What will it take to lead today's FDA?
With a new administration in the White House, the top seat at the FDA is open again. The contrasting attributes of frontrunners Janet Woodcock, MD, acting head and 37-year veteran of the FDA, and Joshua Sharfstein, MD, former FDA principal deputy commissioner during the Obama administration, could lead the agency in two different directions: One squarely focused on public health and the other pushing quality and innovation. Depending on who snags the president-appointed seat at the federal agency, pharma companies might find themselves weighing the innovation, value and health equity of their products differently.
Although the FDA has regular oversight over many products that figure prominently in the health and life of Americans, the pandemic has kept many laser-focused on the agency’s role in regulating drugs—specifically the approval of safe and effective COVID-19 vaccines and treatment. And that could be a point in Woodcock’s favor. After all, she’s approved countless medications, including some of the most innovative cancer therapies, during her tenure. But the pandemic also could benefit from a leader with a solid public health record, and that marks a point in Sharfstein’s favor.
The agency’s leadership has been questionable, if not politically influenced, since Scott Gottlieb stepped down in April 2019. In fact, trust in the agency is at an all-time low. During his time in the public service role, Gottlieb devoted equal time to three ideal areas of focus: to protect, innovate and scale up. Historically, the agency’s focus had been heavily skewed to protect, was slow to innovate and was cautious to scale up. Let’s take a closer look at what sets today’s leading candidates apart, where there’s common ground and whether one of the candidates could once again bring welcome change to the FDA.
To protect
Public protection is deeply embedded in the FDA’s genes, and each FDA commissioner is tasked with setting high-priority initiatives and seeing to it that they’re backed with the right resources. In fact, you could argue that each agency chief’s term of service is measured by how well they protect and promote public health. We’ve also learned that trust is foundational to protection. With the public’s confidence in the FDA undermined, the new leader will be inheriting a relationship that needs mending. So, how do Woodcock and Sharfstein stack up?
I’d say Sharfstein has the edge here. After all, his roots in the public health domain run deep: He stepped down from his post at the FDA to become the secretary of health and mental hygiene for the state of Maryland in 2011. A pediatrician by training, it’s no surprise that children’s health has figured prominently in his public health initiatives, as have HIV, opioid addiction and teen tobacco use. But he’s had his share of failures on his watch, too, like when Maryland’s health exchange crashed on its first day. He’s no stranger to adversity, an attribute that he’d likely need in such a high-profile position.
Sharfstein’s public health background would fit nicely with the likely agenda of Xavier Becerra, Biden’s nomination for HHS Secretary, should he gain Senate approval. His pro-public health mindset could be a boon amid a global public health crisis that revealed a lack of coordination, communication, education and leadership at the top. In fact, he’s talked at length about what can be accomplished for the American people when public health leaders work closely with federal policymakers. And, he wrote the book on how leadership is the key to surviving a public health crisis. No really, he wrote the book.
On the pandemic front, Sharfstein has gone on record agreeing with the emergency use authorization designation extended to COVID-19 vaccines during the previous administration. And neither are strangers to an outbreak, both at the FDA during H1N1. In fact, Sharfstein even stated in a New England Journal of Medicine article that, “The agency's success will be determined by the nation’s access to a safe and effective vaccine.” And both candidates are equally prepared to fend off any political figures who run interference with the FDA’s agenda, a practice that seemed to reach an all-time high during the last administration.
For Woodcock’s part, she knows the agency inside and out and would hit the ground running if appointed to the top seat. She’s demonstrated regulatory flexibility and a willingness to coordinate closely with manufacturers to bring new medicines to market more quickly. The approval of Sarepta Therapeutics’ controversial Duchenne muscular dystrophy medication couldn’t have happened without Woodcock’s resolve and her advocacy of patient needs. It was a breakthrough for the agency, both the start of seeing the drug industry as a partner and giving the patient community a voice at the approval table.
She has a long history of testifying before Congress—and winning. She’s been the target of public scrutiny and hasn’t wavered, but she can admit when she’s made mistakes. Innovation can’t be supported without a bedrock of trust, so only a good protection record will make more innovation possible.
To innovate
The FDA has a history of drafting, piloting and enforcing regulations in a way that enables pharma industry innovation. The new commissioner needs to encourage pharma companies to strive for a higher level of innovation than we’re seeing today.
