Washington, D.C., August 21, 2017 -- Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, today issued the following statement in response to the signing by President Trump of the Food and Drug Administration Reauthorization Act of 2017:
“We thank President Trump for signing the Food and Drug Administration Reauthorization Act (FDARA) into law. This law makes it possible for FDA to continue to review orphan drugs, biologics, and medical devices expertly, quickly, and safely. The law also includes several critical improvements to FDA's review of orphan therapies and medical devices. Several provisions of the new law deal specifically with rare diseases, including the expansion of the FDA Rare Diseases Program; strengthening of the expedited review pathway for breakthrough therapies; development of a process through which real-world evidence can be utilized; continuation of the Patient-Focused Drug Development program; and the creation of the National Evaluation System for health Technology (NEST).
For over two years, NORD has played a central role in ensuring individuals with rare diseases are benefited by the reauthorization of the FDA user fee programs. Starting with testifying at the FDA's July 2015 public meeting that launched the reauthorization process, NORD has worked tirelessly in partnership with the FDA, our Congressional champions, key industry members, and our patient organization colleagues to ensure this law was enacted. We are incredibly thankful to all our partners in this effort, and we look forward to continuing our collaboration as the FDA implements FDARA. As always, we look forward to continued progress in the development and approval of new therapies for patients with rare diseases.”
