Clinical Drug Development for Rare Disease in Neurology (P2.194)

Objective: The goal of this project is to screen the approved orphan drug products in neurology and characterize the contents of the clinical pharmacology packages submitted to the drug applications. In addition, it was intended to identify the characteristics of the efficacy studies that supported the approval of these drug products and identify instances where FDA provided flexibility in reviewing these applications. Background: Drug development for rare diseases in neurology can be very challenging due to small number of patient available for clinical trials, poor understanding of the disease. It’s essential that the FDA recognize the need for flexible regulatory policies for establishing drug safety and efficacy given the consideration of the inherent limitation in rare diseases. Design/Methods: We used publically available databases including Drugs@FDA and orphan drug database, to screen the approved drugs with orphan indication in neurology. We screened the clinical pharmacology packages and efficacy trials from drug labels and assessed multiple elements of clinical trial design including, randomization, control, study design, number of participants, blinding, length of study, number of pivotal studies and number of doses tested. Results: 20% of neurological products approved through 2015 have orphan indications. About 53% of these products had other non-orphan indication and the majority of these products were developed to treat seizures and movement disorders with limited number of drugs developed for other neurological disorders such as head and spinal trauma. 22% of neurological products were approved based on a single, well controlled efficacy trial. Most of the orphan applications used parallel designs and tested only one dose or used titration to effect dosing. Conclusions: Implementation of innovative trial designs and flexible methods of analysis for small clinical trials can improve the drug development for rare disease. Moreover, clinical pharmacology can enhance the quantity and quality of data needed to satisfy the regulatory standards for approval. Disclosure: Dr. Abuasal has nothing to disclose. Dr. Albusaysi has nothing to disclose. Dr. Bhattaram has nothing to disclose. Dr. Uppoor has nothing to disclose. Dr. Mehta has nothing to disclose.