Rare Disease Report

FDA Commissioner Releases Statement Highlighting Plans for Rare Disease Research

FEBRUARY 14, 2018
Mathew Shanley
Late last night, U.S. Food and Drug Administration (FDA) Commissioner Scott Gottlieb released a statement regarding the Administration’s request for new FDA funding to “promote innovation and broaden patient access through competition.”

In the statement, he emphasizes that new scientific opportunities and advances in both manufacturing and commerce will allow for the FDA to find new ways to protect and promote public health. Leveraging these advances, he says, requires investments in regulatory science that will reassure innovators, spur investment in new industries, and provide principles for the safe and effective development of new technologies. These same advances will permit the FDA to support wider availability of treatment and use generic drugs to promote price competition and patient access.

As it pertains to rare disease, Gottlieb hopes to stimulate both investment in and innovation of medical products targeted toward the space. His hope is that the FDA can develop clinical trial networks to create an understanding of the natural history and clinical outcomes in several uncommon conditions.

“The initial focus would be on rare and ultra-rare diseases, where product development can be challenging because of the difficulty of recruiting clinical trials,” he said in his statement. “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. 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.”

Since the FDA appointed Gottlieb as the FDA Commissioner in May 2017, he has made his voice heard throughout the rare disease community. He has actively made plans to streamline the orphan drug review process, and prioritized removing the backlog of orphan drug designation requests.

Other points addressed in Gottlieb’s most recent statement include:
  • Promote Domestic Manufacturing: Advancing Modern Drug and Biological Product Manufacturing Technologies Through the Development of Efficient Regulatory Pathways
  • Advance a New Domestic Drug Industry and Promote Access by Establishing the Outsourcing Facility Sector as a Robust and Reliable Source of Compounded Products
  • Bring MedTech Manufacturing Home: Advance Medical Device Manufacturing and Quality
  • Create a New Medical Data Enterprise: Advance the Use of Real-World Evidence to Improve Human and Animal Health and Support Pre-Market Evaluation and Post-Market Safety
  • Facilitate Growth and Spur Transformation of the Digital Health Technology Industry by Shifting Regulation to an Efficient and Novel Framework for Reliable Post-Market Oversight
  • Create a New Platform for How the Agency More Efficiently Develops and Validates Modern Science-Based Principles for New Drug Development and Shares this Information with Innovators
  • Modernize Generic Drug Development and Review to Enable Increased Competition, Promote Generic Drug Substitution and Provide Affordable Options for American Patients
“As I've previously noted, our work at the FDA is taking place during an inflection point in both science and policy,” Gottlieb concluded. “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.”

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