Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD), discusses his organization’s position on the Orphan Drug Act. NORD is in the process of setting up a Task Force to look at the Orphan Drug Act and determine what, if any, changes may be needed to improve this important piece of legislation passed into law over 35 years ago.

Here are some of the highlights since the Orphan Drug Act was enacted include:

  • As of December 2017, FDA had approved more than 600 orphan products and 4,000 orphan drug designations since 1983.
  • The Orphan Drug Act has been credited with helping to drive innovation in cancer treatment, gene therapies, and has resulted in life-saving enzyme replacement therapies for children and adults with metabolic diseases for which there was previously no treatment.
  • People with rare diseases have new hope that medical researchers one day will develop a treatment for their disease, and hundreds of patient organizations have come into existence to advocate on behalf of the patient communities.
  • The Orphan Drug Act works by providing incentives to the pharmaceutical industry to invest in developing therapies to help small patient populations.
  • Market exclusivity: 7-year market exclusivity for approved orphan drugs or products for the orphan indication
  • Tax incentives: Companies have a tax incentive to invest in research as they can benefit from tax credits (Orphan Drug Tax Credit) for expenses occurred prior to the orphan designation
  • Clinical research subsidies: Orphan Product Grant program provides funding for clinical testing of new therapies to treat and/or diagnose rare diseases
  • User fee exemptions: Orphan drugs and products are exempt from the usual new drug application or “user” fees charged by FDA.
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