Former FDA officials call for more transparency for drug approvals

Regulatory NewsRegulatory News | 15 June 2022 |  By 

The US Food and Drug Administration (FDA) needs to take a more proactive approach in combatting misinformation and communicating decisions to the public when approving drugs, especially when the decision goes against the recommendations of its advisory committees. Not doing so will continue to undermine the public’s trust in the agency, said a panel of former FDA commissioners and others at the Food and Drug Law Institute (FDLI) annual conference on Tuesday.
Califf addresses misinformation
FDA Commissioner Robert Califf also spoke about the perils of misinformation in his keynote address. Califf mentioned that the prevalence of misinformation and disinformation “is putting patients and consumers at risk.”
“The constant stream of information, opinion and, too often, disinformation being produced has eroded trust in societal institutions, including the FDA – and, regrettably, in the science upon which our decisions are based. This affects the scope of our work and it is causing harm to patients and consumers,” Califf said, bemoaning the lack of trust in the government.
FDA transparency
One former official said the agency can expand its reach by using informal methods, such as press announcements, blog posts or tweets in communicating these messages.
The speakers were asked to address how the agency can regain the public’s trust and combat public mistrust of COVID-19 vaccine authorizations, following a series of high-profile decisions that went against votes by the agency’s advisory committees.
The panel included former acting FDA commissioners, Joshua Sharfstein and Stephen Ostroff, Lowell Schiller, former associate commissioner for policy at FDA, and Holly Fernandez Lynch, professor of medical ethics and law at the University of Pennsylvania. The panel was moderated by FDLI President Amy Comstock Rick.
Rick asked the panel to discuss whether FDA should be more transparent in its decision-making following the controversial approval of Biogen’s Alzheimer’s disease drug Aduhelm (aducanumab) last year. The agency granted Aduhelm accelerated approval despite its Peripheral and Central Nervous System (PCNS) Drugs advisory committee voting against the drug’s approval and reassurance from Billy Dunn, director of CDER’s Office of Neuroscience, that the agency was not looking at amyloid plaque reduction as a surrogate for efficacy.
“In general, I feel very strongly that the FDA should be explaining itself on any decision ... I do think that transparency and explanations are extremely important,” Sharfstein said. “When I was at FDA, I helped them work on the transparency initiative, and I have been very outspoken in my writing on the importance of explaining decisions. It is particularly important when you are doing something different from the advisory committee.”
Sharfstein added that a “serious question of integrity was raised when the advisory committee was told that something would not happen and it turned out to happen,” adding that “these questions of integrity are incredibly important to the FDA and the commissioner’s job to protect the integrity of the agency.”
Lynch concurred. “I think it is critically important when the advisory committee was told one thing and other things happened … it was helpful to have the advisory committee’s opinion in the first instance and then when the material information changed the advisory committee should weigh in again.”
When the decision to reject the drug was almost unanimous, as was the case with Aduhelm, “the obligations to explain why FDA went a different way become higher,” said Lynch.
There was another case where FDA rejected a decision made by its advisory committee which brought “a lot of disagreement among the FDA career staff,” said Lynch. She was referring to an FDA advisory committee recommendation in approving Sarepta’s Duchenne muscular dystrophy therapy Vyondys 53 (golodirsen) in 2019. The advisory committee rejected the drug over safety concerns and questions about its efficacy.
Schiller said it is important for the agency to explain the reasons for rejecting these decisions. “Absolutely, explaining that difference of opinion is important.” He said this communication does not have to be formal, and can take the form of press statements, blog posts, tweets or tutorials.
Misinformation fills the void
The panelists said that this void of information invites misinformation about product approvals and the science leading to these approvals.
“This is a difficult time for public health at every level,” responded Sharfstein. “For a number of reasons, the world we live in is very different than when I was at FDA. What goes on in social media and the sheer volume of misinformation that is out there is fueling people’s mistrust.”
Sharfstein said that FDA carefully weighs its message before it goes in the public realm, yet “FDA generally does not say that much until it makes a decision, and in the meantime all this misinformation then gets out there.”
Better messaging would have helped FDA in communicating its thoughts prior to authorizing the COVID-19 vaccine for children, said Sharfstein. He noted that FDA did a “phenomenal job” communicating about its decision to authorize COVID-19 vaccines for adults, but “kind of struggled to be that kind of voice” for pediatric vaccines.
FDA also did a poor job in communicating its thoughts along the way with the Novavax vaccine, said Ostroff. Earlier this month, an FDA advisory committee unanimously recommended authorizing Novavax’s COVID-19 vaccine for adults 18 and older.
“They submitted their applications back in January and it goes into this void, and you have no idea what’s been happening for the past four or five months.” He said that there was a similar information void with Moderna’s EUA requests for pediatric populations.
Ostroff said, “It would be very helpful for the agency” to communicate their thoughts to the public. “You don’t have to give out all the proprietary information, you can say ‘here is what is going on with this,’ so the public has some understanding of what the agency is doing, and it just doesn’t go into this big black box and disappear for some period of time.”
FDLI annual meeting


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