The Orphan Drug Act: the first 7 years
Article Abstract:
An evaluation is presented of several issues raised by the Orphan Drug Act (Public Law 97-414), passed in 1983 to stimulate the development of drugs for treating rare diseases. Such drugs (''orphans'') are not profitable for pharmaceutical companies because of the high research and development costs relative to the number of potential consumers, and the intent of the law was to provide marketing and tax incentives for drug development. The problems faced by companies in developing orphan drugs included not only the small market and considerable costs of development, but the lack of patent protection and the large number of liability claims filed. Each of these points is discussed in detail. The Orphan Drug Act applies to drugs for treating conditions that affect 200,000 or fewer people in the US. The major incentives it provides are seven-year exclusivity (guaranteed to the manufacturer) of a drug designed to treat a particular indication, and tax credits (a fixed percentage on the dollar). Between 1983 and the end of 1989, 42 orphan drugs were developed and approved from 30 firms. Most are for treating conditions that affect fewer than 50,000 people. Controversy has arisen over the issue of market exclusivity, because it can be difficult to decide whether related products are really different products, and whether monopolies, in fact, exist when profits become excessive. Thus, although 25 of 40 orphan drugs have less than $1 million in annual sales, 3 have sales totalling more than $100 million each. These are discussed. Proposed changes in the law have sought to eliminate orphan status for drugs that would serve more than 200,000 people within three years of product approval, and to prevent drugs such as aerosol pentamidine (one of the three very profitable products), designed to treat a disease that affects AIDS patients, from becoming too expensive. However, the bill that would have done this was vetoed by President Bush. The major unresolved issues concern market protection (exclusivity) and pricing; these are discussed. Companies, the public, and patients must all play a role in future considerations of the issue. (Consumer Summary produced by Reliance Medical Information, Inc.)
Publication Name: JAMA, The Journal of the American Medical Association
Subject: Health
ISSN: 0098-7484
Year: 1991
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Rechallenge with crystalline niacin after drug-induced hepatitis from sustained-release niacin
Article Abstract:
Niacin (nicotinic acid), a drug used to regulate blood lipid (fat) levels, is associated with side effects that are harmful to the liver. The drug is available in several forms, including a crystalline form and sustained-release (SR) capsules. The case studies of three patients who developed hepatitis while taking SR niacin are presented. One, a 62-year-old man, had been treated with crystalline niacin for high levels of low-density lipoprotein cholesterol (one form in which cholesterol is stored). He received the drug in this form for five months, then changed to SR niacin. After five days, the patient became nauseated, and fainted after drinking a glass of champagne. Tests for liver function were abnormal, and niacin was withheld. The patient recovered and resumed taking crystalline niacin. The second patient, a 50-year-old woman with familial hypercholesterolemia (high blood cholesterol), took SR niacin and developed impaired liver function. However, she was able to take the crystalline form. Crystalline niacin was prescribed for the third patient, a 47-year-old man, because of elevated cholesterol, but, based on his pharmacist's advice, he changed to the SR form. This led to abnormal liver functions tests and symptoms which disappeared when the crystalline form was re-instituted. In summary, it is likely that niacin will be prescribed more frequently in the future, since the National Cholesterol Education Program calls for aggressive treatment of high cholesterol levels. The cause of niacin-induced hepatitis is not known. However, the ease of access to SR preparations, which do not require a prescription, is cause for concern. (Consumer Summary produced by Reliance Medical Information, Inc.)
Publication Name: JAMA, The Journal of the American Medical Association
Subject: Health
ISSN: 0098-7484
Year: 1990
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Drug allergy
Article Abstract:
A 67-year-old patient taking multiple medications for various conditions developed edema, or swelling of her throat, which was initially ascribed to ampicillin, a derivative of penicillin. Although a penicillin skin patch test indicated a penicillin allergy, her swollen throat was caused by enalapril prescribed to control high blood pressure. Penicillin patch tests can predict 50% to 75% of drug reactions in patients with previous adverse penicillin reactions, but in only 20% of patients with no previous reaction. To find a drug causing a reaction, the physician must list all drugs the patient has received recently and is currently receiving in order of the possibility of causing reactions, and a chronological history of symptoms and medication. Allergic reactions do not usually occur more than 48 hours after the patient has received a drug or cross-reactive compound of similar structure.
Publication Name: JAMA, The Journal of the American Medical Association
Subject: Health
ISSN: 0098-7484
Year: 1992
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