Costs and benefits of early access to new cancer drugs through the US Food and Drug Administration’s accelerated approval pathway: retrospective observational study and economic evaluation
This study examined the trade-off between the benefits of earlier access to cancer drugs through the FDA's accelerated approval pathway and the uncertainty surrounding their clinical efficacy at the time of market entry. Accelerated approval yielded uneven survival returns, with a small number of drugs accounting for the majority of life year gains. Medicare incurred substantial costs for treatments that, in over half of cases, did not provide an overall survival benefit to patients.
Authors
- Anita Katharina Wagner (ORCID: https://orcid.org/0000-0002-1069-6668)
- Rebecca Costa
- Joseph S. Ross (ORCID: https://orcid.org/0000-0002-9218-3320)
- Peter J. Murphy (ORCID: https://orcid.org/0000-0001-8864-1416)
- Huseyin Naci (ORCID: https://orcid.org/0000-0002-7192-5751)
- Yichen Zhang (ORCID: https://orcid.org/0000-0001-7093-1044)
- Mahnum Shahzad (ORCID: https://orcid.org/0000-0002-7170-7228)
Institutions
- Transparency International (CZ)
- Yale New Haven Hospital (US)
- Harvard University (US)
- Peking University (CN)
- Royal Pharmaceutical Society (GB)
- Yale University (US)
- Harvard Pilgrim Health Care (US)
- Yale New Haven Health System (US)
- University of York (GB)
- London School of Economics and Political Science (GB)
Publication Details
- Journal
- BMJ Medicine
- Published
- 2025-12-01
- DOI
- https://doi.org/10.1136/bmjmed-2025-001934
- Primary Topic
- Health Systems, Economic Evaluations, Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- London School of Economics and Political Science
- King's College London