Woodcock has led the agency’s Center for Drug Evaluation and Research not once, but twice. It was during her first run in the post that she introduced a risk management approach to drug safety, modernized drug manufacturing and regulation through the Pharmaceutical Quality for the 21st Century Initiative, and advanced medical discoveries from the laboratory to consumers more efficiently under the Critical Path Initiative. And, she played a role in the approval of the first biotechnology-based treatments for multiple sclerosis and cystic fibrosis during her time as director of the Office of Therapeutics Research and Review in CBER. If the tenure of a leader is to be judged by the organization’s accomplishments during that time, then Woodcock—who’s credited with many of the positive industry-related accomplishments at the FDA—has the edge.
Sharfstein seems to operate at the other end of the spectrum, often erring on the safe side of a drug’s risk and benefit profile. At times, he’s demonstrated an unwillingness to fully understand the complexities of bringing a drug to market and believes that some risks aren’t worth taking.
Woodcock’s experience with the agency has kept pace with, and encouraged, industry innovation. But she’s also outspoken about the pitfalls of pharma’s approach, often speaking out against the high cost of innovation and reminding companies that developing a drug needs to be about improving patient outcomes, not gaining FDA approval.
Woodcock has advice for ensuring medical innovation, too. She believes that researchers can develop wider-reaching treatments by leveraging big data sources like EHRs and by involving practicing physicians in the research. Ultimately, this would speed up the process of discovering and commercializing therapeutic solutions. And as luck would have it, more consolidated data sources could soon be available. Pharma companies could have an opportunity to partner and actualize the agenda that Woodcock would bring to the table
It’s easy to lose momentum when it comes to encouraging healthcare innovation, and it’s extra challenging to help other career officials and politicians think beyond the confines of safety and broad access. Innovation often is perceived as “new” and risky. It’s easy to take your foot off the pedal, and any losses in doing so will largely be felt by future generations. With unfinished business in many of these tech-related areas, the new commissioner will need to have a sense of urgency to carry already established programs across the finish line—and contribute a few ideas of their own.
To scale up
In addition to making ongoing investments in regulatory science designed to protect patients and consumers, it falls on the FDA to establish modern, risk-based regulatory standards to help reduce uncertainty for innovators. Under Gottlieb’s tenure, the FDA doubled-down on the role it must play in advancing cutting-edge science, and the initiative’s future is dependent on the new leadership carrying it forward.
In addition to pushing through several other initiatives designed to bolster the sector, Woodcock has established accelerated pathways and incentives for pharma companies making experimental treatments, most notably new treatments for cancer. This focus aligns with Biden’s “national commitment to end cancer as we know it.”
There’s some evidence that the drug approval process would be less likely to accelerate under Sharfstein’s rule. He also hasn’t shied away from opportunities to criticize the FDA’s tendency to approve drugs without giving the public a view of the supporting evidence and data. More transparency is sure to top his agenda if appointed to the FDA’s top seat. In fact, he’d likely pick up where he left off with the agency’s Transparency Task Force. He has proposed increasing FDA oversight of potentially dangerous drugs on the market and cracking down on incentives that prevent generic competition for common drugs.
But he does have a mind for regulation, too. During his less than two-year stint with the FDA, he and then FDA Commissioner Margaret Hamburg, MD, doubled down on regulating drug, device and food companies. On the health policy front, Sharfstein is expected to push hard for reform. For that, he’d rely on his experience serving as a health policy adviser to California Rep. Henry A. Waxman and on the Democratic staff of the House Government Reform Committee.
Experience is just one measure of success for a new commissioner. Like those who’ve come before, the soon-to-be-appointed leader will come to be known for their own key areas of focus. For example, drug pricing is a hot-button and bipartisan issue, one that Sharfstein has been outspoken on. Another area where a new commissioner could make significant progress is to integrate real-world evidence into regulatory submissions to accelerate approvals.
But most of all, a permanent appointee who knows what it takes to protect, innovate and scale up—and is prepared to do some hard work on the trust front—will serve the FDA well. So long as the commissioner’s main strengths aren’t solely focused on an overly cautious interpretation of protection at the expense of the other two focus areas, the FDA will benefit from a fresh perspective.