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Archive for the ‘FDA Accountability and Transparency’ Category

FDA’s Indifferent Attitude Towards the First Amendment

Monday, April 1st, 2013

The FDA doesn’t care about the First Amendment rights of the companies it regulates. It cares even less about the “free speech” rights of those companies’ sales and marketing representatives.

And why should the agency care? One of FDA’s primary missions is to protect the public health and safety of the American people from illegal, adulterated and misbranded products. Doing so involves restraining food, drug, device and cosmetics companies from committing fraudulent and deceptive acts that are not protected by companies’ commercial free speech rights.  

Nonetheless, FDA Matters envisions opportunities for FDA and industry to broaden permissible product communications. The key is understanding history, not constitutional law.


I recently participated in a forum at American University Washington College of Law on “Evolving First Amendment Protection of Commercial Speech” and offered up my mantra that constitutional analysis is largely irrelevant from FDA’s perspective. I said “largely” because FDA is still part of the federal government and can’t act arbitrarily. On the other hand, the agency mostly operates within the zone in which government is given the most leeway: where public health and safety is at stake and the threat is from commercial (as opposed to individual) speech.

History, not constitutional law, provides the best explanation. The sale of bad food and drugs—often accompanied by slick, deceptive pitches—goes back millennia and was even addressed as a problem in most ancient legal codes.

FDA’s own birth comes from a time when state regulation and inspection of food and drugs was minimal, inconsistent and often corrupt. It is hard for any of us to imagine what an unregulated market in food and drugs is like.  Yet, it is not so long ago.

The 1938 Amendments to the Food, Drug and Cosmetics Act gained popular support in part because of a traveling exhibit that portrayed the death and disability that resulted from patent medicines, counterfeit products, false medical and scientific claims, and adulterated and misbranded products. Lax to non-existent cosmetics standards were particularly singled out for their role in causing burns and blindness, as well as some deaths.

It is nice to imagine that this world is behind us, just an interesting piece of history. But it isn’t.

No industry regulated by FDA is immune from shoddy products, false claims, unscrupulous behavior and greed-induced threats to public health and safety.  I am sure that none of my readers count themselves among these “bad guys,” but they exist in the U.S., as well as globally.

Further, even the great and innovative companies—household names that we view with great trust—have often proven to be quite fallible. A certain amount of informal off-label promotion of drugs and devices is rightfully ignored–when good studies have been published, when the off-label indication is very close to an approved use, when assertions are made with great care about the extent of proven scientific knowledge.

In contrast, most of the off-label promotions that have resulted in billion dollar settlements with big-name drug companies have not been based on such close questions. Nor have they been the result of an individual salesperson crossing the line in some excess of enthusiasm.

Rather, the off-label promotions have been the product of marketing departments and sales managers who encouraged, empowered, or authorized the off-label promotion. There never seems to be a good answer as to who was supervising marketing and sales, which is why so many drug companies are now operating under government-negotiated corporate integrity agreements.

From FDA’s perspective, there are legitimate, well-documented reasons to scrutinize all companies: none are immune from the impulse to over-hype products to expand markets and sales beyond what FDA has approved. This is not a matter of FDA being over-fussy. It is the inevitable conclusion from about 4000 years of human commerce in food and drugs.

In the face of this, the First Amendment really has very little place. Commercial free speech does not extend to misleading statements, blatant fraud, or deception. FDA sees too much of this to ignore. 

FDA Matters believes the agency still has an obligation—but not a constitutional one–to clarify its standards, provide published guidance, and demonstrate acceptance that the Internet has fundamentally changed the nature of product promotion. The lack of FDA guidance on social media (first raised at an agency hearing 15 years ago) is particularly outrageous and the agency’s tendency to create de facto policy with enforcement letters is an abdication of responsibility.

The drug and device industry can also improve the situation. They need to stop looking at the current controversies in product promotion as noble causes involving sacred constitutional rights. If these industries have a claim to better treatment and clearer policies, it needs to be grounded in the contributions they make to improve public health and evidence of serious efforts to rid their companies of unscrupulous promotional practices.


I have written previously on some of the issues in drug and device promotion, as well as about opportunities for FDA and industry to reach accommodation:


Off-Label Promotion: Best Resolved by Congress, Not Courts     December 2012

On December 3, a federal appeals court ruled against one of the FDA’s untouchable restrictions on industry—thou shalt not promote the off-label use of pharmaceutical products. An industry that is little interested in constitutional law suddenly finds itself talking about the First Amendment. At stake: permitting off-label promotion undercuts the incentive for companies to thoroughly investigate the safety and efficacy of a drug for a second or third use.


Off-Label Uses Need to Become On-Label Indications  December 19, 2009

A friend asked: what advice would you give a pharmaceutical company in the late stages of developing a new product that will be widely used off-label? The company’s concern was that FDA might hold the first use to a very high, perhaps unrealistic standard to protect patients that might receive the drug off-label after approval.


Internet Communications: FDA Needs to Divide the Issues to Conquer the Problem  Dec. 2nd, 2009.

Creating an Internet communications policy for regulated medical product companies is so daunting that FDA has largely ignored the responsibility. November’s FDA hearing on social media was an important step, but offered no sign that new policy will be announced anytime soon. FDA needs a different approach. This is not a matter of a large, complicated problem with many facets. Rather, it is a number of smaller problems that can be addressed separately. 


