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J Clin Oncol. 2017 Nov 20;35(33):3737-3744. doi: 10.1200/JCO.2017.73.7916. Epub 2017 Oct 2.

Broadening Eligibility Criteria to Make Clinical Trials More Representative: American Society of Clinical Oncology and Friends of Cancer Research Joint Research Statement.

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Edward S. Kim, Levine Cancer Institute, Carolinas HealthCare System, Charlotte, NC; Suanna S. Bruinooge, Caroline Schenkel, and Richard L. Schilsky, ASCO, Alexandria, VA; Samantha Roberts, Marina Kozak, Jeff Allen, and Ellen Sigal, Friends of Cancer Research; Samantha Roberts, Genentech, Washington, DC; Gwynn Ison, Julia A. Beaver, Rajeshwari Sridhara, and Tatiana M. Prowell, US Food and Drug Administration, Silver Spring; Thomas S. Uldrick and Andrea M. Denicoff, National Cancer Institute, Bethesda, MD; Nancy U. Lin, Dana-Farber Cancer Institute, Boston; Paul J. Hesketh, Lahey Health Cancer Institute, Burlington, MA; Lia Gore, University of Colorado School of Medicine and Children's Hospital Colorado, Aurora, CO; Stuart M. Lichtman, Memorial Sloan-Kettering Cancer Center, New York, NY; Nancy Roach, Fight Colorectal Cancer, Springfield, MO; Elizabeth Garrett-Mayer, Hollings Cancer Center, Medical University of South Carolina, Charleston, SC; Eric Rubin, Merck Research Laboratories, Kenilworth, NJ; and Pratik Multani, Ignyta, San Diego, CA.


Purpose The primary purposes of eligibility criteria are to protect the safety of trial participants and define the trial population. Excessive or overly restrictive eligibility criteria can slow trial accrual, jeopardize the generalizability of results, and limit understanding of the intervention's benefit-risk profile. Methods ASCO, Friends of Cancer Research, and the US Food and Drug Administration examined specific eligibility criteria (ie, brain metastases, minimum age, HIV infection, and organ dysfunction and prior and concurrent malignancies) to determine whether to modify definitions to extend trials to a broader population. Working groups developed consensus recommendations based on review of evidence, consideration of the patient population, and consultation with the research community. Results Patients with treated or clinically stable brain metastases should be routinely included in trials and only excluded if there is compelling rationale. In initial dose-finding trials, pediatric-specific cohorts should be included based on strong scientific rationale for benefit. Later phase trials in diseases that span adult and pediatric populations should include patients older than age 12 years. HIV-infected patients who are healthy and have low risk of AIDS-related outcomes should be included absent specific rationale for exclusion. Renal function criteria should enable liberal creatinine clearance, unless the investigational agent involves renal excretion. Patients with prior or concurrent malignancies should be included, especially when the risk of the malignancy interfering with either safety or efficacy endpoints is very low. Conclusion To maximize generalizability of results, trial enrollment criteria should strive for inclusiveness. Rationale for excluding patients should be clearly articulated and reflect expected toxicities associated with the therapy under investigation.

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