The House Energy and Commerce Committee seems poised to make substantial changes to the Food and Drug Administration’s (“FDA’s”) Accelerated Approval Program. The committee’s Democratic chairman, Frank Pallone, Jr. (D-NJ) and Republican ranking member, Cathy McMorris Rodgers (R-WA) have proposed competing bills that were featured prominently in the Health Subcommittee’s legislative hearing on March 17, 2022.
The Accelerated Approval Program was developed in 1982, largely in response to the HIV/AIDs epidemic, to expedite approval of novel drugs that treat serious conditions with unmet medical needs based on a surrogate endpoint. Drugs that receive accelerated approval must undergo post-approval (Phase IV) 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.
However, concerns have mounted regarding FDA’s ability to remove ineffective drugs from the market, and those concerns were punctuated during a February 3, 2022 Health Subcommittee hearing on the reauthorization of FDA User Fees. Dr. Patrizia Cavazzoni, the Director of the Center for Drug Evaluation and Research at the FDA testified that the program’s existing mechanism to withdraw accelerated approvals is cumbersome, resource intensive, and seldom used.
In response, both the Democratic and Republican leaders of the full Energy and Commerce Committee drafted separate bills proposing changes to the program, both of which would expand FDA’s authority to remove drugs that fail to satisfy post-approval testing requirements. Below is a summary of the competing bills and their key similarities and differences.
The Democratic bill, H.R. 6963, the Accelerated Approval Integrity Act of 2022
House Energy and Commerce Committee Chairman Frank Pallone, Jr. (D-NJ), sponsored H.R. 6963, the Accelerated Approval Integrity Act of 2022, which significantly enhances FDA’s ability to ensure that drugs receiving accelerated approval are providing a clinical benefit by way of new expedited procedures to remove drugs if such benefit is not demonstrated through post-approval studies. The bill would impose a five-year limit for allowing drugs to stay on the market without confirming clinical benefit.
All told, the bill sets forth six instances in which FDA may withdraw accelerated approval using such expedited procedures. For instance, FDA could withdraw a drug if the sponsor fails to conduct post-approval testing, or if the sponsor fails to achieve agreed upon study targets or fails to confirm clinical benefit.
Other changes include the following:
- codifying post-approval testing requirements imposed on drug sponsors, including the requirement to “conduct appropriate, adequate, and well-controlled post-approval studies to verify and describe the predicted effect on irreversible morbidity or mortality or other clinical benefit”;
- requiring that, prior to FDA granting accelerated approval, manufacturers enter into an agreement with FDA explaining how post-approval studies will be conducted (e.g., enrollment targets, milestones, and study design);
- allowing FDA discretion to require that post-approval studies are underway at the time of approval;
- requiring quarterly updates on post-approval studies, including updates on enrollment targets, milestones, and study design;
- detailing expedited procedures for withdrawing approval, which include notice, an opportunity for a written appeal to FDA, and an opportunity for public comment;
- specifying additional situations in which an accelerated approval can be withdrawn; and
- providing for automatic expiration of accelerated approval status if certain post-approval conditions are not met.
The Republican bill, H.R. 6996, the Accelerating Access for Patients Act of 2022
The House Energy and Commerce Committee’s Ranking Member, Cathy McMorris Rodgers (R-WA) has sponsored H.R. 6996, the Accelerating Access for Patients Act of 2022, a counter-proposal to the democratic bill, which grants the FDA the authority to use “expedited procedures” to withdraw a grant of accelerated approval for products that are not demonstrating clinical benefit to patients. However, the bill requires FDA promulgate regulations specifying the expedited procedures to withdraw accelerated approvals.
The bill also includes the following changes:
- requiring FDA to establish procedures regarding the requirement that drug sponsors develop a plan detailing how it will comply with accelerated approval requirements, which includes a “plan for reaching agreement” on the design of a post-approval study, developmental milestones, and a strategy for the inclusion of diverse populations;
- requiring FDA to issue guidance on “the use of novel clinical trial designs” that may be used to satisfy post-market study requirements; and
- requiring FDA to approve or “specify changes” to a post-approval study protocol within 60 calendar days after the protocol is submitted.
Key Similarities and Differences
As noted above, both bills grant FDA the authority to use expedited procedures to withdraw accelerated approval status if post-approval testing requirements are not satisfied, which include failing to conduct required post-approval studies with due diligence. However, the Democratic bill includes two additional conditions, which if not met, may warrant withdrawal of a product. As such, under that bill, FDA may withdraw approval status if a drug sponsor fails to: (i) submit periodic progress reports for post-approval studies; or (ii) achieve agreed upon enrollment targets, milestones, or timely post-approval study completion.
Unsurprisingly, the Republican bill is friendlier to drug manufacturers, as it imposes fewer obligations on drug sponsors with respect to post-approval studies and compliance. The Republican bill places the weight of responsibility on FDA to implement procedures to facilitate and enforce compliance with post-market study requirements.
The Democratic bill, meanwhile, more evenly divides responsibility between FDA and drug manufacturers with respect to post-approval study requirements. For example, the Democratic bill explicitly requires FDA and drug sponsors agree on the approach for post-approval before FDA can grant accelerated approval, while the Republican bill forces FDA to establish procedures regarding the creation of post-approval study plans. The Democratic bill also outlines the expedited procedures for withdrawing accelerated approval, while the Republican bill only requires that expedited procedures include an opportunity for an informal hearing and defers to FDA regarding creation of specific procedures for withdrawal.
Another key difference is that the Democratic bill provides for the automatic withdrawal of accelerated approval status unless the drug sponsor satisfies its post-market testing obligations, whereas the Republican bill does not include any type of automatic expiration.
It is unclear whether either bill will receive enough support to ultimately become law. However, the fact that both parties have acknowledged the need to modify the Accelerated Approval Program suggests this is only the beginning of efforts to refine the program.
Reed Smith will continue to track developments related to the FDA Accelerated Approval Program and this legislation. Please reach out to the health care attorneys at Reed Smith if you have any questions about this topic or other items related to FDA developments.