Discussions at the Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) aimed to understand the meaning of “high-cost technologies,” and to explore mechanisms to increase access to these technologies in publicly funded health systems in the Asia region.

Discussions and presentations at the 2018 HAPF, informed by a literature review and a premeeting survey of HTA agencies and industry, form the basis of this paper.

Challenges payers in the public health system face when investing in high-cost technologies include a lack of data, especially real-world data, affordability, and the budgetary impact of high-cost technologies. Managed entry schemes (MES) are one means to enable earlier access to high-cost technologies, or at reduced cost to the system. Most countries surveyed had used an MES to introduce a new health technology and most industry representatives had experience with financial-based MES, such as discounts or rebates, with most put in place to increase access to pharmaceuticals. Little experience of outcome-based or evidence-generation MES was reported.

Although it is early days in the implementation of MES in Asia, they have the potential to play an important role enabling access to new, mainly pharmaceutical, health technologies. The development of a “road map” of MES in the region should outline the intent and need for a MES, articulating the “rules of engagement” for all stakeholders—patients, providers, payers, and industry—which will assist countries to clearly identify the problem trying to be solved, and how an MES can be part of the solution.

This article retrospectively examines the evolution of rapid assessments (RAs) produced by the Health Technology Assessment (HTA) Program at the Institute of Health Economics over its 25-year relationship with a single requester, the Alberta Health Ministry (AHM).

The number, types, and methodological attributes of RAs produced over the past 25 years were reviewed. The reasons for developmental changes in RA processes and products over time were charted to document the push–pull tension between AHM needs and the HTA Program's drive to meet those needs while responding to changing methodological benchmarks.

The review demonstrated the dynamic relationship required for HTA researchers to meet requester needs while adhering to good HTA practice. The longstanding symbiotic relationship between the HTA Program and the AHM initially led to increased diversity in RA types, followed by controlled extinction of the less fit (useful) “transition species.” Adaptations in RA methodology were mainly driven by changes in best practice standards, requester needs, the healthcare environment, and staff expertise and technology.

RAs are a useful component of HTA programs. To remain relevant and useful, RAs need to evolve according to need within the constraints of HTA best practice.

To develop a definition of “capacity building” relevant to Health Technology Assessment international (HTAi).

A review of capacity building activities undertaken by HTAi members, members of the International Network of Agencies for Health Technology Assessment (INAHTA), and regional HTA networks was compared against general literature on capacity building definitions and frameworks. The findings were reviewed by the HTAi Scientific Development and Capacity Building Committee. Furthermore, the Executive Committee and Interest Groups of HTAi provided input on the draft final paper.

The literature demonstrated the need for a definition of capacity building specific to HTA. In the context of HTAi, it was necessary for the definition to cover (i) the broadest range of HTA-related activities, (ii) multiple stakeholders involved in the HTA process, and (iii) the spectrum of activities that compose capacity building. We propose the following definition of HTA capacity building: The process by which individuals and organizations develop or strengthen abilities related to understanding, providing input to, conducting, or utilizing HTA for health policy and decision making, as well as, developing awareness and support in the environment within which HTA is being used.

A definition of HTA-related capacity building that was intended to provide clarity about what this term means to HTAi was developed. As HTA is context-dependent, a need for further work to develop an operationalization “menu” relevant to the specific needs in which HTA is being used was identified.

Public health (PH) interventions are crucial for ensuring sustainable healthcare infrastructures. Nevertheless, they represent a neglected area in HTA due to various methodological issues and their complex design that goes beyond clinical setting. Our study provides an environmental scan of HTA initiatives related to the assessment of PH technologies on a global level.

The Initiative for Public Health Outcomes Research and Measurement (INPHORM) interest group has conducted a survey among European and international societies, health bodies and networks during September 2018. The questionnaire evaluates what kind of PH technologies and/or interventions have been evaluated in the last five years, or are planned for the future.

Our preliminary findings from November 2018 indicate a total of 94 initiated and 44 completed surveys. Among the completed ones, the majority of respondents came from European countries (36%), followed by North (30%) and South America (16%) countries. Sixty-eight percent of institutions reported engagement in any aspect of HTA in the area of PH (N = 30). Medical aspects of the PH technology are considered by 83 percent of the institutions, followed by organizational impact (67%), economic evaluation (60%) and societal consequences (60%). An average of four PH technologies has been evaluated by the responding institutions in the last five years. In reference to methodological aspects, 90 percent of institutions used a classical HTA approach for evaluating PH interventions, while 40 percent used budget impact analyses. Among the barriers for reaching a decision, conflicting stakeholder priorities, lack of data and clear methodological frameworks were most commonly cited.

Data analysis is currently on-going and final results will be presented during the Cologne meeting. This study will allow to raise awareness about the importance of PH interventions in HTA, identify existing gaps and propose future methodological developments.

