State of the ART? Two New Tools for Risk Communication in Health Technology Assessments

Purpose: Outcomes of health technology assessments (HTA) are uncertain, and decision-making is associated with a risk. This risk, consisting of the probability of making a wrong decision and its impact, is rarely considered in HTA. This hampers transparent and consistent risk assessment and management. The aim of this study was to develop risk communication tools in the context of health technology decision-making under uncertainty. Methods: We performed a scoping review of tools for uncertainty and risk communication within HTA using citation pearl-growing. We developed two tools, drawing on existing publications on risk and uncertainty communication for inspiration. Individual semi-structured interviews with HTA stakeholders were performed to identify potential improvements in usefulness, user-friendliness, and information adequacy. Tools were amended and further evaluated in a real-world HTA and workshop with HTA stakeholders. Results: The identified risk communication tools did not include non-quantified uncertainties, and did not link to risk management strategies. We developed two tools: the Assessment of Risk Table (ART), for a summary of quantified and non-quantified uncertainties and the resulting risk assessment, and the Appraisal of Risk Chart (ARCH), for linking net benefit and risk outcomes to appropriate risk management strategies. Stakeholders appreciated the usefulness of the tools. They also highlighted that more information on local policy options was required for optimal risk management use, and HTA processes may need adapting. Conclusion: The risk communication tools presented here can help assess risk, facilitate communication between analysts and decision-makers, and guide the appropriate use of available risk management strategies

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").

The aim of this letter to the editor is to provide a comprehensive summary of uncertainty assessment in Health Technology Assessment, with a focus on transferability to the setting of rare diseases. The authors of “TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases” presented recommendations for reducing uncertainty in rare diseases. Their article is of great importance but unfortunately suffers from a lack of references to the wider uncertainty in Health Technology Assessment and research prioritisation literature and consequently fails to provide a trusted framework for decision-making in rare diseases. In this letter to the editor we critique the authors’ tool and provide pointers as to how their proposal can be strengthened. We present references to the literature, including our own tool for uncertainty assessment (TRUST; unrelated to the authors’ research), apply TRUST to two assessments of orphan drugs in rare diseases and provide a broader perspective on uncertainty and risk management in rare diseases, including a detailed research agenda

Optimizing the Use of High-Sensitivity Troponin Assays for the Early Rule-out of Myocardial Infarction in Patients Presenting with Chest Pain: A Systematic Review.

BACKGROUND: We assessed the accuracy and clinical effectiveness of high-sensitivity cardiac troponin (hs-cTn) assays for early rule-out of non-ST-segment elevation myocardial infarction (NSTEMI) in adults presenting with acute chest pain. METHODS: Sixteen databases were searched to September 2019. Review methods followed published guidelines. The bivariate model was used to estimate summary sensitivity and specificity with 95% confidence intervals for meta-analyses involving 4 or more studies, otherwise random-effects logistic regression was used. RESULTS: Thirty-seven studies (124 publications) were included in the review. The hs-cTn test strategies evaluated in the included studies were defined by the combination of 4 factors (assay, number of tests, timing of tests, and threshold concentration or change in concentration between tests). Clinical opinion indicated a minimum acceptable sensitivity of 97%. A single test at presentation using a threshold at or near the assay limit of detection could reliably rule-out NSTEMI for a range of hs-cTn assays. Serial testing strategies, which include an immediate rule-out step, increased the proportion ruled out without loss of sensitivity. Finally, serial testing strategies without an immediate rule-out step had excellent sensitivity and specificity, but at the expense of the option for immediate patient discharge. CONCLUSION: Test strategies that comprise an initial rule-out step, based on low hs-cTn concentrations at presentation and a minimum symptom duration, and a second step for those not ruled-out that incorporates a small absolute change in hs-cTn at 1, 2, or 3 hours, produce the highest rule-out rates with a very low risk of missed NSTEMI. PROSPERO REGISTRATION: CRD42019154716