Project Facilitate: An analysis of the increased utilization in the oncology single patient expanded access pathway.

Abstract

11029 Background: FDA Oncology Center of Excellence’s (OCE) Project Facilitate (PF) was launched in June 2019 in response to perceived barriers providers face when accessing Expanded Access (EA). PF staff provide step-by-step support to oncology healthcare providers requesting use of investigational drugs to treat their patients with cancer. Since launch, PF has received an increased number of oncology single patient EA applications each calendar year. An analysis of single patient investigational new drug applications (IND) received was performed over each calendar year, starting June 1, 2020. Methods: Data was extracted from PF’s central database between June 1, 2020 and December 31, 2023. Data collected included IND receipt date, granting date, status, application type, indication, drug name, address of requestor, and patient demographics. The starting date of June 1, 2020 was chosen since it was when PF staff officially started performing clinical review of applications. Results: Of the 2791 oncology single patient expanded access applications extracted, there were 681 (24.4%) emergency INDs (eIND) and 2110 (75.6%) non-emergency single patient INDs (SPI). Applications received by calendar year increased since June 1, 2020 to December 31, 2020 (397, 14.2%), 2021 (682, 24.4%), 2022 (822, 29.5%), and 2023 (890, 31.9%). The top-3 disease areas requested include high grade glioma (262, 9.4%), multiple myeloma (213, 7.6%), and melanoma (186, 6.7%). The top-3 disease areas requested in pediatric patients < 17 years of age (568, 20.4%) include AML (88, 15.5%), high grade glioma (85, 15%), and ALL (47, 8.3%). Race and ethnicity information was provided in 32 applications containing white/Caucasian (24, 75%), Black or African American (4, 12.5%), Asian (2, 6.25%), and Hispanic or Latino (2, 6.25%). Applications were received from 46 states and 1 US territory, Puerto Rico. The top 3 states requestors resided in were CA (346, 12.4%), NY (317, 11.4%), and TX (276, 9.9%). Although rare, 22 (0.79%) applications were denied or placed on clinical hold most commonly due to curative or established therapies being available. Conclusions: Project Facilitate saw a 20.4% increase in oncology single patient expanded access requests received from calendar year 2021 compared to 2022 and an 8.3% increase from 2022 to 2023, equaling a 30.4% increase in 2023 compared to 2021. [Table: see text]