The way choice is presented has an impact on decision-making. This is the case also in the context of neonatal intensive care units (NICUs), particularly in the challenging cases that concern the limit of viability. The objective of this article is to examine the role of nudging in the shared decision-making in neonatology and elaborate on the respective moral challenges.

Nudging is not morally neutral. There are two key sources of ethical issues at the heart of nudging. The first one concerns the lack of transparency, while the second concerns the background value judgments that are imminent whenever nudging is used for achieving a particular end. To solve the underlying conflict, a virtue ethics approach combined with the accountability for reasonableness framework is suggested to guide the use of the tool of nudging.

NICU professionals ought to use the tool of nudging transparently in line with their act of profession and their practically wise judgment.

Generic preference-based (GPB) measures of health-related quality of life (HRQL) are widely used as outcome measures in cost-effectiveness and cost-utility analyses (CEA, CUA). Health technology assessment agencies favor GPB measures because they facilitate comparisons among conditions and because the scoring functions for these measures are based on community preferences. However, there is no gold standard HRQL measure, scores generated by GPB measures may differ importantly, and changes in scores may fail to detect important changes in HRQL. Therefore, to enhance the accumulation of empirical evidence on how well GPB measures perform, we advocate that investigators routinely use two (or more) GPB measures in each study.

We discuss key measurement properties and present examples to illustrate differences in responsiveness for several major GPB measures across a wide variety of health contexts. We highlight the contributions of longitudinal head-to-head studies.

There is substantial evidence that the performance of GPB measures varies importantly among diseases and health conditions. Scores are often not interchangeable. There are numerous examples of studies in which one GPB measure was responsive while another was not.

Investigators should use two (or more) GPB measures. Study protocols should designate one measure as the primary outcome measure; the other measure(s) would be used in secondary analyses. As evidence accumulates it will better inform the relative strengths and weaknesses of alternative GPB measures in various clinical conditions. This will facilitate the selection and interpretation of GPB measures in future studies.

Proceeding from a basic concept underpinning economic evaluation, opportunity cost, this study aims to explain how different approaches to economics diverge quite dramatically in their ideas of what constitutes appropriate valuation, both in principle and practice. Because the concept of opportunity cost does not inherently specify how valuation should be undertaken or specify how appropriate any economic value framework (EVF) might be, the three main economics-based approaches to providing evidence about value for health technology assessment are described.

This paper describes how the three main EVFs—namely, the extra-welfarist, welfarist, and classical—are most typically understood, applied, and promoted. It then provides clarification and assessment of related concepts and terminology.

Although EVFs differ, certain underlying characteristics of valuation were identified as fundamental to all approaches to economic evaluation in practice. The study also suggests that some of the rhetoric and terms employed in relation to the extra-welfarist approach are not wholly justified and, further, that only the welfarist approach ensures adherence to welfare-economic principles. Finally, deliberative analysis, especially when connected with a classical economic approach, can serve as a useful supplement to other analytical approaches.

All three approaches to economic evaluation have something to offer assessment processes, but they all display limitations too. Therefore, the author concludes that the language of economic evaluation should be used with sufficient humility to prevent overselling of EVFs, especially with regard to the qualities of evidence they provide for priority setting processes.

Medical devices (MDs) differ regarding their risk class (I to III), operational area (in-, outpatient), intended use (diagnostic, monitoring, intervention), and with regard to available clinical evidence. Therefore, the market access processes as well as the approach of gaining reimbursement differ significantly. From a variety of potential approaches the underlying analysis illustrates five MD-specific processes.

Based on a systematic search of publicly available regulations the main pathways of potential reimbursement for MDs were evaluated.

MDs to be used in the in-patient setting can be divided into three categories: an innovative MD (a) is exceeding a current reimbursement framework (German Operations and Procedures Key (OPS) / diagnosis related groups (DRG)), (b) falls within an existing reimbursement rate, or (c) the MD is based on a known mode of action (MoA) for which already adequate reimbursement exists. Due to less empirical data from MDs for a) and b), a health technology assessment (HTA) is required before inclusion in a DRG, whereas a MD with known concept (c) will be grouped into existing price structures. Initiators of these processes are hospitals through a so-called NUB application. MDs entering the outpatient sector are covered by another reimbursement catalogue (EBM/GOÄ) and have to pass an assessment by the G-BA (rapid HTA) if based on new MoA (d). Such an assessment can only be initiated by respective umbrella organizations of service providers (e.g. KBV (National Association of Statutory Health Insurance Physicians)). MDs not being positively recommended by the G-BA are not reimbursable. For MDs (e) with known MoA no HTA is required.

