The Orphan Drug Act: Provisions and Considerations
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0092-8615/2000 Copyright 0 2000 Drug Information Association Inc.
THE ORPHAN DRUG ACT: PROVISIONS AND CONSIDERATIONS CARSONR. REIDER,MS, CCRC Department of Neurology, The Ohio State University, Columbus, Ohio
The United States Orphan Drug Act passed in 1983 provides four provisions to spur the development of medications for conditions that might otherwise have been abandoned. The office of Orphan Drug Product Development was established to provide assistance in protocol development, and to administer a grants program. A tax credit incentive also was enacted but the most significant incentive has been the market exclusivity clause. Despite such measures, the act in the wake of technological and economic developments should continually be reexamined to enhance eflciency in development of orphan products, to ensure accessibility of these products to patients, and to minimize economic abuses by developers. Key Words: Orphan Drug Act; Drug development; Rare diseases
INTRODUCTION OVER 20 MILLION AMERICANS suffer from one of approximately 5000 orphan diseases, each affecting a relatively small number of people (1). The United States Orphan Drug Act (ODA) was initiated with the intent to promote the development of pharmaceuticals for orphan diseases-those diseases or conditions that have been classified as “rare,” by defining the conditions and establishing attractive incentives for industry to develop medications for such designations (2). Its impetus was the lack of attention toward these diseases primarily due to the negative balance of development costs versus the fiscal gains once marketed. The notion of high profitability was not seriously considered, and the statutory language as had been written originally addressed an atmosphere and situation of limited profitability. Interest in
Reprint address: Carson R.Reider, MS, CCRC. Department of Neurology, The Ohio State University, 371 McCampbell Hall, 1581 Dodd Drive, Columbus, OH 43210.
such legislation was initiated by the rare disease patient community and spearheaded by a group now known as the National Organization of Rare Disorders (NORD). It was opposed by the Pharmaceutical Manufacturer’s Association (PMA) because of the perceived intrusion by government into their private enterprise, and the fact that agreement might have suggested an admission to their lack of development of products for these so-called orphan diseases. Since its passage in 1983 during the Reagan Administration, the Office of Orphan Products (OPD) of the United States Food and Drug Administration (FDA) has designated over 800 orphan products with over 170 of these receiving FDA marketing approval, benefitting more than eight million Americans. The act was originally designed for unprofitable or unpatentable medicines. In 1984 an amendment shifted the standard designation of orphan drug from profitability to prevalence and the requirement of unprofitability was dropped from the act. The overwhelming concentration of products have been for cancer and genetic diseases, ac-
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