We found a match
Your institution may have rights to this item. Sign in to continue.
- Title
Improving Precision Oncology through Better Designs and Reporting of Biomarker-Driven Randomized Clinical Trials.
- Authors
LoRusso, Patricia M; Freidlin, Boris
- Abstract
Of these biomarker-positive/overall trials, 17 reported positive results for the overall population with 8 showing modest-to-no effect in the biomarker-negative subgroups (hazard ratio [HR] > 0.8): these 8 trials recommended treatment for biomarker-negative patients. It is well known that the biomarker-positive/overall design may result in misguided treatment recommendations, specifically recommending ineffective treatments to the biomarker-negative patients as the overall population results may be driven solely by the biomarker-positive patients ([[1], [3], [5]]). An often used, yet less transparent and indirect approach, is the so-called biomarker-positive/overall design that (in its simplest form) is based on evaluating treatment effect in the biomarker-positive and the overall population (with a treatment recommendation for the biomarker-negative subgroup based on the overall population results). Going forward, however, it is imperative, and should be strongly encouraged by regulatory agencies, that future biomarker-validation studies use biomarker-stratified designs to properly validate the predictive biomarkers by providing reliable treatment-effect estimates in each relevant biomarker subgroup separately.
- Subjects
CLINICAL trials; HEAD &; neck cancer; HORMONE receptor positive breast cancer; IMMUNOTHERAPY; ONCOLOGY
- Publication
JNCI: Journal of the National Cancer Institute, 2023, Vol 115, Issue 2, p122
- ISSN
0027-8874
- Publication type
Article
- DOI
10.1093/jnci/djac212