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Objectives A recent debate in health economics and outcomes research community identified option value as one of the elements warranting consideration in the assessment of medical technologies. To conduct a scoping review of contributions on option value in the healthcare sector and identify relevant conceptual aspects and methods used to incorporate it in standard economic evaluations. Methods A systematic search was conducted up to July 2020 to identify contributions from electronic bibliographic database and gray literature. Data on the proposed definitions of option value, theoretical implications of its use in economic evaluations, and methods used to estimate it were extracted and analyzed. Results We found 57 eligible studies. Three different definitions emerged: insurance value, real option value, and option value of survival. Focusing on the latter (24 studies), we analyzed in depth 8 empirical applications across 7 therapeutic areas. The most relevant methodological challenges were on the perspective used in economic evaluations and how to robustly manage forecasting uncertainty, update cost-effectiveness thresholds, and avoid double-counting issues. For empirical studies assessing the total value of the technology, including option value, estimates ranged from +7% to +469% of its conventional value. Conclusions This review synthesizes theoretical and empirical aspects on option value of healthcare technologies and proposes a terminology to distinguish 3 different concepts identified. Future work should focus primarily on agreeing on whether option value should be included in economic evaluations and, if so, on developing and validating reliable methods for its ex-ante estimation.

Objectives Medical devices are potentially good candidates for coverage with evidence development (CED) schemes, as clinical data at market entry are often sparse and (cost-)effectiveness depends on real-world use. The objective of this research was to explore the diffusion of CED schemes for devices in Europe, and the factors that favour or hamper their utilization. Methods We conducted structured interviews with 25 decision-makers from 22 European countries to explore the characteristics of existing CED programmes for devices, and how decision makers perceived 13 pre-identified challenges associated with initiating and operating CED schemes for devices. We also collected data on individual schemes that were either initiated or still ongoing in the last 5 years. Results We identified seven countries with CED programmes for devices and 78 ongoing schemes. The characteristics of CED programmes varied across countries, including eligibility criteria, roles and responsibilities of stakeholders, funding arrangements, and type of decisions being contemplated at the outset of each scheme. We observed a high variability in how decision makers perceived CED-related challenges possibly reflecting country-specific arrangements and different experiences with CED. One general finding across all countries was that relatively little attention was paid to the evaluation of schemes, both during and at their completion. Conclusions CED programmes for devices with different characteristics exist in Europe. Decision-makers’ perceptions differ on the challenges associated with these schemes. More exchange of knowledge and experience will help decision makers anticipate the likely challenges in CED schemes for devices, and to learn from good practices existing elsewhere.

Differing contexts have greatly influenced HTA development in various countries, with considerable effort recently made by international HTA networks (e.g., EUnetHTA) and the European Union (EU) to make HTA a more coherent, equal, and efficient process. Medical devices (MDs) present particular challenges for HTA because of frequent, rapid innovation, outcomes influenced by end-user competence, dynamic pricing and often low-quality scientific evidence. Our objective is to describe the development, structure and governance of a National HTA Program for MDs (PNHTADM) in Italy, a highly participatory, stakeholder-engaged, evidence-based process to reform a fragmented system of appraisal and approval. Based largely on EUnetHTA methods, the resulting process delineates a standardized system for proposing MDs by any stakeholders, accrediting HTA producers, setting criteria for prioritization and appraisals, and innovatively linking recommendations with coverage, reimbursement and procurement of MDs. Expected benefits include reduced disparities in pricing and reimbursement policies and improved access to new technologies across 21 regional healthcare systems in Italy's decentralized, universal system, complete with provisions to require additional evidence collection and centrally monitor diffusion. Though devised for Italy, the design, resources and underlying analysis provide a framework for other nations seeking to consolidate HTA initiatives, particularly in light of new EU regulation.

