Accelerated-Approval Changes Possible
Duane Wright, the primary analyst for this report.
Duane Wright
Team: Government
BI Senior Government Analyst
Lighter Accelerated-Approval Pathway Reforms Likely by Year End
Biogen, Roche and other drug makers should expect Congress to pass watered-down reforms to the accelerated-approval pathway during the lame duck session, leaving largely intact the program's foundations. More onerous reforms, like sun-setting accelerated approval, are likely off the table as Congress works toward a solution that favors industry. (10/19/22)
1. Watered-Down Accelerated-Approval Reforms Likely
Additional Reading:
CDER List of Accelerated-Approval Drugs and Biologics
Food and Drug Amendments of 2022
We give a 70% probability that Congress passes watered-down reforms to the accelerated-approval pathway during the lame duck session, which should largely favor industry in the long run by removing the overhang of more burdensome changes to the program. We anticipate legislation to require earlier design of confirmatory trials and provide FDA authority to require trials be underway at the time of approval. The legislation will likely allow drugmakers to use real-world evidence to support post-approval studies, filling evidence gaps during the confirmatory trial stage.
Even though Congress passed a FDA reauthorization bill over the summer, there is continuing support for modest reforms to the accelerated-approval pathway with a focus on drugs that spend years on the market without post-approval evidence. (10/19/22)
2. Significant Legislative Reforms Off the Table
Additional Reading:
Accelerated-Approval Payment Reforms
Accelerated-Approval Integrity Act of 2022
More onerous legislative reforms to the accelerated-approval pathway are likely off the table, maintaining a level of predictability for the program. Reforms such as automatic expiration of accelerated approval for drugs without completed confirmatory trials, or granting FDA authority to withdraw approval of an accelerated-approval product that fails to meet enrollment targets, milestones, or timely study completion, aren't in play. The latter could have been a high bar for companies given the number of trials that fail to meet clinical trial-enrollment timelines -- 80% by some estimates -- and fail to meet clinical trial-enrollment targets -- almost 50%.
Limiting Medicaid or Medicare reimbursement until confirmatory trials are completed and traditional approval is granted also appears unlikely. (10/19/22)
3. Administrative Reforms Possible
Additional Reading:
Executive Order on Lowering Drug Prices
Comprehensive Plan for Addressing High Drug Prices
It's possible the Biden administration could enact regulatory reforms that use innovative payment arrangements for drugs approved through the accelerated pathway. We're keeping an eye on Biden's drug pricing Executive Order (EO) that requires the Centers for Medicare and Medicaid Innovation to outline payment models that will test the feasibility of lowering costs for innovative drugs used in Medicare and Medicaid. Though no new authority is granted in the EO, it signals additional steps the administration may take to lower drug costs that go beyond the drug-pricing provisions in the Inflation Reduction Act. We anticipate the recommendations to be smaller scale in nature, with a potential focus on drugs approved through the accelerated-approval pathway by linking reimbursement to outcomes. (10/19/22)
