Gottlieb Calls for $400 Million in Expanded FDA Funding in Part to Stimulate Investment in Rare Diseases

Rare Daily Staff

U.S. Food and Drug Administration Commissioner Scott Gottlieb called for developing clinical trial networks to improve the understanding of the natural history and clinical outcomes of rare diseases as part a series of initiatives that would be supported by an estimated $400 million increase in the agency’s funding under the Trump administration’s newly released budget requests

Gottlieb outlined a series of initiatives the additional funding would help support in a statement released on the FDA’s website.

With regard to the development of clinical trial networks to create natural histories and clinical outcomes of rare diseases, Gottlieb said the initial focus would be on rare and ultra-rare diseases where product development can be challenging because of the difficulty of recruiting patients for clinical trial.

“The FDA has already invested, on occasion, in the development of natural history models for a small subset of rare diseases and has expertise in this area,” he said. “The FDA would stimulate medical product development for rare diseases by expanding and enhancing the understanding of rare diseases and the research and drug development processes in this space.”

The other measures outlined in the statement include a range of initiatives that Gottlieb said would support novel medical technology, address public health priorities, and promote price competition and patient access. They include efforts to advance manufacturing technologies, advance the use of real-world evidence, facilitate the growth of digital health technology, modernize generic drug development and review, and create new ways to develop and validate new drug development.

“As I’ve previously noted, our work at the FDA is taking place during an inflection point in both science and policy,” said Gottlieb. “There’s perhaps never been a better moment in the history to be engaged in public health, and to be leveraging the capabilities of the FDA to support new investment and product innovation.

February 13, 2018
Photo: Scott Gottlieb, FDA Commissioner