Reducing clinical trial eligibility barriers for patients with MDS: an icMDS position statement

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.

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Publication Details

Journal
Blood
Published
2025-03-27
DOI
https://doi.org/10.1182/blood.2023023717
Citations
6
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
10.62
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article

Reducing clinical trial eligibility barriers for patients with MDS: an icMDS position statement

Andrew H. Wei, María E. Figueroa, Rami S. Komrokji, Gail J. Roboz et al.
6 citations
Blood
Acute Myeloid Leukemia Research
10.62
article

Reducing clinical trial eligibility barriers for patients with MDS: an icMDS position statement

Andrew H. Wei, María E. Figueroa, Rami S. Komrokji, Gail J. Roboz, Uma Borate, Maximilian Stahl, Pierre Fenaux, Hetty E. Carraway, Jane E. Churpek, Stephen D. Nimer, Fabio Efficace, Omar Abdel‐Wahab, Ying Huang, Robert P. Hasserjian, David Andrew Sallman, Naval Guastad Daver, Amy Elizabeth Dezern, Daniel T. Starczynowski, Peter L. Greenberg, Eric Padron, Jacqueline Boultwood, Rena J. Buckstein, Zhuoer Xie, Michael Robert Savona, Richard F. Little, Guillermo Sanz, Stephanie Halene, Mikkael Aaron Sekeres, Sanam Loghavi, Tae Kon Kim, Olatoyosi Odenike, Elizabeth A. Griffiths, Guillermo Garcia‐Manero, Christopher Simon Hourigan, Rafael Bejar, Alan F. List, Mina L. Xu, Mrinal M. Patnaik, Ravindra Majeti, Uwe Platzbecker, Allyson Waller, Andrew M. Brunner, Jan Philipp Bewersdorf, John M. Bennett, Justin Taylor, Matteo Giovanni Della Porta, Valeria Santini, Rina Li Welkie, Steven D. Gore, Amer M. Zeidan, Kelly Pugh
article en
6 citations

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.

BloodVol. 145(13)
Cincinnati Children's Hospital Medical Center (US), Roswell Park Comprehensive Cancer Center (US), Cleveland Clinic (US), The Royal Melbourne Hospital (AU), Sunnybrook Health Science Centre (CA), Mayo Clinic (US), Memorial Sloan Kettering Cancer Center (US), Humanitas University (IT), NewYork–Presbyterian Hospital (US), The University of Texas MD Anderson Cancer Center (US), University of Wisconsin–Madison (US), Harvard University (US), University of Miami (US), The University of Melbourne (AU), Walter and Eliza Hall Institute of Medical Research (AU), Université Paris Cité (FR), Cornell University (US), The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (US), Peter MacCallum Cancer Centre (AU), New York Hospital Queens (US), Instituto de Salud Carlos III (ES), Health Sciences Centre (CA), WinnMed (US), Yale Cancer Center (US), University of Rochester Medical Center (US), Hospital Universitari i Politècnic La Fe (ES), Moffitt Cancer Center (US), Yale University (US), University of Oxford (GB), University of Chicago (US), Blood Cancer UK (GB), Massachusetts General Hospital (US), Presbyterian Hospital (US), Assistance Publique – Hôpitaux de Paris (FR), University of Wisconsin Carbone Cancer Center (US), Precision BioSciences (United States) (US), Azienda Ospedaliero-Universitaria Careggi (IT), Dana-Farber Cancer Institute (US), University Hospital Leipzig (DE), Biomedical Research Institute (US), National Cancer Institute (US), Hôpital Saint-Louis (FR), Johns Hopkins Hospital (US), Istituti di Ricovero e Cura a Carattere Scientifico (IT), Center for Outcomes Research and Clinical Epidemiology (IT), Sylvester Comprehensive Cancer Center (US), Centro de Investigación Biomédica en Red de Cáncer (ES), Sidney Kimmel Comprehensive Cancer Center (US), University of Florence (IT), The Ohio State University (US), Vanderbilt University Medical Center (US), Leipzig University (DE), Stanford University (US)
Reduced inequalities
Openalex Percentile: Top 2%
Acute Myeloid Leukemia Research
10.62
DOIOpen Access PDFOpenAlex
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