This issue of Clinical Trials contains the proceedings of the sixth in a series of symposia held at the University of Pennsylvania on current statistical issues in clinical trials. This symposium, held in spring 2013, focused on dynamic treatment regimes, a topic highly relevant to the ever-increasing concerns about evaluation of treatment strategies in chronic diseases.
The concept of dynamic treatment regimes is an entirely new way of approaching trial design. Instead of focusing on a question that may address only a very small window of time in a patient’s treatment, the dynamic treatment regime (or Sequential Multiple Assignment Randomization Trials (SMART)) considers long-term treatment strategies for individuals who may regularly require new treatment options over time. The goal is to optimize treatment for individual patients while allowing assessment of different strategies over time. Use of such designs in medical research is in its infancy; the most experience has been in the area of substance abuse, but it has potential applications in many areas of medicine, as discussed by the speakers. The presentations and panel discussions offer a very broad spectrum of viewpoints and emphases from both long-time and more recent researchers, and from methodologists as well as potential users of such study designs (see Table). Expert panels provided thoughtful commentary on the presentations, and there were provocative and insightful questions and comments from the floor; these discussions are included along with the papers.
Table.
2014 Conference on Emerging Statistical Issues in Biomarker Validation for Clinical Trials
| Faculty | Institution | Topic |
|---|---|---|
| Phil Lavori | Stanford University | Introduction to Dynamic Treatment Strategies and Sequential Multiple Assignment Randomization |
| Yingqi Zhao | University of Wisconsin-Madison | Estimation of optimal dynamic treatment regimes |
| Bibhas Chakraborty | Columbia University | Inference about the expected performance of a data- driven dynamic treatment regime |
| Discussion Panel | Keaven Anderson (Merck); Marshall Joffe (Penn); Michael Kosorok (UNC) | |
| Linda Collins | Pennsylvania State University | Optimization of behavioral dynamic treatment regimens based on the sequential multiple assignment randomized trial (SMART) |
| Erica Moodie | McGill University | Simulating Sequential Multiple Assignment Randomized Trials to Generate Optimal Personalized Warfarin Dosing Strategies |
| Kelley Kidwell | University of Michigan | SMART Designs in Cancer Research: Past, Present and Future |
| Discussion Panel | Christy Chuang-Stein (Pfizer); Dean Follman (NIAID); Estelle Russek- Cohen (FDA); Rick Chappell (Wisconsin) | |
| Closing Remarks | Susan Ellenberg (University of Pennsylvania) | |
The Penn conference series is aimed at facilitating advances in approaches to the statistical design, implementation and analysis of clinical trials. Earlier conferences in this series focused on proof-of-concept studies and ‘go–no go’ decision making [1], development of targeted therapies [2], comparative effectiveness studies [3], emerging statistical issues in the conduct and monitoring of clinical trials [4], and biomarker validation [5]
Acknowledgments
The Center for Clinical Epidemiology and Biostatistics at the University of Pennsylvania provided scientific and logistical support and the American Statistical Association and the Society for Clinical Trials provided in-kind support for the conference.
Funding
Funding for the conference was made possible (in part) by award CA132565-06 from the National Cancer Institute. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. Government.
Footnotes
Conflict of Interest
None declared.
References
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