FDA analysis of expanded access use in pediatric patients from 2015 to 2020.

Abstract

1529 Background: Expanded Access is a regulatory mechanism that enables patients with a life-threatening condition or serious disease to receive treatment with an investigational drug outside of a clinical trial when no comparable or satisfactory alternative options are available. FDA has decades of experience with expanded access, but little has been reported about its use in pediatric cancer patients. Methods: FDA’s central electronic database was queried for single-patient investigational new drug (spIND) applications submitted to the Office of Oncologic Diseases between January 2015 through December 2020. Data collection included IND receipt date, IND type/status, drug name, and patient demographics. Duplicate or exempt INDs, those cancelled by the physician-sponsor before initiating therapy, and those requested for indications that occur almost exclusively in adults (e.g. lung cancer) or were missing patient age were excluded. Results: Of 2,901 unique spINDs granted, 534 (18%) were for patients less than 18 years of age. The pediatric population was 57% male, median age 6.0 years (range 0.1 to 17); race/ethnicity were reported in <1%. Patients were treated in 132 zip codes across 39 states; one-quarter of submissions were from 5 large academic hospitals. Central nervous system tumors were the most common indication (Table 1). A total of 98 unique drugs were requested, with 1 to 73 spINDs for each drug; approximately 50% were for tyrosine kinase inhibitors, 25% for other small molecules, and the remainder for immunotherapies and other drug types. Median time for FDA to grant was 1 day. Follow-up information was provided for 75% (annual report or withdrawal letter); 1/3 were withdrawn within 1 year. Over the last 2 years, utilization of the program increased by 120%. Conclusions: While approximately 1% of all cancers per year are diagnosed in children under 17 years of age, 18% of spINDs over the last five years were for pediatric patients. Although utilization of this program for children is robust, efforts are needed to assess its impact on patient outcomes and ensure its availability to patients, families, and institutions more widely. These data highlight interest within the pediatric oncology community in accessing innovative therapies, which supports early investigation of promising new drugs in children.[Table: see text]