Expanded access for cancer patients: The 5-year FDA CBER experience.

Abstract

e18158 Background: Expanded access (EA) refers to the use of an investigational drug when the primary purpose is to diagnose, monitor, or treat a patient’s disease rather than to generate scientific data that is generally derived from clinical trials. FDA has long facilitated expanded access to investigational drugs for patients with serious or immediately life-threatening diseases and who lack therapeutic alternatives. EA requests include single-patient use protocols under existing INDs, single-patient investigational new drug applications (INDs or SPIs), and intermediate-size or treatment protocols/INDs. Methods: We searched the Center for Biologics Evaluation and Research (CBER) internal databases and reviewed all EA requests for cancer patients to receive investigational products regulated by CBER's Office of Tissues and Advanced Therapies (OTAT) from 2014-2018. These products included cellular and gene therapies, cancer vaccines and microbe-based therapies. Results: From 2014-2018, OTAT received 395 EA requests for solid tumor (ST) or hematologic malignancies (HM). One-hundred ninety-seven (197) requests were for individuals who could not be enrolled in an ongoing oncology clinical trial to receive an investigational agent (single-patient use protocols). Of 197 requests, 7 (3%) were either withdrawn or canceled by the treating physicians (sponsors). The remaining 190 (100%) were approved. One hundred seventy-three (173) EA requests, 102 for ST and 71 for HM, came from sponsors to make available to patients an investigational agent for which there were no ongoing clinical trials (SPIs). Of 173 requests, 45 (26%) were withdrawn or cancelled by the sponsors. Of the remaining 128, 127 (99%) were approved. One request was denied. Twenty-five of 395 EA requests were for intermediate-size or treatment protocols/INDs. Conclusions: From 2014-2018 there has been an increase in oncology EA requests for products regulated by CBER/OTAT, which pose unique challenges for evaluating their risks and benefits. Despite the complexities of these novel biological products, nearly all EA requests were approved.