INTRODUCTION: Performance-based risk-sharing agreements (PBRSAs), between payers, health care providers, and technology manufacturers can be useful when there is uncertainty about the (cost-) effectiveness of a new technology or service. However, they can be challenging to design and implement. AREAS COVERED: A total of 18 performance-based agreements were identified through a literature review. All but two of the agreements identified were pay-for-performance schemes, agreed between providers and payers at the national level. No examples were found of agreements between health care providers and manufacturers at the local level. The potential for these local agreements was illustrated by hypothetical case studies of water quality management and an integrated chronic kidney disease program. EXPERT OPINION: Performance-based risk-sharing agreements can work to the advantage of patients, health care providers, payers, and technology manufacturers, particularly if they facilitate the introduction of technologies or systems of care that might not have been introduced otherwise. However, the design, conduct, and implementation of PBRSAs in renal care pose a number of challenges. Efforts should be made to overcome these challenges so that more renal care patients can benefit from technological advances and new models of care.

In the drive towards faster patient access to treatments, health technology assessment (HTA) agencies are increasingly faced with reliance on evidence from surrogate endpoints, leading to increased decision uncertainty. This study undertook an updated survey of methodological guidance for using surrogate endpoints across international HTA agencies. We reviewed HTA and economic evaluation methods guidance from European, Australian and Canadian HTA agencies. We considered how guidelines addressed the methods for handling surrogate endpoints, including (1) level of evidence, (2) methods of validation, and (3) thresholds of acceptability. Across the 73 HTA agencies surveyed, 29 (40%) had methodological guidelines that made specific reference to consideration of surrogate outcomes. Of the 45 methods documents analysed, the majority [27 (60%)] were non-technology specific, 15 (33%) focused on pharmaceuticals and three (7%) on medical devices. The principles of the European network for Health Technology Assessment (EUnetHTA) guidelines published in 2015 on the handling of surrogate endpoints appear to have been adopted by many European HTA agencies, i.e. preference for final patient-relevant outcomes and reliance on surrogate endpoints with biological plausibility and epidemiological evidence of the association between the surrogate and final endpoint. Only a small number of HTA agencies (UK National Institute for Care and Excellence; the German Institute for Medical Documentation and Information and Institute for Quality and Efficiency in Health Care; the Australian Pharmaceutical Benefits Advisory Committee; and the Canadian Agency for Drugs and Technologies in Health) have developed more detailed prescriptive criteria for the acceptance of surrogate endpoints, e.g. meta-analyses of randomised controlled trials showing strong association between the treatment effect on the surrogate and final outcomes. As the decision uncertainty associated with reliance on surrogate endpoints carries a risk to patients and society, there is a need for HTA agencies to develop more detailed methodological guidance for consistent selection and evaluation of health technologies that lack definitive final patient-relevant outcome evidence at the time of the assessment.

In this viewpoint, we discuss the approach promoted by the Italian Drug Agency (AIFA) to the assessment of the value of new pharmaceuticals in the Italian Healthcare Service. On top of traditionally acknowledged components, such as quality adjusted life years gained and net costs, the overall value framework might include other elements such as productivity and adherence, equity, severity of disease, reduction in uncertainty, spillover effects. There is a residual dimension in the value framework that may capture the option value or reduction in fear of contagion for infectious disease treatments. We debate measurement issues on these elements of value and discuss open issues from a methodological and policy standpoint.

In this commentary, we discuss early stage assessments of innovative medical technologies both in terms of methods applied as well as their use in healthcare decision-making. We argue that cost-effectiveness alone may be too reductive if taken as the only decision rule, and it would benefit from being used within a broader evaluation framework. We discuss innovative methods which may contribute to better estimate the potential costs and consequences of a technology in the absence of solid clinical data, as frequently the case in early assessments. Finally, we comment on the potential synergies which may take place should early economic models be used not only by technology developers alone but as a negotiating base during early dialogues with health technology assessment (HTA) bodies.

