Advancing Treatments for Rare Diseases

November 09, 2011

The Congressional Rare Disease Caucus held a briefing in November 2011 to educate the staff of Members of Congress on the experiences of rare disease patients and their families, as well as rare disease treatment development.

Dr. Tim Cote, Chief Medical Officer of the National Organization for Rare Disorders (NORD) hosted the briefing. In his opening remarks, he discussed how orphan drug products are transformative for patients of rare diseases, and that they present a public health opportunity. The speakers that followed continued to highlight the importance of increasing the speed of achieving Food and Drug Administration (FDA) approval, and increasing incentives for pharmaceutical companies to invest in rare disease treatment research. Below is a list of the speakers followed by a summary of their remarks.

Ann Pariser, M.D., Associate Director for Rare Diseases, FDA, Center for Drug Evaluation and Research (CDER), Office of New Drugs, Rare Diseases Program – Outlined FDA initiatives to reduce the time it takes for an FDA approval for rare disease treatments, and demonstrated that as a result, more orphan drug products have been approved. These initiatives include:

  • Fast track designation
  • Accelerated approval
  • Priority review
  • Expanded access
  • Critical Path Initiative orphan drug designation
  • CDER Rare Disease Program (in development)

Steve Groft, Pharm.D. – Director, Office of Rare Diseases Research (ORDR), National Institutes of Health – discussed the Rare Disease Clinical Research Network which has increased the number of researchers with experience collaborating in multi-center and international research projects.

Parents of children suffering from rare diseases discussed their experiences with treating their children for a rare disease, including developing their own molecular compounds because the medication wasn’t available due to lack of investment by pharmaceutical companies. They also discussed their ideas for increasing the speed of FDA approvals for rare disease drugs, such as establishing an Accelerated Approval process for Ultra-Rare Disease Treatments.

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