Orphan Drug Products Research Articles

Radiopharmaceuticals as orphan drugs

Rare diseases and conditions are defined as diseases or conditions that affect less than 200,000 individuals in the United States. Because of the limited population suffering from any given rare disorder, there is little economic motivation for the development of drug products for its diagnosis or treatment. Also, frequently these rare diseases are misdiagnosed or undiagnosed due to a lack of familiarity with their symptoms and course. Thus, these rare disorders are commonly referred to as orphan diseases or conditions. To address this problem, legislation has been enacted that provides for incentives for the development of drug products for rare diseases or conditions (i.e., orphan drug products). Nuclear medicine scientists can take advantage of these incentives in the development of orphan radiopharmaceuticals for the treatment or diagnosis of rare diseases or conditions. Nuclear medicine physicians should know where information on orphan diseases and orphan radiopharmaceuticals can be obtained to maximize the care of their patients.

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.

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.

Survey indicates 133 orphan drug products in development for 96 relatively rare disorders

Survey indicates 133 orphan drug products in development for 96 relatively rare disorders

Survey Indicates 133 Orphan Drug Products in Development for 96 Relatively Rare Disorders

ABSTRACT EIGHTY-FIVE companies have 133 drugs in various stages of development for 96 rare diseases.This is the first comprehensive listing of orphan drugs since the Orphan Drug Act became law in 1983. The list was compiled by the Pharmaceutical Manufacturers Association in response to a request by the National Commission on Orphan Diseases (JAMA. 1989;261: 2938).More Than One Disease Some drugs are being studied for more than one rare disease. Of the 299 orphan drug designations granted by the Food and Drug Administration, 172 are in development and 39 designations involving 36 drugs have been approved.The 88 remaining designations are in early preclinical trial, are diagnostic agents, or are no longer under active investigation, says Gerald J. Mossinghoff, Pharmaceutical Manufacturers Association president. There are 64 research projects in the final stages of development and 40 of these are currently being reviewed for approval by the Food and Drug

The National Information Center for Orphan Drugs and Rare Diseases: Information Service on Treatment for 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.

Mechanistic studies of acetamide hepatocarcinogenicity.

Feeding male Leeds rats a diet containing 5 % acetamide (AA) for 17 months resulted in the development of hepatic cell neoplasms in all animals (Flaks et al., 1983). Many studies have reported that AA does not show genotoxic effects. However, Pienta et al. (1977) found that AA transformed Syrian hamster embryo cells in culture. N-Hydroxyacetamide (N-OH-AA), a possible metabolite of AA, has been approved by the US Food and Drug Administration under the orphan drug products program for treatment of infection-induced renal stones (in: Putcha et al., 1984). This study was undertaken attempting to shed light on the mechanism of AA hepatocarcinogenicity.

Developments Related to Orphan Drug Products

Historical and current initiatives in orphan product development are reviewed. FDA defines an orphan product as one that is potentially useful but is inadequately tested or unavailable because of limited commercial interest. From 1970-79,24% of 174 new chemicals approved by FDA were drugs of limited commercial value. The FDA Bureau of Drugs convened two interagency task forces in the 1970s to explore problems in developing orphan products. Recent initiatives in the private sector, including the PMA Commission on Drugs for Rare Diseases the Generic Pharmaceutical Industry Association's Institute for Orphan Drugs are described Commitments from manufacturers to develop 14 orphan products have been received by FDA since May 1982. Current activities within the public sector are described, including the responsibilities of the DHHS Orphan Products Board and the FDA Orphan Products Development Office. Provisions of the Orphan Drug Act passed on January 4,1983, that provide assistance to researchers and financial incentives to manufacturers are outlined. Current initiatives in the private and public sectors indicate a willingness to facilitate the development and availability of drugs for rare diseases.

Issues Aired Related to Orphan Drug Products at Washington Hearing

Journal Article Issues Aired Related to Orphan Drug Products at Washington Hearing Get access American Journal of Hospital Pharmacy, Volume 40, Issue 2, 1 February 1983, Pages 176–189, https://doi.org/10.1093/ajhp/40.2.176 Published: 01 February 1983

The Orphan Drug Products—Another Solution?

The Orphan Drug Products—Another Solution?