The National Organization for Rare Disorders (NORD) is a non-profit voluntary health agency dedicated to the identification, treatment and cure of rare "orphan" diseases. NORD represents more than 25 million Americans with over 6,000 rare "orphan diseases." Under the federal Orphan Drug Act of 1983, a rare disease is defined as a health condition that affects fewer than 200,000 Americans. Because each disease affects a small number of people, there generally is little commercial interest in performing research or developing new products to diagnose or treat these diseases. Moreover, academic scientists believe it is easier for them to obtain government research grants for the study of more prevalent diseases, so they sometimes avoid applying for grants to study rare diseases.
NORD's mission is to promote the diagnosis, treatment, and cure of rare disorders through programs of education, research, advocacy, and service to families and health professionals. NORD's Clinical Research Grant Program, which includes both general and disease-specific grants, provides small "seed money" grants to academic scientists studying new treatments or diagnostic tests for rare diseases. The small clinical trials supported by NORD's research grants provide preliminary data indicating that a treatment (drug, device, or medical food) may be safe and effective when used for a larger number of patients. Researchers can then use the preliminary data to apply for larger multi-year government grants, or to attract a commercial sponsor who will manufacture the orphan product and get it approved for marketing by the Food & Drug Administration (FDA). When very little is known about a rare disease NORD research grants may support more basic research studying possible treatments that are not yet ready for human testing. NORD's research review guidelines follow those of the National Institutes of Health (NIH), and administrative costs are kept to a minimum.
Requests for proposals (RFP's) are issued on a cyclical basis. Adequate funds must be in place before preparations to initiate a research grant begin. Therefore, to maximize the full benefits of international advertisements and wide circulation of the funding opportunity, donors should understand that it takes several months after an RFP is issued, until the grant is awarded and actual research can begin. The outline below depicts a typical cycle:
October - January Prepare to initiate Request For Proposals (RFP)
January - March Issue RFP
April/May Deadline for preliminary proposals. NORD Medical Advisory Committee narrows applicants and invites finalists to submit detailed proposals.
July/August Deadline for full proposals from finalists.
Peer review of final proposals.
September/October Winners selected and awards announced.
SELECTION OF GRANTEES
The initial Request for Proposals asks scientists to submit a brief description (abstract) of their proposed research project along with their professional qualifications and a draft budget. Once that information is received, NORD's Medical Advisory Committee (composed of leading academic scientific experts) reviews all of the applications and selects finalists. The finalists are invited to submit a full grant application describing their proposed project in detail. NORD's Medical Advisory Committee then reviews the full grant applications (peer review) and ranks them through a scoring system. The highest scoring grant applications are recommended for funding to NORD's Board of Directors who votes on the final grant awards.
Depending upon the amount of money available for grants, one or more applications are chosen by the MAC for the grant award. Interim progress and budget reports are required every six months from the investigators, which are also reviewed and approved by NORD's Medical Advisory Committee. It is possible, however, that some researchers may require more or less time for completion of their research project.
NORD's grant review process is based on the same peer review system utilized by the National Institutes of Health (NIH). Scoring of grants is done individually by each medical expert, and scores are added together to determine the final ranking of each proposal. Any reviewer with a conflict of interest is excused from voting on a specific proposal. It is not possible for donors to signify how they want their donations spent (e.g., on a genetic test rather than development of a treatment), or which researcher should be funded. The grant selection process is unbiased and independent; awards are based solely on scientific merit. Only the best research projects will be funded.
By encouraging research applicants to compete against each other on an international basis, NORD's Research Grant Program funds only the best proposals that are most likely to result in research breakthroughs that can be applied to the diagnosis or treatment of people with rare disorders.
All research contributions are restricted by NORD and can be used for no other purpose without the donor's permission. Donations to NORD's Research Fund are tax deductible under IRS law. Interest cannot be accrued on gifts to the research Fund. Accumulated amounts for each disease-specific fund over $1,000 are published in NORD's newsletters, Orphan Disease Update.
If you are interested in initiating a research grant, or making a donation to NORD's Clinical Research Grant Program, please contact Nicole Woodage, Research Grant Administrator, by email: firstname.lastname@example.org or by calling 203-744-0100. Your gifts enable NORD to continue its vitally important research program aimed at promoting the diagnosis, new treatments, and cures for rare "orphan
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