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ERIC Number: EJ959412
Record Type: Journal
Publication Date: 2011-Dec
Pages: 3
Abstractor: ERIC
Reference Count: N/A
ISSN: ISSN-0046-9157
Families Seek a Greater Role in Search for Rare Disease Treatments
Abbott, Natalie
Exceptional Parent, v41 n12 p35-37 Dec 2011
Of the nearly 7,000 rare diseases identified by the National Institutes of Health (NIH), only a few hundred currently have treatments. The development of therapies for rare diseases is often hampered by the special challenges of conducting the needed studies for rare disease drugs and medical devices, such as small numbers of patients and the fact that patients are scattered around the world. However, the National Organization for Rare Disorders (NORD) believes that patients and families of patients with rare disorders and chronic conditions belong to a highly-motivated population who can help drive the search for safe, effective treatments. A nonprofit organization established in 1983 that serves as the voice of rare disease patients and families in the U.S., NORD is seeking a stronger role for the patient community to accelerate the development of new therapies. One area of particular interest at this time is the definition of risk. NORD believes patients and families affected by rare and/or chronic diseases may define relative risks and potential benefits of therapies in development differently from people with more common conditions. The organization is asking the Food and Drug Administration (FDA) to take this group's increased risk tolerance into account when reviewing potential new drugs and medical devices for rare disorders and chronic conditions. NORD, along with 30 of its advocacy partners, recently sent a letter to key officials at FDA seeking more frequent and regular opportunities to provide input on decisions related to risk/benefit for new therapies in development and being reviewed by FDA.
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Publication Type: Journal Articles; Reports - Descriptive
Education Level: N/A
Audience: N/A
Language: English
Sponsor: N/A
Authoring Institution: N/A
Identifiers - Location: United States