(011) Does Expedited Regulatory Approval Predict the Therapeutic Value of Drugs? A Systematic Review

Background: Expedited approval pathways are used by regulators to introduce promising new drugs onto the market more quickly than the standard approval process would allow. While this is intended to give patients access to potentially improved or life saving therapeutic options earlier, there is some uncertainty about whether these pathways have been successful in approving drugs with additional clinical benefit and acceptable safety profiles.

Objectives: This review aims to examine the safety, effectiveness, and cost effectiveness of drugs approved via expedited approval pathways as compared with drugs approved by standard approval pathways.

Methods: Observational studies were included if they evaluated drugs approved via expedited approval pathways, and assessed effectiveness, safety, or cost effectiveness. Medline, Embase, and Scopus were searched for articles published from January 1992 to May 2023. Risk of bias was assessed using the relevant checklists from the Joanna Briggs Institute.

Results: Forty-one articles were included. Most assessed oncology treatments and provisional approvals that were based on surrogate outcomes, phase II or interim phase III trial data. The US Food and Drug Administration was the most studied regulator, included in 23/41 (56%) articles, followed by the European Medicines Agency (15/41; 37%), Health Canada (9/41; 22%), and the Australian Therapeutic Goods Administration and Swissmedic included in one article each. Drug effectiveness was the most common outcome (15/41; 37%). Results were mixed, with some drugs failing to demonstrate safety and effectiveness advantages in a post-approval setting.

Conclusions: Expedited pathways aim to bring potentially important or life-saving treatments to patients faster. However, research on post-approval outcomes has shown that treatments approved via these pathways frequently are no better than what is currently available, and that uncertainties may remain about effectiveness. These results call into question the criteria used by regulators to grant rapid approval, as many of the medicines approved via these pathways have not lived up to expectations of substantial health benefits.