Characterising uncertainty in the assessment of medical devices and determining future research needs

Decisions about the adoption of medical interventions are informed by evidence on their costs and effects. For a range of reasons, evidence relating to medical devices may be limited. The decision to adopt a device early in its life cycle when the evidence base is least mature may impact on the prospects of acquiring further evidence to reduce uncertainties. Equally, rejecting a device will result in no uptake in practice and hence no chance to learn about performance. Decision options such as ‘only in research’ or ‘approval with research’ can overcome these issues by allowing patients early access to promising new technologies while limiting the risks associated with making incorrect decisions until more evidence or learning is established. In this paper, we set out the issues relating to uncertainty and the value of research specific to devices: learning curve effects, incremental device innovation, investment and irrecoverable costs, and dynamic pricing. We show the circumstances under which an only in research or approval with research scheme may be an appropriate policy choice. We also consider how the value of additional research might be shared between the manufacturer and health sector to help inform who might reasonably be expected to conduct the research needed

Nature and reporting characteristics of UK health technology assessment systematic reviews

BACKGROUND: A recent study by Page et al. (PLoS Med. 2016;13(5):e1002028) claimed that increasing numbers of reviews are being published and many are poorly-conducted and reported. The aim of the present study was to assess how well reporting standards of systematic reviews produced in a Health Technology Assessment (HTA) context compare with reporting in Cochrane and other 'non-Cochrane' systematic reviews from the same years (2004 and 2014), as reported by Page et al. (PLoS Med. 2016;13(5):e1002028). METHODS: All relevant UK HTA programme systematic reviews published in 2004 and 2014 were identified. After piloting of the form, two reviewers each extracted relevant data on conduct and reporting from these reviews. These data were compared with data for Cochrane and "non-Cochrane" systematic reviews, as published by Page et al. (PLoS Med. 2016;13(5):e1002028). All data were tabulated and summarized. RESULTS: There were 30 UK HTA programme systematic reviews and 300 other systematic reviews, including Cochrane reviews (n = 45). The percentage of HTA reviews with required elements of conduct and reporting was frequently very similar to Cochrane and much higher than all other systematic reviews, e.g. availability of protocols (90, 98 and 16% respectively); the specification of study design criteria (100, 100, 79%); the reporting of outcomes (100, 100, 78%), quality assessment (100, 100, 70%); the searching of trial registries for unpublished data (70, 62, 19%); reporting of reasons for excluding studies (91, 91 and 70%) and reporting of authors' conflicts of interests (100, 100, 87%). HTA reviews only compared less favourably with Cochrane and other reviews in assessments of publication bias. CONCLUSIONS: UK HTA systematic reviews are often produced within a specific policy-making context. This context has implications for timelines, tools and resources. However, UK HTA systematic reviews still tend to present standards of conduct and reporting equivalent to "gold standard" Cochrane reviews and superior to systematic reviews more generally

Social values and their role in allocating resources for new health technologies

Every healthcare system faces unlimited demands and limited resources, creating a need to make decisions that may limit access to some new, potentially effective technologies. It has become increasingly clearer that such decisions are more than technical ones. They require social value judgements - statements of the public’s distributive preferences for healthcare across the population. However, these value judgements largely remain ill-defined. The purpose of this thesis was to explicate distributive preferences of the public to inform funding/coverage decisions on new health technologies. It contains six papers. The first comprises a systematic review of current coverage processes around the world, including value assumptions embedded within them. The second paper presents findings from an expert workshop and key-informant interviews with senior-level healthcare decision-makers in Canada. A technology funding decision-making framework, informed by the results of the first paper and the experiences of these decision-makers, was developed. Their input also highlighted the lack of and need for information on values that reflect those of the Canadian public. The third paper provides a systematic review of empirical studies attempting to explicate distributive preferences of the public. It also includes an analysis of social value arguments found in appeals to negative coverage decisions. From the results of both components, possible approaches to eliciting social values from the public and a list of factors around which distributive preferences may be sought were compiled. Such factors represented characteristics of unique, competing patient populations. Building on findings from the third paper, the fourth paper describes a citizens’ jury held to explicate distributive preferences for new health technologies in Alberta, Canada. The jury involved a broadly representative sample of the public, who participated in decision simulation exercises involving trade-offs between patient populations characterized by different combinations of factors. A list of preference statements, demonstrating interactions among such factors, emerged. The fifth and sixth papers address methodological issues related to citizens’ juries, including the comparability of findings from those carried out in the same way but with different samples of the public, and the extent to which they changed the views of individuals who participate in them

Role of centralized review processes for making reimbursement decisions on new health technologies in Europe

