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NORD Submits Citizen's Petition to FDA Regarding Orphan Drug Reviews

National Organization for Rare Disorders Calls for Policy on Products for Rare Diseases

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.

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 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."

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Don't Cut Life-saving Funding for NIH!

Congress is gearing up to reduce federal funding for medical research at the National Institutes of Health (NIH). This effort will slow research progress that offers hope to the more than 133 million people living with a chronic disease or disability. The National Health Council and other members of the health care community ask you to join us and oppose cuts in federal funding of the NIH. Sign this petition to tell Congress that you support life-saving research.

OHF is proud to be part of the Rare Kidney Stone Consortium

Its Mission
Rare Kidney Stone Consortium

The Consortium facilitates cooperative exchange of information and resources among investigators, clinicians, and patients, and researchers in order to improve care and outcomes for patients with rare stone diseases. The consortium promotes ready availability of diagnostic testing, pooling of clinical experiences, and availability of tissue banks in order to advance the science. Our goals are to:

  • Establish and expand registries and collaborate with patient organizations for the rapid dissemination of knowledge
  • Stimulate generation of testable hypotheses regarding mechanisms of renal injury in these diseases through registry findings, tissue resources, and pilot projects
  • Develop cohorts of well-characterized patients for future clinical studies
  • Attract and train investigators to rare diseases research in nephrology

Consortium Structure

The Rare Kidney Stone Consortium is an organization of diverse participants and independent efforts representing four major diseases of hereditary nephrolithiasis and headed up by Dawn S. Milliner, MD, and John C. Lieske, MD

  • Primary Hyperoxaluria Program Director | Dawn S. Milliner, MD | bio
  • Cystinuria Program Director | David Goldfarb, MD | bio
  • Dent Disease Program Director | John Lieske, MD | bio
  • APRT Deficiency Program Director | Vidar Edvardsson, MD | bio

The OHF is a 501 (c)(3), public charity.
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