Introduction: Specific guidance and examples for health technology assessment (HTA) of medical devices are scarce in medical device development. A more intense dialogue of competent authorities, HTA agencies, and manufactures may improve evidence base on clinical and cost-effectiveness. Especially as the new Medical Device Regulation requires more clinical evidence. Methods: We explore the perceptions of manufacturers, competent authorities, and HTA agencies towards such dialogues and investigate how they should be designed to accelerate the translational process from development to patient access using semi-structured interviews. We synthesized the evidence from manufacturers, competent authorities, and HTA agencies from 14 different jurisdictions across Europe. Results: Eleven HTA agencies, four competent authorities, and eight manufacturers of high-risk devices expressed perceptions on the current situation and the expected development of three types of early dialogues. Discussion: The MDR has to be taken into account when designing the early dialogue processes. Transferring insights from medicinal product regulation is limited as the regulatory pathways differ substantially. Conclusion: Early dialogues promise to accelerate the translational process and to provide faster access to innovative medical devices. However, health policy-makers should promote and fully establish regulatory and HTA early dialogues before introducing parallel early dialogues of regulatory, HTA agencies, and manufacturers. For initiating change, the legislator must create the legal basis and set the appropriate incentives for manufacturers.

Objectives Early rescue surfactant therapy using less invasive surfactant administration (LISA) can reduce the need for mechanical ventilation and avoid complications in preterm infants with respiratory distress syndrome. The purpose of this study was to estimate the budget impact of LISA compared with management based on continuous positive airway pressure (CPAP) alone and rescue surfactant therapy in case of CPAP failure. Methods A budget impact model was built comparing LISA with CPAP alone in order to estimate the potential resource consumption and budget impact from the perspective of the National Health Service in England. A literature review was conducted to populate the model. Deterministic and probabilistic sensitivity analyses were conducted to characterise the existing uncertainty and to explore the contribution of individual model parameters to the overall budget impact. Results Early rescue with LISA is expected to reduce resource consumption and costs compared with conservative therapy based on CPAP alone for preterm infants born at 25–32 weeks gestation. Savings are higher for preterm infants of 25–28 weeks (expected budget impact −£5146 per case, 95% credible interval (CrI) −£22 403 to £13, probability of being cost saving 97.4%) than for preterm infants of 29–32 weeks (−£176, 95% CrI −£4279 to £339, probability of being cost saving 85%). The impact of bronchopulmonary dysplasia (BPD) and intraventricular haemorrhage on resource consumption and the expected reduction in the incidence of BPD with LISA are the most influential parameters on the budget. Conclusions Early rescue with LISA used in preterm infants with respiratory distress syndrome and fraction of inspired oxygen ≥0.3 is expected to be cost saving compared with management based on CPAP alone, particularly in those born at 25–28 weeks gestation.

Objectives: Coverage with evidence development (CED) schemes are particularly relevant for medical devices (MDs), since clinical evidence is often limited at the time of launch and their long-term (cost- ) effectiveness heavily depends on how they are adopted into routine clinical practice. The objective of this study was to identify and describe the challenges that payers and manufacturers might face when assessing the desirability of, choosing the research design for, implementing, and evaluating CED schemes for MDs. Methods: A systematic literature review was performed on six databases following PRISMA guidelines. Two independent reviewers assessed the eligibility of studies based on predefined criteria and extracted data from the included articles by using a pre-defined extraction template. The data were synthesised in a narrative review. Results: The systematic search yielded 4293 articles of which 27 were eligible for inclusion. We identified 20 challenges that are associated with CED schemes for MDs. Five of these challenges relate directly to the characteristics of MDs, and hence are specific to MDs. These challenges concern deciding on whether a CED scheme is required, understanding the relevant uncertainties and risks, identifying meaningful out- comes, defining an adequate duration for a scheme, and market entry of new technologies. Conclusions: Payers and manufacturers of MDs have to address the identified challenges to improve a CED scheme’s chance of success. This can be further improved by public sharing of information about the outcome of applied schemes and way in which stakeholders have addressed the challenges they faced when applying a CED scheme.

