A review of studies examining the relationship between progression ...

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ABOUT THE DECISION SUPPORT UNITThe Decision Support Unit (DSU) is a collaboration between the Universities of Sheffield, York andLeicester. We also have members at the University of Bristol, London School of Hygiene andTropical Medicine and Brunel University. The DSU is commissioned by The National Institute forHealth and Clinical Excellence (NICE) to provide a research and training resource to support theInstitute's Technology Appraisal Programme. Please see our website for further informationwww.nicedsu.org.ukThe production of this document was funded by NICE through its Decision Support Unit. The views,and any errors or omissions, expressed in this document are of the authors only. NICE may takeaccount of part or all of this document if it considers it appropriate, but it is not bound to do so.AcknowledgementsWe thank Oriana Ciani, Bhash Naidoo and Allan Wailoo for their helpful peer review comments andJenny Dunn for formatting the report. The content of this report is the responsibility of the authorsalone.2
EXECUTIVE SUMMARYIntroductionProgression-free survival (PFS), time-to-progression (TTP) and overall survival (OS) are commonlyused endpoints in randomised controlled trials (RCTs) and observational studies of treatments formetastatic disease in solid tumour cancers. Within health economic models of cancer interventions,disease- and treatment-related health states are often defined according to whether the patient hasexperienced disease progression since starting a particular line of therapy. Such models typicallyestimate the mean sojourn time in these health states and therefore information is needed on bothPFS/TTP and OS. PFS or TTP are sometimes regarded as valid surrogate outcomes whenestablishing the clinical benefit of a treatment in the absence of mature data on OS, but an estimateof OS is still needed within the economic analysis. Some quantification of the relationship betweenPFS/TTP and OS may be used to populate the economic model as an alternative to directlymodelling OS from the trial data.ObjectivesThe aim of this review was to examine the evidence available concerning the relationship betweenPFS/TTP and OS in advanced or metastatic cancer, with a view to determining the extent to whichPFS/TTP can be considered a robust surrogate endpoint for OS.MethodsA review was conducted of papers using meta-regression or other meta-analytic techniques toexamine the statistical relationship between OS and either PFS or TTP. Studies were identifiedthrough citation searching as a systematic search was not considered feasible. The review was notrestricted to any particular cancer type but instead included any form of cancer in which thetreatment intent is palliative rather than curative and therefore the surrogate outcome of interest isprogression-free rather than disease-free survival. The main focus was therefore advanced ormetastatic cancer.ResultsNineteen papers were included in the review covering eight different tumour types. Four studies usedaggregate data from multiple trials to examine the relationship between PFS and OS for individualtrial arms. Seven studies used individual patient data to examine the relationship between PFS andOS for individual patients. Thirteen studies examined the trial level relationship between treatment3
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