Office of Orphan-Products Development

Since it was created in 1982, the FDA s Office of Orphan Products Development has been dedicated to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. To locate such products, the Office of Orphan Products Development interacts with the medical and research communities, professional organizations, academia, and the pharmaceutical industry, as well as rare disease groups. The Office of Orphan Products Development administers the major provisions of the Orphan Drug Act, which provide incentives for sponsors to develop products for rare diseases. 

The Orphan Drug Act of 1983, which amended the 1938 Federal Food, Drug, and Cosmetic Act, provides incentives for the development of drugs for treatment of diseases affecting fewer than 200,000 patients in the USA. The FDA maintains an office of Orphan Product Development to provide special assistance and grants to scientists with an interest in these products. Information on orphan drugs is also available from The National Organization for Rare Disorders. 

Submission of an orphan application does not require a formal FDA preapplication meeting, though the Office of Orphan Products Development will generally be helpful in responding to clarification questions via telephone. The application—generally a notebook tabbed to separate the application sections and the enclosed references—is submitted in duplicate, with an accompanying cover letter. 

Marlene E. Haffner, MD, MPH, Director, Office of Orphan Products Development Food and Drug Administration, HF-35 5600 Fishers Lane, Room 6A55 Rockville, MD 20857... 

The estimated 130h pertains to Sec. Sec. 316.20,316.21, and 316.26. These apply primarily to initial applications/requests seeking orphan-drug designation. Many applications/requests received in the Office of Orphan Products Development contain multiple volumes include an exact duplicate copy of the original and may include 50 or more documented references. Additional information is requested when an application/request is denied. The sponsor usually supplies the requested information in the form of an amendment. 

The regulations require that a permanent resident of the United States must act as the sponsor s agent upon whom service of all processes, notices, orders, decisions, requirements and other communications may be made on behalf of the sponsor. The name of the agent must be provided to Office of Orphan Products Development (HF-35), Food and Drug Administration, 5600 Fishers Lane, Rockville, MD 20857 (5). 

FDA Office of Orphan Products Development, www. fed.gov/orphan/about/progovw.htm (accessed Feb 2000). 

Within the Act itself, the four major benefits are (1) the period of marketing exclusivity, which may be considered as a type of patent (2) the tax benefits on clinical trials between the date of orphan drug designation and NDA approval and (3) the FDA s Office of Orphan Products Development grants to support clinical trials on orphan drugs. A fourth benefit of protocol assistance, from the FDA, was always available for important new drugs (as well as others) and is important, but not necessarily new. Nonetheless, it is useful to call attention to this provision. 

Rear-Admiral Marlene Haffner MD MPH FRCP, Director of the FDA s Office of Orphan Products Development, has summarized this issue best by saying For a difference to be a difference it must make a difference (personal communication). A lower cost of a second form of a medicine, or of the... 

Each of these subdivisions is typically further subdivided. For example, CBER has offices of Management, Compliance, Therapeutic Research and Review, Vaccines Research and Review, Establishment Licensing and Product Surveillance, Blood Products and Communications and Training. Each are typically led by career civil servant Office Directors, although, currently, the Office of Orphan Product Development is headed by a Rear-Admiral from the US Public Health Service. 

In the United States, from January 1983 to June 2004, a total of 1,129 different orphan drug designations were granted by the Office of Orphan Products Development (OOPD), and 249 orphan drags received marketing authorization in the United States. In contrast, in the decade prior to 1983, fewer than ten such products came to market. 

The code will be broken and data will be analyzed when the 12-mo follow-up has been completed for all patients. At that time, DHA therapy will be offered to all patients who desire to receive it. Clinical and laboratory studies will be monitored by an independent data and safety monitoring committee. This committee will also perform an interim analysis when 30 patients have completed assessment and has permission to terminate the study if there are significant favorable findings or unanticipated adverse outcomes. A determination of statistical power indicates that this sample size is sufficient to detect a major effect (0.862 standard deviations). With a more modest effect of 25%, however, the statistical power diminishes to 60%. The study has been approved by the Institutional Review Board at the Johns Hopkins Medical Institutions and is supported by a research grant from the Office of Orphan Products Development at the Food and Drug Administration. Fifty-two patients are enrolled in the study at this time. The code has not yet been broken. No adverse effects attributable to the medication have been observed. 

SOURCE Office of Technology Assessment, 1993. Based on data supplied by the U.S. Department of Health and Human Services, Food and Drug Administration, Office of Orphan Product Development, 1992. 

The Orphan Drug Act authorized grants for clinical research on potential orphan products, and one of its more recent amendments (Public Law 100-290) extended this authority to preclini-cal studies. These grants represent a direct subsidy for orphan drug R D.31 The Office of Orphan Products Development administers the program in a manner parallel to other Public Health Service grants. 

Office of Orphan Products Development U.S. Food and Drug Administration Rockville, MD... 

The FDA s Office of Orphan Products Development grants to support clinical trials on orphan drugs (although this is a relatively small amount of money each year). 

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