In July 2013, the Food and Drug Administration (FDA), provided guidance on the modifications to the Orphan Drug Act (ODA) (Pub. L. 97-414, 21 CFR Part 316).
The ODA was originally promulgated in 1983 to address pharmaceutical treatment options for rare diseases and conditions in the United States. Such conditions include myoclonus and muscular dystrophy. According to a Seattle Times article, "30 years ago, Congress passed the Orphan Drug Act as a way to lure pharmaceutical companies to develop drugs for rare diseases that had been "orphaned" - abandoned or ignored because they were unprofitable." While some contend that this provision has been exploited, there are, as Congress recently found, benefits as a matter of public policy.
After Congressional consideration regarding the market patent protection, tax incentives, and decreased testing time, the law stands, as amended. The Congressional rationale included the following: Diseases exist in this country that are rare and need to be addressed; drugs that would be adequate have not been developed; pharmaceutical companies have less of a chance of recouping the R&D cost because of the limited patient population; and from a public policy standpoint, it is important to provide "changes and incentives for the development of orphan drugs."
For physicians who treat patients with rare conditions, there are some important reasons to be familiar with the ODA. First, an alternative treatment may exist. Second, a patient or family member may find something on the Internet. And, the cost of these drugs is often expensive. A patient may have the option of subsidies through the manufacturer to either offset the cost or have it covered completely. These discussions can be intense and the more educated the physician is, the more he/she will be able to treat the patient.