Modelling the Survival Outcomes of Immuno-Oncology Drugs in Economic Evaluations: A Systematic Approach to Data Analysis and Extrapolation

Eddie Gibson (), Ian Koblbauer, Najida Begum, George Dranitsaris, Danny Liew, Phil McEwan, Amir Abbas Tahami Monfared, Yong Yuan, Ariadna Juarez-Garcia, David Tyas and Michael Lees
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Eddie Gibson: Wickenstones Ltd
Ian Koblbauer: Wickenstones Ltd
Najida Begum: Wickenstones Ltd
George Dranitsaris: Augmentium Pharma Consulting Inc.
Danny Liew: Monash University
Phil McEwan: Health Economics and Outcomes Research Ltd
Amir Abbas Tahami Monfared: Bristol-Myers Squibb
Yong Yuan: Bristol-Myers Squibb
Ariadna Juarez-Garcia: Bristol-Myers Squibb
David Tyas: Bristol-Myers Squibb
Michael Lees: Bristol-Myers Squibb

PharmacoEconomics, 2017, vol. 35, issue 12, No 6, 1257-1270

Abstract: Abstract Background New immuno-oncology (I-O) therapies that harness the immune system to fight cancer call for a re-examination of the traditional parametric techniques used to model survival from clinical trial data. More flexible approaches are needed to capture the characteristic I-O pattern of delayed treatment effects and, for a subset of patients, the plateau of long-term survival. Objectives Using a systematic approach to data management and analysis, the study assessed the applicability of traditional and flexible approaches and, as a test case of flexible methods, investigated the suitability of restricted cubic splines (RCS) to model progression-free survival (PFS) in I-O therapy. Methods The goodness of fit of each survival function was tested on data from the CheckMate 067 trial of monotherapy versus combination therapy (nivolumab/ipilimumab) in metastatic melanoma using visual inspection and statistical tests. Extrapolations were validated using long-term data for ipilimumab. Results Modelled PFS estimates using traditional methods did not provide a good fit to the Kaplan–Meier (K–M) curve. RCS estimates fit the K–M curves well, particularly for the plateau phase. RCS with six knots provided the best overall fit, but RCS with one knot performed best at the plateau phase and was preferred on the grounds of parsimony. Conclusions RCS models represent a valuable addition to the range of flexible approaches available to model survival when assessing the effectiveness and cost-effectiveness of I-O therapy. A systematic approach to data analysis is recommended to compare the suitability of different approaches for different diseases and treatment regimens.

Date: 2017
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DOI: 10.1007/s40273-017-0558-5

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