The FDA's insider review on aducanumab was all about politics, not science, and it threatens patients and the biopharma industry alike
Bioregnum Opinion Column by John CarrollThe FDA deserves kudos for drawing a line in the sand over their review of Covid-19 vaccines. They’ve made it clear — after deep skepticism began to appear following the debacle over convalescent plasma — just what they will need to see before handing out an EUA, thus saving the agency’s rep for enforcing standards essential to maintaining public trust in drugs.
That same integrity, though, has gone missing from the FDA’s stance on Biogen’s Alzheimer’s drug aducanumab. Despite a statistical analysis that highlights the conflicting nature of the Biogen $BIIB data, a post hoc dumpster dive operation to find supportive data and a move to shine up small cohort results as backup proof of efficacy, the FDA division responsible for Alzheimer’s chose to endorse adu for use among millions of patients.
We’ve been here before. The Sarepta approval leading to marketing authorization — with a label that clearly states their first, very expensive, Duchenne MD drug remains experimental — is a permanent black eye when it comes to industry standards. What followed just made it all much, much worse — and appears to be about ready to happen again in an infinitely larger patient population.
At the time, as I found out fairly recently, the FDA’s Janet Woodcock repeatedly told colleagues that an approval for Sarepta was a once-off decision. She signed off on biomarker data from a tiny study one time. The next time Sarepta would have to do something like a walk test with more boys. She wanted real data.
Only that never happened. What did happen: The FDA regulators in charge adopted the biomarker data on dystrophin production as a low bar for an approval, and once they got a naysayer out of the way who handed out a CRL for their second app, the new regulators in charge waved it through.
So we shouldn’t consider this some sort of once-off just to give Alzheimer’s patients a drug they can use until something better comes along.
This is a precedent that can be used again and again, lowering the bar on Alzheimer’s data that others can jump over as well.
What else did we learn from Sarepta?
One, when you catch a ‘Hail Mary’ pass, there’s no need to do something like actually meet FDA timelines for a full pivotal trial to confirm the weak signs of efficacy you’re seeing. The biotech drug their heels on that forgotten front for years, and the FDA did nothing about it.
We see this sort of thing regularly. Once a drug wins any kind of quick OK, it doesn’t always just disappear once the required Phase III followup — inevitably requiring years — turns out to be a flop.
An unmet medical need like Alzheimer’s is a terrible thing. But when FDA standards go in the trash bin on the way into an advisory panel meeting, it hurts everyone — patients and industry alike. If you destroy patients’ trust in regulatory decisions, as the FDA appears determined to do here, you undermine all marketed therapeutics.
We’ll find out what the FDA panel thinks of aducanumab later today. Most drug developers already know that the FDA is being pushed by political reasons here. The science simply doesn’t back it up.
