Orphan disease

The emphasis of the program is to support projects that complement, rather than compete with the private sector. Some examples are projects related to high-risk and novel targets, orphan diseases, and areas in which the monetary return may not be immediately clear. 

Seven years exclusive market following the approval of a product for an orphan disease. 

Orphan diseases are those that affect such a small number of patients that the market caimot sustain the cost of research to find treatments. For some time, the agency, the industry and patient support groups had recognised the problem indeed, the FDA worked with various drug firms to find homes for potentially valuable orphan products. Nevertheless, the economics worked against those with rare diseases. 

Freidreich s ataxia, Striimpell-Lorrain disease, Fewy body dementia, myopathies, certain neuropathies, sleeping sickness (tsetse) are just a few of these orphan diseases which certainly merit more aggressive research. 

I telephoned Max Tishler, outlined the situation, and he said he would call back that afternoon. Max contacted the Merck Medical Department, who stated that Wilson s Syndrome was a rare condition affecting only about 50 to 100 patients a year, and was terminal. However, Max was able to launch a crash program to prepare penicillamine at Merck and get quick FDA approval under the "orphan disease category, in spite of the unpromising commercial outlook. 

Where a drug is not developed into a usable medicine because the developer will not recover the costs then it is known as an orphan drug, and the disease is an orphan disease the sufferer is a health orphan. Drugs for rare diseases inevitably must often be licensed on less than ideal amounts of clinical evidence. 

Scheinberg, I.H., Walshe, J.M., Eds. Orphan Diseases and Orphan Drugs, Manchester University Press Manchester, UK, 1986. 

Market exclusivity is awarded for certain uses for a drug product, such as to treat or prevent orphan diseases. In this case, the most powerful incentive for a company to develop a product with a small target population is the Orphan Drag Act s market exclusivity clause. 

The special populations covered in the following chapters include the four major demographic segments the elderly, women, children and major ethnic groups and although any smaller grouping of people or diseases may be labeled special , only renal and hepatic patients and orphan disease... 

In the United States, rare disease is defined as a disease with a prevalence of less than 200000 patients. Some countries have defined a rare disease based on a prevalence of 0.1-0.5% of the population. A rare disease is sometimes referred to as an orphan disease. An orphan drug is defined as a drug to treat a rare disease. The term orphan drug originated from the belief that there were drugs that no pharmaceutical sponsor wanted to develop and market, and thus they were like homeless orphans. 

Burley D. 1986. The decline and fall of thalidomide . In Orphan Diseases and Orphan Drugs, Scheinberg IH (ed.). Manchester University Press (in association with The Fulbright Commission) London. 

Cosier,. I.M. Recombinant Erythropoietin Orphan Product with a Silver Spoon. Intern. J. Technol. Assess. Health Care 1992, 8 (4), 635-646. 45. Scheinberg, I.H., Walshe, J.M. Eds. Orphan Diseases and Orphan Drugs Manchester University Press Manchester, UK, 1986. 

In its recent proposed regulations (adopted as final in December 1992, the FDA makes clear that the 200,000 prevalence figure means 200,000 affected persons in the United States at the time that the orphan-drug designation request is made (not 200,000 new cases annually) and that a drug would remain an orphan drug even if the disease or condition ceases to an orphan disease or condition because of increased prevalence in order to protect a sponsor s good-faith investment (56 FR 3339) ... 

We present in this Chapter an overview on these orphan diseases from the perspective of opportunities offered by developments in lipidomics. The severity of these conditions, which are usually progressively debilating, become manageable by therapies that considerably delay the appearance of the consequent irreversible damage. 

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