Economic Evaluation of Medical Devices

There are a number of challenges in the economic evaluation of medical devices (MDs). They are typically less regulated than pharmaceuticals, and the clinical evidence requirements for market authorization are generally lower. There are also specific characteristics of MDs, such as the device–user interaction (learning curve), the incremental nature of innovation, the dynamic nature of pricing, and the broader organizational impact. Therefore, a number of initiatives need to be taken in order to facilitate the economic evaluation of MDs. First, the regulatory processes for MDs need to be strengthened and more closely aligned to the needs of economic evaluation. Second, the methods of economic evaluation need to be enhanced by improving the analysis of the available clinical data, establishing high-quality clinical registries, and better recognizing MDs’ specific characteristics. Third, the market entry and diffusion of MDs need to be better managed by understanding the key influences on MD diffusion and linking diffusion with cost-effectiveness evidence through the use of performance-based risksharing arrangements

Endovascular versus surgical creation of arteriovenous fistula in hemodialysis patients: cost-effectiveness and budget impact analyses

Objectives: The aim of the present study was to perform cost-effectiveness and budget impact analyses comparing endovascular arteriovenous fistula creation to surgical arteriovenous fistula creation in hemodialysis patients from the National Healthcare Service (NHS) perspective in Italy. Methods: A systematic literature review has been conducted to retrieve complications’ rates after arteriovenous fistula creation procedures. One study comparing endovascular arteriovenous fistula creation, performed with WavelinQ device, to the surgical approach through propensity score matching was preferred to single-arm investigations to execute the economic evaluations. This study was chosen to populate a Markov model to project, on a time horizon of 1 year, quality adjusted life years and costs associated with endovascular arteriovenous fistula (WavelinQ) and surgical arteriovenous fistula options for both cohorts of incident and prevalent hemodialysis patients. Results: For both incident and prevalent hemodialysis patients, endovascular arteriovenous fistula creation, performed with WavelinQ, was the dominant strategy over surgical arteriovenous fistula approach, showing less cost and better patients’ quality of life. Compared to the current scenario, progressively increasing utilization rates of WavelinQ over surgical arteriovenous fistula creation in the next 5 years in incident hemodialysis patients are expected to save globally 30–36 million euros to the NHS. Conclusion: Endovascular arteriovenous fistula creation performed with WavelinQ could be a cost-saving strategy in comparison with the surgical approach for patients in hemodialysis. Future studies comparing different devices for endovascular arteriovenous fistula creation versus the surgical option would be needed to confirm or reject the validity of this preliminary evaluation. In the meantime, decision-makers can use these results to take decisions on the diffusion of endovascular procedures in Italy

Investigating patients’ preferences to inform drug development decisions: novel Insights from a discrete choice experiment in migraine

Abstract: There is limited evidence on the scope and overall benefit of patient-centred drug development decisions. The present study assessed patients’ preferences for the characteristics of an ideal migraine treatment through a discrete choice experiment in order to inform decision-making and drug development processes. We investigated the preferences according to five treatment attributes identified from a systematic literature review and two focus group elicitations. The heterogeneity of preferences was also investigated. Overall, the respondents considered the presence of adverse events, duration of treatment effect, reduction of symptom intensity, speed of effect and cost born by the patient as the most relevant treatment features. As expected, the patients preferred treatments with lower levels of adverse events and costs and treatments with greater speed, duration of treatment effect and effectiveness in reducing symptom intensity. There was significant preference heterogeneity only for the presence of adverse events. Compared to men, women had significantly higher preferences for quicker treatment effect and limited adverse events and reported higher preferences for costly treatments. The results of our survey help address research and development strategies in the pharmaceutical industry and public policy regarding treatments that are clinically effective and responsive to the needs expressed by patients

what type of clinical evidence is needed to assess medical devices

The objective of this mini-review is to discuss the role of real-world studies as a source of clinical evidence when experimental studies, such as randomised controlled trials (RCTs), are not available. Waiting for RCT evidence when the technology is diffusing could be anti-economical, inefficient from the policy perspective and methodologically questionable.We explain how real-world studies could provide relevant evidence to decision makers. Matching techniques are discussed as a viable solution for bias reduction.We describe a case study concerning a cost-effectiveness analysis based on real-world data of a technology already in use: Mitraclip combined with medical therapy versus medical therapy alone in patients with moderate-to-severe mitral regurgitation. The CEA has encountered the scepticism of most reviewers, due not to the statistical methodology but to the fact that the study was observational and not experimental. Editors and reviewers converged in considering real-world economic evaluations premature in the absence of a RCT, even if in the meantime the technology had been implanted >30 000 times. We believe there is a need to acknowledge the importance of real-world studies, and engage the scientific community in the promotion and use of clinical evidence produced through observational studies

Mapping health-related quality of life scores from FACT-G, FAACT, and FACIT-F onto preference-based EQ-5D-5L utilities in non-small cell lung cancer cachexia.

