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Report on Research on Rare Diseases in Children: FY 2000 to FY 2005

Office of Dietary Supplements (ODS), Office of Disease Prevention, Office of the Director, NIH

Overview of ODS Rare Diseases in Children Research Activities, FY 2000–FY 2005

The mission of ODS is to coordinate research at NIH on dietary supplements and to provide the public, practitioners, and other stakeholders with reliable information about these ingredients. Dietary supplements (which include vitamins, minerals, herbals, botanicals, metabolic intermediates, amino acids, and a number of other ingredients) are regulated by the FDA under the terms of the Dietary Supplement Health and Education Act (DSHEA) of 1994. DSHEA stipulates that products in this category may not be marketed for the treatment, prevention, mitigation, or cure of any disease. The DSHEA stipulation makes it appear that these ingredients should not be relevant to the management of children with rare diseases. In reality, there are two issues that make us reconsider the relevance of dietary supplement ingredients to rare diseases in children:

  • Some of the ingredients marketed in the dietary supplement category are also available as orphan drugs or as over-the-counter preparations.

  • Parents of children with a number of rare diseases (examples include, but are not limited to, Zellweger syndrome and related peroxisomal disorders, mitochondrial abnormalities, and some forms of attention deficit hyperactivity disorders [ADHDs]) look for answers to their children's care in unconventional places, including the use of ingredients, or combinations of ingredients, that are available as dietary supplements.

ODS, in collaboration with NICHD and other Institutes, organized a workshop on "Dietary Supplement Use in Children" held at NIH in February 2001. Ms. Abby Meyers, President of the National Organization of Rare Disorders, spoke about "Valid Medical Uses for Supplements" and emphasized that products marketed as orphan drugs for rare disease treatment (such as carnitine for a number of metabolic conditions) can often be obtained as dietary supplements, off the shelf, in food stores, pharmacies, and nutrition stores, but that these products are not in any way controlled for quality in the manner that the orphan drug version is. Sadly, some parents cannot afford the "orphan drug" version and settle, incautiously, for the dietary supplement version. ODS will continue to encourage research into the efficacy and safety of ingredients sold as dietary supplements, recognizing that these products should not be considered substitutes for orphan drugs.

Although dietary supplements cannot be marketed for disease treatment, this fact does not prevent consumers from using them in this manner when they are desperate for solutions to their children's intractable disease. Because of the rapid dissemination of information (through parent support groups and via the Internet) about unconventional treatments, all too often the use of these treatments far outstrips the scientific studies to support that use. ODS will continue to support research that is aimed at demonstrating efficacy and safety, in collaboration with other NIH Institutes and other Federal agencies.

While ODS does not have a specific research agenda in mind for the study of dietary supplement ingredients that are relevant to rare diseases in childhood, there is a general strategy that ODS will use to support research in this area:

  • Establish the base of scientific studies in the area. This often requires an evidence-based review of the existing literature in order to identify gaps in knowledge about ingredients used in particular clinical situations. The ODS evidence-based review program for dietary supplements is guided by a Federal working group (including NIH, FDA, Agency for Healthcare Research and Quality [AHRQ], as well as other Department of Health and Human Services [DHHS] and non-DHHS members) that assists ODS in setting priorities and providing guidance to AHRQ's Evidence-Based Practice Centers, which conduct the reviews.

  • Work with NIH Institutes, other Federal agencies, and private-sector organizations to support research activities that might include basic biological studies, clinical studies, epidemiologic surveys, and outcomes research.

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Last Reviewed: May 15, 2003
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