Analysis of FDA's Accelerated Approval Program Performance December 1992-December 2021.

Area of Science:

• Pharmacoeconomics and Drug Approval Processes
• Regulatory Science and Policy

Background:

• The accelerated approval pathway allows earlier drug access based on surrogate endpoints.
• Criticisms include lower regulatory standards, delayed withdrawals, and inadequate confirmatory trials.
• This pathway has been in place for 30 years, requiring ongoing evaluation.

Purpose of the Study:

• To examine the status of confirmatory studies for drugs approved via accelerated approval.
• To assess the overall performance and success of the FDA's accelerated approval program over three decades.

Main Methods:

• Analysis of confirmatory study status for drugs approved between December 1992 and December 2021.
• Review of FDA actions regarding drugs failing to confirm clinical benefit.
• Evaluation of program performance based on conversion to traditional approval and trial completion.

Main Results:

• The accelerated approval program has demonstrated considerable success.
• Half of accelerated approvals were converted to traditional approval within a median of 3.2 years.
• Recent FDA actions indicate effective management of non-compliant drugs.

Conclusions:

• The accelerated approval program is largely successful and effectively managed.
• Proposed program changes should consider the cumulative 30-year experience.
• Focus should be on overall program performance, not isolated outlier cases.