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PubMedDate not supplied

Orphan products: drugs and devices for rare diseases.

The issues of orphan products include an inadequate definition of orphan status that hampers implementation of the legislation, difficulties in evaluating the products themselves in order to ensure safety and effectiveness, neglect in developing orphan devices, and conflict over creating unnecessary monopolies and excessive profits. However, it has been demonstrated that the ODA is effective in stimulating the development of these much needed drugs and devices. The next efforts therefore can be directed toward increasing the effectiveness and ensuring the equity of the program.

Equipment and Supplies↗
PubMed1992

Evaluation of orphan products by the U.S. Food and Drug Administration.

Orphan drug products generally are used in treating or preventing rare diseases. The small number of patients available for study may create special problems in the evaluation of these products. This paper examines some of the special problems that are associated with the design and implementation of studies to evaluate the safety and efficacy of orphan drugs. The U.S. Food and Drug Administration (FDA) has not established special criteria for evaluating orphan drugs per se, but the FDA has been flexible in evaluating drug products that present special problems, especially when these products are for treatment of serious of life-threatening illnesses. The FDA and other U.S. governmental agencies also have taken steps to promote the development and availability of drugs for rare diseases, including making these products available to patients who are in need, even before the drugs have full FDA marketing approval.

Drug Approval↗
PubMed1985

Orphan drugs: the question of products liability.

Orphan drugs, essential for the treatment of persons with rare diseases, generally are unprofitable for manufacturers to develop and market. While congressional and administrative efforts to promote the development of orphan drugs have met with modest success, application of products liability doctrine to orphan drug sponsors could subvert those efforts. This Note describes the provisions of the Orphan Drug Act and analyzes products liability law with respect to orphan drug litigation. It argues that the goals of tort law support the imposition of liability for design defect, failure to warn and negligence in testing. Finally, the Note acknowledges that liability costs create disincentives for orphan drug development and suggests mechanisms for reducing manufacturers' liability concerns.

Drug Industry↗
PubMed1989

Information service on treatment for rare diseases: the National Information Center for Orphan Drugs and Rare Diseases.

The amount of information available on rare diseases and the drug products used to treat them increases every year. The Orphan Drug Act, enacted in 1983, not only stimulated the development of orphan-drug products but also empowered the Food and Drug Administration to assist in the dissemination of information on these products to all health-care professionals. As the health-care professionals legally responsible for the dispensing of all drugs, pharmacists especially must be as informed as possible about any medication dispensed by them to their patients. The growing list of orphan-drug products makes this an almost impossible task. To assist in accessing this information, a new resource has been created.

Drug Industry↗
PubMed1990

An overview of the FDA publication Approved Drug Products with Therapeutic Equivalence Evaluations.

The history, purpose, and content of FDA's Approved Drug Products with Therapeutic Equivalence Evaluations (the "Orange Book") are described. When cost guidelines established in the 1970s for Medicare reimbursement prompted most states to permit generic drug product selection, an official list of interchangeable drug products was needed. Regulations for determining bioavailability and bioequivalence were published in 1977, and FDA first published Approved Drug Products in 1980. The Orange Book includes products that have been fully reviewed by FDA for both safety and effectiveness and for which new drug applications (NDAs) or abbreviated new drug applications (ANDAs) and special applications (From 5s or 6s) for antibiotics have been approved. Pursuant to the 1984 Drug Price Competition and Patent Term Restoration Act, FDA added approved nonprescription products to the Orange Book along with information about patents and periods of marketing exclusivity. Orphan drug products and certain testing and application procedures are also provided. Equivalence evaluations are provided in the Orange Book only for multisource prescription drug products that contain the same active ingredients and can be expected to have the same clinical effect when administered to patients under the conditions specified in the labeling. A coding system is used to indicate the products' equivalence evaluations according to FDA criteria, and indexes by product name and manufacturer are included. Monthly Cumulative Supplements are issued. The Orange Book contains public information and advice, but it is not an official national compendium; FDA has no position on state regulation of drug product selection by pharmacists. The equivalence evaluations do not relieve practitioners from exercising care in prescribing and dispensing products according to patients' individual needs.

