Gottlieb Highlights New FDA Rare Disease Efforts to Address Rare Disease Challenges

Rare Daily Staff

U.S. Food and Drug Administration Commissioner Scott Gottlieb in a blog entry today outlined several steps the agency is taking to “support and expedite” the development of rare disease products ahead of World Rare Disease Day.

“FDA is committed to doing its part to facilitate continued progress toward more treatments and even potential cures for rare diseases,” Gottlieb said. “New scientific opportunities enabled by advances in cell and gene therapy hold out more opportunities to develop these potential cures. With efficient regulation, proper incentives for product development, and the continued support of patients, providers, and innovators; we’re more able to pursue these opportunities than ever before.”

Gottlieb announced a new pilot program for more efficient orphan designation requests. This includes a new fillable form that he said will make the submission process easier for sponsors to complete designation requests and make it more efficient for the FDA to review. There is also an online tutorial to guide sponsors through the submission process. The agency has also developed a new inter-center consultation process to streamline and standardize its communications process.

The Commissioner separately announced a new memorandum of understanding with the National Organization for Rare Disorders to conduct outreach with FDA’s new Patient Affairs Staff on ways to enhance the incorporation of patient experience into regulatory discussions. As part of that process, the agency is planning a joint series of pilot listening sessions on rare diseases.

“We recognize that early and iterative engagement can improve clinical and regulatory understanding of diseases and conditions, provide a common understanding of the most urgent patient needs, and inform drug development programs,” he said.

Last, Gottlieb noted the agency is planning a public meeting to help it prepare for the changing landscape of orphan drug development posed by the growth in targeted therapies and molecularly defined diseases. It will seek input on complex scientific and regulatory issues related to cancer drugs and biologics that target a tumor’s specific genetic features rather than its location in the body. As part of that, it will also consider the appropriate application of orphan incentives to this new paradigm of drug development, and how we apply designations to these indications.

The agency has set up a webpage to highlight the scope of FDA’s rare disease activities.

February 26, 2018
Photo: FDA Commissioner Scott Gottlieb