Key Issues of Economic Evaluations for Health Technology Assessment in China: A Nationwide Expert Survey.

OBJECTIVES: Health technology assessment (HTA) is increasingly crucial in medicine price negotiations in China, yet previous appraisals revealed national discrepancies on key economic evaluation issues: willingness-to-pay threshold, pricing models for multi-indication medicines, and comparator selection principles. This study aimed to collect expert opinions on these issues for future HTA evaluations. METHODS: A nationwide anonymous web-based survey encompassing experts across academia, HTA, consultancy/contract research organization/industry, service provider, and payer. In 2023, a generic invitation containing a web link to the questionnaire was disseminated via WeChat using convenience and snowball sampling. Agreement rates for questionnaire views were analyzed using descriptive statistics. The relationship between participants' responses and demographics was examined using appropriate logistic models. RESULTS: A total of 303 responses were received from experts in 34 cities. Key expert views include a suggested base willingness-to-pay threshold ranging from 0.5 to 1.5 times gross domestic product (52.1% agreement); elevated thresholds for childhood diseases, rare diseases, end-of-life diseases, and first-in-class medicines (>78.0% agreement); a single pricing model for multi-indication medicines (60.4% agreement); consideration of multiple medicines as comparators (79.9% agreement); and avoiding the use of centrally procured medicines as comparators for medicines with a time to market of less than 3 years (71.0% agreement). Participants who are service provider had lower odds of selecting higher thresholds (odds ratio 0.26; P < .01) than responders from consultancy/contract research organization/industry. CONCLUSIONS: Expert views indicate the need for substantial changes in China's current HTA methods, highlighting the need for increased investment in HTA processes and expertise cultivation.

[The economic prerequisites of implementation of innovative technologies of mass-spectrometry in diagnostic of bacterial infections].

The article presents the results of clinical economical analysis, based on "cost-effectiveness" technology, of MALDI-TOF MS, innovative medical technology of express identification of microorganisms, calculation of incremental indicator and application of notion "willingness-to-pay-threshold". Due to the extension of sanctions against the Russian Federation, this medical equipment for national laboratories becomes difficult to access and expensive, that conditions necessity to scientifically substantiate economical effectiveness of implementation of expensive innovative MALDI-TOF MS technology as instrument to contain global antibiotic resistance increase. The understanding of importance of express identification of microorganisms as well as other positive effects that can be achieved by using modern medical equipment on the basis of mass spectrometry results in improving medical care quality, increasing reputation level of medical institution, greater commitment of physicians and patients to microbiological analysis with purpose of prescription of rational antibiotic therapy and improving population health.

Approaches to Capturing Value in Oncology.

This article sets out to describe different value frameworks in the field of new developments in oncology. Since the costs of new oncological therapies follow a steep path, their implementation and financing demand a thorough assessment. This is an ambitious task due to the complex nature of oncological treatments within overall health policy. Five value frameworks were reviewed: European Society for Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale, American Society of Clinical Oncology (ASCO) Value Framework (version 2.0), National Comprehensive Cancer Network (NCCN) Evidence Blocks, Memorial Sloan Kettering Cancer Center DrugAbacus, and the Institute for Clinical and Economic Review Value Assessment Framework. They are all based on a large set of criteria. However, all these frameworks differ considerably in their outcomes. Among the main differences one has to cite are the inclusion of costs and the use of different outcomes, as well as the fact that they address different target stakeholders, etc. Despite these shortcomings, the value frameworks serve the necessity to introduce more rationality in health decision making seen from the perspective of physicians, patients, and financing bodies.

Willingness to Pay (WTP) for Newer Treatment Options for Diabetes: A Study Among Patients at a Tertiary Care Centre.

Introduction Type 2 diabetes mellitus (T2DM) poses a substantial burden globally and particularly in India, affecting health, finances, and overall quality of life. The management of this condition relies on lifestyle modifications and advanced pharmacological interventions, with emerging drugs showing promise in areas such as administration, side effects, efficacy, and cardiovascular benefits. However, their market penetration is hindered by high costs. Understanding the target population's expectations and willingness to pay (WTP) for these drugs is crucial. WTP, a key concept in behavioral science, reflects the maximum price consumers are willing to pay for a product, aiding in healthcare cost-effectiveness evaluations. Despite its relevance, only one WTP study has been conducted in the Indian context for diabetes. This study explores WTP for two novel drugs: oral semaglutide and icodec (weekly insulin). Material and methods This observational study, conducted in a diabetes specialty clinic and telemedicine facility in All India Institute of Medical Sciences, Bhopal, India, involved adults (18-80 years) diagnosed with T2DM. Data collection adhered to ethical guidelines, and participants provided written informed consent. Face-to-face interviews were employed to gather socio-economic, demographic, and medical details. Participants estimated their WTP for oral semaglutide and weekly insulin, considering reference ranges for existing antidiabetic treatments. Statistical analyses, including t-tests and analysis of variance, explored sociodemographic and clinical factors influencing WTP. Results Of 105 approached patients, 87 (74.3%) participated. The majority were males (55.2%) with an average age of 57.2 years. The average WTP for oral semaglutide was INR 9.35±5.66 per pill, significantly lower than its market price (INR 315). For weekly insulin (icodec), the WTP was INR 157.25±112.60 per dose. Subgroup analyses revealed no significant correlations based on sociodemographic or clinical parameters. Conclusion This study demonstrated the feasibility of WTP assessments in an Indian outpatient setting, revealing a substantial cost disparity between patients' WTP for oral semaglutide and its market price. The findings underscore the importance of considering WTP in introducing new diabetes medications in India, offering valuable insights for healthcare decision-makers and developers.

