Project Summary & Abstract The Patient Advocacy Committee of the Consortium for Pediatric Cellular Immunotherapy supported by the NCATS U01 to “Accelerate cellular immunotherapy for treatment of life-threatening disorders” has been focused on retrospectively examining whether or not there is equitable enrollment by underrepresented populations such as lower socioeconomic and minority populations in cellular therapy trials across the member institutions. Concern exists that unique, identifiable patient cohorts may face barriers to recruitment and/or enrollment on these trials. For instance, referring clinicians may be limiting patients’ access to the consortium institution trials due to inadequate insurance status, English language proficiency, geography, family characteristics or other factors. Thus, it is important to understand the referring clinician perspective to determine how these clinicians decide whom to refer and therefore whether there are limitations impacting referral and study enrollment patterns. Furthermore, to date, the Committee has focused on patients and families that have enrolled on cellular therapy trials. We understand, however, that it is vital to prospectively analyze the perspectives and experiences of those families unable to enroll and those who encounter barriers to participation. Further elucidating clinician- and family-level factors that may influence referral patterns can help with future approaches to clinical trials for emerging therapies to improve equity to study participation. Therefore, through this supplemental bioethics application, we aim to: 1) understand how clinicians make patient referral decisions for specialized pediatric immunotherapy trials and determine how these decisions might contribute to inequitable access; and 2) identify the key barriers to recruitment and enrollment in cellular therapy trials, as identified by families. These data will inform policies to ensure that resources and systems are in place to optimize equitable participation through robust trial design and that recruitment and enrollment for novel emerging therapies for pediatric diseases are equitably accessible. Finally, this work will generate pilot data for a subsequent R01 application that proposes to longitudinally examine the family experience and clinician referral patterns across novel pediatric emerging therapies, including cellular and gene therapies, across multiple consortia.

Project Narrative We will evaluate referring clinicians’ limitations regarding referrals for participation in cellular immunotherapy clinical trials to understand where and how these affect the referral process and to ensure equitable trial design of future trials conducted by the Consortium for Pediatric Cellular Immunotherapy. Similarly, understanding the perspectives and experiences of families that do and do not enroll on such trials will also provide a wealth of data to help define the resources, structure, and methodology necessary to ensure equitable future recruitment and enrollment. These analyses should inform the development of a framework that we will employ to optimize recruitment and participation practices across cellular therapy trials and in other novel pediatric therapies; the overarching aim of this proposal is to ensure that future advances in cell therapies for children are accessible to—and benefit—all eligible pediatric patients across the full socioeconomic and ethnic spectrum.