Sunday, September 11, 2011


The National Organization for Rare Disorders (NORD) has submitted a Citizen’s Petition to the Food and Drug Administration (FDA) requesting that a documented policy be established regarding the review of potential treatments for people with rare diseases.


“Today, most of the nearly 7,000 rare diseases do not have an FDA-approved treatment,” said Peter L. Saltonstall, president and CEO of NORD, a nonprofit organization representing the one in 10 Americans affected by rare diseases. “Basic research and clinical trials needed to develop treatments for these diseases are complicated by the fact that there are few patients and they are geographically dispersed.”

“NORD recognizes and applauds FDA’s historical flexibility in the regulatory review of submissions for articles that treat rare disease.” Now, he added, NORD is asking for FDA documentation to guide future drug reviews and specifically to:

•acknowledge that clinical trials for most orphan drugs are fundamentally different from trials for drugs for common conditions

•acknowledge that FDA review of orphan drugs is therefore different from the review of other products

•document that, while the need to demonstrate both effectiveness and safety remains unchanged, it will now be official FDA policy to apply special flexibility in the review of orphan drugs

NORD was established in 1983 by patient advocates seeking to enhance awareness of rare diseases and to improve the lives of those affected. Today, NORD provides services that include advocacy, education, research grants, and patient assistance programs. It represents 160 disease-specific member organizations, as well as all individuals and families affected by rare diseases.

Real treatment for Rare Diseases are not OTC (over the counter) available -- NORD is fighting for help for our loved ones.

Visit the NORD site to see how you can help and to read the Petition.

And take a minute to sign the ongoing Rare Disease Day Petition -- a click on the hands will take you there!

With respect to all on this very special day.  Those loved, those lost, those remembered.