Sharpless Flags Patient Engagement for Rare Disease Therapies

Acting FDA Commissioner Ned Sharpless emphasized the agency’s commitment to patient engagement and data collection to promote rare disease therapies, in remarks at a National Organization for Rare Diseases (NORD) meeting in Washington, D.C.

“To most effectively support the development of treatments, patients should be involved in the development process to inform our understanding of any given rare disease,” he said, at NORD’s rare diseases and orphan products summit.

The agency is developing a series of four patient-focused drug development guidance documents that will “support systematic approaches for gathering useful patient and caregiver input,” he said.

Sharpless also stressed the importance of data collection in rare disease therapies, and said the agency is prioritizing data modernization and integration. “We must turn big data into big smart data,” he said.