Objectives:Current health technology assessment (HTA) methods guidelines for medical devices may benefit from contributions by biomedical and clinical engineers. Our study aims to: (i) review and identify gaps in the current HTA guidelines on medical devices, (ii) propose recommendations to optimize the impact of HTA for medical devices, and (iii) reach a consensus among biomedical engineers on these recommendations.Methods:A gray literature search of HTA agency Web sites for assessment methods guidelines on devices was conducted. The International Federation of Medical and Biological Engineers (IFMBE) then convened a structured focus group, with experts from different fields, to identify potential gaps in the current HTA guidelines, and to develop recommendations to fill these perceived gaps. The thirty recommendations generated from the focus group were circulated in a Delphi survey to eighty-five biomedical and clinical engineers.Results:Thirty-two panelists, from seventeen countries, participated in the Delphi survey. The responses showed a strong agreement on twenty-seven of thirty recommendations. Some uncertainties remain about the methods to accurately assess the effectiveness and safety, and interoperability of a medical device with other devices or within the clinical setting.Conclusions:As medical devices differ from drug therapies, current HTA methods may not accurately reflect the conclusions of their assessment. Recommendations informed by the focus group discussions and Delphi survey responses aimed to address the perceived gaps, and to provide a more integrated approach in medical device assessments in combining engineering with other perspectives, such as clinical, economic, patient, human factors, ethical, and environmental.

Introduction This study systematically reviews costing studies of seasonal influenza-like illness (ILI) in high-income countries. Existing reviews on the economic impact of ILI do not report information on drug consumption and its costs, nor do they provide data on the overall cost per episode. Methods The PRISMA-P checklist was used to design the research protocol. Studies included were cost of illness analysis (COI) and modeling studies that estimated the cost of ILI episodes. Records were searched from January 2000 to December 2016 in electronic bibliographic databases including Medline, Embase, Science Direct, the Cochrane Library, the Centre for Reviews and Disseminations of the University of York, and Google scholar. References from the included studies were hand-searched for completion. Abstract screening, full-text analysis and data extraction were performed by two reviewers independently and discrepancies were resolved by discussion with a third reviewer. A standardized, pre-piloted form was used for data extraction. All costs were converted to 2015 US$ Purchasing Power Parities. Results The literature search identified 5,104 records. After abstract and title screening, 76 studies were analyzed full-text and 27 studies were finally included in the review. Full estimates of the cost per episode range from US$19 in Korea to US$323 in Germany. Particularly, the cost per episode of laboratory confirmed influenza cases was estimated between US$64 and US$73. Inpatient and outpatient services account for the majority of the costs. Differences in the estimates may reflect country-specific characteristics, as well as other study-specific features including study design, identification strategy of ILI cases, study populations and types of costs included in the analysis. Children usually register higher costs, whereas evidence for the elderly is less conclusive. Patients risk-profile, co-morbidities and complications are the other important cost-drivers. None of the papers considered appropriateness in resource use (e.g. abuse of antibiotics). Despite cost of illness studies have ultimately a descriptive role, evidence on (in)appropriateness is useful for policy-makers.

ObjectivesTo explore treatment behaviours in a cohort of Italian patients with hereditary angioedema due to complement C1-inhibitor deficiency (C1-INH-HAE), and to estimate how effects and costs of treating attacks in routine practice differed across available on-demand treatments.DesignCost analyses and survival analyses using attack-level data collected prospectively for 1 year.SettingNational reference centre for C1-INH-HAE.Participants167 patients with proved diagnosis of C1-INH-HAE, who reported data on angioedema attacks, including severity, localisation and duration, treatment received, and use of other healthcare services.InterventionsAttacks were treated with either icatibant, plasma-derived C1-INH (pdC1-INH) or just supportive care.Main outcome measuresTreatment efficacy in reducing attack duration and the direct costs of acute attacks.ResultsOverall, 133 of 167 patients (79.6%) reported 1508 attacks during the study period, with mean incidence of 11 attacks per patient per year. Only 78.9% of attacks were treated in contrast to current guidelines. Both icatibant and pdC1-INH significantly reduced attack duration compared with no treatment (median times from onset 7, 10 and 47 hours, respectively), but remission rates with icatibant were 31% faster compared with pdC1-INH (HR 1.31, 95% CI 1.14 to 1.51). However, observed treatment behaviours suggest patterns of suboptimal dosing for pdC1-INH. The average cost per attack was €1183 (SD €789) resulting in €1.58 million healthcare costs during the observation period (€11 912 per patient per year). Icatibant was 54% more expensive than pdC1-INH, whereas age, sex and prophylactic treatment were not associated to higher or lower costs.ConclusionsBoth icatibant and pdC1-INH significantly reduced attack duration compared with no treatment, however, icatibant was more effective but also more expensive. Treatment behaviours and suboptimal dosing of pdC1-INH may account for the differences, but further research is needed to define their role.