Tania Stafinski1, Devidas Menon2, Caroline Davis1, Christopher McCabe31Health Technology and Policy Unit, 2Health Policy and Management, School of Public Health, University of Alberta, Edmonton, Alberta, Canada; 3Academic Unit of Health Economics, Leeds Institute for Health Sciences, University of Leeds, Leeds, UKBackground: The purpose of this study was to compare centralized reimbursement/coverage decision-making processes for health technologies in 23 European countries, according to: mandate, authority, structure, and policy options; mechanisms for identifying, selecting, and evaluating technologies; clinical and economic evidence expectations; committee composition, procedures, and factors considered; available conditional reimbursement options for promising new technologies; and the manufacturers' roles in the process.Methods: A comprehensive review of publicly available information from peer-reviewed literature (using a variety of bibliographic databases) and gray literature (eg, working papers, committee reports, presentations, and government documents) was conducted. Policy experts in each of the 23 countries were also contacted. All information collected was reviewed by two independent researchers.Results: Most European countries have established centralized reimbursement systems for making decisions on health technologies. However, the scope of technologies considered, as well as processes for identifying, selecting, and reviewing them varies. All systems include an assessment of clinical evidence, compiled in accordance with their own guidelines or internationally recognized published ones. In addition, most systems require an economic evaluation. The quality of such information is typically assessed by content and methodological experts. Committees responsible for formulating recommendations or decisions are multidisciplinary. While criteria used by committees appear transparent, how they are operationalized during deliberations remains unclear. Increasingly, reimbursement systems are expressing interest in and/or implementing reimbursement policy options that extend beyond the traditional "yes", "no", or "yes with restrictions" options. Such options typically require greater involvement of manufacturers which, to date, has been limited.Conclusion: Centralized reimbursement systems have become an important policy tool in many European countries. Nevertheless, there remains a lack of transparency around critical elements, such as how multiple factors or criteria are weighed during committee deliberations.Keywords: reimbursement, centralized review, health technologies, Europ

HTA Implementation Roadmap in Central and Eastern European Countries.

The opportunity cost of inappropriate health policy decisions is greater in Central and Eastern European (CEE) compared with Western European (WE) countries because of poorer population health and more limited healthcare resources. Application of health technology assessment (HTA) prior to healthcare financing decisions can improve the allocative efficiency of scarce resources. However, few CEE countries have a clear roadmap for HTA implementation. Examples from high-income countries may not be directly relevant, as CEE countries cannot allocate so much financial and human resources for substantiating policy decisions with evidence. Our objective was to describe the main HTA implementation scenarios in CEE countries and summarize the most important questions related to capacity building, financing HTA research, process and organizational structure for HTA, standardization of HTA methodology, use of local data, scope of mandatory HTA, decision criteria, and international collaboration in HTA. Although HTA implementation strategies from the region can be relevant examples for other CEE countries with similar cultural environment and economic status, HTA roadmaps are not still fully transferable without taking into account country-specific aspects, such as country size, gross domestic product per capita, major social values, public health priorities, and fragmentation of healthcare financing

When does NICE recommend the use of health technologies within a programme of evidence development?

This article is made available through the Brunel Open Access Publishing Fund. This article is distributed under the terms of the Creative Commons Attribution Noncommercial License which permits any noncommercial use, distribution, and reproduction in any medium, provided the original author(s) and the source are credited.This article has been made available through the Brunel Open Access Publishing Fund.Background: There is growing interest internationally in linking reimbursement decisions with recommendations for further research. In the UK, the National Institute for Health and Clinical Excellence (NICE) can issue guidance to approve the routine use of a health intervention, reject routine use or recommend use within a research programme. These latter recommendations have restricted use to ‘only in research’ (OIR) or have recommended further research alongside routine use (‘approval with research’ or AWR). However, it is not currently clear when such recommendations are likely to be made. Objectives: This study aims to identify NICE technology appraisals where OIR or AWR recommendations were made and to examine the key considerations that led to those decisions. Methods: Draft and final guidance including OIR/AWR recommendations were identified. The documents were reviewed to establish the characteristics of the technology appraisal, the cost effectiveness of the technologies, the key considerations that led to the recommendations and the types of research required. Results: In total, 29 final and 31 draft guidance documents included OIR/AWR recommendations up to January 2010. Overall, 86 % of final guidance included OIR recommendations. Of these, the majority were for technologies considered to be cost ineffective (83 %) and the majority of final guidance (66 %) specified the need for further evidence on relative effectiveness. The use of OIR/AWR recommendations is decreasing over time and they have rarely been used in appraisals conducted through the single technology appraisal process. Conclusion: NICE has used its ability to recommend technologies within research programmes, although predominantly within the multiple technology appraisal process. OIR recommendations have been most frequently issued for technologies considered cost ineffective and the most frequently cited consideration is uncertainty related to relative effectiveness. Key considerations cited for most AWR recommendations and some OIR recommendations included a need for further evidence on long-term outcomes and adverse effects of treatment.Medical Research Counci

