Congratulations Dr. Michael Okun on the publication of “Reforming the Process for Deep Brain Stimulation and Neurologic Device Approval in Rare Diseases,” in the November 9th issue of JAMA.
Traditionally, we have developed and offered neurologic therapies for the “many” (ie, individuals with epidemiologically more prevalent disorders), but the possibility exists to serve “the few” who have more infrequently occurring diseases and therefore represent smaller patient populations. The US Orphan Drug Act of 1983 defined a rare disease or condition as any pathology affecting fewer than 200 000 people. Pharmaceutical companies can request an orphan drug designation for the use of specific drugs and/or biologics in treating rare conditions. Incentives for obtaining this status include tax credits for human clinical trials, access to an Orphan Product Grants Program, waiver of the prescription drug user fee of $3 million, and market exclusivity for 7 years.1 Unfortunately, the process for neurotechnological devices that are developed and/or can be used for rare diseases or conditions does not entail, or benefit from, the same approval process.