DIA/FDA Biostatistics Industry and Regulator Forum

This session will provide an overview of challenges and opportunities for statisticians in the design and analysis of studies evaluating rare disease therapies.

Recent years have seen an increase in rare disease designations and therapies development for patients with rare diseases. There also is increased urgency to consider innovative methods to improve the development of treatments in this overwhelmingly unmet area. In his remarks in February 2018, FDA Commissioner Scott Gottlieb announced several initiatives to support research and development of therapies for rare diseases. “FDA is committed to do what we can to stimulate the development of more products by improving the consistency and efficiency of our reviews, streamlining our processes, and supporting rare diseases research.”

Statisticians have an opportunity and obligation to lead efforts cross-functionally and across various stakeholders toward improved and innovative designs and analysis methods to speed rare disease therapies to patients. Rare diseases present unique challenges and opportunities for statistical innovation. Some of these were recently discussed at the FDA-funded Duke-Margolis public workshop in March 2018. These include adapting innovations from other therapies to the rare disease setting such as using master protocols, using SMART trials, or borrowing data from non-randomized studies. They also include leveraging rich longitudinal information from disease registries and natural history studies to improve study design planning, recruitment, endpoint selection, and follow-up.