We are back with a rundown of pertinent developments in the healthcare and pharmaceutical space, this week through a regulatory and reimbursement lens. Some major developments are: lawmakers seeking to make changes to the FDA's Accelerated Approval Pathway, FDA advisory panel for Amylyx ALS drug and Gilead and J & J join the 340B dispute.
Lawmakers are facing off over the future of the FDA’s accelerated approval pathway, due to Biogen’s approval of its contentious Alzheimer’s drug, Aduhelm. A recent House Energy and Commerce Committee hearing, originally to highlight roughly 20 different health policy measures, became a battleground for the accelerated approval program. Accelerated approval allows drugs that treat serious conditions and fill an unmet medical need approval based on a surrogate or an intermediate clinical endpoint. Drugs that receive accelerated approval must undergo post-approval studies to confirm the intended clinical benefit. If the clinical testing does not demonstrate the intended clinical benefit, FDA has mechanisms to remove the drug from the market. Republicans, regarding Biogen’s approval as a triumph, are hoping to streamline the pathway for more approvals. Democrats are hoping to make it easier for the FDA to crack down on companies that don’t quickly follow up their initial, accelerated approvals with studies proving the drug’s benefit.
The Democratic bill, H.R. 6963, the Accelerated Approval Integrity Act of 2022, focuses on ensuring timeliness for confirming clinical benefit and increasing FDA discretionary authority for withdrawal of approval. The bill would impose a five-year limit for allowing drugs to stay on the market without confirming clinical benefit. Further, the bill would allow the FDA to withdraw approval status if a drug sponsor fails to: submit periodic progress reports for post-approval studies or achieve agreed upon enrollment targets, milestones, or timely post-approval study completion. The Republic bill, H.R. 6996, the Accelerating Access for Patients Act of 2022, in contrast to the Democratic legislation, focuses on the FDA implementing procedures to facilitate and enforce compliance with post-market study requirements. The bill would require the FDA to establish procedures regarding the requirement that drug sponsors develop a plan detailing how it will comply with accelerated approval requirement, issue guidance on for post-market study requirements; and require the FDA to approve or “specify changes” to a post-approval study protocol within 60 calendar days after the protocol is submitted.
It’s still not clear whether either accelerated approval bill will ultimately become law. However, the legislations’ inclusion in the hearing Thursday is a signal that lawmakers could tack either or both onto the forthcoming FDA funding agreements that Congress must reauthorize by the end of September. Several witnesses and lawmakers explicitly suggested Thursday that the legislation under consideration could pass alongside those agreements.
Less than a year following Aduhelm’s contentious approval, the FDA may soon approve another drug for a deadly neurodegenerative disease. The FDA is set to meet next week, March 30th to review evidence from a small, mid-stage study of Amylyx Pharmaceuticals’ drug for ALS, or amyotrophic lateral sclerosis. Regulators told Amylyx last year it would need to conduct a large, confirmatory study before seeking approval, but after months of intense lobbying by ALS patients and their representatives in Congress, the agency permitted submission of the drug based on the smaller study. The FDA traditionally requires two large, late-stage studies for approval. For deadly diseases like cancer, one study showing promising early results is often accepted. The FDA has approved only two therapies for ALS. The more effective one extends life by several months, and many experts say Amylyx’s drug is likely to show a similar benefit.
Gilead and Johnson & Johnson are the latest manufacturers to join the 340b dispute by cutting off sales of 340B-discounted products to covered entities’ contract pharmacies. 340B requires manufacturers to offer product discounts to safety net providers in exchange for participation in Medicare and Medicaid. However, the program’s integrity is under question due to contract pharmacy transactions that are not required to be consistent with the 340B statute. Manufacturers want claims level data as a contingency of contract pharmacies dispensing drugs, the claims data could be used to ensure aversion of duplicate discounts or diversion. Covered entities that provide claims level data, will be able to continue to use the contract pharmacies of their choice. Covered entities that decide not to participate in this integrity program, and do not have an in-house pharmacy, will have the option to select a single contract pharmacy location. There are currently 16 pharmaceutical companies restricting 340B discounts.