Direct Incorporation of Expert Opinion into Parametric Survival Models to Inform Survival Extrapolation. (April 2023)

• Record Type: Journal Article
• Authors: Cooney, Philip; White, Arthur
• Is Part Of: Medical decision making. Volume 43:Number 3(2023)
• Page Start: 325
• Page End: 336
• Publication Date: 2023-04
• Subjects: survival models -- extrapolation -- expert opinion; Medical policy -- Periodicals; Clinical medicine -- Decision making -- Periodicals; Medicine -- Periodicals; Médecine clinique -- Prise de décision -- Périodiques; 362.1
• DOI: 10.1177/0272989X221150212
• Languages: English
• ISSNs: 0272-989X

Abstract

Background: In decision modeling with time-to-event data, there are a variety of parametric models that can be used to extrapolate the survival function. Each model implies a different hazard function, and in situations in which there is moderate censoring, this can result in quite different survival projections. External information such as expert opinion on long-term survival can more accurately characterize the uncertainty in these extrapolations. Objective: We present a general and easily implementable approach to incorporate various types of expert opinions into parametric survival models, focusing on opinions about survival at various landmark time points. Methods: Expert opinion is incorporated into parametric survival models using Bayesian and frequentist approaches. In the Bayesian method, expert opinion is included through a loss function and in the frequentist approach by penalizing the likelihood function, although in both cases the core approach is the same. The issue of aggregating multiple expert opinions is also considered. Results: We apply this method to data from a leukemia trial and use previously elicited expert opinion on survival probabilities for that particular trial population at years 4 and 5 to inform our analysis. We take a robust approach to modeling expert opinion by using pooled distributions and fit a broad class of parametric models to the data. We also assess statistical goodness of fit of the models to both the observed data and expert opinion. Conclusions: Expert opinions can be implemented in a straightforward manner using this novel approach; however, more work is required on the correct elicitation of these quantities. Highlights: Presentation of a novel and open-source method to incorporate expert opinion into decision modeling. Extends upon earlier work in that expert opinion can be incorporated into a wide range of parametric models. Provides methodological guidance for directly including expert opinion in decision modeling, which is a research focus area in NICE TSD 21. 1