Barriers to implementing expanded access programs in oncology: A multidisciplinary perspective.

Sherry Fu UT Southwestern Medical Center, Dallas, TX info_outline Sherry Fu, Maishara Muquith, Erin Fenske Williams, Larry D. Anderson, Jr, Tian Zhang, Shaalan Beg

Authors Sherry Fu UT Southwestern Medical Center, Dallas, TX info_outline Sherry Fu, Maishara Muquith, Erin Fenske Williams, Larry D. Anderson, Jr, Tian Zhang, Shaalan Beg Organizations UT Southwestern Medical Center, Dallas, TX, University of Texas Southwestern Medical Center, Dallas, TX, Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center, Dallas, TX, Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, Dallas, TX Abstract Disclosures Research Funding No funding received None. Background: Expanded access (EA) is a pathway to make investigational drugs available to patients in need but who are unable to join a trial. Despite the rapid approval time for expanded access requests by the Food and Drug Administration, EA protocols ( > 1 patient) and single-use EA have limited utilization due to barriers affecting physicians, pharmacists, and clinical research office staff. We examined factors limiting the broad implementation of EA at an NCI-designated comprehensive cancer center. Methods: Semi-structured interviews were conducted with oncologists (5), pharmacists (2), and research operations leadership (1) involved with applying for and facilitating expanded access at our institution. Interviews were transcribed verbatim and grouped into themes by 2 independent coders. Results: At our institution, recent EA requests for adult patients with cancer involved anti-viral and CAR-T therapies. Participants agreed that increased EA utilization benefits patients and potentially the institution, provided the workflow is not burdensome. Major barriers include gaps in physician knowledge of institutional processes and lack of response from drug sponsors on ability to supply drug. For EA protocols ( > 1 patient), there was lack of reimbursement for time and resources, particularly for regulatory coordination and pharmacy services, which often competed with clinical trial needs. EA requests are part of standard clinical care but may require reporting patient-level data to sponsors and resources similar to a clinical trial. This contributes to unclear delineation of roles between research and clinical staff, which delays communication with the pharmacy and adverse event reporting to sponsors. Across all departments, there was a desire for a centralized office or dedicated individual to support EA processes. Conclusions: Oncology professionals recognize the benefit of EA for patients with cancer. However, utilization is limited by various challenges including lack of clearly defined roles and financial support. Suggested improvements include establishing a centralized resource at the institutional level to streamline workflows. External parties such as industry, payers and advocacy groups can also enhance EA activity through defined protocols and resources for administrative support.