Join Dr. Janet Woodcock and Dr. Marshall Summer as they discuss their two review papers in Clinical and Translational Science on rare disease trial design: “Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model is Crucial” and “The Placebo Effect in Rare Disease Clinical Trials: Measurement Impact and Statistical Approaches for Patient as Own Control Designs.” They delve into why traditional randomized controlled trials can be infeasible for small, heterogeneous rare disease populations, how patient-as-own-control and other alternative statistical designs can add rigor while reducing false negatives, as well as practical ways to address placebo effects, bias, and endpoint selection, emphasizing natural history studies and patient-centered input. You can find more information about The Haystack Project mentioned in this episode here: https://haystackproject.org/.
