As Edward R. Murrow reminds us, “Anyone who isn’t confused really doesn’t understand
the situation. The “existential crisis” that currently pervades the Food and Drug Administration (FDA) is real – but not particularly helpful to dwell on. It’s time to clear our heads, stop complaining, and start planning for what comes next.
What comes next is the development of means strategies and tactics that allow the FDA to remain a potent force for medical progress. In his inaugural remarks to staff, the agency’s newly confirmed Commissioner, Dr. Martin Makary, spoke about the importance of medical innovation. His many peer-reviewed articles and books reinforce his reputation as a change agent, a supporter of 21st century regulatory science. Now he can put those words into action. But will he be allowed to do so?
Dr. Makary is a man of high ethical standards and impressive intelligence, but actions speak louder than words. Here are some things to watch for as he begins his tenure as our nation’s top regulator of products that comprise nearly 20 cents of every dollar spent by US consumers and, more importantly, the future of medical innovation.
New Hires. Priority #1 for the Commissioner Makary is recruiting elite talent to replace Drs. Peter Marks (formerly Director of the Center for Biologics Evaluation and Research) and Janet Woodcock (formerly the agency’s Principle Deputy Commissioner). These scientists presided over the reviews and approvals of new therapies for serious and life-threatening conditions such as diabetes, Alzheimer’s Disease, Parkinson’s Disease, Muscular Dystrophy and many others (including vaccines) and expedited the introduction of generic drugs and biosimilars – a key strategy for lowering costs and increasing access to prescription medicines. Regardless of any forthcoming reorganizations, Dr. Makary must be allowed to recruit and hire the best and the brightest.
21st Century Regulatory Science. Over the last 25 years, the FDA has made tremendous strides transforming the way regulated products are developed, evaluated, and manufactured. This “critical path initiative” has (across presidential administrations of both parties and multiple commissioners) diagnosed the reasons for the widening gap between basic scientific discoveries that have unlocked the potential to prevent and cure some of today's biggest killers and their translation into innovative medical treatments. It also reinforces the importance of the agency being both partner to and regulator of the industries it regulates. It’s a difficult tightrope. How will Dr. Makary handle this highwire balancing act? Are we willing to have important new medicines and medical devices available in Europe years earlier than in the United States? Importantly, will Dr. Makary embrace the evolving, dynamic role of the patient voice in the medical products review process. We cannot leave these questions to the Magic Eight Ball.
Battling the Status Quo. Being the regulatory global “gold standard” doesn’t mean hewing to outdated ideas and practices. The critical path notwithstanding, convincing FDA staff (at all levels and all product centers, from medicines to medical devices, tobacco to food and cosmetics) to lean in is a tough challenge. Inside the FDA, those who fear of change often disguise themselves as “Guardians of the Gold Standard.” As commissioner, Dr. Makary must be both cheerleader and Chief Innovation Officer. His career has been all about challenging the status quo. Minus aggressive leadership, it is likely that FDA review teams will revert to the “tried and true,” abandoning still nascent regulatory approaches that have already delivered exciting new gene therapies for Sickle Cell Disease, treatments for many cancers, Friedreich's ataxia, Duchenne muscular dystrophy, and other rare and orphan diseases. The status quo is a harsh commandant and can only be overcome through the strong leadership of a true believer such as Dr. Makary.
A RIF Raft. The recent reduction in force (RIF) has denuded many crucial FDA functions. While “Less is More” aptly describes Mies van der Rohe’s philosophy of Bauhaus architecture, it isn’t a scientific approach to forward looking healthcare technology regulation. Not all non-product review functions can be centralized under one big tent. Perhaps Commissioner Makary’s toughest assignment will be to convince Secretary Kennedy to allow the FDA to appropriately staff key areas including 21st century clinical development policy, drug shortage prevention, oversight of drug marketing practices (including advertising), expanded access to experimental medicines, Freedom of Information Act requests, and advisory committee administration. Crucially, Dr. Makary must claw back some basic communications capabilities. One of the Commissioner’s most important missions is to reestablish public trust in the FDA. After all, if Americans don’t have trust in the regulatory process, they won’t have faith in the products the agency regulates. Advancing health literacy starts at the top and must be one of the highest priorities. This cannot be done without a dedicated FDA communications team.
Owning Medical Innovation. As Chief Innovation Officer, Commissioner Makary’s words and deeds will determine whether medical innovators are willing to invest billions of dollars in development programs for new medicines and equally large financial commitments for clinical programs investigating additional uses of existing products. Dr. Makary’s use of the Bully Pulpit (externally) as well as his administrative direction (internally) will make all the difference.
An existential crisis is a period of deep questioning and reflection about the fundamental nature of one's life, purpose, and existence, often leading to feelings of anxiety, despair, or confusion – and all of those things are in play today inside the FDA, the industries it regulates, and among healthcare professionals and patients. The best way to address such a crisis is to focus on practical, non-political ideas and practices. And those ideas can come from many places. Under Dr. Makary’s leadership, it’s time to set aside politics, recognize priorities, reorganize assets, and design concrete plans for practical change. Now is not the time to seek shelter in the caves of caution and complacency.
Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest and a Visiting Professor at the University of Paris School of Medicine.