Off-Label Promotion and Whistleblowing    September 9th, 2009

Whistleblowing and off-label promotion of drugs and devices have become hot topics because of the September 2 Pfizer settlement with the federal government. While none of my views are specific to Pfizer, the company’s settlement provides an opportunity to comment on off-label promotion….and to encourage bio-pharma and medical device companies to engage in deeper soul-searching. 

FDA’s E-Mail Scandal: First Impressions

Monday, July 23rd, 2012

According to the New York Times, FDA collected more than 75,000 employee e-mails in an effort to identify leaks of confidential trade secret information. At some point, a narrow, possibly legitimate inquiry into a handful of scientists at the Center for Devices and Radiological Health (CDRH) turned into a massive e-mail surveillance of selected individuals and their contacts.

So far, FDA is not contrite. FDA’s position, while still not quite official, appears to be: we tried to accommodate these individuals’ complaints within the personnel and dispute resolution systems. We had legitimate concerns that trade secrets were leaving FDA in their correspondence with third parties.

FDA Matters believes we don’t know the whole story yet.

The immediate perception is that the agency–in the name of protecting trade secrets–targeted internal critics and found ways to monitor their strategy and actions.

Fueling concerns is that a number of the monitored e-mails were sent to media, the President, and Members of Congress. Among others, Republican Senator Charles Grassley and Democratic Congressman Chris Van Hollen, have expressed concerns that correspondence with their staffs had been part of the surveillance.

Further, the surveillance appears to have included correspondence with the Office of Special Counsel, an independent federal investigative and prosecutorial agency with jurisdiction to oversee whistleblower complaints. Understandably, they are unhappy when they perceive that other federal agencies are interfering in their investigations.

In short—even with Congressional recess coming up and a national election—this issue is not likely to go away.

Every day FDA receives confidential trade secret information that it is legally obligated to protect. Any individual failing to do so is open to penalties—I assume both criminal and civil. To me, the key paragraph of the NY Times story is this one:

F.D.A. officials went to the inspector general at the Department of Health and Human Services to seek a criminal investigation into the possible leak, but they were turned down. The inspector general found that there was no evidence of a crime, noting that “matters of public safety” can legally be released to the news media.


Undeterred, agency officials began the electronic monitoring operation on their own.

If true, this is quite damning of the agency and provokes the usual question of why senior FDA officials did not exercise more restraint and better judgment.  

However, the agency’s unofficial semi-response suggests that this is not the full picture. At some point, the Office of General Counsel (OGC) at FDA became involved and authorized surveillance. Assuming this is true, did OGC have the authority to do so…and did they know that they were authorizing a broad surveillance? (I refuse to think of 75,000 e-mails as a narrow search).

According to an on-line Wall Street Journal article, the key individual in this case is a “serial whistleblower” (my term, not theirs), having filed lawsuits at two previous jobs. Supposedly, in both cases, the allegations of institutional misconduct were not proven in court, but he received settlements for “wrongful terminations” that followed his whistleblowing. What happened previously and elsewhere is irrelevant, except perhaps to remind us how hard it is for government agencies and public entities to fire an employee who they believe to be a disruptive force.

There is no way that FDA can look good if it is seen as approving devices that should not be on the market, squelching internal scientific disagreements, pursuing vendettas against its employees, or interfering with the prerogatives of Congress and the Office of Special Counsel.

In the face of all of this—the allegations and FDA unwillingness or inability to respond fully–it is hard not to worry about the agency. It is an institution that badly needs public and congressional support to do its job, especially when its responsibilities are growing and its budget isn’t.

FDA Matters hopes that Commissioner Hamburg and her senior staff are able to respond more fully and “on the record” in a way that helps stakeholders, Congress, the media and OSC understand why the extensive surveillance became necessary and what public purposes it served.


Here is more background, provided as a courtesy by BioCentury:  http://www.biocenturytv.com/freecontent/tbr_072312.pdf.

Some of the other relevant documents available only through subscription trade media:


1/ FDA’s response to Senator Grassley is available from Inside Health Policy (www.insidehealthpolicy.com, by subscription) at: FDA letter to Grassley.


2/ According to Dickinson’s FDA Webview (www.fdaweb.com, by subscription) Commissioner Hamburg on 7/17/12 sent the following email to all employees:


Over the past few days, a handful of stories, first appearing in The New York Times, were written regarding the agency’s monitoring of the computers of five CDRH employees.


I want to reinforce that the FDA’s greatest strength is our people. I value the expertise, professionalism and dedication each of you brings to this agency every day. There is no greater mission than that with which we are charged – protecting the public health. I have great confidence in FDA’s leadership and employees, and we have worked hard to foster a culture where differing opinions on scientific data, regulatory issues and product approvals may be expressed freely. In addition, employees have avenues available to them to voice their concerns both inside and outside the organization without disclosing proprietary information that is protected by law from unauthorized disclosures. I want to reiterate the FDA's commitment to protecting the rights of whistleblowers who are doing a service by bringing public safety concerns to the forefront. It is only in working together with the highest levels of professionalism and ethics that we will continue to achieve our mission.


