Pharmaceutical companies are constantly researching and developing new medications to treat medical conditions, and new drugs come on the market frequently. People who have rare diseases or disorders, however, have not had as much research attention in past decades. This is because their numbers are small and therefore the potential market for new drugs to treat them (commonly referred to as "orphan drugs") is also small. A rare disease occurs in less than 200,000 individuals in the United States, or less than 5 per 10,000 individuals in the European Union. Government regulatory agencies in the United States and the European Union have thus taken steps to reduce this disparity and offers some financial incentive for the pharmaceutical companies to devolep medication for these diseases or conditions.
The U.S. Office of Orphan Product Development
The U.S.FDA is responsible for ensuring the safety and efficacy of medications on the market in the United States. The FDA established the Office of Orphan Product Devolepment (OOPD) to facilitate the development of orphan drugs (and other medical products for rare disorders), including offering research grants.
Orphan drugs, like other medications, still have to be proven that they are safe and effective through research and clinical trials before the FDA will approve them for marketing.
European Union and Orphan Product Development
Like the U.S. Congress, the EU government recognized the need to increase research and development of orphan drugs. The Regulation on Orphan Medicinal Products, passed by the European Council, provides incentives for the development of orphan drugs.Companies with an orphan designation for a medicinal product benefit from incentives such as:
Marketing authorisation (10-year marketing exclusivity)
Financial incentives (fee reductions or exemptions);