Orphan Drugs: The Current Situation in the United States, Europe, and Asia
- PDF / 283,301 Bytes
- 4 Pages / 504 x 719.759 pts Page_size
- 25 Downloads / 188 Views
0092-8615/97 Copyright Q 1997 Drug Information Association Inc.
ORPHAN DRUGS: THE CURRENT SITUATION IN THE UNITED STATES, EUROPE, AND ASIA ABBEYS. MEYERS President, National Organization of Rare Disorders, Inc. (NORD), New Fairfield, Connecticut
The American Orphan Drug Act has spurred international activities on orphan disease research and orphan drug research and development (R&D).Legislation has been enacted in Singapore and Japan, and is pending in Europe. Rare disease research facilities and information centers are operational in Scandinavia and Europe, facilitating the needs of patients, families, and medical personnel. Yet, the absence of a harmonized international orphan drug development program still hinders the wise use of economic and human resources in the most optimal and eflcient manner. Ke.y Words: Asia; Europe; Orphan diseases; Orphan drugs; United States
THE ORPHAN DRUG ACT appeared to be a benign piece of legislation in 1983 when it was enacted into federal law. The legislation was conceived by the rare disease patient community for the purpose of enticing pharmaceutical manufacturers into developing drugs that have little commercial value due to their limited markets. The pharmaceutical industry, through the Pharmaceutical Manufacturer’s Association (PMA) trade group, opposed enactment of the law because it perceived it as government intrusion into private enterprise. The industry also felt that support of the concept would be an admission that orphan drugs existed. Most of the large research intensive companies insisted that they were voluntarily developing orphan drugs as
Presented at the DIA Annual Meeting “The Challenge of Worldwide Pharmaceutical Development in an Era of Regulatory Change: Accelerated Approval with Quality and Contained Cost,” June 9-13, 1996. Reprint address: Abbey S. Meyers, President, National Organization for Rare Disorders, Inc. (NORD), 100 RI 37, PO Box 8923, New Fairfeld, CT 068128923.
a public service and that the patient community was inventing a problem that did not actually exist. The “patient community,” as it existed at the time, was an informal coalition of rare disease support groups that later united into the federation that is now known as the National Organization for Rare Disorders (NORD). NORD is dedicated to the identification, treatment, prevention, and cure of orphan diseases through programs of education, support services, and research. NORD still has significant political influence today on matters pertaining to health policy in the United States. For the first two to three years after enactment of the Orphan Drug Act, there was slow progress in development of new orphan drugs. Then the biotechnology industry realized that the seven years exclusivity provisions of the law could act as surrogate patents. Since patentability of biotech products was (and continues to be) weak, the seven years of exclusivity could be used to prevent competition while disputed patents were being finalized andor litigated.
Downloaded from dij.sagepub.com at NORTHWEST
Data Loading...
