Reducing clinical trial eligibility barriers for patients with MDS: an icMDS position statement
Uma Borate, Kelly Pugh, Allyson Waller, Rina Li Welkie, Ying Huang, Jan Philipp Bewersdorf, Maximilian Stahl, Amy E. DeZern, Uwe Platzbecker, Mikkael A. Sekeres, Andrew H. Wei, Rena Buckstein, Gail J. Roboz, Michael R. Savona, Sanam Loghavi, Robert P. Hasserjian, Pierre Fenaux, David A. Sallman, Christopher S. Hourigan, Matteo Giovanni Della Porta, Stephen D. Nimer, Richard F. Little, Valeria Santini, Fabio Efficace, Justin Taylor, Guillermo Garcia‐Manero, Olatoyosi Odenike, Tae Kon Kim, Stephanie Halene, Rami S. Komrokji, Elizabeth A. Griffiths, Peter L. Greenberg, Mina L. Xu, Zhuoer Xie, Rafael Bejar, Guillermo Sanz, Mrinal M. Patnaik, María E. Figueroa, Hetty E. Carraway, Omar Abdel‐Wahab, Daniel T. Starczynowski, Eric Padron, Jacqueline Boultwood, Steven D. Gore, Naval Daver, Jane E. Churpek, Ravindra Majeti, John M. Bennett, Alan F. List, Andrew M. Brunner, Amer M. Zeidan
Abstract
ABSTRACT: Excessively restrictive inclusion and exclusion criteria in clinical trials are one of many barriers to clinical trial enrollment for patients with myelodysplastic syndromes/neoplasms (MDSs). Many organizations are developing efforts to increase clinical trial eligibility; yet, several recent publications focused on patients with MDS suggest that many patients with this disease may be excluded from clinical trials unnecessarily. Clinical trial eligibility should reflect the phase of the study and risks of the agent being studied. Phase 3 trials should be less restrictive than early-phase trials to represent the real-world population as closely as possible. We hypothesize that many clinical trials, particularly phase 3 trials, have unnecessarily restrictive eligibility criteria. This study aims to evaluate the most common eligibility criteria according to phase of trial and to determine whether criteria correspond with drug safety signals. We identified MDS clinical trials registered on ClinicalTrials.gov from 1 January 2000 to 1 September 2023 and analyzed the eligibility criteria of 191 therapeutic MDS trials. We found that categorical inclusion and exclusion criteria are remarkably similar in representation across trial phases. Additionally, only 13% of trials are concordant with drug safety signals, suggesting that the eligibility criteria are often arbitrary. On behalf of the icMDS (International Consortium for Myelodysplastic Syndromes), an association of international MDS experts, we provide a position statement on restrictive eligibility criteria for MDS clinical trials that should be avoided with the aim of removing barriers to clinical trial enrollment.