A Case Study Of Surrogate Outcomes For Overall Survival In The Treatment Of Chronic Myelogenous Leukemia

Exeter, UK – Ideally, licensing and value-for-money policy decisions on medications should be based on clinical trials that show benefits to final patient health outcomes, such as overall survival.  In order to speed up the approval process, however, those decisions often rely on surrogate or intermediate outcomes, such as laboratory measures, which are easier and cheaper to obtain.  Understanding and characterizing the relationship between the surrogate and the final outcome is an important step to pursing reliable, evidence-based decision making on health technologies.

In the article “Complete Cytogenetic Response and Major Molecular Response as Surrogate Outcomes for Overall Survival in First-Line Treatment of Chronic Myelogenous Leukemia: A Case Study for Technology Appraisal on the Basis of Surrogate Outcomes Evidence,” published in Value in Health, the authors describe how two surrogate markers for patients with chronic myeloid leukemia were validated as surrogate outcomes for overall survival.

Results of this study supported decisions issued by the National Institute of Health and Care Excellence on the public funding of three drug treatments for patients with chronic myeloid leukemia in England and Wales.

Oriana Ciani, lead author and PhD candidate at the University of Exeter Medical School, comments: “Whilst it is recommended that acceptance of surrogate outcomes be based on several randomised controlled trials reporting a strong association between the treatment effect on both the surrogate and final outcomes, in the absence of such evidence, this case study shows that a sound and thorough validation may lead to the approval of a drug for a specific health condition.” The authors also recommend developing consensus on methods to incorporate the uncertainty associated with surrogate outcomes in future cost-effectiveness analyses.


Value in Health (ISSN 1098-3015) publishes papers, concepts, and ideas that advance the field of pharmacoeconomics and outcomes research as well as policy papers to help health care leaders make evidence-based decisions. The journal is published bi-monthly and has over 8,000 subscribers (clinicians, decision makers, and researchers worldwide).

International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that strives to increase the efficiency, effectiveness, and fairness of health care resource use to improve health.

For more information: www.ispor.org

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