Single-Arm Trials Can Provide Randomized Real-World Evidence: The Random Invitation Single-Arm Trial Design
They show how RISATs allow fairer access to participation, improve applicability and generalizability of results compared with traditional single-arm trials, and provide a form of randomized real-world evidence. At negligible added cost beyond already existing infrastructure, this approach may catalyze the early generation of evidence of higher value than that produced with traditional single-arm trials, increasing the credibility and validity of accelerated drug approval processes and enabling better health care decisions.
Authors
- Benjamin Kasenda (ORCID: https://orcid.org/0000-0003-0110-8585)
- Perrine Janiaud (ORCID: https://orcid.org/0000-0001-7684-8014)
- Steven N. Goodman (ORCID: https://orcid.org/0000-0002-3872-5723)
- Atle Fretheim (ORCID: https://orcid.org/0000-0002-6268-7966)
- John P. A. Ioannidis (ORCID: https://orcid.org/0000-0003-3118-6859)
- Lars G. Hemkens (ORCID: https://orcid.org/0000-0002-3444-1432)
Institutions
- Norwegian Institute of Public Health (NO)
- University Hospital of Basel (CH)
- Stanford University (US)
Publication Details
- Journal
- Annals of Internal Medicine
- Published
- 2025-07-14
- DOI
- https://doi.org/10.7326/annals-24-02979
- Primary Topic
- Statistical Methods in Clinical Trials
- Type
- article
- Field-Weighted Citation Impact
- 0.00