Challenges of Rare Disease Drug Development Necessitate New Strategies, Report Says

Rare disease drug development now accounts for nearly one third of all drugs in active R&D worldwide, but challenges to developing these therapies will require the adoption of new strategies, according to the Tufts Center for the Study of Drug Development.

“Our research on rare disease development programs suggests that sponsor companies are encountering unprecedented operating challenges in this area,” said Ken Getz, associate professor and director of sponsored research at Tufts CSDD, who led the analysis. “Smaller market opportunities and longer development cycle times—driven in large part by difficulties identifying investigators and recruiting rare disease patients—will necessitate increased use of data and analytics and more flexible and mobile clinical trial models.”

In the center’s July/August 2019 Impact Report, it reports that new drug approvals for rare diseases doubled to 58 percent in 2018 from 29 percent in 2010. But the report finds clinical development through approval phase for rare disease drugs takes four years longer on average than those for non-rare diseases.

Phase 1 clinical trials for rare diseases, on average, engaged six times the number of investigative sites to recruit a quarter of the number of patients, compared with those for non-rare diseases.

Photo: Ken Getz, associate professor and director of sponsored research at Tufts CSDD

Author: Rare Daily Staff