Regarding the recent news stories, I want to stress that the FDA’s ability to fulfill our mission of protecting and promoting public health necessarily relies upon our ability to protect confidential information. Protecting such information in our possession allows us to work with industry and other stakeholders to ensure the quality of FDA-regulated products and the integrity of FDA-decision-making.


In 2010, the agency initiated monitoring limited to the government-owned computers of five CDRH employees. The impetus for the monitoring was a March 2010 New York Times article and a letter from GE Healthcare that indicated a pattern of unauthorized disclosures of confidential information related to pending medical device applications and submissions over the course of more than a year. The intent of the monitoring was to determine whether confidential commercial information had been inappropriately released and to stop any further unauthorized disclosures, since any such disclosures are a violation of the law.


Although the FDA, as with other federal agencies, has the discretion to conduct appropriate monitoring of government computers, we do so only in very limited circumstances. We do not take lightly the decision to monitor government computers.


I hope this note offers a bit more context to the situation. We will continue to update you as best we can. Please be assured that your work and your opinions are appreciated.

OMB, HHS, or FDA: Who Really Makes FDA’s Decisions?

Monday, April 23rd, 2012

OMB, White House staff, and the Secretary of HHS review many FDA decisions and their oversight sometimes alters FDA’s positions. This was chronicled in a recent NY Times article and generated a number of editorial comments criticizing anyone tampering with FDA’s integrity. However, the individuals named in the article have the authority (on behalf of the President) to question FDA’s judgments.  

More importantly, FDA Matters observes that the vast majority of FDA-related decisions–and virtually all of the science-related decisions–are made by the FDA. The key is the strong public health and scientific expertise of FDA staff and the credibility this brings to any scientifically-based agency decision.  

The President is in charge.  The President’s responsibility is to “faithfully execute the laws” of the United States. The task is enormous, requiring a $3 trillion annual budget and 1.3 million civilian employees*.  Delegation is necessary and is controlled by having a rigidly hierarchical structure of government that assures, to the extent possible, that decisions made by subordinate departments and agencies reflect the law and the President’s policies.

Department secretaries—those primarily responsible for carrying out the President’s policies—report directly as part of the President’s Cabinet. They also report indirectly through the Office of Management and Budget (OMB), which is the primary administrative structure for assuring that the Executive Branch makes decisions consistent with the President’s wishes. 

FDA is part of the government, not separate from it. The very nature of government makes FDA’s independence an illusion**. The Commissioner is not elected; she is appointed by the President and the Secretary of Health and Human Services. Ultimately, all decisions are the President’s (and the Secretary of HHS’ acting on his behalf).

While oversight and review of FDA’s decisions by White House staff, OMB and the Secretary of HHS is quite real, it is also legitimate. Those named in the NY Times article all have the authority to question FDA’s judgments before they become final.  The positions they took may have been unwise, but they did not exceed their responsibilities.

Oversight and review of FDA is limited in its scope and impact. While FDA cannot escape oversight within the Executive Branch, the NY Times article cited only five examples among thousands of decisions FDA makes every year.

None of the five cases involved overriding the scientific and medical expertise of FDA. Two of the five were labeling issues (caloric content of movie popcorn, sunscreens with relatively low SPF factors). The other three were access issues (emergency hormonal contraceptives, the continued marketing of an asthma inhaler containing fluorocarbons, and whether FDA should waive enforcement against pharmacy compounding of a specific, newly-approved drug).

The most serious of the five was the decision on access to emergency hormonal contraceptives, where it is alleged that HHS overrode FDA’s scientific judgment. Without defending HHS’s actions, it is relevant that neither biological nor medical science was involved. Rather the FDA “science” involved label comprehension studies (can adolescents under-17 understand and properly follow the directions on the label).  As an aside: decisions concerning emergency hormonal contraceptives have an almost-unique history of getting FDA leadership in hot water within the Executive Branch and with Congress and the public. 

FDA’s medical and scientific expertise protects virtually all of its medical and scientific decisions. Since OMB and HHS oversight of FDA is continuous, there are undoubtedly other examples where FDA has compromised or yielded. Given FDA's public accountability for its decisions (correspondence, hearings, reports, advisory committees), there can't be many instances in which the agency's scientific or medical judgments are overruled and it is not publicly known. So, I can’t say that FDA’s scientific judgments are never overruled, but it certainly appears to be rare.  

The alleged problem of intrusions on FDA’s scientific integrity may appear larger because of a misunderstanding about the nature of FDA. Not all FDA decisions are based on scientific or medical expertise. For example, requiring caloric labeling of movie popcorn might create consistency of government policy across multiple food service settings, but it's a policy judgment, not a decision based on science or medicine.

In fact, there is very good reason why White House staff, OMB and HHS will never significantly affect FDA scientific and medical decisions: they lack the credibility and scientifically-trained manpower to do it.


* Federal government expenditures and number of civilian employees are for 2011. Civilian employees are expressed as full-time equivalents and exclude US postal workers. Source: http://www.whitehouse.gov/omb/budget/Historicals

** Proposals have been made to  re-create FDA as an “independent agency”  that reports directly to the President and not through a Cabinet-level department, much like the U.S. Environmental Protection Agency. However, EPA’s experience is fairly clear: reporting directly to the President and OMB….does not free you from having your decisions questioned and sometimes overruled.

