FDA’s advisory committee plans are disappointing

Vowing to ‘optimize’ committee process via bureaucratic tweaks, agency leaders avoid difficult decisions needed for real reform

Frustrations over the quality of the scientific and medical advice provided by FDA advisory committees have been building for years, along with concerns about their independence and objectivity. So when FDA Commissioner Rob Califf announced in the summer of 2023 a “stem-to-stern” review of advisory committee procedures, patient advocacy groups, biopharma innovators, supporters and critics of FDA all cheered.

FDA officials and people who care deeply about the agency disagree about how advisory committee meetings should be conducted, especially whether and when votes should be taken.

FDA officials, including center directors, have publicly complained that conflict of interest policies prevent the agency from recruiting committee members with necessary expertise, especially for products that treat rare diseases.

There’s raging debate within the agency and among people who care deeply about its actions on how advisory committee meeting conduct should be changed, especially whether and when advisers should be asked to vote.

One sign that things are out of whack is the cottage industry of consultants, many of them former agency employees, that has evolved to help biopharma companies prepare for meetings. Despite spending a great deal — those that can afford it invest $1 million or more — preparing for meetings, companies often find that the fate of their medicines is determined by under-prepared committee members who engage in discussions that wander into irrelevant topics. The result can be shallow advice about life-and-death issues.

Ensuring that advisory committees learn from patients or their caretakers, and from physicians who treat them, is essential. Yet, patients who participate in open hearings usually only represent those who believe they have or will benefit from a product; those who may not have benefited rarely testify. Committee members have no way to determine if speakers are representative of the affected population, or if they have been recruited and coached directly or indirectly by product sponsors.

Advisory committee meetings are important not only because they inform FDA decisions, but also because they inform the public. At their best, they bring out the complexity of drug development and showcase the wisdom required to make good regulatory decisions. At their worst they are superficial performances with predetermined outcomes.

When he announced his intention to come up with ways to improve the process, BioCentury commended Califf, writing that successfully reforming advisory committees would improve the quality of FDA decisions and enhance public trust.

The news that Califf and two other FDA officials, Principal Deputy Commissioner Namandjé Bumpus and Emily Helms Williams, director of Advisory Committee Oversight and Management Staff, had written a commentary about advisory committees for Nature Medicine about advisory committees was, of course, of great interest.

Readers eager to discover how the agency’s leadership planned to make good on the commitment to improve advisory panels skipped past the overview of the advisory committee process and legal authorities and went straight to the highlighted box titled “Focus areas for the optimization of FDA advisory committees.”

The first item on the list is: “Modernize systems to reduce paperwork burden and streamline processes, including by updating forms for expedited digital signature and processing and exploring system improvements for SGE onboarding and internal processes.”

Huh?

Cutting red tape, using digital signatures and “exploring” ways to improve onboarding special government employees may set some civil servant’s heart fluttering, but it is a letdown for anyone hoping for bold change.

Maybe Califf et al. saved the best for last?

The sixth and last item on the list is “Obtain input on FDA use of ACs from all interested parties through a public listening session and docket for public comment.”

You know how sometimes when you write a to-do list you put something on there that you’ve already done so you can cross it out and start with a sense of accomplishment? That’s what this looks like.

As the commentary reports, FDA has already held a public listening session and opened and closed a docket for public comment. It heard a lot of ideas, many of them pushing and pulling the agency in different directions.

So, what about items 2-5? There’s not much there either.

Conflicting views

As comments submitted to FDA show, there’s no shortage of opinions about how advisory committees should be reformed. There is also no agreement.

One of the most contentious issues involves voting.

Califf has said publicly that he’d like to see fewer committee votes. Focusing on votes, he says, distracts attention from discussion and leads to misperceptions that advisory committees are decision-making bodies.

Richard Pazdur, director of FDA’s Oncology Center of Excellence, has argued forcefully in favor of retaining votes. They provide essential clarity, he says.

The Nature Medicine comment restates both points of view, but it gives no indication whether FDA will change its policies to make votes more or less common.

The agency also fails to take a stand on how it will fix the bias in the public testimony portion of committee meetings, focusing on what it can’t do instead of what it could. “Public commenters are not screened for COI,” the agency notes, referring to conflict of interest, and urges them not to spread inaccurate information, “although there is currently no way to enforce this.”

Another contentious issue the agency sidesteps is whether there is a need to get people with more relevant expertise onto committees, and if so, how to do it.

About half of the therapies FDA approves are orphan drugs. For some rare conditions, every expert clinician has participated in an industry-sponsored trial.

In comments submitted to FDA’s docket, biopharma companies and some patient groups urged FDA to use its power to grant waivers from conflict-of-interest restrictions more frequently to ensure that world class experts serve on committees.

Consumer groups demanded that FDA completely stop granting waivers, arguing that they corrupt the advisory process.

I’ve proposed some ideas about how to reform FDA’s systems for obtaining external expertise, and I acknowledge that the agency’s leaders are in a better position to come up with improvements. Leadership is required because it isn’t possible to get everyone, even everyone at FDA, to agree on the path forward.

In the face of such contradictory views, a policy based on consensus will focus on non-controversial process changes, like using digital signatures.

FDA leaders are aware of the perils of lowest common denominator decision-making.

Very often, the agency must make difficult decisions in the absence of perfect information, knowing that whatever it decides will disappoint a lot of people. That’s unavoidable. FDA’s job isn’t to find consensus or win popularity contests, it is to make the best, evidence-based decision possible and then explain it.

That’s what the agency’s leaders should do when it comes to reforming the advisory committee process: take steps to dramatically improve the quality of the advice it receives, even if those steps ruffle some feathers, and explain its actions.

Signed commentaries do not necessarily reflect the views of BioCentury.