CONTEXT: Continuing the theme of under-representation in clinical trials that develop new treatments, especially in oncology, this article reports on the discussion at the European Hematology Association (EHA) 2021 Virtual Congress. Speakers highlighted the disparity between typical multiple myeloma patients (frail, elderly and with a number of comorbidities) and those patients carefully selected for inclusion in RCTs for pivotal approval studies. The conclusion seems to be to include real-world studies in the overall body of evidence for a new compound. The alternative, less optimistic, view is that new compounds once approved, will not show the benefits demonstrated during the approval process.
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1. “Following Davies, Ajai Chari, MD, professor of medicine, director of clinical research in the Multiple Myeloma Program, and associate director of clinical research at Mount Sinai, reviewed outcomes of patients who were eligible for RCTs vs those who were ineligible.”
2. “During a session at the European Hematology Association 2021 Virtual Congress, speakers discussed the differences in how RCTs translate to the real world, which patients in real-world practices would be eligible or ineligible, and the real-world outcomes and challenges in multiple myeloma (MM).”
3. “Randomized controlled trials (RCTs) may be the gold standard for trials, but the stringent eligibility criteria often mean that many patients who more closely represent real-world populations are ineligible for the trials.”
4. “In multiple studies, “the patients who were ineligible for the clinical trials have worse outcomes by either time-to-next therapy, overall survival, or progression.””
5. “Randomized clinical trial representativeness and outcomes in real-world patients: comparison of 6 hallmark randomized clinical trials of relapsed/refractory multiple myeloma.”