Background Unhealthy diet is a risk factor for adverse health outcomes. Reformulation of processed foods has the potential to improve population diet, but evidence of its impact is limited. The purpose of this review was to explore the impact of reformulation on nutrient intakes, health outcomes and quality of life; and to evaluate the quality of modelling studies on reformulation interventions. Methods A systematic review of peer-reviewed articles published between January 2000 and December 2017 was performed using MEDLINE, ScienceDirect, Embase, Scopus, Cochrane, and the Centre for Reviews and Dissemination of the University of York. Additional studies were identified through informal searches on Google and specialized websites. Only simulation studies modelling the impact of food reformulation on nutrient intakes and health outcomes were included. Included articles were independently extracted by 2 reviewers using a standardized, pre-piloted data form, including a self-developed tool to assess study quality. Results A total of 33 studies met the selected inclusion criteria, with 20, 5 and 3 studies addressing sodium, sugar and fats reformulation respectively, and 5 studies addressing multiple nutrients. Evidence on the positive effects of reformulation on consumption and health was stronger for sodium interventions, less conclusive for sugar and fats. Study features were highly heterogeneous including differences in methods, the type of policy implemented, the extent of the reformulation, and the spectrum of targeted foods and nutrients. Nonetheless, partial between-study comparisons show a consistent relationship between percentages reformulated and reductions in individual consumption. Positive results are also shown for health outcomes and quality of life measures, although comparisons across studies are limited by the heterogeneity in model features and reporting. Study quality was often compromised by short time-horizons, disregard of uncertainty and time dependencies, and lack of model validation. Conclusions Reformulation models highlight relevant improvements in diets and population health. While models are valuable tools to evaluate reformulation interventions, comparisons are limited by non-homogeneous designs and assumptions. The use of validated models and extensive scenario analyses would improve models’ credibility, providing useful insights for policy-makers. Review Registration A research protocol was registered within the PROSPERO database (ID number CRD42017057341). Electronic supplementary material The online version of this article (10.1186/s40795-018-0263-6) contains supplementary material, which is available to authorized users.

Health Technology Assessment (HTA) has been defined as a ‘multidisciplinary approach studying the clinical, economic, social and ethical implications of development, diffusion and use of health technology’. While the general definition of HTA is widely accepted and its role in policy making is increasingly established in EU countries, the currently adopted methodological framework for HTA does not fully encounter the challenges rising from different types of health technologies, such as medical devices. This paper provides i) an introduction to the HTA methodology, highlight on ii) specific challenges medical devices pose in addition to other health technologies (i.e. short lifecycle and rapid changes, clinical outcomes often depend on training and experience of operator, dynamic pricing), iii) current HTA practices for medical devices and iv) the results of a FP7 funded project, “Methods for Health Technology Assessment of Medical Devices: a European Perspective” (MedTecHTA n. 305694) completed in December 2015. The general objective of MedtecHTA was to enhance HTA methods for medical devices that would acknowledge complexities rising from their integration into clinical practice and to develop recommendations for a wide range of stakeholders in the field. Overall, this paper provides a summary of the current and expected future trends in the assessment of medical devices technology to inform coverage and reimbursement decisions in healthcare within Europe and beyond.