The State of the FDA—January 2012

Tuesday, January 24th, 2012

FDA is the only federal agency that touches the lives of every American several times every day. Its remarkably broad mandate includes all medical products and 80% of the nation’s food supply, plus countless other products. Despite this, when the President delivers his State of the Union (SOTU) address to Congress this week, it is unlikely that FDA will rate a mention.

FDA Matters will instead provide its second annual “State of the FDA.” As reflected in last week’s column, FDA did well in 2011, but one year’s progress does not change the continued precarious state of the FDA. Here is our analysis:

Strengths: FDA’s most important strength is the dedication of the agency’s staff  and the leadership of Commissioner Hamburg’s team. In the current environment, their efforts are invisible to the public they serve and largely unappreciated. If safe foods and safe and effective medications are important to you, say “thank you” to the FDA staff that make it possible.

The agency’s independence is another key strength of the FDA. At the moment, many FDA observers don’t see it that way, viewing the HHS Secretary’s decision to overrule FDA on Plan B as evidence that the agency is weak and dependent. However, Plan B is a ‘one-of-a –kind” controversy, presenting uniquely difficult and combustible issues that aren’t present in 99.9 percent of FDA’s decisions. If you look at the totality of FDA actions, the agency is remarkably independent from HHS and the White House. Rather than a weakness, this is one of the agency’s strengths.

Weaknesses: Despite a number of recent, laudable efforts at improvement, the FDA is still disorganized and largely ineffective in communicating its messages to the public, media, stakeholders and Congress. Notably, an analysis published in the journal, Medical Care, last week concluded that: although some [FDA] communication efforts had a strong and immediate effect, many had little or no impact on drug use or health behaviors and several had unintended consequences.

FDA’s information technology (IT) systems continue to be grossly inadequate for an agency with such large, far-flung and complex responsibilities. Some progress has been made with analytic data bases, such as the Sentinel program to track post-market safety, and with data bases that improve the flow of information within the agency and between field and headquarters. The October 2011 appointment of a new chief information officer with industry experience is a hopeful sign.

Opportunities: The promise of science has never been brighter. And Dr. Hamburg, to her credit, has made it a priority to improve the agency’s scientific bench strength—better credentials, better training and better tools.

This provides FDA and the medical products industries with the opportunity to forge a new “social contract” with regard to scientific standards and product approvals. FDA must commit to becoming less formalistic and bureaucratic in its dealings with companies. It must demonstrate (not just accept) that advancing medical innovation is an integral part of the FDA’s role in promoting public health. In turn, industry needs to accept that “science, fairly evaluated within predictable guidelines,” is an appropriate expectation as opposed to a system based on short-cuts to market and ill-defined, “leap of faith” assumptions about safety and efficacy. In addition, industry bashing of FDA needs to end. It is counterproductive to everyone’s interests.

Equally promising is the opportunity to significantly upgrade the safety of the American food supply. Even with the devotion of FDA staff to this cause, we are lucky that the reported levels of foodborne disease and product adulteration are not higher. The year-old Food Safety Modernization Act (FSMA) is, by general agreement, a blueprint for moving to a new level, one where a safer food supply reflects smart decisions.

Threats: The largest threat to FDA is inadequate funding. As science has become more complex, industry more global and information more integral to every human interaction, FDA oversees a rapidly expanding portfolio of products and responsibilities. Even without the threat of budget cuts facing all federal agencies, it would be hard to grow the FDA budget enough to stay ahead.

A related threat is the potential for massive expansion of FDA’s unfunded mandates during Congressional reauthorization of the drug and medical device user fee programs. FDA is almost certain to be given new (and needed) authority for drug import inspections and drug shortages. In addition, Congress will consider and most likely pass a dozen or more other new programs or significant changes in FDA regulation. FDA will almost certainly have to implement these new requirements without additional appropriations. 


For those who may be interested, here is a link to “The State of the FDA—January 2011” http://www.fdamatters.com/?p=1240.

Understanding FDA—Its Strength Is Its People

Monday, December 26th, 2011

Science is essential and rules and regulations must be followed…but ultimately it is the people of FDA who determine whether the agency functions well and acts in the best interests of the American people.  For that reason, a recurrent theme in FDA Matters is the importance of FDA employees. Their jobs are much more difficult than most of us imagine.

Consider the oft-expressed paradigm: FDA is committed to science-based decisionmaking. Yet, science doesn’t exist in a pure, understandable, easily accessible and unassailable form. Neither do laws and regulations. While law and science are fundamental to FDA decisions, only people can actually make the decisions.

During 2011, FDA Matters explicitly looked at the role of staff three times.

The first column asked: Will the Real FDA Please Stand Up?  It was in response to a widely circulated e-mail appealing for public support to prevent the firing of “courageous, honest FDA scientists who risked their careers to save lives by informing Congress about serious safety concerns involving dangerous, FDA-approved drugs and medical devices.”

Whistle-blowing is sometimes necessary. However, the image of a corrupt, politicized FDA cynically suppressing dissent and putting the American people at risk is inconsistent with the FDA that I have known and followed for over 30 years. Yet, I acknowledged that the claims made in the e-mail are persistent and come from many sources.

Exploring this in the column, I concluded that FDA is making progress in handling dissent and in encouraging managers to be more open-minded. While FDA makes mistakes, there is no conspiracy. The agency is fully committed to serving the American people. 

