October 15, 2018
“The FDA is committed to helping advance the development of treatments for rare diseases, where there remain many unmet medical needs. Developing a drug or biologic for a rare disease can be especially challenging. It’s important that the agency continues to provide clear information to drug developers so that they can engage in productive conversations with the FDA early in the process,” said FDA Commissioner Scott Gottlieb, M.D. “The draft guidance issued today will assist sponsors of drug and biological products for the treatment of rare diseases in conducting more efficient and productive pre-investigational new drug application meetings. The draft guidance discusses important issues commonly encountered in the early phases of rare disease drug development. 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. 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. By providing greater clarity and direction to drug developers, we hope to help reduce the barriers of bringing new treatments to patients with rare diseases.”
The FDA today issued a draft guidance for industry, Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings, to assist sponsors of drug and biological products for the treatment of rare diseases in conducting more efficient and productive 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 The draft guidance specifically discusses issues commonly encountered in the early phases of rare disease drug development.
The draft guidance clearly lays out the topics that should be covered in a pre-IND meeting between the FDA and a sponsor and addresses the following important topics:
Regulatory considerations across various FDA disciplines including product quality, nonclinical, clinical pharmacology and clinical.
Additional considerations including use of expedited programs for serious conditions, combination products/companion diagnostics, orphan drug incentives, pediatric studies and data standards.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.