COMMENTARY

The Important Traits Needed for an FDA Commissioner

Jonathan Sackner-Bernstein, MD

Disclosures

March 30, 2021

The Biden administration is in the process of reviewing potential candidates for the next Commissioner of the US Food and Drug Administration (FDA). It's one of the most important healthcare positions in the United States, and every potential candidate is being carefully scrutinized.

Jonathan Sackner-Bernstein, MD

I've been a patient, a physician, and a drug/device developer, as well as an FDA official, and I believe that the next FDA Commissioner should embody several characteristics in addition to scientific, clinical, and public health expertise.

Clear and Transparent Communicator

The FDA Commissioner must focus resources on clear, transparent, and consistent communication, coupled with accepting responsibility for action/inaction, with the understanding that emerging scientific data can change what and how the FDA communicates. This must be a priority if the FDA wants to be considered a trusted source of information.

Several surveys demonstrate that the public doesn't adequately understand the work of the FDA. While it's important for the public to understand what it means for a drug to be FDA-approved, communication needs are much more extensive.

As an example, consider the events following the National Resources Defense Council's submission to the FDA in 2008 seeking tighter regulation of bisphenol A (BPA) in food and food containers. By 2014, the FDA eliminated the regulations that allowed for use of BPA in products used by infants, including baby bottles, infant formula containers, and sippy cups, while also reporting that there was insufficient evidence to persuade the FDA to change its determination of safe human exposure.

The way the FDA communicated these apparently conflicting conclusions — that it should be banned from products used by infants yet there was not enough evidence to revise FDA's assessment of the safety of BPA — did not make sense to the public.

In some cases, the law may prohibit public communication of certain facts by the FDA, particularly when based on proprietary information that belongs to a person or company. But that's only one type of question the public deserves to have addressed.

Another example of the importance of good communication concerns the perception of appropriate or inappropriate relationships between the FDA and/or its personnel and the industry that it regulates. Often, close collaboration can enable the process to work better. For instance, if a device or drug developer gets specific feedback on requirements, it can address those requirements in a timely manner.

However, if the relationship appears to show that something other than data led to a particular decision, that's a problem. To date, the FDA has not explained the parameters that control the relationships with regulated industry in a way that the public can understand. And such relationships can be powerful in helping to improve the efficiency of product development without exposing the public to unacceptable risk. This is an example of the communication responsibility facing a commissioner.

Successful communication also requires accepting responsibility. The case can be made that the FDA contributed directly to the opioid crisis. Is it enough for the Agency to wait a decade or longer to act, after the problems of opioid use and abuse were apparent?

Policy changes by the FDA would be embraced, appreciated, and probably have greater impact if the Agency were to acknowledge and accept responsibility for its errors.

Persuasive Leader

Decision-making defines the impact of the FDA as a practical guide to the regulatory processes and requirements. With rare exception, these decisions are made by the reviewers, not FDA's leadership. If a policy is to be implemented as intended, the commissioner and FDA leadership must understand how to persuade the reviewers to make decisions according to the intent of the policy.

The approval of eteplirsen for muscular dystrophy is an example of how a disagreement between reviewers and leadership failed to be resolved because of inability to be persuasive. Documents available for review suggest that there was relative uniformity of opinion by reviewers and their supervisors, with a conclusion that the drug should not be approved. That stood in contrast to the view of Center for Drug Evaluation and Research leadership that the drug should be approved.

Functionally, the disagreement between the reviewers and the center director was adjudicated by the center director rather than through an independent panel, with approval directed by the center director and allowed to stand by the commissioner. Independent of whether the process was designed or implemented correctly, this is an example of failure of persuasion skills — and a lesson on the importance of such a characteristic in the commissioner.

Flexible Intellect: The Ability to Change Direction When Necessary

COVID-19 is teaching us again the importance of flexibility for a commissioner as much as for a government agency. Whether the infectious agent is the polio virus, HIV, or a coronavirus, we've learned that rapid responses are difficult if there has been no advance planning. And while major public health needs such as obesity, neurodegenerative disease epidemics, and the war on cancer may not feel like they require such immediate response, addressing these problems requires intellectual flexibility for science and process as much as for managing FDA employees, patients, and industry.

In response to the HIV/AIDS crisis in the 1980s, Congress established the mechanism for accelerated approval of drugs and biologics in 1992. The statute and regulations establish FDA power to lower the evidentiary requirements for approval when a drug or biologic addresses an unmet public health need. While this path to market was developed in response to the HIV/AIDS crisis, it has been used widely over the past 20 years for a broad range of oncology products.

While some are critical of this path because of concern that the benefit-risk ratio is not sufficiently defined, review at a public workshop verified that accelerated approval is critically important to favorably affect public health. An emerging challenge is FDA's Breakthrough Device and Drug designations. The accelerated approval path relies on surrogates that the FDA believes sufficiently demonstrate likely clinical benefit.

But when a Breakthrough product is a breakthrough because data suggest that it can address a previously unmet need, there will be no validated surrogate. Thus, the FDA must assure that implementation of these regulatory pathways is internally consistent. The next commissioner must determine how to use these mechanisms more broadly, with the flexibility required to stimulate development of new products — including medical devices — to address unmet public health needs.

What can help the next commissioner lead the FDA onto a new and effective path for positively influencing public health? Perhaps it is as simple as adding a dimension to the usual assessment of benefit-risk of a new product, to include the risk that patients face without any new products. Perhaps the new CMS policy of automatically paying for Breakthrough devices for 4 years — providing time for the manufacturer to develop data to justify continuing to pay for such devices — is a template for time-limited approval of new drugs and biologics.

Irrespective of which mechanisms or paths are implemented, a commissioner must be primarily wed to what the data support. Thus, implementing a new policy that fails would certainly be problematic, but less so when a commissioner can identify and react to the failure promptly, transparently acknowledge it, understand why it happened, and use the experience to define new opportunities.

Cautious Risk-taker

Intellectual flexibility can impact public health faster, better, and/or more widely only when FDA leadership can manage risk-taking in a cautious manner. That caution can't serve as a barrier, but rather as a guide to take risks in a manner where potential gain outweighs potential risk, particularly by understanding how to identify and react to failure as quickly as possible, as well as how to shift any initiative to maximize likelihood of success. The FDA can learn from the scientists, trialists, and companies it regulates.

On a day-to-day basis, a commissioner needs to understand how to ask challenging if not contrarian questions. Doing so can stimulate novel thinking and creative action.

Perhaps one example of where such an approach could be valuable is for the FDA to ask its experts and consultants how and what to communicate to the public about medical cannabis use. Although the FDA recently released a draft guidance focused on clinical research of cannabis products, with over two thirds of US states allowing medical marijuana, a trusted source of science-based information is critical. Such a role seems to be mandated by the FDA's own mission statement.

So while there are laws that prevent the FDA from regulating cannabis products, the public needs science-based information, whether it's a favorable or unfavorable assessment of cannabis use. And no federal agency can do that better than the FDA.

Scientific/Public Health Expert

Selecting the next FDA Commissioner is far more complex than merely compiling a list of characteristics. However, such characteristics in the context of scientific and public health expertise would enable FDA leadership to set a tone that is aligned with the mission of its career employees and its legal responsibilities. If coupled with high-level skills in data collection, analysis, and interpretation, both for scientific and process related questions, such a commissioner would serve as a leader who would allow the FDA to maintain and strengthen its position as the world's leading voice for managing and regulating public health needs.

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