Adaptation of Agile Methodologies to Rare Diseases Drug Development

Executive Summary

There are an estimated 7000 rare diseases (RD) that affect 25 million Americans, most of which have unmet medical needs. RD drug development presents many challenges, such as small populations for clinical study, poorly understood diseases, few disease experts, and financial impediments to investment, among others. New approaches to expedite RD research through basic and translational phases and into the clinic are needed, as well as novel clinical designs to assess the efficacy of candidate therapeutics. The IT industry has adopted “agile” software development practices that use incremental and iterative methods to encourage adaptive planning, evolutionary development, continuous improvement, and rapid and flexible response to change. We propose adapting agile practices from the IT industry to RD drug development.

Team Members

Anne Pariser (team lead), NIH / NCATS
Tiina Urv, NIH / NCATS

Milestones

August 2017: Project selected into the HHS Ignite Accelerator
September 2017: Time in Accelerator Began
December 2017: Time in Accelerator Ended