Optimizing Precision Medicine Cancer Clinical Trials

ABSTRACT

With the advent of precision medicine, getting the right treatment for the right patient at the right time has illuminated a variety of challenges and opportunities for innovation in trial design and conduct. Although there is no onesize- fits-all approach to precision medicine, a number of approaches, particularly basket and umbrella trial platforms that permit simultaneous evaluation of multiple treatments in multiple patient cohorts, have evolved to improve trial efficiency. The novel coronavirus pandemic has illuminated the need for, and feasibility of, conducting trials with fewer requirements, greater flexibility, and more decentralization. Through the lens of precision medicine cancer clinical trials, successes and challenges will be discussed to share practical solutions of how to improve evidence generation in the era of precision medicine. Through discussion of precision medicine cancer clinical trials within the United States, this session will provide an overview of how best to optimize these clinical trials. This session comprises the following three main topic areas matching treatments to patients, including the use of novel patient identification strategies, genomic matching rules, molecular tumor boards (MTBs), decision-support tools, incorporating precision medicine trials into a research portfolio, and how to overcome challenges;accelerating evidence development, including the use of adaptive trial designs, cohort management strategies and data sharing plans;and improving diversity of trial participants and increasing generalizability of study results through expanded eligibility criteria and site selection strategies. This topic will be explored through 90 min of invited talks and a panel discussion with Q&A. Moderator and session chair, Richard L Schilsky, MD, FACP, FSCT, FASCO, will introduce the session and speakers and provide an introduction on the basics of precision oncology. Timothy Cannon, MD, a medical oncologist and clinical trial researcher, will present a case study of two patients with the same alteration and discuss how the care for each differed based on access to a precision medicine cancer clinical trial. Dr. Cannon will also discuss how to implement precision medicine trials within a research portfolio and how to identify patients at a site. Christine Walko, PharmD, BCOP, FCCP, a pharmacist and researcher, will then discuss the basics of matching therapies to genomics, how to use decision support tools, the role of an MTB, and identifying therapeutic options for patients. Edward S Kim, MD, MBA, FACP, FASCO, a medical oncologist, will talk about MTBs from a clinician perspective and precision medicine cancer clinical trials from a community practice perspective. Dr. Kim will also speak about how to extend the research team to create an adequate community research portfolio and about using broader eligibility criteria that might facilitate enrollment of diverse populations. Jane Perlmutter, PhD, MBA, FASCO, a cancer survivor and patient advocate, will discuss what pragmatic trials are, how they increase the opportunity for diversity and generalizability, and patient perspectives regarding pragmatic and precision cancer clinical trials. Susan Halabi, PhD, FASCO, FSCT, a researcher and professor of biostatistics and bioinformatics, will talk about the best ways to design an adaptive trial, especially its role in real-world settings, how these types of designs can be used for efficient signal finding in rare populations, and how these trials can create opportunities for data sharing and collaboration. Pam K Mangat, MS, a research scientist and the Director of Clinical Research for American Society of Clinical Oncology's (ASCO) Targeted Agent and Profiling Utilization Registry (TAPUR) Study, will discuss the operations behind a precision medicine study, advantages and disadvantages of a pragmatic trial, cohort closing and collapsing rules to help with management of large numbers of small cohorts, and contributing knowledge to other trials. The session will conclude with a panel di cussion moderated by session chair, Richard L Schilsky, MD, FACP, FSCT, FASCO, for approximately 10 min, with 5 min for a Q&A session.