability" also means something different in city government than it does at a university. Public health departments are on the frontlines for all public health decisions--from the availability of flu vaccine to monitoring restaurants for sanitary conditions. Leadership must persevere in the chaotic environment of a big city. Every day brings unpredictable events generated by external forces. Success is achieved by having a tight organizational structure and "standard operating procedures" (SOPs) that cover nearly every potential challenge. In most situations, employees, stakeholders, mayors, and councilmen know what to expect. .

Unpredictable external events play a much smaller role in the life of an academic medical center. The combination of a medical school, a teaching hospital, and biomedical research labs is considered one of the most complex systems in our society. Leadership is more inwardly focused, trying to make the different components work together….and work well. Organizational predictability almost always take a backseat to people management, work flow and revenue generation.

My point is that Drs. Hamburg and Sharfstein have been schooled in the value of creating predictability. Generally speaking, their predecessors were not.

Predictability is in short supply at FDA currently--because the new leadership team is identifying the agency's internal and external problems and constructing appropriate solutions. The "new normal" that eventually emerges will be: more focused in the face of a broadening mission; more committed to serious enforcement; and more dedicated to innovation that is consistent with public health and science.

In the process, agency policies and actions will be increasingly based on SOPs, public and industry guidances, and clear articulation of scientific and legal positions. These will be implemented by a larger, tighter organization that will be more consistent in its decisions.

Although some of this evolution will be painful for FDA-regulated industries….they will eventually benefit from greater predictability and less ad hoc decisionmaking at FDA. And all stakeholders benefit from the increased public credibility that FDA will earn when its decisions are easier to understand, better grounded in science and public health and more predictable.

Steven

Public Health Leadership at FDA

Dr. Hamburg and Dr. Sharfstein have a style and approach that is already distinct from their predecessors. One of my earliest columns, two weeks before Dr. Hamburg's swearing in, explored why.

I predicted it would take 6 months for the patterns to be clear. We are halfway there, still evolving, and right on track. Do you agree?

Steven

From May 15, 2009.

The world will soon realize that the new FDA leadership–Dr. Hamburg and Dr. Sharfstein– are cut from an entirely different mold than their predecessors. When Dr. Hamburg is sworn in, she will formally begin an era of public health leadership at the agency. FDA staff and agency stakeholders will eventually come to appreciate that this difference is good for FDA.

It is a perfect time to put the agency in the hands of experienced public health leaders with real world experience. The shift will be both interesting and salutary. Notably, there will be a consistent standard in decisionmaking. The answer to every question and pressing issue will be: we will explore what is right from a public health perspective, and then act accordingly.

FDA staff and agency stakeholders argue for their position by saying they are advancing the public health, while secretly believing that other factors will drive the final decision. It is disarming, then, for the Commissioner to actually treat "public health benefit" as the agency's North Star.  Of course there will be many disputes, but everyone will have to build their rationale on public health grounds, knowing that it is the real basis of decisionmaking.

Several misunderstandings drive concerns about public health leadership at FDA. Public health is about helping people and communities to get healthy and stay healthy. Prevention is preferred because it preserves health, while therapies "only" restore health. Preferring prevention is not the same as being against therapy. Public health is not anti-therapeutic nor could any FDA commissioner be anti-therapeutic.

Public health does not require safety to be an absolute value that cannot be offset by other considerations. Innovation to restore health is just as much a public health value as safety. Dr. Hamburg has affirmed this.

What it means to run a big city health department has also been misunderstood. The imperative to act is immediate and real, but you learn that "what appears to be real" has to be examined before any decisions are made. Nothing you're told can be relied upon until it had been rechecked and sometimes double and triple re-checked. An over-simple example: reports about unsanitary conditions in a restaurant might just be from the eatery across the street that has lost business.

Dr. Hamburg and Dr. Sharfstein have limited track records on FDA issues. This uncertainty breeds anxiety. Six months from now, everyone will see that the agency is being run by steady, pragmatic leaders. Indeed, it is quite difficult to run the public health department of a large city without these virtues.