Roundtable Session Proposal
Title: "Derivations of real-world data (RWD) oncology endpoints: challenges and opportunities."
Roundtable Leader:
Ilana Trumble, PhD
303-817-4892
[email protected]Proposal:
The role of real-world data (RWD) in the design and execution of clinical trials is ever increasing. Of note is the recent surge in the use of RWD to build external control arms, especially for rare disease or biomarker driven oncology trials where a perceived effective standard of care does not exist (Izem et al., 2022; Majumdar et al., 2022; Mishra-Kalyani et al., 2022). However, RWD can be useful more broadly to a clinical development program, beyond external controls. For example, RWD can be used to validate associations between short-term response-based endpoints and long-term survival endpoints. RWD can be used to assess endpoint differences between similar classes of drugs, for example, to differentiate between multiple PD(L)-1 agents. Such use cases require careful derivations of endpoints, often involving making clinically reasonable assumptions, a detailed understanding of the RWD patient experience, and knowing the limitations of translating the RWD experience to clinical trials. In this roundtable session, we will discuss challenges and opportunities specific to the use of RWD data, particular as related to endpoint derivations to inform clinical development in the Phase I to III oncology setting.
The roundtable discussion will benefit from the participation of statisticians from industry, regulatory agencies, and academia. Topics may include: challenges, tradeoffs, and solutions when defining clinical, pathological, and survival endpoints for RWD; challenges, tradeoffs, and solutions when defining I/E criteria for RWD; the use of RWD to justify novel oncology endpoints; regulatory agencies' considerations when assessing the use of RWD in oncology trial design and analysis; the importance of involving multidisciplinary teams in decision making and strategies for effectively collaborating with those teams; participants own experiences and advice pertaining to the use of RWD to inform oncology trials; and how RWD and these discussions have the potential to create positive impacts on global health.
About the Roundtable Leader:
Dr. Ilana Trumble is a Principal Statistician in the Oncology Biostatistics division of GSK. As a member of the Disease Area Strategy team, Dr. Trumble uses RWD to make trials more efficient and less risky for patients. Dr. Trumble obtained her degree in Biostatistics from the Colorado School of Public Health.
References
Izem, R., Buenconsejo, J., Davi, R. et al. Real-World Data as External Controls: Practical Experience from Notable Marketing Applications of New Therapies. Ther Innov Regul Sci 56, 704–716 (2022). https://doi.org/10.1007/s43441-022-00413-0
Majumdar, A., Davi, R., Bexon, M. et al. Building an External Control Arm for Development of a New Molecular Entity: An Application in a Recurrent Glioblastoma Trial for MDNA55. Stat Biosci 14, 285–303 (2022). https://doi.org/10.1007/s12561-022-09337-7
Mishra-Kalyani PS, Amiri Kordestani L, Rivera DR, Singh H, Ibrahim A, DeClaro RA, Shen Y, Tang S, Sridhara R, Kluetz PG, Concato J, Pazdur R, Beaver JA. External control arms in oncology: current use and future directions. Ann Oncol. 2022 Apr;33(4):376-383. https://doi.org/10.1016/j.annonc.2021.12.015. Epub 2022 Jan 10. PMID: 35026413.