My second column, People, Not Science, Make Decisions, looked at why decisions take so long, especially product approvals. When asked about bottlenecks at FDA, I had to admit that people slow the process down. I pointed to three factors that cause delays:

  • FDA lacks sufficient resources.  FDA has been dramatically understaffed for decades, although better funding in recent years has improved the situation. Even still, new demands on FDA are increasing faster than staff can be added.
  • FDA’s culture promotes intensive scientific discussion.  Objective science-based decisionmaking is a goal, not a reality. People arrive at decisions with a bundle of perceptions and experiences that render their judgment subjective. Yet, they wouldn’t have much to contribute without those experiences.
  • Science has become more complex and results often unpredictable. Life sciences’ products are increasingly based on cutting edge discoveries. It takes more time, more thought and more knowledge on FDA’s part to make a good decision about them. 

Finally, I devoted a third column, FDA and Things that Might Go Bump in the Night, to exploring what happens when FDA staff has to decide the fate of breakthrough technology that could bring great benefit or great sorrow to humankind. In the current context, I had nanotechnology, genetically-engineered (GE) animals, and synthetic biology in mind.

Thirty years ago, FDA gave a cautious “yes” to the first biotechnology studies that eventually became important medical products to treat cancer, arthritis, MS and many other diseases. The world is a better place as a result.

In retrospect, the decision was right, but at the time, this wasn’t clear. Scary visions of mad scientists and technology run amok were powerful forces in the early days of biotechnology. In response, FDA staff took great care in setting up an appropriate regulatory environment.

Conclusion: Based on my experiences working with FDA and as expressed in these three columns, I believe that FDA staff–the people contributing to and making the decisions at FDA–are its strength. While they are human and make mistakes, FDA’s employees are smart, conscientious and committed.   


Beyond Plan B: Scientific Integrity and a Possible Third Class of Drugs

Monday, December 12th, 2011

Patient access to the emergency hormonal contraceptive “Plan B One-Step” has been one of the most combustible issues ever faced by FDA. It received more attention last week when FDA approved expanded access for adolescents under 17 and HHS Secretary Katherine Sebelius promptly overruled the agency because she found inadequate scientific support for the decision. (For my readers outside the US, here is a New York Times article that provides background).

FDA Matters wonders: has FDA’s scientific decisionmaking authority been thwarted in some lasting way, as some have claimed? Is it time to reconsider proposals for a new category of drugs for which pharmacists are the gatekeeper?

Under the Federal Food, Drug and Cosmetic Act and other laws, the Secretary of HHS has the legal authority to make virtually all decisions within the Department’s jurisdiction. Most of this authority has been delegated to subordinates, including the FDA Commissioner, although the delegation can be withdrawn at any time. No one remembers an instance in which the Secretary explicitly overruled FDA.

Was there a lapse in communications between FDA and HHS? Normally, the Secretary and the Commissioner (or their staffs) discuss controversial decisions before they are made and a compromise reached. Had this occurred, FDA would have announced that compromise as the agency’s decision and taken the heat for ignoring its advisors and staff.  

The alternative explanation, which I hope is true, is that there was an understanding that FDA’s voice should be heard and its integrity preserved, while the Secretary would take the heat for the decision by overruling the agency. This would be consistent with an approach that Commissioner Hamburg has championed: that government should be more honest and transparent about disagreements, as long as everyone understands that someone with decisionmaking authority will actually make a decision.

Either way, it is important to recognize that Plan B has proven to be a “one-of-a-kind” controversy, presenting uniquely difficult issues that aren’t present in 99.9% of FDA’s decisions. I see no reason for commentators to be writing, as one did:  “FDA’s medical and scientific integrity has been forever blighted by these frankly political decisions.”

Secretary Sebelius is not going to make a habit of questioning the scientific support for FDA’s decisions. Nor have we any reason to fear the death of FDA’s integrity or to conclude that it can no longer be a scientifically-driven regulatory agency.

Leaving aside the merits of either FDA or HHS’s position, the Plan B controversy provides an impetus to consider whether there should be a third class of drugs that are neither prescription-only (Rx) nor over-the-counter (OTC). The day after the HHS decision, John Jenkins, director of FDA’s Office of New Drugs, suggested just such a connection between Plan B and a potential third class of drugs known as “behind the counter” drugs (BTC).   

BTC, which has been discussed for decades, relies upon pharmacists to dispense these drugs without a prescription and independent of a physician-office visit. Pharmacists are the most widely distributed of all health care professionals, as well as the most readily accessible. BTC would empower them to provide additional education, monitor appropriate use and discourage misuse for a wide variety of drugs. 

Currently, OTC products containing pseudoephedrine are handled as BTC because of their potential use in making methamphetamine. According to Dr. Jenkins, statins are potential candidates for BTC status, as might other drug classes where petitions for Rx to OTC switches have been denied but there is still potential for safe patient self-treatment without a physician office visit.

In sum, accessibility to the emergency contraceptive Plan B continues to be a disruptive and controversial issue for FDA. However, this is a unique issue and there should be no continuing damage to FDA because the agency was overruled by HHS. A third class of drugs could evolve out of this that might be beneficial to patients across a number of diseases and drug classes, including hormonal contraceptives.



