The National Institute of Health estimates that about 25 million Americans suffer from rare or ‘orphan’ diseases (“FAQ About Rare Diseases”). An orphan disease can be defined either as a disease that is often neglected by physicians or one that affects only a small number of people. J.K Aronson, the chairman of the editorial board for the British Journal of Clinical Pharmacology said that in the US it is defined as one that affects “fewer than 200,000 individuals”, though internationally these numbers vary greatly (Aronson). These diseases can be mild or life threatening, but in either case it can be difficult for a drug to be developed to treat the disease. Scientists and companies face a constant struggle between increasing the quality of, and potentially saving, lives and the cost-ineffective realities of creating a drug that will not sell in high quantities due to the small number of people who require it.
In 1983 the United States passed the Orphan Drug Act, which was intended to create incentives for the pharmaceutical industry to research and develop treatments for orphan diseases. This act gave tax breaks to companies doing this research as well as creating a 7 year long period during which there could only be one treatment per disease on the market, thereby eliminating competition. Since this act was passed, the FDA has approved around 200 treatments for orphan diseases (“FAQ About Rare Diseases”). Despite this, many orphan diseases have no treatment approved by the FDA. In fact 95% of these diseases have no approved treatments (“Rare Diseases: Facts and Statistics”). This lack of treatment is due to the fact that pharmaceutical companies recognize that, even with these additional benefits, by pure cost-effective analysis it is not economically lucrative to create orphan drugs.
Works Cited
Aronson, JK. “Rare Diseases and Orphan Drugs.” British Journal of Clinical Pharmacology 61.3 (2006): 243-245. Web
“FAQ About Rare Diseases.” NIH, n.d. Web.
“Rare Diseases: Facts and Statistics.” Global Genes. Web.
In 1983 the United States passed the Orphan Drug Act, which was intended to create incentives for the pharmaceutical industry to research and develop treatments for orphan diseases. This act gave tax breaks to companies doing this research as well as creating a 7 year long period during which there could only be one treatment per disease on the market, thereby eliminating competition. Since this act was passed, the FDA has approved around 200 treatments for orphan diseases (“FAQ About Rare Diseases”). Despite this, many orphan diseases have no treatment approved by the FDA. In fact 95% of these diseases have no approved treatments (“Rare Diseases: Facts and Statistics”). This lack of treatment is due to the fact that pharmaceutical companies recognize that, even with these additional benefits, by pure cost-effective analysis it is not economically lucrative to create orphan drugs.
Works Cited
Aronson, JK. “Rare Diseases and Orphan Drugs.” British Journal of Clinical Pharmacology 61.3 (2006): 243-245. Web
“FAQ About Rare Diseases.” NIH, n.d. Web.
“Rare Diseases: Facts and Statistics.” Global Genes. Web.