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User’s Perspective on Use of External Information for Rare Disease Clinical Trials (304251)

Keywords: Historical Information; Bayesian Framework; Rare Diseases; Clinical Trials

Rare disease clinical trials have several challenges that make them more difficult to conduct as compared to clinical trials entailing common diseases. Small patient populations and the desire by patients to receive active therapy while on a clinical trial make it much more likely that rare disease trials be conducted without placebo control groups. An alternative to a placebo control group is the use of existing information on a group of similar patients to provide a historical control. Usage of external but relevant information, not only reduces the cost of the clinical trial, but also helps to increase the speed of the trial allowing for estimating safety and efficacy with fewer resources. Regulatory agencies are increasingly interested in utilizing innovative methods in design and analysis of rare diseases trials. While there are many available methods, including Bayesian framework, to combine information from historical control, each approach has its own advantages and disadvantages. In this presentation we compare a few different methods both in the presence of current control and without and provide a user’s perspective on few different scenarios.