NORD's request is being made in conjunction with a report submitted by an FDA committee to Congress on June 27, 2011: Improving the Prevention, Diagnosis and Treatment of Rare and Neglected Diseases.
While expressing concern that currently no policy specific to orphan drugs exists, NORD praises FDA's history of flexibility in the review of "orphan" (for rare diseases) products, noting that rare diseases present special challenges to researchers seeking to develop therapies.
"Today, most of the nearly 7,000 rare diseases do not have FDA-approved treatments," 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."
Saltonstall said that "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
At that hearing, NORD Board Chair Frank Sasinowski presented testimony on the fact that so many rare diseases still have no treatment. He said NORD feels that the "low-hanging fruit" have now been harvested by medical researchers but that "the vast majority of therapies" remain out of reach of patients with rare diseases. Many of those patients, he added, are children suffering with chronic and lifelong genetic diseases.
Sasinowski noted in his testimony that focus groups and other studies conducted by NORD had identified uncertainty in the review process as a deterrent to the development of treatments for rare diseases.
Saltonstall said that NORD's request for a documented FDA policy on review of orphan drugs is consistent with a report issued last October by the Institute of Medicine, Rare Diseases and Orphan Products: Accelerating Research and Development.
Citizen Petitions may be submitted to FDA by individuals or organizations requesting specific actions to issue, amend, or revoke regulations. In this case, NORD is asking that a specific policy related to orphan drugs be included in the guidance document that FDA must issue no later than December 27, 2011, following the June report to Congress.
The June report was submitted to Congress in response to legislation, specifically Section 740 of the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act, 2010 (P.L. 111-80).
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 more than 150 disease-specific member organizations, as well as all individuals and families affected by rare diseases. NORD's website is at www.rarediseases.org.
