An article titled “Accelerated Approval as the New Norm in Gene Therapy for Rare Diseases” from DIA Global Forum digital magazine’s May 2025 issue explores the evolving landscape of regulatory pathways for gene therapies, particularly those targeting rare diseases. The piece highlights how accelerated approval processes, originally designed to expedite access to promising treatments for life-threatening conditions, are increasingly becoming standard practice in the gene therapy arena.
Rationale for Accelerated Approval
Gene therapies hold transformative promise for rare diseases that often lack effective treatments, but traditional drug approval pathways are challenging due to small patient populations and urgent unmet needs. Accelerated approval pathways, used by agencies like the FDA and EMA, allow earlier access by basing approval on surrogate or intermediate endpoints likely to predict clinical benefit, rather than long-term outcome data. This approach has enabled several gene therapies for conditions such as spinal muscular atrophy and inherited retinal diseases to reach patients sooner, often relying on biomarkers or functional improvements.
Challenges and Considerations
These approvals, however, frequently come with post-marketing requirements to confirm safety and efficacy in the real world. Additional challenges remain, including the validation of surrogate endpoints, the complexity of conducting confirmatory studies in rare populations, and the risk that real-world outcomes may differ from early predictions. Ensuring stakeholder collaboration and transparency is crucial to maintain high standards for safety and efficacy.
Regulatory Evolution and Future Outlook
Regulatory agencies are evolving their frameworks, embracing flexible trial designs, increased use of real-world evidence, and innovative long-term follow-up strategies. As a result, accelerated approval is becoming the primary pathway for gene therapies targeting rare diseases, balancing the need for timely patient access with robust oversight.
