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Aug. 21, 2017

TOPIC: Advocacy, Featured News, Medical

NORD Issues Statement in Response to the Signing by President Trump of the Food and Drug Administration Reauthorization Act of 2017

Posted by Christina Jensen

Washington, D.C., August 21, 2017Peter 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.”

2 Responses to “NORD Issues Statement in Response to the Signing by President Trump of the Food and Drug Administration Reauthorization Act of 2017”

  1. Bethany says:

    Dear President Trump,
    As one who suffers from several very rare diseases, I greatly appreciate the interest and effort you are putting into addressing this growing problem. There exists so few treatments for some of the rarest diseases that exist. One disease I have affects one per one million patients world wide. This disease is called “Stiff Person Syndrome” and has greatly reduced the quality of my life.
    The FDRA sounds as if It might help me as well as those in my position. Thank you much for your interest and efforts into this matter. I look forward to seeing the progress being made in this situation.

  2. Pat Baumgartner says:

    That is the best news for all of us who are stricken with Myasthenia Gravis an Autoimmune Disease. This disease attacks all the muscles. It is very rare with only around 70,000 cases known in the world, there is very little research being done! Hopefully this is going to help us! Please remember this disease in research, MYASTHENIA GRAVIS! Thank you!