Scientific Advances in Adaptive Clinical Trial Design

DESCRIPTION (provided by applicant): It is widely accepted that the biomedical research community is seeking dependable and reliable ways to improve clinical trial research. Adaptive clinical trial designs provide the flexibility to adjust trial procedures based on data reviewed at interim stages. However, the wide variety of proposed and/or in-use modifications has met with varying levels of support. Hence, such designs present both opportunity and challenge to the critical task of testing new human therapeutics. Industry clinical investigators are actively exploring the benefits and issues associated with use of adaptive designs. For example, a Pharmaceutical and Manufacturers of America (PhRMA) working group has engaged FDA and other regulatory agencies in a series of discussions about adaptive designs. Due to this industry-wide interest and a growing body of science, FDA plans to publish draft guidelines on adaptive trials in fall 2008. It is vital to bring the perspective of those working on publicly funded clinical trial research to this important conversation. An NIH-focused workshop on adaptive designs and how their use in this setting differs from in an industry setting is timely and important. The workshop, Scientific Advances in Adaptive Clinical Trial Design, will provide an important forum for bringing together NIH researchers from across the Institutes and Centers with representatives from the Food and Drug Administration (FDA), as well as professional societies and patient advocates to explore the potential utility of adaptive designs. The 11/2 -day workshop, purposely limited to 50 attendees, is designed to be a springboard for action. The attendees, "operational" experts in clinical trial research, will be introduced through presentations and Q&A sessions to definitions, case studies, and perspectives of various stakeholders. They will then be challenged in a half-day of breakout sessions to explore the short- and long-term options for use of adaptive designs. Through proceedings, journal articles, a video archive of the presentations, and a dedicated Web site on adaptive designs, the workshop will extend beyond the initial dialogue and create a foundation for future NIH action. A preliminary Web site with references is already available and will be an ongoing resource for NIH-funded investigators and others. PUBLIC HEALTH RELEVANCE: This collaborative workshop on adaptive clinical trial designs offers a forum for NIH and academic communities to explore ways of improving clinical trial research by reviewing data at multiple points during the trial, sometimes within shortened time frames and with more efficient use of fewer resources. If it's possible to overcome the real and perceived obstacles to these innovative design concepts, the benefit to public health will be readily measurable. This workshop is a needed step in that direction.