Mapping benefits, risks, and challenges of expedited drug approval programs

Objective: This scoping review identified the benefits, risks, and challenges associated with expedited drug approval programs implemented by regulatory agencies. Methods: A systematic search was conducted in Embase, Scopus, PubMed, Lilacs, Cochrane, and Google Scholar, complemented by gray literature, to identify reviews published in English, Spanish, or Portuguese. Data on regulatory agencies, drugs, and approval programs were extracted. Results: Ten regulatory agencies were identified, and their expedited programs were grouped into regulated programs and exceptional-use programs. Regulated programs included priority review, flexible efficacy and safety requirements, accelerated development, collaborative review, and reliance on other agencies’ decisions. Exceptional-use programs addressed high unmet medical needs, public health emergencies, and negative impacts on healthcare coverage. The main benefit was the reduction in approval times (e.g., FDA: average 6.9 months). Among the risks, notable issues included uncertainty stemming from the use of surrogate endpoints, a higher incidence of problems in pivotal trial design (4.06 times more than in regular approvals), noncompliance with post-marketing study requirements after a median of 7.25 years, and the conversion of accelerated approvals into regular approvals without confirmed clinical benefit (79% in the case of the FDA). Challenges included persistent clinical uncertainty, confusion between “early approval” and “timely access”, high drug prices, poor cost–effectiveness ratios, conceptual high opportunity costs, and reliance on FDA/EMA decisions—an especially critical issue for middle- and low-income countries. Conclusion: Regulatory frameworks should optimize expedited approval processes to ensure genuine clinical benefit while safeguarding healthcare systems sustainability.