Orphan diseases affect too few people to interest the investment of capital in research for cures and therapies. There are various definitions of what precisely constitutes an orphan disease or a rare disorder.
Orphan drugs are pharmaceutical medications developed to treat orphan diseases. The dilemma is that pharmaceutical companies claim they cannot make a profit in their production, so in the USA they are given huge tax credits - and allowed to hold the patent for 20 years (three times longer than the patent for the average drug). Nonetheless, these orphan prescriptions are priced above $400,000 a year per patient. And some are priced at $1 million per treatment. The FDA has allowed stem cells and natural gene therapies, or biologics, to be labelled as orphan drugs and allowed the same commercial benefits. In 2020, gene therapies make up 60% of orphan drugs and comprise 75% of the biggest individual drug revenues.
Rare disorders strike people of all ages, races and ethnic background. Many of these disorders are genetic, for example: Tay Sachs disease, Huntington's disease and cystic fibrosis. Some disorders are acquired such as AIDS and legionnaires' disease. A number of rare disorders fall within the realm of widespread health conditions such as heart disease and cancer.
The Canadian Organization for Rare Disorders claims that one in twelve individuals in Canada have been diagnosed with a rare disorder. There are more than 5,000 rare disorders that, taken together, affect approximately 2.5 million Canadians. A rare or "orphan" disease affects fewer than 20,000 people in Canada.
A rare or "orphan" disease affects fewer than 200,000 people in the United States. There are more than 5,000 rare disorders that, taken together, affect approximately 20 million Americans. One in every 12 individuals in this country has received a diagnosis of a rare disease.