Development and Validation of the TRansparent Uncertainty ASsessmenT (TRUST) Tool for Assessing Uncertainties in Health Economic Decision Models

Abstract

Background An increasing number of technologies are obtaining marketing authorisation based on sparse evidence, which causes growing uncertainty and risk within health technology reimbursement decision making. To ensure that uncertainty is considered and addressed within health technology assessment (HTA) recommendations, uncertainties need to be identified, included in health economic models, and reported. Objective Our objective was to develop the TRansparent Uncertainty ASsessmenT (TRUST) tool for systematically identifying, assessing, and reporting uncertainties in decision models, with the aim of making uncertainties and their impact on cost effectiveness more explicit and transparent. Methods TRUST was developed by drawing on the uncertainty and risk assessment literature. To develop and validate this tool, we conducted HTA stakeholder discussion meetings and interviews and applied it in six real-world HTA case studies in the Netherlands and the UK. Results The TRUST tool enables the identification and categorisation of uncertainty according to its source (transparency issues, methodology issues, and issues with evidence: imprecision, bias and indirectness, and unavailability) in each model aspect. The source of uncertainty determines the appropriate analysis. The impact of uncertainties on cost effectiveness is also assessed. Stakeholders found using the tool to be feasible and of value for transparent uncertainty assessment. TRUST can be used during model development and/or model review. Conclusion The TRUST tool enables systematic identification, assessment, and reporting of uncertainties in health economic models and may contribute to more informed and transparent decision making in the face of uncertainty.

Building a trusted framework for uncertainty assessment in rare diseases: suggestions for improvement (Response to “TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases”)

“…Assessment of impact (based on quantitative analysis if available or subjective opinion) can help in prioritising those uncertainties that should be analysed further. This step can be aided by tools such as the TRUST tool [ 1 ]. Ideally, all uncertainties are quantified in a decision model and incorporated in the probabilistic analysis [ 4 ].…”

“…Assessment of impact (based on quantitative analysis if available or subjective opinion) can help in prioritising those uncertainties that should be analysed further. This step can be aided by tools such as the TRUST tool [ 1 ].…”

“…Our group of authors have developed a tool for identifying, assessing and reporting uncertainty, the TRansparent Uncertainty ASsessmenT (TRUST) tool [ 1 ], independently of the research presented by Annemans & Makady and published earlier in 2020. Based on our own research in this area, we think that the article could have benefitted from more detail, clarification and placement in the quickly developing literature in this field.…”

Exploratory, Participatory and Iterative Assessment of Value: A Response to Recent Commentaries

“…3 This does, however, require a broader notion of uncertainty than imprecision. 13,14 The Notion of Value Many of the commentaries rightfully discussed our strong focus on cost-effectiveness. Of course, as we mentioned in the original paper, value of an innovative technology is much more than cost-effectiveness alone.…”

Health technology assessment of treatment for peripheral arterial disease

“…Guidance regarding the use of applicable methodologies, such as the selection of uncertainties, the use of expert elicitation, the aggregation of elicited and existing data, the use of model averaging, and reporting the results in a comprehensive and transparent manner was missing and perceived as a barrier to the comprehensive uncertainty assessment. The use of existing tools such as TRUST for the assessment of uncertainties (20), and EXPLICIT for the elicitation of expert opinion (21) were perceived as facilitators. The study provided an example of how comprehensive uncertainty assessment can be conducted by utilizing existing tools and available sources of expert knowledge.…”