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Federal regulatory agencies can’t stand still. They must evolve with industries they regulate.

No agency faces more pressure to do this than the Food and Drug Administration. Charged with simultaneously protecting the public’s health from unsafe products and promoting public health by accelerating access to new treatments, it regulates about 20 cents of each dollar in the U.S. economy, all while responding rapidly to emerging health challenges like the disease caused by the novel coronavirus, Covid-19, and new vaping technologies that may reduce risks for smokers but increase nicotine use by youth.

At this time of new challenges for an agency led by a newly confirmed commissioner, we provide some context and highlight some emerging opportunities for the FDA to fulfill its mission, and truly change the lives of patients. We do so from the perspective of a patient advocate (E.S.) who knows what a difference innovative approaches at the FDA can make, and a former commissioner (M.M.) who has seen the ongoing pressures facing the agency’s staff in fulfilling its evolving mission.


We have both seen how the FDA can benefit from new science and technologies, along with continuously increasing experience with what works and what doesn’t in regulation, to implement new tools and refined approaches to protect and promote the health of Americans.

A good place to start is placing recent regulatory debate about the FDA in context. One constant at the FDA is criticism from both sides: blaming the agency for not demanding enough evidence and taking enough time to assure safety, while simultaneously disparaging its requirements for standing in the way of speedy access to life-improving therapies. The agency has always worked to balance these perspectives.


For example, some recent critiques of the agency have focused on “process” metrics — how many patients are included in preapproval trials, for example, or the share of product submissions that get approved, or the share of products approved through accelerated approval pathways. These are potentially informative and important indicators, but they aren’t what really matter when assessing a public health agency. Instead, the focus should be on public health: Are medical products becoming safer, or less safe? Are regulatory actions leading to better outcomes for patients?

These counts play into the ongoing debate about how the FDA is balancing speed and safety. But in a science-based agency, the goal isn’t to keep processes the same — it’s about regulating better. Just as the technologies the agency regulates have evolved, so have opportunities for innovation in the science of regulation and in the development of medical products. These innovations make faster regulatory action possible, but even more importantly they can make the development process for medical products faster and more efficient while maintaining the gold standard for assuring safety and effectiveness.

For example, the FDA has implemented a breakthrough therapy designation that directs more resources to interacting with companies developing new kinds of interventions for unmet medical needs throughout the development process. That guidance should enable reductions in development time. On average, drugs that received the breakthrough therapy designation had 2.2 years shorter premarket development time and are approved nearly three months ahead of the Prescription Drug User Fee Act goal date compared to drugs that did not receive breakthrough designations.

It is possible to create outcome-oriented measures of the impact of reforms like these on the agency’s mission. Since 1992, the FDA has granted accelerated approval to 66 new molecular entities for the treatment of cancer. More than half of these drugs transitioned to full approvals following the confirmation of benefit in follow-up studies. More recent approvals have shown benefit in different treatment settings or have studies underway to verify clinical benefit. It’s important to note that only six oncology drugs have been modified or withdrawn since accelerated approval was established.

The FDA’s ability to regulate the safety of these drugs has been reinforced by major innovations in its expanding active surveillance system, which allows for large-scale analysis of the use and adverse events associated with drugs and biologics on an ongoing, national basis. In the past decade, the FDA has developed this capacity thanks to the Sentinel Initiative. This system has substantially enhanced the FDA’s ability to identify and act in a timely manner on drug safety problems, and to assess more comprehensively whether suspected safety concerns are real.

These improvements in the use of real-world data provide a foundation for understanding the impact of treatments in real patients — not just those in clinical trials — and the FDA is thoughtfully approaching scientifically valid ways to expand such evidence.

Strong bipartisan support for the agency has been key to enabling these advances, from approving additional resources to recruit more scientific expertise to placing greater focus on innovative regulatory sciences, encouraging more opportunities to provide clear guidance for industry, and providing for significantly improved post-market surveillance systems. The FDA has used these resources to improve its scientific expertise in areas such as statistical analysis, electronic data use, and the understanding of disease and treatment mechanisms.

Thanks to the additional resources and progress in the science of product development and regulation, how the FDA regulates today is more advanced than it was a decade or two ago. The agency needs support to continue these steps, enabling better regulation that keeps up with scientific development in the coming decade.

As we look toward the FDA of the 2020s that can achieve further progress in addressing urgent health threats and pressing unmet patient needs, it makes sense to build on what has worked to improve public health outcomes.

First, as scientific advances continue to expand the scope and complexity of products that the agency regulates, the FDA needs more resources to support its mission. Along with our colleagues in the scientific, patient advocacy, academic, medical, and industry communities, we have advocated for providing more resources to the FDA so it may operate as effectively and efficiently as possible. Still, talented professionals are leaving the agency due to financial reasons, and new capabilities must be developed.