In Praise of Predictability: A Virtue Revisited

Monday, October 24th, 2011

More than two years ago, FDA Matters suggested that one of FDA Commissioner Hamburg’s goals would be making it easier for stakeholders to anticipate agency actions. I was looking beyond transparency (see what FDA is doing and has done). I hoped the agency would focus on the larger virtue of predictability (being able to anticipate FDA’s standards and actions).

The task has proven larger than I imagined and the volume of issues facing the agency has made progress hard. However, the agency has taken many steps forward, even as it has been pushed backward on a number of occasions. Is the glass half empty or half full?

I believed that, after the initial stage of setting new policies and assembling her team, one of Dr. Hamburg’s priorities would be to provide more guidance (published and spoken), explain more actions, and rationalize and coordinate more regulatory processes. With regard to this goal of predictable, science-based regulation, progress has definitely been made.

Once completed and enacted, the 2012 round of user fee legislation will further contribute a degree of predictability. The five-year renewal cycle allows industry and FDA to re-work ground rules for the product review process (note: these negotiations are intended to change process, not standards).  

So why doesn’t it feel like FDA is making more progress in becoming predictable and science-based in its regulatory decisionsmaking?

First, a degree of uncertainty is inherent in the nature of FDA’s work. I’ve explored this theme in two recent columns. One discussed the role of FDA staff and the necessity that People, Not Science, Make Decisions. The other examined the biological complexity that makes it impossible to provide guidance that anticipates all situations and outcomes (FDA Approvals and the Failure of the “Human Body as Machine” Metaphor).

Second, certain well-publicized areas of conflict between FDA and industry have tended to exaggerate the overall degree of unpredictability. For example, FDA faced a decision last year on three weight-loss drugs—and approved none, despite credible claims that the drugs met FDA standards.  The agency’s reasons satisfied some observers, but not others. This particular situation became something of a poster-child for critics who say that FDA is unpredictable in its decisionmaking.

The ongoing struggle between FDA and the medical device industry has also focused attention on claims that FDA’s regulatory decisions are not predictable. I have chronicled the often-heated debate in a number of columns, most recently Medical Device Melodrama: A Great Story With a New Plot Twist.

Medical devices were a conflict waiting to happen. It is not because FDA is disinterested in improving predictability in this area. Rather, the approval process is uniquely adapted to the nature of medical devices (wide range of risks, short innovation cycle for product improvements) and almost impossible to explain to someone not already familiar with medical device regulation. Ironically, the two years spent (thus far) on improving the medical device process began with FDA unexpectedly approving a device based on irregular agency procedures.  

Increasing predictability and science-based decisionmaking are still hallmarks of Dr. Hamburg’s agenda. A recent example is FDA’s new initiative to spur biomedical innovation and improve the health of Americans. Streamlining and reforming FDA regulation is one of the initiative’s goals. 

If FDA has not become more predictable in your area of interest, keep encouraging FDA to make it so. The agency is listening and making progress. They need feedback on where processes have broken down or do not produce predictable decisionmaking. In the quest to achieve this goal, I believe the glass is half-full.


People, Not Science, Make Decisions

Monday, August 8th, 2011


People constantly make decisions. We choose vanilla instead of chocolate or a job in DC instead of a job in Texas. Can’t decide? Then, maybe, flip a coin.


Yet, the most important decisions—such as FDA approvals–can’t be treated so cavalierly. While scientific evidence and good judgment are necessary to make these choices, people make the decisions.


To FDA Matters, the people making the decisions at FDA are its strength. They are smart, conscientious and committed. Yet, when asked about bottlenecks at FDA, I have to admit that people slow the process down. There are good reasons why this is so.  


FDA lacks sufficient resources.  FDA has been dramatically understaffed for decades. Better funding in recent years has improved the situation, but not nearly enough. Also, it seems like new demands on FDA are increasing faster than staff can be added.


Many FDA employees have overwhelming workloads and inadequate time to refresh their scientific knowledge or broaden their thinking. As a result, higher levels of evidence and “certainty” become necessary to reach decisions. Getting to “yes,” particularly for product approvals, often consumes a great deal more time than telling a sponsor “no” or asking for more information. Hiring more staff would reduce or at least stabilize workloads*.


FDA’s culture promotes intensive scientific discussion.  Objective science-based decisionmaking is a goal, not a reality. People arrive at decisions with a bundle of perceptions and experiences that render their judgment subjective. On the flip side, they probably wouldn’t have much to contribute to the decisionmaking process without those experiences.


This is true for FDA, where agency staff—all with subjective views–interact and struggle to come to a clear decision.  If six or seven well-trained, highly-disciplined FDA scientists examine a pre-clinical or clinical trial, there are certain to be multiple views about the meaning of the data It is a slow path to a consensus, if one can be forged at all.   


Delay can increase further if the agency—as it has in the past—expects a single conclusion to emerge from such staff engagement. One of Commissioner Hamburg notable efforts has been to manage dissent (and let it be expressed in public), rather than always force consensus decision-making.


Science has become more complex and results often unpredictable. Life sciences’ products are increasingly based on cutting edge discoveries. It takes more time, more thought and more knowledge on FDA’s part to make a good decision about them. No sponsor feels good when FDA doesn’t know the science behind their product. Delays are inevitable while FDA catches up.


