Overestimated treatment effects in randomised phase II trials: What's up doctor?
European Journal of Cancer
MetadataShow full item record
Michiels, S., & Wason, J. (2019). Overestimated treatment effects in randomised phase II trials: What's up doctor?. European Journal of Cancer, 123 116-117. https://doi.org/10.1016/j.ejca.2019.09.023
Phase II clinical trials of experimental treatments play an essential role in drug development. Historically, trials were conducted sequentially, starting from a phase I trial in which dose and safety are evaluated advancing to phase II, looking for some sign of efficacy on a short-term end-point such as tumour shrinkage to screen out inefficacious experimental agents, and to large-scale randomised phase III trials evaluating properly the efficacy of a new treatment on objective clinical end-points. To expedite this process, phase II trials historically used single-arm designs to treat patients with experimental therapies only . However, the overall success rate of drug development programs in oncology from the start of phase I to registration has been estimated recently to be only 3.4%, much lower compared with that of other diseases . Part of this low success rate has been argued to be due to suboptimal use of randomised trial designs in the phase II space.
Cancer Research UK (18113)
Embargo Lift Date
External DOI: https://doi.org/10.1016/j.ejca.2019.09.023
This record's URL: https://www.repository.cam.ac.uk/handle/1810/298852
Attribution-NonCommercial-NoDerivatives 4.0 International
Licence URL: https://creativecommons.org/licenses/by-nc-nd/4.0/