Note: OPD funding is included Human Drugs budget.

The Orphan Products Grants Program provides funding for clinical research on products to prevent and treat rare diseases and conditions. OPD's program has funded 356 grants since 1984. Appropriations for this program have been virtually static for the last decade while indirect and grantee costs have escalated. This has put pressure on the management of the existing grants to ensure that limited funds are wisely applied. OPD received 105 grant applications 1997 of which 87 were deemed deserving of funding after rigorous peer review; 27 of these were funded. For the 1998 cycle, 97 applications are being reviewed. Twenty-one (21) products have achieved FDA marketing approval through studies funded in whole or in part by this Grants Program.

Increased program interest and activity results from recent Congressional initiatives which enhance the value of orphan designation. Renewal of the Federal tax credit associated with orphan product designation, establishing it with a "carry-back/carry forward" eligibility and making it permanent, is one factor. Another is an exemption from the marketing application user fees for these designated products incorporated in the renewal of the agency's prescription drug user fee authority.

JUSTIFICATION OF BASE

FDA continues to carry out a program encouraging the development of drugs, biologicals, medical devices and medical foods for rare diseases and conditions. In 1998, Office of Orphan Products Development (OPD) met with 20 potential sponsors of orphan products to provide guidance on the development of these products as well as guidance on orphan product "designation" applications and issues. OPD also participated in 60 FDA meetings with sponsors about ongoing development of orphan products in 1998. Significant increases in interest in the program, informational meetings, applications, and designation reviews are anticipated for 1999 and 2000.

Historically 80 percent of the applications result in designations. Thus, the inventory of designations is expected to increased by 300, an increase of 60 percent. Based on recent experience, it is anticipated that there will be 100 applications in 1998, 130 in 1999, and 150 in 2000, up from an average of 70 per year since 1990. Timeliness of the designation review process is important to sponsors which make product development decisions based on whether or not a product receives orphan designation. FDA will continue to explore means to streamline the designation review process to ensure that the increased workload does not adversely affect designation application review times.

During 1998, FDA reviewed 118 (up 64 percent from 72 the previous year) new sponsor requests for designation of drugs as orphan products. As part of the request for designation, sponsors are required to submit data that adequately demonstrates the use of their product for diseases or conditions affecting fewer than 200,000 people in the U.S., or that meets economic qualifications. Based on OPD reviews, 67 drugs and biological products received designation as orphan products during the year. FDA estimates that past levels of sponsor orphan designation applications may double by the year 2000.

Designation of medical device applications for the Humanitarian Use Devices (HUD) exemption is a new function of the orphan product program. The designation request to become a HUD is the first part of a two-step process which requires sponsors to demonstrate that a device affects fewer than 4,000 individuals in the United States. Ten HUD applications were reviewed in 1998, bringing the 3-year total to 30. The HUD exemption was enacted in the Safe Medical Devices Act of 1990.

Under the FDA Orphan Products Grants Program $5,659,528 was awarded for 20 new studies, and 8 competing continuation studies in 1998. These studies were designed to provide information on human safety and effectiveness of products for orphan diseases and conditions like: congential lactic acidema, neoplastic meningitis, leukemia, autism, and Tourette's syndrome. Additionally, $6,027,472 was spent during the year for noncompeting continuation studies begun in prior years.

In 1998, 18 designated orphan drugs were approved for marketing (* indicates funding in part by FDA's grant program):

• Aldesleukin -- Treatment of metastatic melanoma.
• Basiliximab -- Prophylaxis of solid organ rejection.
• Filgrastim -- Reduction in the duration of neutropenia, fever, antibiotic use, and hospitalization, following induction and consolidation treatment for acute myeloid leukemia.
• Hydroxyurea -- Treatment of patients with sickle cell anemia as shown by the presence of hemoglobin S.
• Infliximab* -- Treatment of moderately to severely active Crohn's disease for the reduction of the signs and symptoms, in patients who have an inadequate response to conventional therapy; and treatment of patients with fistulizing Crohn's disease for the reduction in the number of draining enterocutaneous fistula(s).
• Lamotrigine -- Treatment of Lennox-Gastaut syndrome.
• Lepirudin -- Treatment of heparin-associated thrombocytopenia type II.
• Mafenide acetate -- For use as an adjunctive topical solution antimicrobial agent to control bacterial infection when used under moist dressings over meshed autografts on excised burn wounds.
• Modafinil -- Treatment of excessive daytime sleepiness in narcolepsy.
• Octreotide -- Treatment of acromegaly.
• Octreotide -- Treatment of severe diarrhea and flushing associated with malignant carcinoid tumors.
• Octreotide -- Treatment of diarrhea associated with vasoactive intestinal peptide tumors (VIPoma).
• Pilocarpine HCL -- Treatment of xerostomia and keratoconjunctivitis sicca in Sjogren's syndrome patients.
• Rifapentine -- Treatment of pulmonary tuberculosis.
• Sacrosidase* -- Treatment of congenital sucrase-isomaltase deficiency.
• Thalidomide -- Treatment of erythema nodosum leprosum.
• Thyrotropin alpha -- As an adjunct in the diagnosis of thyroid cancer.
• Valrubicin -- Treatment of carcinoma in situ of the urinary bladder

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