Second, the FDA should continue its steps to enable public input and dialogue. During the past decade, it has become one of the most open agencies to invite input from the public, patients, and academic and industry experts. This should be the basis for further growth.

Approaches used by the FDA’s Oncology Center of Excellence to make trials more efficient and bring needed products to patients faster — products that are demonstrably improving patient outcomes — could be a model for expanding such steps for public input and engagement with professional communities. The oncology center has shown that such transparency helps focus regulatory approaches and enhance shared understanding of the relevant science and its intersection with patient priorities. Such transparent processes to inform an independent FDA should not be convoluted with outside sources “influencing” the agency.

Third, the agency needs more support to modernize how it collects and uses the vastly expanding sources of digital health information. This includes promoting interoperability to obtain electronic data seamlessly from clinical trials, including trials and studies in real-world settings, to reduce development costs and generate a broader range of evidence about medical products throughout their life cycles. The FDA’s recently announced technology modernization plan reflects this opportunity for continued progress in evidence-based regulation.

Fourth, strong leadership that respects the bipartisan support for the agency’s commitment to science-based regulation should continue. The FDA’s current strength is based on the public service of thousands of dedicated employees, reinforced by committed leadership. The recently confirmed commissioner, Stephen Hahn, a well-respected oncologist, has expressed his commitment to this critical tradition.

As the agency’s importance continues to grow, it faces new challenges. Drug pricing is a significant issue, and there is more the FDA can do to improve access to medications by promoting competition involving generics and biosimilars and facilitating efforts to address questions about costs and cost-effectiveness that are important to payers, clinicians, and patients. Although addressing costs and pricing are not directly part of the FDA’s mission, the infrastructure used to improve evidence on safety and effectiveness, such as an interoperable infrastructure to learn more about products in real-world settings, can also be used to address questions about cost-effectiveness. The FDA must also act swiftly to better address the youth vaping epidemic and the premature deaths caused by tobacco use, the ongoing opioid epidemic, and the need for effective diagnostics and treatments for the new coronavirus.

There will be further public health crises and opportunities ahead, along with new ways in which the agency can prepare to address them. By following the four steps we outlined and focusing on outcomes, the FDA can move forward with better regulatory capabilities that match the evolving biomedical and data sciences and achieve the health improvements that matter most to patients and all Americans.

Mark McClellan, M.D., is the director of the Robert J. Margolis Center for Health Policy and professor of business, medicine, and health policy at Duke University. He served as FDA commissioner from 2002 to 2004, and is an independent board member of Cigna and Johnson and Johnson. Ellen V. Sigal, Ph.D., is founder and chair of Friends of Cancer Research, an advocacy organization based in Washington, D.C.

  • I’m genuinely curious: What does the word “independent” mean to imply when stating in McClellan’s bio he “is an independent board member of Cigna and Johnson and Johnson”?

    • Kristina

      You raise an extremely valid question and a good one. Mark has been part of the FDA task force responsible for drug shortages that has today delivered a BIG CIRCLE WITHOUT CENTER.
      I call him part of elite group who claim to be independent but show up at every task force FDA has. Look at November/December 2018 meeting on drug shortages.
      Most are part of “scratch my back brigade”. Thank you for asking.

      Following is the note I sent to Mark this morning.
      Hi Mark

      I read your perspective. You have some points but unfortunately I don’t think USFDA has the guts and stomach to be creative and aggressive, two characteristic needs today.

      FDA needs to create processes that will approve e.g. ANDAs in 90 days.

      They need to stop sales and distribution of less than quality drugs.

      Since FDA is squeezed for money all the time, it’s revolving door and pharma lobby have made FDA essentially a puppet. They have more desk jockeys than people who are hands on experienced and can take it forward.

    • Thank you for this info, Girish. As a drug safety advocate whose teen’s death was precipitated by a risky and wrongfully prescribed FDA black box drug, one given without any informed consent, it is always disconcerting to peel back the onion and see the tangled web of conflicts and systemic deceit that increases risks and harms to all Americans.

      As a trained journalist, it is disconcerting to see that the author of this article, and the STAT news editorial staff, have not provided an answer to my original question.

      Readers have a right to know what, exactly, the word “independent” as it pertains to pharmaceutical company board membership is meant to imply.

  • Thank you for this thoughtfully conceived and well-articulated opinion piece.

    The FDA must not submit to bureaucratic intransigence, institutional obstinance or a backward-facing rule-making posture in a kinetic, ever-changing medical technological landscape.

    As the authors so aptly imply, the “this is the way we do things because this is the way we’ve always done things” ethic of regulation is antiquated, anachronistic and counterproductive.

    Progress has been made and more progress remains to be made. In the area of stem cell policy – on which I write – this is particularly true. Revolutionary, paradigm-shifting technologies may require revolutionary, paradigm-shifting policies. The FDA must not fear change.

    Flexibility, adaptability, alacrity and open-mindedness are watchwords to be embraced. Industry is ready. More importantly, the people are ready.

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