Project facilitate: A review of the FDA oncology center of excellence expanded access pilot program.

Abstract

7023 Background: Expanded Access (EA), also known as “compassionate use,” is a regulatory pathway in which a patient with an immediate life-threatening condition or disease can gain access to an investigational product for treatment when no satisfactory therapy is available. Oncology practices may lack the regulatory experience or administrative support to use EA. In response, FDA OCE launched Project Facilitate (PF), a call center to assist oncology healthcare providers requesting EA. An analysis of single-patient investigational new drugs (IND) was performed to assess the first 10 months of PF compared to the period prior to its launch. Methods: Preliminary data was extracted from the FDA’s central database that yielded 719 single-patient INDs between May 31-November 30, 2018 & 2019 in the Office of Oncologic Diseases (OOD). Data collected included IND receipt date, acknowledgment date, application status, drug name, underlying malignancy of patient, address of requesting physician, withdrawal date, and patient demographics. A manual review of INDs was performed to assess for actual processing dates and to capture demographics not captured by the database. A total of 28 INDs were excluded due to duplications, cancellation by Sponsor prior to issuance of FDA decision, or coding errors in the database. Industry denial explanations were reported by the provider by emails. Results: Data from 692 INDs were analyzed and 692 (100%) were granted safe to proceed. The median processing time was 1 day (mean=2) in 2018 and 1 day (mean= 1.5) in 2019. Our findings indicate that the volume of oncology EA requests increased by 76 (19%) in 2019 vs 2018. A total of 207 unique drugs were requested. Malignancies most frequently involved included: Acute myeloid leukemia (n = 84, 8.3%), soft tissue sarcoma (n = 77, 7.6%), and non-small cell lung cancer (n = 60, 5.9%). States with the highest requests included: California (n = 82, 11.8%), New York (n = 81, 11.7%), and Massachusetts (n = 42, 6.1%). A majority of requests were from major academic centers (77%). All denied requests (N = 9) by industry were due to company’s decision to not provide products outside of a clinical trial. Conclusions: The positive trends in decreased processing times and increased number of requests are consistent with OCE’s mission to improve efficiency of the EA program and ensure equitable access to all oncology patients. [Table: see text]