Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy

Daniel Tobias Michaeli (), Thomas Michaeli, Sebastian Albers, Tobias Boch and Julia Caroline Michaeli
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Daniel Tobias Michaeli: National Center for Tumor Diseases, Heidelberg University Hospital
Thomas Michaeli: University Hospital Mannheim, Heidelberg University
Sebastian Albers: Klinikum Rechts Der Isar, Technical University of Munich
Tobias Boch: University Hospital Mannheim, Heidelberg University
Julia Caroline Michaeli: LMU University Hospital

The European Journal of Health Economics, 2024, vol. 25, issue 6, No 6, 979-997

Abstract: Abstract Background Over the past decades, US Congress enabled the US Food and Drug Administration (FDA) to facilitate and expedite drug development for serious conditions filling unmet medical needs with five special designations and review pathways: orphan, fast track, accelerated approval, priority review, and breakthrough therapy. Objectives This study reviews the FDA’s five special designations for drug development regarding their safety, efficacy/clinical benefit, clinical trials, innovation, economic incentives, development timelines, and price. Methods We conducted a keyword search to identify studies analyzing the impact of the FDA's special designations (orphan, fast track, accelerated approval, priority review, and breakthrough therapy) on the safety, efficacy/clinical benefit, trials, innovativeness, economic incentives, development times, and pricing of new drugs. Results were summarized in a narrative overview. Results Expedited approval reduces new drugs’ time to market. However, faster drug development and regulatory review are associated with more unrecognized adverse events and post-marketing safety revisions. Clinical trials supporting special FDA approvals frequently use small, non-randomized, open-label designs. Required post-approval trials to monitor unknown adverse events are often delayed or not even initiated. Evidence suggests that drugs approved under special review pathways, marketed as “breakthroughs”, are more innovative and deliver a higher clinical benefit than those receiving standard FDA approval. Special designations are an economically viable strategy for investors and pharmaceutical companies to develop drugs for rare diseases with unmet medical needs, due to financial incentives, expedited development timelines, higher clinical trial success rates, alongside greater prices. Nonetheless, patients, physicians, and insurers are concerned about spending money on drugs without a proven benefit or even on drugs that turn out to be ineffective. While European countries established performance- and financial-based managed entry agreements to account for this uncertainty in clinical trial evidence and cost-effectiveness, the pricing and reimbursement of these drugs remain largely unregulated in the US. Conclusion Special FDA designations shorten clinical development and FDA approval times for new drugs treating rare and severe diseases with unmet medical needs. Special-designated drugs offer a greater clinical benefit to patients. However, physicians, patients, and insurers must be aware that special-designated drugs are often approved based on non-robust trials, associated with more unrecognized side effects, and sold for higher prices.

Keywords: Orphan designation; Fast track; Accelerated approval; Priority review; Breakthrough therapy; Clinical trial; Innovation; US food and drug administration; European medicines agency; Drug development; Special designation; Safety; Efficacy; Healthcare policy; Pharmaceutical policy; Drug price (search for similar items in EconPapers)
JEL-codes: I00 I1 I13 I18 (search for similar items in EconPapers)
Date: 2024
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DOI: 10.1007/s10198-023-01639-x

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