Within the next few weeks, the Food and Drug Administration (FDA) is set to make an extraordinary decision: Whether or not to grant approval for Biogen’s experimental and highly controversial Alzheimer’s drug aducanumab. This would be the first treatment, ever, to be approved to treat the underlying causes of Alzheimer’s disease rather than just its symptoms. That could spell the difference between, say, controlling mental or physical outbursts related to Alzheimer’s versus actually declining the rate of mental decline associated with the brain-eating disease.
The thing is, that monumental process is almost certainly bound to play out without a full-time FDA commissioner in place. It’s one of the key public health positions that President Joe Biden’s administration has yet to fill, to the consternation of some public health experts who point to cuts made to the agency during the Trump administration and the challenges of the COVID-19 pandemic, and could have rippling effects for drug development in multiple therapeutic spaces. And, given the high-stakes nature of a pioneering Alzheimer’s drug approval, all eyes will be on how the powers that be balance hard science, the recommendations of its outside advisers, and political pressure from patient advocacy and drug industry groups.
That’s where things get a bit uncomfortable from a political lens. Right now, Janet Woodcock, a physician and long-time FDA veteran, is serving as the agency’s acting commissioner. She’s been with the FDA since 1986, served a number of stints as director of the agency’s Center for Drug Evaluation and Research (CDER), as was appointed the interim position at the agency’s top perch on the day of President Biden’s inauguration. But her experience in the role has brushed up against controversial decisions over her decades-long tenure, including pressing for the approval of treatments that outside advisers to the FDA have deemed ineffective and the approval of new opioid drugs.
“In its opioid decision-making, Dr. Woodcock, and the division she supervised, consistently put the interests of opioid manufacturers ahead of public health, often overruling its own scientific advisors and ignoring the pleas of public health groups, state Attorneys General, and outraged victims of the opioid crisis,” wrote a collection of 28 medical and public advocacy groups in a letter to Biden urging the president to appoint a different permanent commissioner for the FDA. The grievances include Woodcock’s role in getting the opioid painkiller Zohydro approved despite an overwhelming consensus by outside advisers that it could be unsafe for the public.
The FDA isn’t obligated to follow the advice of these third-party expert panels, but it usually does. And, beyond the Zohydro and other opioid-related decisions, Woodcock has been at the center of several drug approval controversies, including by leading the charge to get rare disease firm Sarepta’s controversial Duchenne muscular dystrophy drug, Exondys 51 (which goes by the generic name eteplirsen), an FDA green-light despite little evidence of its efficacy.
And that’s where the approach FDA leadership takes to drug approvals becomes so significant. The agency is technically only supposed to consider safety and efficacy in its decision-making. But examples such as Sarepta’s success show that a concerted political campaign by patient advocacy groups and drug makers themselves can influence the process. And as Biogen’s aducanumab heads for its so-called PDUFA date in June, when the ultimate decision on the drug’s fate will be made, those kinds of considerations will be front and center.
Alzheimer’s support groups have long been gearing up for this fight. As the day of reckoning looms for Biogen, whose drug was thoroughly trashed by outside FDA advisers for what they said was a shaky interpretation of clinical trial data, the Alzheimer’s Association has enlisted the likes of actor Samuel L. Jackson to make a last-minute plea for the treatment. After all, something is better than nothing, the thinking goes.
Those are decisions which will be influenced by the top levers of government, making a permanent FDA appointment all that more crucial. A recent Wall Street Journal report suggests that President Biden’s list of potential candidates is only growing. But a steady and lasting hand will be critical for ensuring trust in the sorts of difficult decisions the agency will have to make with coronavirus, opioids, Alzheimer’s, rare diseases, and beyond.
