Primary and Secondary Outcomes in Oncology Clinical Trials: Definitions and Uses

Authors

  • António Vaz-Carneiro Centro de Estudos de Medicina Baseada na Evidência. Faculdade de Medicina. Universidade de Lisboa. Lisboa. Portugal.
  • Ricardo da Luz Centro de Estudos de Medicina Baseada na Evidência. Faculdade de Medicina. Universidade de Lisboa. Lisboa. Portugal.
  • Margarida Borges Centro de Estudos de Medicina Baseada na Evidência. Faculdade de Medicina. Universidade de Lisboa. Lisboa. Portugal.
  • João Costa Centro de Estudos de Medicina Baseada na Evidência. Faculdade de Medicina. Universidade de Lisboa. Lisboa. Portugal.

DOI:

https://doi.org/10.20344/amp.5286

Abstract

Introduction: The proof of efficacy from a therapeutic intervention in oncology must be defined through well conducted clinical trials. One of the most important methodological issue is the outcome selection needed to calculate measures of association allowing definition of clinical efficacy.
Material and Methods: We designed a narrative revision based on some of the international regulatory instructions from drug agencies, as well as consensus papers from scientific oncology societies, listing and critically assessing each outcome used in oncology clinical trials.
Results: We identified as being the most important outcomes in oncology trials the overall survival, the progression free survival/ disease-free survival, the toxicity, the quality of life/patient- reported outcomes and the objective response rate.
Discussion: The selection of the primary outcome must be based on therapeutic efficacy as well as toxicity, expected survival, alternative drug regimens and even disease prevalence.
Conclusion: The selection of efficacy outcomes for clinical trials in oncology is very important and its selection must be well justified, and depends on the type of disease, the patients and the drug being studied.
Keywords: Clinical Trials as Topic; Neoplasms; Medical Oncology; Treatment Outcome.

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Author Biographies

António Vaz-Carneiro, Centro de Estudos de Medicina Baseada na Evidência. Faculdade de Medicina. Universidade de Lisboa. Lisboa. Portugal.

Ricardo da Luz, Centro de Estudos de Medicina Baseada na Evidência. Faculdade de Medicina. Universidade de Lisboa. Lisboa. Portugal.

Margarida Borges, Centro de Estudos de Medicina Baseada na Evidência. Faculdade de Medicina. Universidade de Lisboa. Lisboa. Portugal.

João Costa, Centro de Estudos de Medicina Baseada na Evidência. Faculdade de Medicina. Universidade de Lisboa. Lisboa. Portugal.

Published

2014-08-04

How to Cite

1.
Vaz-Carneiro A, Luz R da, Borges M, Costa J. Primary and Secondary Outcomes in Oncology Clinical Trials: Definitions and Uses. Acta Med Port [Internet]. 2014 Aug. 4 [cited 2024 Nov. 25];27(4):498-502. Available from: https://actamedicaportuguesa.com/revista/index.php/amp/article/view/5286

Issue

Section

Review Articles