Publication TypeJournal Article
Year of Publication2015
AuthorsIvanova, A, K M. Anderson, Gary L. Rosner, and E Rubin
JournalStat Biopharm Res
Date Published2015

We congratulate the authors on their comments on innovative approaches to drug development that fall out of the traditional mold and may result in more quickly bringing safe and effective treatments to patients. Changes in the overall clinical develop approach are most relevant to "breakthrough" therapies, which have generally yielded exceptional efficacy data in early clinical studies, motivating exploration of accelerated development and regulatory approaches, as well as a potential ethical need for crossover upon progression in randomized controlled studies (Horning et al., 2015). As is clear from the manuscript, it will be important to develop an understanding of what works well and where the pitfalls in new approaches are. We comment briefly on the four topics mentioned by the authors, combining comments on items 2 and 3: 1) non-proportional hazards, 2) interpretability of extended Phase I trials, 3) single-arm trials as a basis for approval, and 4) recent innovations in trial design.

Alternate JournalStat Biopharm Res
Original PublicationCommentary on "Current Statistical Challenges in Oncology Clinical Trials in the Era of Targeted Therapy" by R. Sridhara, K. He, L. Nie, Y.L Shen, and S. Tang.
PubMed ID30416674
PubMed Central IDPMC6223633
Grant ListP01 CA142538 / CA / NCI NIH HHS / United States