FDA Issues Draft Guidance to Support Development of Rare-Disease DrugsBy
The US Food and Drug Administration (FDA) has issued a draft guidance to help companies developing drug products for rare diseases conduct more efficient pre-investigational new drug application (pre-IND) meetings. The draft guidance specifically discusses issues commonly encountered in the early phases of rare disease drug development.
The draft guidance, Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings, lays out the topics that should be covered in a pre-IND meeting between the FDA and a sponsor. It also addresses the following topics: regulatory considerations across various FDA disciplines, including product quality, nonclinical, clinical pharmacology, and clinical; and additional considerations, including use of expedited programs for serious conditions, combination products/companion diagnostics, orphan drug incentives, pediatric studies, and data standards.
“Although these issues are encountered in other drug-development programs, they’re frequently more difficult to address in the context of a rare disease than in the setting of a common disease where there’s typically more widespread medical experience,” said FDA Commissioner Scott Gottlieb, in an October 15, 2018 agency statement. “Our experience shows that early and substantive engagement with FDA review teams can help increase the quality of applications and the odds of success with development programs. We encourage sponsors to engage the agency early in the regulatory process, especially when it comes to drugs targeted to rare conditions.”