Rare Daily Staff

The U.S. Food and Drug Administration issued a draft guidance addressing the role of meetings with the agency prior to filing an application to begin human clinical trials in the hopes of providing greater direction to drug developers that could help reduce the barriers of bringing new rare disease therapies to market.

A drug developer must submit an Investigational New Drug (IND) application and obtain agency approval prior to starting human clinical trials. A so-called pre-IND meeting is often the first communication between a drug developer and the FDA regarding the development program for an experimental drug or a new indication for an approved drug.  During pre-IND meetings, sponsors can discuss with the agency the unique challenges of rare disease drug development and where regulatory flexibility can be justified.

Developing drugs for rare diseases can be particularly challenging because of the small patient populations and the limited understanding there may be of a condition. Producing statistically significant results and finding meaningful endpoints can be a point of contention between regulators, researchers, drug developers, and the patient community.

Earlier this month the issue of the need for more frequent and earlier communications between the FDA and rare disease drug developers became an issue of concern during a U.S. Senate subcommittee hearing. Mallory Factor, CEO of IntraBio, discussed his testimony on this week’s RARECast podcast.

The new draft guidance describes frequently encountered issues to consider in early drug development and pre-IND meetings including topics related to pharmaceutical quality, nonclinical evaluation, clinical pharmacology, and clinical development including early phase study designs and statistical analysis plans.

“The draft guidance issued today will assist sponsors of drug and biological products for the treatment of rare diseases in conducting more efficient and productive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,” said Scott Gottlieb, commissioner of the FDA. “The draft guidance discusses important issues commonly encountered in the early phases of rare disease drug development.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.”

The agency is seeking comments on the draft guidance for the next 60 days.

October 17, 2018
Photo: FDA Commissioner Scott Gottlieb

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