Journal
JOURNAL OF HEPATOLOGY
Volume 74, Issue 6, Pages 1483-1488Publisher
ELSEVIER
DOI: 10.1016/j.jhep.2021.01.033
Keywords
Hepatocellular carcinoma; Cholangiocarcinoma; RECIST; Treatment; Precision oncology; Trial design; Evidence-based medicine
Categories
Funding
- Instituto de Salud Carlos III [PI18/00768]
- AECC [PI044031]
- WCR (AICR) [16-0026]
- Instituto de Salud Carlos III
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Clinical trials rely on strict definitions and criteria, but real-world clinical practice may not follow the same guidelines, leading to discrepancies in patient selection, disease assessment, and treatment decisions. Understanding these differences is crucial in refining criteria for drug activity, toxicity, and treatment failure in order to achieve evidence-based clinical practice and precision oncology.
The design and execution of clinical trials relies on strict definitions and criteria to avoid heterogeneous decisions by investigators at different sites. Ideally, definitions and decision making in clinical practice should mimic those implemented in trials, but this is not the case. Target populations are narrowly defined in trials, with the goal of evaluating activity and toxicity, and ultimately, demonstrating a survival benefit. In real-world practice, patients may not fit into the stringent inclusion/exclusion criteria of clinical trials. The evaluation of activity may also differ and the common policy to stop therapy upon progression may not be followed if progression is minor. Indeed, registration of progression may not reflect treatment failure or resistance. Parameters such as response according to RECIST criteria, time to progression and progression-free survival are not fully informative and cannot be assumed as a definitive surrogate for survival, which is the hardest endpoint in therapeutic cancer studies. This difference is because of the varying methods used to evaluate drug activity and tumour evolution, which ultimately dictates patient outcome. This expert opinion exposes the current discrepancies between research trials and clinical practice. Understanding the origin and limitations of such a conundrum should be the first step in refining the criteria that define drug activity, toxicity and treatment failure. Otherwise, evidencebased clinical practice and precision oncology will be an unattainable reality. (c) 2021 European Association for the Study of the Liver. Published by Elsevier B.V. All rights reserved.
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