Alzheimer’s Can Follow HIV And Become Chronic Condition If Science Advances, Woodcock Argues
Executive Summary
The acting US FDA commissioner equates drug development in the neurodegenerative diseases to work on HIV.
Janet Woodcock believes that neurodegenerative diseases like Alzheimer’s and amyotrophic lateral sclerosis (ALS) can eventually become chronic conditions if the science continues to develop.
The acting US Food and Drug Administration commissioner, making her first appearance on Capitol Hill in the role, equated drug development in the area to what was seen in HIV, saying during a 10 June Senate subcommittee hearing on the fiscal year 2022 budget request that the agency will remain flexible with approval and other requirements to help new treatments advance.
“We will do everything we can to move these therapies along and be very flexible,” Woodcock said during the Senate Appropriations Subcommittee on Agriculture, Rural Development, FDA, and Related Agencies hearing. “However, we need good science because we need treatments that are going to work to advance treatment of this disease.”
Woodcock said the neurodegenerative space remains in “a more primitive area of science,” compared to cancer and HIV at the moment, in part because there is no understanding why the disease occurs in patients.
“We’re still in what we call empirical development to some extent, which means ‘Just try it and see if it works,’ rather than we are trying to interrupt a very specific pathway and we understand its role in bringing on the disease,” she said.
Woodcock added that during the AIDS crisis, patients clamored for any treatment that could help them and were willing to accept a lot of risk. She said that because the agency was willing to allow some drugs on the market earlier than normal, the field advanced.
“Now because of that flexibility and that effort we put in, living with HIV is a chronic disease,” Woodcock said. “That’s where we’d like, as a step one, to get some of these neurodegenerative diseases.”
At the same time, the FDA cannot allow therapies on the market where efficacy is unknown because “that’s not how medical science really gets to a point that really helps people like we have with HIV, like we’re getting there with cancer,” Woodcock said.
Aduhelm Approval Encourages More Drug Development, Senator Says
The comments are telling in the in the wake of the FDA’s accelerated approval of Biogen, Inc.’s Aduhelm (aducanumab) as an Alzheimer’s treatment despite questions about its efficacy. Woodcock met a largely friendly subcommittee on the subject, despite the substantial amount of criticism from stakeholders and others about the approval.
In fact, Sen. Jerry Moran, R-KS, complimented and thanked the agency for making the drug available.
“I’m convinced that with more timely approvals, that there will be more private investment in research to find these life-saving and life-improving drugs,” he said.
“A lot of research goes on elsewhere and the reward for that research comes in a drug that is approved for the market,” Moran said. “So sending a message to the market that there is a process by which there is a reward, a return on investment, is very valuable, I suppose you could say to the company, but much more important to me is to individuals who suffer from Alzheimer’s and their families.”
Indeed, patients and advocacy groups heavily lobbied the FDA for Aduhelm’s approval, including after a largely negative FDA advisory committee meeting. (Also see "Patient Support May Have Helped Push Aduhelm Toward Approval" - Pink Sheet, 7 Jun, 2021.)
The reward for research that Moran discussed is among the problems stakeholders have raised with the product. Its $56,000 per year wholesale acquisition cost could cripple health care budgets. (Also see "All Alzheimer’s Eyes On Medicare: CMS Will Take The Lead On Tailoring Access To Aduhelm" - Pink Sheet, 8 Jun, 2021.)
Senate Finance Committee members also discussed Aduhelm’s approval during a separate same-day hearing, but with a much more critical reaction to the price – and FDA leadership. (See sidebar.)
Budget Increases For Product Safety, Data Modernization Highlighted
The FDA requested $6.53bn in FY 2022, which would be a 7.9% increase over the total enacted for FY 2021. The agency wants $322m in additional budget authority, about a 9.8% increase, and $155.3m in additional user fee dollars, a 5.6% increase, over FY 2021.
Presidential budget requests typically amount to a wish-list for funding, but offer a glimpse at administration and agency priorities. Among them is the FDA’s intention to quickly ramp up its inspection program to more normal levels. (Also see "US FDA’s Inspection Volume Projected To Return To Normal Levels In FY 2022 As Backlog Looms" - Pink Sheet, 2 Jun, 2021.)
Woodcock said the domestic facility inspection program should be back to full capacity this summer, but foreign inspection work will continue to depend on the state of the coronavirus pandemic around the world.