Drug Information Services↗
PubMed2000

A proposal to improve the supply of orphan drugs.

Industrial medicinal products are not always available for the treatment of some specific patients or diseases. Appropriate research on orphan drugs, neglected doses or administration routes and patients' sensitivity to excipients, as an example, might lead to interesting results in this respect. In the United States, a specific legislation on orphan drugs dates back to 1983; in the European Union, regulations are currently being studied. The industrial production of orphan drugs might be improved following the adoption of adequate incentives. New regulations should also take care of neglected doses, and stimulate the preparation of galenic products, as an example by including a number of monographs on orphan drugs in Pharmacopoeias and National Formularies. As a matter of fact, magistral and officinal preparations made in community and hospital pharmacies in accordance with a medical prescription or with the prescription of a Pharmacopoeia or an Official Formulary, can represent an effective alternative. In this paper, the regulations enforced in the USA and Japan, the European situation, possible incentives to the industry and the role of galenic products will be discussed.

Drug and Narcotic Control↗
PubMed1998

Clinical studies of safety and effectiveness of orphan products; availability of grants; request for applications--FDA. Notice.

The Food and Drug Administration (FDA) is announcing changes to its Orphan Products Development (OPD) grant program for fiscal year (FY) 1999. The previous announcement of this program, which was published in the Federal Register of July 9, 1997, is superseded by this announcement. In the future, a new announcement will be published annually.

Clinical Trials as Topic↗
PubMed1999

Clinical studies of safety and effectiveness of orphan products; availability of grants; request for applications. Department of Health and Human Services (HHS), Public Health Service (PHS), Food and Drug Administration (FDA). Notice.

The Food and Drug Administration (FDA) is announcing changes to its Orphan Products Development (OPD) grant program for fiscal year (FY) 2000. The previous announcement of this program, which was published in the Federal Register of August 5, 1998 (63 FR 41855), is superseded by this announcement. In the future, a new announcement will be published annually.

Clinical Trials as Topic↗
PubMed1985

Orphan drug development in the United States.

Drug research and development in the U.S. tends to focus on drugs to treat common diseases because of the anticipated return on investment. To stimulate pharmaceutical manufacturers to pursue the development of drugs for rare conditions, the Orphan Drug Act was enacted by Congress on January 4, 1983. Under the provisions of this Act, the FDA can make recommendations on the investigations necessary for marketing approval; exclusive marketing privileges can be obtained; tax credits for expenses incurred are allowed; availability of orphan drugs on an investigational basis is encouraged; and the Orphan Product Board is established for the coordination of research efforts and their reimbursement. The effects of this legislation are evident in the continuing increase in orphan drug designations.

Drug Evaluation↗
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↗
PubMed1995

The economics of orphan drug policy in the US. Can the legislation be improved?

This review of the US Orphan Drug Act (ODA) 1983 outlines how the ODA is intended to stimulate orphan drug research and development of drugs for rare diseases. We also evaluate the effectiveness of the ODA in the past decade and provide recommendations for ODA improvements in the future. The economic incentives embedded in the ODA are presented in a simple economic model, in which a guarantee of market exclusivity plays a central role in encouraging firms to pursue the development of orphan products. Some evidence suggests that this provision has been a major impetus for the rise in orphan drug applications and designations in the last decade. Market exclusivity is the key incentive for orphan drug research, and should be retained. Concerns about a limited number of highly successful 'blockbuster' orphan drugs should be evaluated in terms of the useful economic incentives. In the future, exceptionally high profits could be limited by more precise evaluation of disease prevalence, elasticity of demand, and the other uses of orphan compounds. We further recommend an expansion of the ODA tax credits and research grants programme and targeting of 'priority' diseases. We conclude that the ODA has been a valuable legislative initiative, but it can be strengthened with some simple extensions of the current incentives that it contains.

Drug Industry↗