The FDA Drug Act provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor. For a drug to qualify for approval both the drug and the disease or condition must meet certain criteria specified in the FDA’s implementing regulations. FDA approval qualifies the sponsor of the drug for various development incentives, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received FDA designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.
Each designation request must stand on its own merit. Sponsors requesting designation of the same drug for the same rare disease or condition as a previously designated product must submit their own data and information in support of their designation request. The granting of a designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. Safety and effectiveness of a drug must be established through adequate and well-controlled studies.
On December 23, 2015, the DEA eased some of the regulatory clinical requirements impose by the Controlled Substance Act (CSA) for those who are conducting FDA-approved trials on cannabidiol (CBD), an extract of the cannabis plant. These modifications will facilitate the exploration of CBD, a compound which has already shown significant medicinal promise, and which has generated compelling interest in exploring its therapeutic potential.