Medical and scientific information is rarely simple. Sometimes a protocol has a sound hypothesis, great supporting science, and logical inferences from similar studies, etc. It feels like running the clinical trials is merely a formality. …and yet the product ultimately turns out to have little efficacy or unexpected safety problems. Every FDA reviewer knows: assumptions based on early data can often be wrong. Biological complexity can, indeed, lead to surprises.


Conclusion. There is much that can be done to improve the FDA regulatory pathways, particularly for approvals, even though bottlenecks are inevitable.


FDA staff makes the decisions, which is how it should be. As individuals, their decisions cannot be isolated from workload, culture or complexity. Faster is possible, but it is important that people slow the process enough to be sure that decisions are both scientific and sound.




*   Adding new staff eventually helps. However, it can take upwards of two years for new hires to be trained, integrated, knowledgeable and experienced enough to lessen the workloads of others.


Complexity, Uncertainty, Unpredictability: Not Necessarily Bars to FDA Approvals        July 17th, 2011

In most discussions of science and medicine, there is an implicit assumption that the human body is a machine—complex and biological, but still a machine. If we could only understand all the mechanisms, processes and parts of that machine, then we could prevent and cure disease. Yet, the further we travel into the biology of life, the more complexity we find and the less certainty and predictability.


“The human body as a machine” is a metaphor, not a fact. Once we accept this, FDA Matters believes we can become liberated from unrealistic expectations about medical discovery and FDA’s role as a gatekeeper for new products that benefit patients.  Read the rest of this entry


The State of the FDA—January 2011      January 16th, 2011

FDA’s touches the lives of every American at least 6 to 10 times each day. The agency oversees 80% of the nation’s food supply, all of human/animal medical products and cosmetics, and almost all radiation-emitting devices. Altogether, the agency is responsible for about 20% of all consumer dollars spent in the United States.


With the President set to deliver his State of the Union address to Congress in 10 days, it seemed a good time for FDA Matters to provide its view of the “State of the FDA.” At the beginning of 2011, the agency is doing well, but has a lot of catching-up to do and faces a number of threats. Read the rest of this entry

FDA, Reorganization and the Four Crises

Sunday, July 24th, 2011


In previous posts, FDA Matters has expressed its disdain for efforts to solve problems by reorganizing government agencies. So, it may be surprising that I am giving Commissioner Hamburg an “A” for her recent reorganization of FDA’s senior management.


In the reorganization, she is addressing four crises that beset the agency: industry discontent with the medical product review process; public concern about import safety; implementation of the Food Safety Modernization Act; and Congressional concerns that the agency is inefficient in its use of resources. The new structure should drive better decisonmaking and greater productivity…. at a time when the agency is struggling to fulfill its growing mission and faces the potential for budget cuts.  


Until the beginning of this year, Dr. Hamburg has relied upon Dr. Joshua Sharfstein, a trusted deputy and alter-ego, to assist in managing the agency. In addition, he had served as a buffer between the agency and external forces, particularly Congress.  Instead of replacing Dr. Sharfstein, the Commissioner has chosen a new organizational approach that responds to the agency’s most difficult challenges:


Crisis #1: Growing discontent among the medical product industries. They allege that unreasonable FDA requirements and the agency’s overly-cautious approach to potential safety issues are keeping innovative and effective products from becoming available to patients. For more details, see my columns at:  http://www.fdamatters.com/?p=1428 and http://www.fdamatters.com/?p=1401.


Response: Create a new Deputy Commissioner for Medical Products and Tobacco and appoint Stephen P. Spielberg, MD., PhD, a distinguished physician and researcher and former dean of Dartmouth Medical School. He also spent 11 years in industry, working for Merck and then J&J.


In his new role, Dr. Spielberg will oversee the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research, the Center for Devices and Radiological Health and the Center for Tobacco Products.

The intent is for him to serve as a “senior partner” to the four center directors, facilitating decisions that might otherwise wait for the Commissioner. When appropriate, he would also stand-in for the Commissioner on their behalf. Right now, these center directors can’t be getting very much of Dr. Hamburg’s time and they are quite vulnerable on the Congressional side.


In addition to the stature he brings, Dr. Spielberg’s bio describes his research interests as: mechanisms of idiosyncratic adverse drug reactions, human pharmacogenetics and personalized medicine, and pediatric clinical pharmacology. This background is germane to the areas of industry concern about pre- and post-market review of medical products and also positions him to be one of the agency’s chief advocates for improvements in regulatory science.

Crisis # 2: The safety and quality of imported food and medical products.  The American people and Congress want safe products and expect FDA to use its very limited resources to make it so. For more details, see my column at: http://www.fdamatters.com/?p=1408.


Response: Create a new Deputy Commissioner for Global Regulatory Operations and Policy and appoint Deborah Autor, now Director of CDER’s Office of Compliance. Her “directorate” will oversee the Office of Regulatory Affairs (ORA) and the Office of International Programs.


The intent is to increase coordination and greatly reduce the number of decisions that would otherwise wait for the Commissioner’s availability. Just as importantly, the new structure brings together FDA’s overseas relationship and capacity-building successes with a tougher, more regulatory posture to assure that imports meet the same standards for safety and quality as domestic goods.


