Ahmad Halwani, Assistant Professor, Division of Hematology and Hematologic Malignancies, Huntsman Cancer Institute, University of Utah Healthcare, Salt Lake City, USA, examines the "mostly unmet need for research that aims to further our understanding of the comparative effectiveness of cancer treatments in real world settings" in this OncLive article:
You may have read about poorer results being achieved in real life compared to clinical trials. Ahmad Halwani explains why this happens. It's a bit technical but we need to understand that this difference is real and the reasons why, to better understand how we, personally are likely to fare in comparison to a published trial's typical outcomes. "Clinical research in oncology has typically relied on prospective, often randomized, clinical trials as the gold standard methodology to generate evidence of the benefits and risks of novel treatments. Prospective clinical trials rely on a number of elements to achieve a high level of internal validity: strict eligibility criteria, consistent treatment delivery, predefined endpoints, and a priori statistical power considerations to accept or reject the null hypothesis. Randomization ensures the exchangeability of patients in different treatment arms and allows causal inference.
The resulting high internal validity of prospective clinical trials is often achieved at the expense of external validity, or applicability of evidence to patients in real world settings. Stringent eligibility criteria often lead to exclusion of patients with comorbidities. As a result, trials often accrue patients who are younger and healthier than patients in actual clinical practice. Patients who belong to ethnic minorities are also under represented in clinical trials. The technical and regulatory challenges involved in the conduct of clinical trials often lead to a predominance of patients treated in academic settings as opposed to community settings."
As a result of these differences, the need to complement our understanding of the efficacy of cancer treatments as studied in randomized controlled trials (RCTs) with evidence of effectiveness in a real world setting is increasingly recognized. This type of research is typically referred to as comparative effectiveness research (CER), population research, or outcomes research. While not as common as explanatory RCTs, and at times lagging behind RCTs addressing the same question, CER studies are now starting to address the evidence gap."
In conclusion, he draws from the reported impact of dose reductions on CLL Progression Free Survival in studies from Israel (Cllcanada recently posted about this study), France and Germany and looks at the promise offered by the widespread adoption of electronic health records.
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