Estimation of Value-Based Price for Five High-Technology Medical Devices Approved by a Regional Health Technology Assessment Committee in Italy.

Background and objectives Value-based pricing (VBP) is used quite frequently for medicines, but its application to medical devices is very limited. The objective of the present study was to conduct a pilot experience of systematic estimation of the value-based price of medical devices from the perspective of our national health system. Our experience was focused on high-technology devices (class IIb/III and active implantable). The objective was to evaluate the applicability of VBP in a real-world setting and to estimate the value-based price of devices in all cases where this estimation was feasible. Methods The dataset analysed in this work consists of 24 new devices approved consecutively in the Tuscany region over the period from January 2020 to December 2021. Since the calculation of value-based price requires the availability of a cost-effectiveness analysis, we searched for this information for each of these devices. The Cost-Effectiveness Analysis (CEA) Registry of Tufts Medical Center (US) and the health technology assessment (HTA) reports of our region were considered adequate sources of these data. Standard equations of cost-effectiveness were applied to determine the value-based price for these devices, and these prices were compared with the corresponding real prices charged in our region. Results We found adequate information for five devices (21%) out of the total of 24. In three of these cases, the published analysis taken as a reference was based on Markov modelling. The comparison between value-based prices and real prices generally showed an acceptable concordance, though with a couple of outliers. An important finding is that, in a large proportion of cases (79%), the information needed for this calculation was lacking. Conclusion To our knowledge, this is the first experience in which an institution of the healthcare system has tried a systematic application of VBP in the field of high-technology devices. Our results are encouraging and suggest a wider application of cost-effectiveness in this field.

Generating, Presenting, and Interpreting Cost-Effectiveness Results in the Context of Uncertainty: A Tutorial for Deeper Knowledge and Better Practice.

This tutorial aims to help make the best available methods for generating and presenting cost-effectiveness results with uncertainty common practice. We believe there is a need for such type of tutorial because some erroneous practices persist (e.g., identifying the cost-effective intervention as the one with the highest probability to be cost-effective), while some of the more advanced methods are hardly used (e.g., the net loss statistic 'NL', expected net loss curves and frontier). The tutorial explains with simple examples the pros and cons of using ICER, incremental net benefit and NL to identify the cost-effective intervention, both with and without uncertainty accounted for probabilistically. A flowchart provides practical guidance on when and how to use ICER, incremental net benefit or NL. Different ways to express and present uncertainty in the results are described, including confidence and credible intervals, the probability that a strategy is cost-effective (as usually shown with cost-effectiveness acceptability curves (CEACs)) and the expected value of perfect information (EVPI). The tutorial clarifies and illustrates why EVPI is the only measure accounting fully for decision uncertainty, and why NL curves and the NL frontier may be preferred over CEACs and other plots for presenting cost-effectiveness results in the context of uncertainty. The easy calculations and a worked-out real-life example will help users to thoroughly understand and correctly interpret key cost-effectiveness results. Examples with mathematical calculations, interpretation, plots and R code are provided.

When is enough evidence enough? - Using systematic decision analysis and value-of-information analysis to determine the need for further evidence.

Decision analysis (DA) and value-of-information (VOI) analysis provide a systematic, quantitative methodological framework that explicitly considers the uncertainty surrounding the currently available evidence to guide healthcare decisions. In medical decision making under uncertainty, there are two fundamental questions: 1) What decision should be made now given the best available evidence (and its uncertainty)?; 2) Subsequent to the current decision and given the magnitude of the remaining uncertainty, should we gather further evidence (i.e., perform additional studies), and if yes, which studies should be undertaken (e.g., efficacy, side effects, quality of life, costs), and what sample sizes are needed? Using the currently best available evidence, VoI analysis focuses on the likelihood of making a wrong decision if the new intervention is adopted. The value of performing further studies and gathering additional evidence is based on the extent to which the additional information will reduce this uncertainty. A quantitative framework allows for the valuation of the additional information that is generated by further research, and considers the decision maker's objectives and resource constraints. Claxton et al. summarise: "Value of information analysis can be used to inform a range of policy questions including whether a new technology should be approved based on existing evidence, whether it should be approved but additional research conducted or whether approval should be withheld until the additional evidence becomes available." [Claxton K. Value of information entry in Encyclopaedia of Health Economics, Elsevier, forthcoming 2014.] The purpose of this tutorial is to introduce the framework of systematic VoI analysis to guide further research. In our tutorial article, we explain the theoretical foundations and practical methods of decision analysis and value-of-information analysis. To illustrate, we use a simple case example of a foot ulcer (e.g., with diabetes) as well as key references from the literature, including examples for the use of the decision-analytic VoI framework by health technology assessment agencies to guide further research. These concepts may guide stakeholders involved or interested in how to determine whether or not and, if so, which additional evidence is needed to make decisions.