Activities per year
Abstract
Method: Using published response data for a HIT, a Bayesian hierarchical model (BHM) was used to identify heterogeneity in response and to estimate the probability of response for each histology included in single-arm studies which informed the submission for the HIT, larotrectinib. The probability of response for a ‘new’ histology was estimated. Results were inputted into a simplified response-based economic model using hypothetical parameters. Histology-independent and histology-specific incremental cost-effectiveness ratios (ICERs) accounting for heterogeneity were generated.
Results: The results of the BHM show considerable heterogeneity in response rates across histologies. The predicted probability of response estimated by the BHM is 60.9% (95% CrI 16.0; 91.8%), lower than the naively pooled probability of 74.5%. A mean response probability of 56.9% (0.2; 99.9%) is predicted for an unrepresented histology.
Based on the economic analysis, the probability of the hypothetical HIT being cost effective under the assumption of identical response is 78%. Allowing for heterogeneity, the probability of various approval decisions being cost effective ranges from 93% to 11%.
Conclusions: Central to the challenge of reimbursement of HITs is the potential for heterogeneity. This study illustrates how heterogeneity in clinical-effectiveness can result in highly variable and uncertain estimates of cost-effectiveness. This analysis can help understand the consequences of histology-independent vs histology-specific decisions.
Original language | English |
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Pages (from-to) | 1-14 |
Number of pages | 14 |
Journal | Medical Decision Making |
Volume | 41 |
Issue number | 2 |
Early online date | 13 Jan 2021 |
DOIs | |
Publication status | E-pub ahead of print - 13 Jan 2021 |
Bibliographical note
© The Author(s) 2021.Activities
- 1 Oral presentation
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Basket Weaving: Can Bayes Help Us Develop a Better Path for Evaluating Efficacy in Basket Trials?
Sofia Dias (Speaker)
15 Nov 2023Activity: Talk or presentation › Oral presentation
File
Projects
- 1 Finished
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NETSCC: TARS - Production of Technology Assessment Reviews for the NIHR
Stewart, L. (Principal investigator), Bojke, L. (Co-investigator), Craig, D. (Co-investigator), Eastwood, A. J. (Co-investigator), Griffin, S. (Co-investigator), Higgins, J. P. T. (Co-investigator), Palmer, S. J. (Co-investigator), Rothery, C. (Co-investigator), Sculpher, M. (Co-investigator), Soares, M. O. (Co-investigator), Spackman, E. (Co-investigator), Walker, S. M. (Co-investigator), Woods, B. (Co-investigator) & Woolacott, N. F. (Co-investigator)
1/04/16 → 31/03/22
Project: Research project (funded) › Research