ArticleMedical decision making : an international journal of the Society for Medical Decision Making2021
Exploring Heterogeneity in Histology-Independent Technologies and the Implications for Cost-Effectiveness.
Article in Medical decision making : an international journal of the Society for Medical Decision Making, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
8 citing papers in PubMed, 1 synthesis or guideline pooled it, 17 citations in OpenAlex.
- Multi-indication Evidence Synthesis in Oncology Health Technology Assessment: Meta-analysis Methods and Their Application to a Case Study of Bevacizumab.Medical decision making : an international journal of the Society for Medical Decision Making · 2025Pooled it
- Extrapolation of Time-to-Event Survival Outcomes of Histology-Independent Therapies Using a Bayesian Hierarchical Model.Medical decision making : an international journal of the Society for Medical Decision Making · 2026Article
- A conceptual health economic modelling framework to assess the cost-effectiveness of molecular target-driven treatment regimens in oncology.Acta oncologica (Stockholm, Sweden) · 2026Article
- A Review of Heterogeneity in Comparative Economic Analysis, with Specific Considerations for the Decentralized US Setting and Patient-Centered Care.PharmacoEconomics · 2025Review
- Advances in Addressing Patient Heterogeneity in Economic Evaluation: A Review of the Methods Literature.PharmacoEconomics · 2024Review
- Bayesian Hierarchical Modelling for Histology-Independent Time-to-Event Outcomes in the NICE Single Technology Appraisal of Pembrolizumab for Solid Tumours with MSI-H/dMMR: External Assessment Group Perspective.PharmacoEconomics · 2024Article
- Evaluating treatments in rare indications warrants a Bayesian approach.Frontiers in pharmacology · 2023Article
- Clinical and cost outcomes following genomics-informed treatment for advanced cancers.Cancer medicine · 2021Article
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
backgroundThe National Institute for Health and Care Excellence and a number of international health technology assessment agencies have recently undertaken appraisals of histology-independent technologies (HITs). A strong and untested assumption inherent in the submissions included identical clinical response across all tumour histologies, including new histologies unrepresented in the trial. Challenging this assumption and exploring the potential for heterogeneity has the potential to impact upon cost-effectiveness.
methodUsing 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 accounting for heterogeneity were generated.
resultsThe 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% credible interval 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%.
conclusionsCentral 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 improve understanding of the consequences of histology-independent versus histology-specific decisions.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.