The United States Food and Drug Administration (USFDA) has released (05 December, 2024) the draft guidance "Expedited Program for Serious Conditions – Accelerated Approval of Drugs and Biologics." This document outlines the FDA's policies and procedures for granting accelerated approval to drugs intended to address serious or life-threatening conditions. It also describes the conditions under which the approval may be expedited or withdrawn, aligning with regulatory updates from the Consolidated Appropriations Act, 2023.
Accelerated approval is one of the FDA's expedited programs aimed at addressing unmet medical needs in treating severe conditions. It allows for approval based on surrogate or intermediate clinical endpoints that are reasonably likely to predict clinical benefits. This pathway expedites access to therapies while ensuring post-approval studies confirm their effectiveness.
The purpose of this guidance is to provide information on FDA's policies and procedures for accelerated approval as well as threshold criteria generally applicable to concluding that a drug is a candidate for accelerated approval. Additionally, this guidance discusses the procedures for expedited withdrawal of approval of an approved product under accelerated approval and Congress's revisions through the Consolidated Appropriations Act, 2023.
The accelerated approval process includes several considerations:
Endpoints for Approval: Surrogate endpoints (e.g., biomarkers) and intermediate clinical endpoints (e.g., early disease responses) are crucial in demonstrating efficacy. Sponsors are encouraged to discuss novel endpoints with the FDA early in development.
Evidentiary Criteria: Approvals require substantial evidence of safety and effectiveness. The surrogate or intermediate endpoints must have a scientifically plausible connection to the clinical outcome.
Confirmatory Trials: Sponsors must conduct post-approval studies to verify clinical benefits, with trials ideally underway at the time of application. The FDA sets clear milestones for these studies, including enrollment targets and completion timelines.
Other Conditions: Drug labeling for accelerated approvals must include information on the drug’s limitations, and sponsors are required to submit promotional materials and periodic progress reports.
The FDA may expedite the withdrawal of an approval if:
Confirmatory trials fail to verify the anticipated clinical benefits.
The sponsor fails to conduct trials diligently.
Evidence demonstrates the drug is unsafe or ineffective.
The sponsor disseminates false or misleading promotional materials.
Confirmatory trials serve as a critical post-approval requirement, ensuring that drugs demonstrating preliminary benefits fulfill their clinical promises. Sponsors must develop these trials in consultation with the FDA, incorporating robust protocols and timely reporting mechanisms. Sponsors are encouraged to engage with the FDA early to address regulatory expectations, especially regarding endpoint selection and trial design.
References and Further Reading
Draft Guidance Document: Accelerated Approval – Expedited Program for Serious Conditions
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