Trends in the design of FDA registrational randomised clinical trials in haematology and oncology: a 20-year cohort study
Most clinical trials leading to FDA approval for cancer-directed therapies employ escalation designs with composite time-to-event endpoints. Designs reflecting paradigm shifts in therapy (in-class switches and out-of-class switches) are sparsely used. This approach raises the concern that trials may increase therapeutic toxicity without delivering meaningful improvements in overall survival or quality of life.
Authors
- Charles Milrod (ORCID: https://orcid.org/0000-0002-2305-6299)
- Агеносов Михаил Петрович (ORCID: https://orcid.org/0009-0006-8236-5952)
- Wayne H. Liang (ORCID: https://orcid.org/0000-0003-2354-9787)
- Jeremy L. Warner (ORCID: https://orcid.org/0000-0002-2851-7242)
- Elizabeth J. Davis (ORCID: https://orcid.org/0000-0001-7626-2435)
- Carolyn Petersen (ORCID: https://orcid.org/0000-0002-2136-1273)
- Talal Hilal (ORCID: https://orcid.org/0000-0001-8249-4128)
- Sam Rubinstein (ORCID: https://orcid.org/0009-0006-9258-3873)
- Peter J. Yang (ORCID: https://orcid.org/0000-0003-4820-9543)
Institutions
- Northside Hospital (US)
- Mayo Clinic (US)
- University of North Carolina Health Care (US)
- Brown University (US)
- Massachusetts General Hospital (US)
- Mayo Clinic in Florida (US)
- Children's Healthcare of Atlanta (US)
- Vanderbilt University Medical Center (US)
Publication Details
- Journal
- BMJ Connections Oncology
- Published
- 2025-12-01
- DOI
- https://doi.org/10.1136/bmjconc-2025-000063
- Primary Topic
- Statistical Methods in Clinical Trials
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Institutes of Health