Research

NORD Research Program Policy

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.

PURPOSE
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.

DONATING FOR RARE DISEASE RESEARCH
Donors may contribute to NORD's General Research Fund, or they may target their donation to research on a specific disorder. Donations made to NORD's General Research Fund will be spent on medical research and related activities that encourage increased research, but are not restricted to one disease. NORD encourages potential donors interested in disease-specific research to conduct fundraising efforts so that a sufficient amount of money is made available, within a reasonable period of time, to attract the highest quality research proposals. Medical research is expensive so a minimum of $35,000 is required to fund one grant. When this target amount is reached, NORD begins the process of issuing a request for research proposals.

In the past, when donors gave small sums restricted to research on specified diseases, the gifts languished unspent for several years because sums were too low to support medical research. Therefore, NORD's Board of Directors decided if the funds donated to a specific disease do not reach the following amounts within the specified period of time, the funds will be transferred to the following:

• If the total of donated funds for research on a specific disease is less than $1,000 after two years without significant donations, the fund will be transferred to general research and related activities.
  
  
• If the total of donated funds for research on a specific disease is more than $1,000 but less than $5,000 after four years, the money will be transferred to research on related diseases. (e.g., neurological, metabolic, connective tissue diseases, etc.)
  
  
• If the total of donated funds for research on a specific disease is more than $5,000 but less than $10,000 after six years, the money will be transferred to research on related diseases.
  
  
• If the total of donated funds for research on a specific disease is more than $10,000 but less than $15,000 after eight years, the money will be transferred to research on related diseases.
  
  
• If the total of donated funds for research on a specific disease is less than $25,000 in ten years, the money will be transferred to research on related diseases.

Exceptions can be made if NORD sees continued donor activity for a specific disease, indicating that there is a determined effort to reach the minimum $35,000 goal, in a reasonable period of time. In many instances, families will launch fundraising campaigns in their communities that raise several hundred dollars at a time. NORD encourages these activities and recognizes that it can take several years to reach the necessary sum that triggers a request for research proposals.

COSTS
A minimum of $35,000 is required to trigger a request for research proposals. Out of each grant awarded between $30,000 and $50,000, NORD uses only $5,000 to cover the administrative expenses and direct costs of initiating a request for proposals (RFP). Grants awarded over $50,000 use 10% for administrative expenses. Costs of the RFP include advertisements in leading medical journals and a mailing to every university-affiliated hospital and medical school in the United States. A search for researchers who have published journal articles about a specific disease in the last 2 - 5 years is conducted in order to reach the most expert scientists. Additionally, research institutions in Canada and Europe may also be notified of the RFP.

Each research proposal is reviewed by NORD's Medical Advisory Committee, which recommends funding for the highest scored proposals. After the grant is awarded, NORD monitors the progress of the research, processing biannual reports to NORD's Medical Advisory Committee. Any restricted funds that are not spent during a grant cycle are returned to the restricted research fund for that disease. Administrative fees are not taken until a grant has been awarded, regardless of the number of RFP's issued.

GRANT CYCLES
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:

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 Stefanie Putkowski, RN, Research Grant Administrator, by email: research@rarediseases.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 diseases."