For a successful market launch including sufficient reimbursement not only the market potential, but also the specific regulatory pathways have to be considered carefully. New and innovative MDs in the outpatient sector may have a longer application process to gain a positive reimbursement decision than MDs used in inpatient setting.

Stroke is a major cause of lasting disability worldwide. Virtual reality (VR) training has been introduced as a means of increasing the effectiveness of rehabilitation by providing large doses of task-related training with many repetitions and different modes of feedback. As VR is increasingly used in neurorehabilitation, cost considerations are important.

A cost-analysis was conducted based on the Virtual Reality for Upper Extremity in Subacute stroke (VIRTUES) trial, a recent international randomized controlled observer-blind multicenter trial. Average therapist time required per therapy session may differ between VR and conventional training (CT), leading to potential cost savings due to a therapist being able to supervise more than one patient at a time. Exploratory cost analyses are presented to explore such assumptions.

Based on our calculations, VR incurs extra costs as compared with CT when the same amount of therapist contact is provided, as was the case in VIRTUES. However, the exploratory analyses demonstrated that these costs may be rapidly counterbalanced when time for therapist supervision can be reduced.

Extra costs for VR can be outweighed by reduced therapist time and decreasing VR system costs in the nearer future, and not least by increased patient motivation.

To report from the Scientific Development and Capacity Building Committee of Health Technology Assessment International (HTAi) on activities that are being undertaken within HTAi regarding the promotion of scientific rigor in the field of health technology assessment (HTA).

Retrieval of definitions of HTA that the SDCB committee considered reflective of the current practice of HTA, followed by a narrative synthesis of the core components of HTA.

Several definitions of HTA have been provided, all sharing the notion that HTA is the formal, systematic, and transparent inquiry into the meaning and value, broadly defined, of health technologies, when used in specific patient populations.

Many frameworks and tools have been developed for assessing the quality of specific tasks that may be conducted in the context of HTA. Collating such frameworks and tools is likely to be helpful in developing standards and in providing guidance as to how the scientific quality of HTA may be secured. Two current trends in HTA were noted: a stronger health systems focus, and the need to involve stakeholders throughout the HTA process. A wider systems’ perspective requires that plausible alternative scenarios are being developed, and wide consultation of various stakeholders is a prerequisite to the development of such scenarios with data from various sources.

Current trends in HTA will lead to different demands on the HTA expert. The task of this emerging policy professional would be not just to provide technical information for problem-solving, but also to combine it with a new function of facilitating public deliberation and learning.

Population-based colorectal cancer (CRC) screening programs that use a fecal immunochemical test (FIT) are often faced with a noncompliance issue and its subsequent waiting time (WT) for those FIT positives complying with confirmatory diagnosis. We aimed to identify factors associated with both of the correlated problems in the same model.

A total of 294,469 subjects, either with positive FIT test results or having a family history, collected from 2004 to 2013 were enrolled for analysis. We applied a hurdle Poisson regression model to accommodate the hurdle of compliance and also its related WT for undergoing colonoscopy while assessing factors responsible for the mixture of the two outcomes.

The effect on compliance and WT varied with contextual factors, such as geographic areas, type of screening units, and level of urbanization. The hurdle score, representing the risk score in association with noncompliance, and the WT score, reflecting the rate of taking colonoscopy, were used to classify subjects into each of three groups representing the degree of compliance and the level of health awareness.

Our model was not only successfully applied to evaluating factors associated with the compliance and the WT distribution, but also developed into a useful assessment model for stratifying the risk and predicting whether and when screenees comply with the procedure of receiving confirmatory diagnosis given contextual factors and individual characteristics.

Canada has a long history of the use of clinical evidence to support healthcare decision making. Given improvements in data holdings and analytic capacity in Canada and stakeholder interest, the purpose of this study is to reflect on perceptions of the value of real-world evidence in pricing and reimbursement decisions, barriers to its optimal use in pricing and reimbursement, current initiatives that may lead to its increased use, and what role the pharmaceutical industry may play in this.

To capture stakeholder perceptions, ninety-one participants identified as key stakeholders were identified according to background roles and geography and invited to participate in four round table discussions conducted under Chatham House rule. Important themes emerging from these discussions included: (i) the need to understand what “real world” evidence means; (ii) barriers to using real world evidence from differences in access, governance, inter-operability, system structures, expertise, and quality across Canadian health systems; (iii) differing views on industry's role.

The use of real-world data in Canada to inform pricing and reimbursement decisions is far from routine but nascent and slowly increasing. Barriers, including interoperability concerns, may also apply to other federated health systems that need to focus on the networking of healthcare administrative data across provincial jurisdictional boundaries. There also appears to be a desire to see better use of pragmatic trials linked to these administrative data sets. Emerging initiatives are under way to use real world evidence more broadly, and include identification of common data elements and approaches to networking data.