Ageing, urban marginality, and health in Ghana

The world’s population is rapidly ageing. Global estimates for the next three decades indicate a two-fold increase in the population of older adults aged ≥60 years. Nearly 80% of this growth will occur in low and middle-income countries in Asia and sub-Saharan Africa, where population health is already under threat from poverty, degraded environments, and deficient healthcare systems. Although the world’s poorest region, sub-Saharan Africa, ironically, will witness the fastest growth in older populations, rising by 64% over the next 15 years. Indications are that the majority of this population will live in resource-poor settings, characterized by deficient housing and neighbourhood conditions. Yet, very little research has systematically examined the health and wellbeing of older adults in such settings. Drawing on the ecological theory of ageing, the present study explores the living conditions and quality of life of elderly slum dwellers in Ghana, a sub-Saharan African country with a growing population of older adults. Data collection was undertaken in two phases in two environmentally contrasting neighbourhoods in Accra, Ghana. In Phase 1, we carried out a cross-sectional survey of older adults in a slum community (n = 302) and a non-slum neighbourhood (n = 301), using the World Health Organization quality of life assessment tool (WHOQoL-BREF). The survey data were complemented in Phase 2 with qualitative interviews involving a sample of community dwelling older adults (N = 30), health service providers (N = 5), community leaders (N = 2), and policymakers (N = 5). Preliminary analysis of the survey data revealed statistically significant differences in the social and environment domains of quality of life, while the qualitative data identified multiple health barriers and facilitators in the two neighbourhoods. Insights from the research are expected to inform health and social interventions for older slum dwellers in Ghana

CJCheck Stage 1: development and testing of a checklist for reporting community juries – Delphi process and analysis of studies published in 1996–2015

Background: Opportunities for community members to actively participate in policy development is increasing. Community/Citizen’s Juries (CJs) are a deliberative democratic process aimed to illicit informed community perspectives on difficult topics. But how comprehensive these processes are reported in peer-reviewed literature is unknown. Adequate reporting of methodology enables others to judge process quality, compare outcomes, facilitate critical reflection, and potentially repeat a process. We aimed to identify important elements for reporting CJs and develop an initial checklist, and to review published health and health policy CJs to examine reporting standards. Design: Using literature and expertise from CJ researchers and policy-advisors, a list of important CJ reporting items was suggested and further refined. We then reviewed published CJs within the health literature and used the checklist to assess the comprehensiveness of reporting. Results: CJCheck was developed and examined reporting of CJ planning, juror information, procedures and scheduling. We screened 1711 studies and extracted data from 38. No studies fully reported the checklist items. The item most consistently reported was juror numbers (92%, 35/38) while least reported was availability of expert presentations (5%, 2/38). Recruitment strategies were described in 66% of studies (25/38) however, the frequency and timing of deliberations was inadequately described (29%, 11/38). Conclusions: Currently CJ publications in health and health policy literature are inadequately reported, hampering their use in policy-making. We propose broadening the CJCheck by creating a reporting standards template in collaboration with international CJ researchers, policy-advisors and consumer representatives to ensure standardised, systematic and transparent reporting

Sustainable Financing of Innovative Therapies: A Review of Approaches

The process of innovation is inherently complex, and it occurs within an even more complex institutional environment characterized by incomplete information, market power, and externalities. There are therefore different competing approaches to supporting and financing innovation in medical technologies, which bring their own advantages and disadvantages. This article reviews value- and cost-based pricing, as well direct government funding, and cross-cutting institutional structures. It argues that performance-based risk-sharing agreements are likely to have little effect on the sustainability of financing; that there is a role for cost-based pricing models in some situations; and that the push towards longer exclusivity periods is likely contrary to the interests of industry

Defining the role of the public in Health Technology Assessment (HTA) and HTA-informed decision-making processes

First published online: 10 March 2020OBJECTIVES: The terminology used to describe community participation in Health Technology Assessment (HTA) is contested and frequently confusing. The terms patients, consumers, public, lay members, customers, users, citizens, and others have been variously used, sometimes interchangeably. Clarity in the use of terms and goals for including the different groups is needed to mitigate existing inconsistencies in the application of patient and public involvement (PPI) across HTA processes around the world. METHODS: We drew from a range of literature sources in order to conceptualize (i) an operational definition for the "public" and other stakeholders in the context of HTA and (ii) possible goals for their involvement. Draft definitions were tested and refined in an iterative consensus-building process with stakeholders from around the world. RESULTS: The goals, terminology, interests, and roles for PPI in HTA processes were clarified. The research provides rationales for why the role of the public should be distinguished from that of patients, their families, and caregivers. A definition for the public in the context of HTA was developed: A community member who holds the public interest and has no commercial, personal, or professional interest in the HTA process. CONCLUSIONS: There are two distinct aspects to the interests held by the public which should be explicitly included in the HTA process: the first lies in ensuring democratic accountability and the second in recognising the importance of including public values in decision making.Jackie Street, Tania Stafinski, Edilene Lopes and Devidas Meno