Rare Disease R&D is Active Development Area: Tufts

The Tufts Center for the Study of Drug Development says rare disease drug development is one of the most active and fastest growing areas in drug research and development, with nearly one-third of all products in the global R&D pipeline targeting treatments for rare disease indications. But the latest Tufts Impact Report (subscription or purchase required) says that rare disease development “presents scientific and operational challenges that will necessitate novel clinical development strategies, operating procedures, and solutions.”

The report shows that the share of new drug approvals for rare diseases in 2018 has doubled since 2010. Some one-third of all drugs in active R&D worldwide target rare diseases, defined as medical conditions affecting 200,000 or fewer people in the U.S. Tufts says this represents nearly 3,500 small and large molecules targeting rare diseases in 2018, more than double the 1,530 in 2010.

More than half (58%) of all new drugs and biologics approved by FDA in 2018 were for rare diseases, up from one-third only a few years ago, the report says.

In the last 25 years, Tufts reports, there was a strong upswing in the number of FDA orphan drug designations, going from 301 in 1994-98 to 1,800 in 2014-18. In that same time period there was a significant increase in orphan drug approvals, from 66 in 2003-08 to 316 in 2014-18.

Sustained R&D investment in rare diseases is driving later stage clinical trial activity, Tufts says. Between 2014 and 2018, nearly 4,100 FDA-regulated clinical trials were initiated worldwide for rare disease treatments. Some 84% of all new clinical trials initiated for rare diseases are now in Phase 1 and 2 clinical development. Phase 2 and Phase 3 clinical trial starts for rare diseases are increasing at twice the rate as those for Phase 1 trials.

The report also finds that overall development durations for rare diseases take four years longer than for all other diseases. Rare disease clinical durations take 131 months on average, 68% longer than for non-rare diseases and 41% longer than for all cancer-related diseases. However, regulatory review durations are four months faster for rare disease drug applications, compared to review time for non-rare diseases.

Tufts also says that the size and scope of clinical trials varies widely by phase for rare versus non-rare diseases, and that patient enrollment for rare disease trials is harder, but retention rates are higher.