BACKGROUND: Health-related quality of life (HRQoL) measurements from disease-specific tools cannot be directly used in economic evaluations. This study aimed to develop and validate mapping algorithms that predicted EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) utilities from Functional Assessment of Anorexia-Cachexia Therapy (FAACT) and Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and their common component (Functional Assessment of Cancer Therapy-General-FACT-G) in patients with non-small cell lung cancer cachexia. METHODS: Data were collected on five occasions over a 12-week period in two multicenter placebo-controlled trials. EQ-5D-5L utilities were calculated using both English and Dutch value sets. The study sample was divided into development and validation datasets according to patients' geographical residence. Generalized estimating equations were applied to five different sets of independent variables including overall, Trial Outcome Index (TOI), and individual subscales results. The best performing models were selected based on mean absolute error (MAE) and root-mean square error (RMSE). RESULTS: EQ-5D-5L and FAACT/FACIT-F results were available for 96 patients. The developed algorithms showed a good predictive performance, with acceptable MAE/RMSE and small differences between mean observed and predicted EQ-5D-5L utilities. In FACT-G models, Physical Well-Being had the highest explanatory value, while Emotional Well-Being did not significantly affect the EQ-5D-5L score; Anorexia-Cachexia and Fatigue subscales were highly statistically significant in FAACT and FACIT-F models, respectively, as well as the TOI scores. The Eastern Cooperative Oncology Group status was included as covariate in all models. CONCLUSION: The developed algorithms enable the estimation of EQ-5D-5L utilities from three cancer-specific instruments when preference-based HRQoL data are missing

Pushing the boundaries of evaluation, diffusion, and use of medical devices in Europe: Insights from the COMED project

The field of medical devices has attracted considerable interest from scholarly research in health economics in recent years. Medical devices are indispensable tools for quality health care delivery, but their assessment and appropriate use pose significant challenges to healthcare systems. More research is needed to overcome existing gaps associated with evaluation of digital technologies, address challenges in the use of real-world data in generating evidence for decision-making and to uncover drivers of variation in access to medical devices across countries. Furthermore, the translation of the results and recommendations stemming from research projects into health technology assessment practices needs to be strengthened. The European Union (EU) project COMED aimed to address these gaps by improving existing research and developing new research streams on the methods for evaluation and diffusion of medical devices. The project also intended to provide directly applicable policy advice and tools to inform decision-making, with the aim of impacting public health in the EU. This Health Economics Supplement, together with references of other published outputs of the project, is intended to be the main source for researchers and policy makers seeking information on the COMED project

Real-World Data for the Evaluation of Transarterial Radioembolization versus Sorafenib in Hepatocellular Carcinoma: A Cost-Effectiveness Analysis

Abstract Objectives To perform a cost-effectiveness analysis comparing the use of transarterial radioembolization (TARE) with that of sorafenib in the treatment of patients with intermediate or advanced hepatocellular carcinoma (HCC) according to the Barcelona Clinic Liver Cancer staging system. Methods Patient-level data were consecutively recorded and collected at three oncology centers in Italy. A propensity score matching was performed to compare patients with similar clinical characteristics who underwent TARE or sorafenib treatment. Clinical data from the matched cohorts were used to populate a Markov model to project, on a lifetime horizon, life years, quality-adjusted life years, and economic outcomes associated with TARE and sorafenib for both intermediate and advanced HCC stages. Results Starting from data covering 389 and 241 patients who underwent TARE and sorafenib treatment, respectively, the propensity score matching yielded a total of 308 matched patients. For intermediate-stage patients, the model estimated for TARE versus sorafenib an incremental cost-utility ratio of €3,302/QALY (incremental cost-effectiveness ratio of €1,865 per life year gained), whereas for patients in advanced stage TARE dominated (lower costs and greater health improvements) compared with sorafenib. Conclusions From an Italian health care service perspective, TARE could be a cost-effective strategy in comparison with sorafenib for patients with intermediate or advanced HCC. The results from forthcoming randomized controlled trials comparing TARE with sorafenib will be able to confirm or reject the validity of this preliminary evaluation. In the meantime, decision makers can use these results to control and coordinate the diffusion of the technology

Distinguishing features in the assessment of mHealth apps

Introduction: The unparalleled surge in digital health adoption during the COVID-19 pandemic has emphasized the potential of mHealth apps. However, the quality of available evidence is generally low, and regulatory frameworks have focused on apps with medical purposes only, overlooking apps with significant interactions with patients that may require stronger oversight. Areas covered: To support this expanded evidence generation process, we identified the reasons that distinguish mHealth apps compared to medical devices at large and that should differentially feature their assessment. mHealth apps are characterized by the iterative nature of the corresponding interventions, frequent user interactions with a non-linear relationship between technology usage, engagement and outcomes, significant organizational implications, as well as challenges associated with genericization, their broad diagnostic potential, and price setting. Expert Opinion: The renewed reliance experienced during the pandemic and the unprecedented injection of resources through recovery instruments can further boost the development of apps. Only robust evidence of the benefits of mHealth apps will persuade health-care professionals and beneficiaries to systematically deploy them. Regulatory bodies will need to question their current approaches by adopting comprehensive evaluation processes that adequately consider the specific features of mHealth apps

Distinguishing features in the assessment of mHealth apps

Introduction: The unparalleled surge in digital health adoption during the COVID-19 pandemic has emphasized the potential of mHealth apps. However, the quality of available evidence is generally low, and regulatory frameworks have focused on apps with medical purposes only, overlooking apps with significant interactions with patients that may require stronger oversight. Areas covered: To support this expanded evidence generation process, we identified the reasons that distinguish mHealth apps compared to medical devices at large and that should differentially feature their assessment. mHealth apps are characterized by the iterative nature of the corresponding interventions, frequent user interactions with a non-linear relationship between technology usage, engagement and outcomes, significant organizational implications, as well as challenges associated with genericization, their broad diagnostic potential, and price setting. Expert Opinion: The renewed reliance experienced during the pandemic and the unprecedented injection of resources through recovery instruments can further boost the development of apps. Only robust evidence of the benefits of mHealth apps will persuade health-care professionals and beneficiaries to systematically deploy them. Regulatory bodies will need to question their current approaches by adopting comprehensive evaluation processes that adequately consider the specific features of mHealth apps