Crisis #3: Implementation of the new Food Safety Modernization Act to create a sophisticated risk-based food safety system. This is a complex multi-faceted task being made more difficult by inadequate funding.


Response: Continue the existing position of Deputy Commissioner for Foods, which oversees the Center for Food Safety and Applied Nutrition and the Center for Veterinary Medicine. Michael Taylor, who will continue in this post, has already demonstrated the advantages of Dr. Hamburg’s new organizational approach….by his general leadership and his representation of the agency with Congress on food issues.


Crisis #4: FDA’s ability to sustain and grow its FY 2011 funding level is being challenged in the Congressional appropriations process.


Response: Create a new Office of Operations, headed by a Chief Operating Officer (COO) to oversee human resources, facilities, information technology and finance. This will strengthen the agency’s ability to respond to Congress on administrative matters and, in particular, assure Congress that the agency is under tight fiscal management.


Conclusion: FDA has many problems, some of which are reaching crisis-proportion. While more resources are necessary, good leadership is essential.


Regardless of the demands, Commissioner Hamburg can never have more than 24 hours each day to address the agency’s needs. The new organizational arrangement—with four deputy commissioners providing span of control over most of the agency–seems well-suited to address this limitation.




Here is a link to read the Commissioner’s message to agency employees conveying the new organizational structure:  http://carl1anderson.wordpress.com/2011/07/14/major-reorganization-at-fda/.


I would hope in the future that FDA would post these types of communications directly onto the agency website, rather than relying on the Commissioner’s messages to be reprinted in newsletters and blogs.

Will the Real FDA Please Stand Up?

Sunday, January 23rd, 2011

This e-mail grabbed my attention this week:

TAKE ACTION: FDA Whistleblowers being Fired – HELP THEM.

[Organization] has received an appeal for help to prevent the firing of courageous, honest FDA scientists who risked their careers to save lives by informing Congress about serious safety concerns involving dangerous, FDA-approved drugs and medical devices.

FDA employees should not be afraid to speak honestly and freely about misconduct that threatens the health and safety of all Americans.

This isn’t the FDA that FDA Matters has known and followed for 30 years. Yet claims like the one in this e-mail are persistent and come from many sources.

Many FDA critics—inside and outside the agency—believe the FDA is corrupt, industry-beholden, and arbitrary in its decisions. They allege that the agency is insufficiently concerned about safety and that managers have too much power to overrule staff and suppress dissent.

FDA Matters has itself raised questions about workplace problems at FDA. There are links below to columns on "Dissent and Efficiency: Difficult Trade-offs for FDA," "Why Do Some People Dislike the FDA?" and "FDA: A Hit and a Miss" (about Avandia). Nobody would take the position that all 12,000 FDA employees are happy, fulfilled or satisfied with their work.

Nonetheless, I have always found FDA employees to be committed to the American people and dedicated to the public health needs of patients and consumers.

After spending four years as a government manager in the 1980’s and over a decade as a manager in the private sector, I believe FDA is like most companies or organizations: encompassing a broad range of competency and commitment and having its fair share of job dissatisfaction. From my government experience, I also remember how hard it can be to re-assign government workers who lack commitment or are incompetent or disruptive.

So, would the real FDA please stand up? Is it my very positive experiences or the dark accusations of ethical lapses, industry coziness and harmful suppression of disagreements? How can two such disparate views co-exist?

Forming a consensus and "speaking with one voice" are logical and sensible for FDA, but not an accurate reflection of what usually happens when well-trained, analytically-oriented people with different perspectives gather to make a decision. For example, reviewers focused on the risk-benefit of a medical product are often at odds with reviewers whose focus is safety.

The situation can become more confused when data is open to different interpretation. In such a clash, some people will feel they were not heard or that their views were not considered seriously enough. This is understandable and inevitable. Further, no one can deny that FDA has made some bad decisions and might have done better if it had listened to dissenting views.

However, aggrieved employees may also feel that malicious agency thinking and dictatorial managers have kept their views from becoming the FDA’s position. It is this generalization–from a single instance to the entire agency–that fosters the corrupt image of FDA propounded by agency critics.

I am not persuaded that these critics are right. FDA is making progress in handling dissent and in encouraging managers to be more open-minded. As happened with the Avandia decision, the agency is trying to be honest about disagreements. I am still impressed by the FDA’s self-evaluation of its poor performance in the ReGen medical device approval.

To me, these are hopeful signs, as well as indications that FDA values the nation’s public health above all other interests. FDA makes mistakes, but there is no conspiracy. The agency is fully committed to serving the American people. 


Some related columns:

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

Why Do Some People Dislike FDA?
October 15th, 2009

Yesterday, I received separate posts from three organizations that are anti-industry, one of which dislikes FDA and one of which hates FDA. They are not alone in these feelings. There are many groups and individuals who believe that industry and physician professional societies run FDA. I don’t accept their premise or the “facts” from which they launch attacks. Read the rest of this entry

FDA: A Hit and A Miss
September 26th, 2010

FDA Matters has watched FDA handle the Avandia decision differently from any prior controversy. I like the new approach. In the same week, FDA provided a status report on its long-overdue social media and Internet communications policy. Because the agency’s efforts have been glacial, the prospect of useful guidance is dim. I think this is a serious problem. Read the rest of this entry

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