US FDA Accelerated Approval for 48 Non-oncology Drugs

The FDA approval process is multi-faceted and involves several aspects. One of these aspects is accelerated approval, which is meant to approve drugs to treat severe or fatal diseases. According to a JAMA Network Open publication, between 1992 and 2018, the FDA approved 48 non-oncology drugs despite later required amendments and warnings.

Researchers in the publications state, “in 1992, Congress authorized the creation of the US Food and Drug Administration (FDA) accelerated approval program to address unmet medical needs via rapid drug development in the wake of the HIV/AIDS crisis. Under the program, drug approval can be based on surrogate measures deemed reasonably likely to project actual clinical endpoints (e.g., symptom change and mortality), including intermediate clinical endpoints and biomarkers.”

This study was classified as a retrospective cohort study and used data from June 1992 to May 2018, made public by the FDA. Researchers looked at the time difference between accelerated approval and any regulatory outcomes issued.

Investigators found that the 48 drugs granted accelerated approval for non-oncological indications addressed 57 conditions, and approximately 40% of those approved drugs were for HIV treatment.

Of the 57 non-oncology indications, 75% obtained regular approval, with the average approval time being approximately 53.1 months following accelerated approval. Additionally, nearly half of the indications required a post-approval label modification, which occurred, on average, over 20 years later.

Approximately 20% of indications granted accelerated approval that had required confirmatory trials did not meet post-approval requirements.

Based on the data collected, researchers in the publication were able to conclude that “although the program expedited the approval of non-oncology drug indications by a median (IQR) of 53.1 (26.8–133.2) months, safety-related label modifications were often added in boxed warnings after approval, and clinical efficacy was sometimes not confirmed. The study findings and long follow-up period suggest that comprehensive evaluation of such drugs may take more than a decade.”

Based on these results, the FDA should consider stricter evaluation of drugs and their indicated uses prior to granting accelerated approval. Prescribers and drug distributors should clarify to patients taking these medications that the approval was accelerated. They should monitor patients closely and report any adverse side effects to the FDA and the drug manufacturer.