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At least 19 recordsLinked to original sources
PubMed1995

Rare diseases, drug development, and AIDS: the impact of the Orphan Drug Act.

The Orphan Drug Act provides public subsidies and incentives to spur the development of drugs for rare diseases--drugs that the private sector might otherwise consider unprofitable to produce. Although the act has achieved numerous successes, the high prices and extraordinary sales generated by some orphan drugs lead to a pivotal policy question: how can the act be used to meet the legislative goal of stimulating drug development for small patient populations without resulting in prices that make drugs inaccessible? This question is explored using the example of AIDS drugs, many of which received subsidies under the act, to illustrate central points. The history of the act, its weaknesses, and strategies for reform are described as well.

AIDS-Related Opportunistic Infections↗
PubMed1992

Evolution and current status of the Orphan Drug Act.

The 1983 Orphan Drug Act was designed in response to market and regulatory disincentives that limited industrial interest in developing drugs needed by people in the United States with rare diseases and conditions. These disincentives evolved from changes in the pharmaceutical industry and in regulatory testing requirements. In the eight years since the Act and subsequent amendments have been in effect, the law has been associated with the new development and approval of 40 marketed drugs and 12 biologicals to treat rare (orphan) diseases. An additional 281 drugs and 141 biologicals have been entered into development and designated as orphans. Finally, the law has mandated exploration of whether the incentives of the Act are necessary and appropriate for stimulating industrial development of orphan medical drugs and devices. Despite this progress, controversies have arisen over three profitable orphan products that have benefited from the law's provisions. This has created the need for continued assessment of the Act's benefits and costs.

Drug Approval↗
PubMed1999

Is orphan drug status beneficial to tropical disease control? Comparison of the American and future European orphan drug acts.

UNLABELLED: OBJECTIVES To quantify past outcomes of tropical pharmacology research and development (R & D) and to assess past benefits of the American orphan drug act and potential benefits of the future European orphan drug regulation on tropical diseases. METHODS: This paper presents two analyses: a 1983-97 retrospective study of the United States Orphan Drug Act concerning rare diseases and a prospective study of the European Proposal for a Regulation Concerning Orphan Drugs and its possible impact on tropical diseases. RESULTS: Different programmes have in the past tried to stimulate R & D in this area, but results remain limited. Of 1450 new chemical entities marketed between 1972 and 1997, 13 were specifically for tropical diseases and considered as essential drugs. Between 1983 & 1997, the US Orphan Drug Act approved 837 drugs and marketing of 152 new molecular entities (NMEs). Three NMEs have been designated for malaria and human African trypanosomiasis. Seven others, already commonly used in tropical diseases, received either orphan designation or an orphan approval for another indication. Pharmaceutical companies benefit from the US framework only when the US market exclusivity clause was applicable. Future European orphan drug regulation appears to be similar to the US Orphan Drug Act. CONCLUSION The orphan drug programmes relating to rare diseases have met with some success. Considering tropical diseases rare diseases seems inadequate to boost pharmaceutical R & D. However, some provisions of the European text may be relevant to tropical diseases, admitting the need for a more specific rule for evaluations of this kind of drug and recognizing the existence of 'diseases of exception'.

Europe↗
PubMed1991

The Orphan Drug Act. The first 7 years.

The 1983 Orphan Drug Act sought to increase market incentives and decrease regulatory barriers for products used to treat rare ("orphan") diseases. Major provisions included market exclusivity, tax credits, and regulatory process clarifications. This analysis compares pre- and post-Act industry and government data to examine changes associated with the law. While industry sponsored 34 marketed and 24 experimental orphan drugs in the 17 years prior to the Act, it has sponsored 39 of 42 marketed orphan products in the 7 years since the Act. An additional 301 experimental products have orphan designation. While 25 of 40 marketed orphan products reportedly had annual sales of less than $1 million, product sales for three conditions are more than $100 million annually. This prompted changes in the law, passed by Congress in 1990, but vetoed. Overall, the law has been associated with an increase in orphan product development. The law's costs and benefits to companies, patients, and the public should be examined if future changes are proposed.

Drug Industry↗
PubMedDate not supplied

Orphan Drug Act on congressional agenda.

In 1983, Congress passed the Orphan Drug Act to encourage development of pharmaceuticals used in the treatment or diagnosis of diseases that occur so rarely that there is no financial incentive for development of these so-called "orphan drugs." The Act established, among other things, exclusive marketing rights on unpatentable orphan drugs for a period of seven years and a tax credit equal to 50 percent of the cost of conducting human clinical trials. It also authorized the Secretary of Health and Human Services (HHS) to make grants and enter into contracts to reduce the costs of clinical testing in orphan drug development.

Drug Industry↗
PubMed1992

Creating the costliest orphan. The Orphan Drug Act in the development of Ceredase.

The FDA recently approved Ceredase, a new treatment for Gaucher's disease, under the provisions of the Orphan Drug Act. Ceredase is unusually expensive, but there are no satisfactory alternative therapies. It appears likely that Ceredase would not have become available without the protection of the Orphan Drug Act, but its expense and the lack of information about its long-term effects on health raise questions about whether the ODA provides appropriate incentives to develop cost-effective technologies.

Adolescent↗
PubMedDate not supplied

The Orphan Drug Act and the Federal Government's Orphan Products Development Program.

Through the combined efforts of agencies and organizations in the public and private sector, drugs have been made available that would not have been at hand without a specific focus on the orphan drug issue. It is anticipated that these cooperative efforts will continue beyond the first enthusiastic burst engendered by the inception of new and interesting activities.

Drug Industry↗
PubMed1998

A cross-national comparison of orphan drug policies: implications for the U.S. Orphan Drug Act.

Six countries--Canada, France, Japan, Sweden, the United Kingdom, and the United States--were studied to compare public policies affecting the development and marketing of pharmaceuticals for rare diseases (i.e., orphan drugs). Information was obtained from a variety of published and unpublished sources, including interviews with public policy and pharmaceutical experts in each country. This article presents different approaches to encouraging the development of orphan drugs while ensuring access by regulating their prices. Additionally, the article describes access to orphan drugs as promoted by special coverage for population subgroups, disease categories, and/or specific drugs. Not all efforts to increase access to orphan pharmaceuticals have been the result of government action, as illustrated by the proliferation of for-profit organizations that specialize in orphan drugs. The many policy options from other countries identified in this study are especially relevant, given increasing calls for reform of the U.S. Orphan Drug Act.

Canada↗
PubMed1990

Orphan Drug Act.

Explore the source record for details and available documents.

Drug Industry↗