Orphan diseases are rare diseases, such as Lou Gehrig's disease, Huntington's disease, and Tourette's syndrome, that effect less than 200,000 individuals each. There are about 7,000 such diseases classified, affecting approximately 25 million Americans. Diagnosing such rare diseases is difficult, taking months and sometime years, leaving individuals with orphan diseases feeling helpless and isolated. Aside from this fact, health care provisions for these individuals have been lacking in the past, with little to no access to adequate prescription drugs or therapies, as well as a preponderance of outrageous health insurance quotes, and even a refusal of treatment.

The lack of adequate health care for people with orphan diseases, specifically with regards to prescription drug coverage, stems from the rarity of the diseases themselves. With little market and demand for such prescription drugs and therapies, pharmaceutical companies have had little incentive or desire to invest resources into researching treatment options. The Orphan Drug Act of 1983 along with more recent health care reforms, however, have made treatment and prescription drugs much more accessible for people with orphan diseases. One of the main reasons reform has been so helpful to the development of orphan drugs is that pharmaceutical companies now have access to funding and incentives for research and development of prescriptions specifically for orphan diseases. This had led to more than 250 approved products for orphan disease treatment since the passing of the Orphan Drug Act. There are now patient assistance programs and affordable health insurance quotes available to people with orphan diseases so they can gain access to the treatments and prescription that are available, albeit some are in limited supply. Additionally, the inception of an Orphan Products Board in the Department of Health and Human Services will ensure that people affected with orphan diseases will have a voice and representation when it comes to the research and development of drugs and treatments for them.

More recently the FDA has recognized over 200 treatments and drug therapies that are used for other more common diseases that may have an impact on orphan diseases as well. They are now pushing for pharmaceutical companies to renew their research into orphan diseases, and how current medicines and therapies may be used for treatment as well. This has the potential to increase the health care options for people with orphan diseases, but also to increase the profits of pharmaceutical companies by marketing their drugs for a wider variety of purposes. Moreover, research into these rare diseases may play a part in solving more common diseases, which should be the final goal of the health care industry.