Pages Menu
Categories Menu

October 2018 Newsletter: Advocacy

October 2018


Marc C. Patterson, MD, FRACP, FANA, FAAN, Professor of Neurology, Pediatrics and Medical Genetics at Mayo Clinic College of Medicine and member of NNPDF’s Advisory Committee recently testified at the U.S. Senate Committee Hearing on Health, Education, Labor, and Pensions. At the hearing entitled Rare Diseases: Expediting Treatments for Patients Dr. Patterson stressed the importance of accelerating the approval of drugs by the FDA to treat rare and ultra-rare diseases.

Recommendations for specific measures regarding drug approval for rare and ultra-rare diseases to provide FDA regulators with a more refined set of tools to benefit the under-served population included:

  1. Requiring the FDA to accept alternative study designs that are better suited for these small, inhomogeneous, populations.
  2. Requiring the FDA to accept the results of well-conducted clinical trials supervised by national regulatory agencies outside the United States, or by such agencies acting in concert with the FDA.
  3. Requiring the FDA to work with national patient organizations, academic medicine, industry and other international regulatory agencies, to develop disease registries, ideally patient owned and managed, containing secure, professionally entered and patient/parent entered data, which will be used to enhance understanding of natural history, to develop outcome measures, and to support clinical trials.

To watch Dr. Patterson’s recorded testimony click here. To view the complete written testimony click here.

To make your voice heard on this issue contact your legislator!