TRLS-01. FDA ANALYSIS OF EXPANDED ACCESS USE IN PEDIATRIC PATIENTS WITH CNS TUMORS FROM 2015 TO 2020

Abstract

Abstract Few therapies are approved for children with central nervous system (CNS) tumors, and their prognosis is often poor. Expanded Access (EA) is a regulatory mechanism enabling patients with life-threatening or serious diseases to receive treatment with investigational drugs outside of clinical trials when no comparable or satisfactory options are available. FDA’s central database was queried for single-patient investigational new drug (spIND) applications submitted to the Office of Oncologic Diseases between January 2015-December 2020. Data collection included IND date/type/status, drug name, patient demographics. Duplicate/exempt INDs, those cancelled by physician-sponsor before initiating therapy, and those for indications occurring almost exclusively in adults or missing patient age were excluded. Of 2,901 unique spINDs granted, 534 (18%) were for patients <18 years old; of those, 229 (43%) were for patients with nervous system tumors. Demographics included: 53% male, median age 5.0 years (range 0.3-17); race/ethnicity reported in <1%. Patients were treated in 78 zip codes across 33 states; one-third of submissions were from 5 large academic hospitals. Tumor types included low-grade glioma (71), plexiform neurofibroma (35), high-grade glioma (32), DIPG/DMG (30), ATRT (27), medulloblastoma (11), ependymoma (11), other CNS tumors (12). Thirty-eight unique drugs were requested (1 to 56 spINDs per drug); 60% for kinase inhibitors, 30% for other small molecule inhibitors, and the remainder for immunotherapies/other drug types. Median time for FDA to review and deem safe to proceed was 1 day. While pediatric CNS tumors are rare, these patients made up almost half of spINDs granted for pediatric patients. Ongoing efforts are needed to assess the impact of EA on patient outcomes and ensure availability to patients, families, and institutions more widely. These data highlight interest within the pediatric neuro-oncology community in accessing innovative therapies, which supports early investigation of promising new drugs for these patients.