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.

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.

As more health technology assessment (HTA) bodies seek to implement patient involvement, there is a desire to learn from other HTA bodies about their experiences and understand what approaches can be used and which ones make a real difference to HTA. This is difficult, as the impact of patient involvement in HTA is not well documented. This study aims to promote further discussion about the ways in which patient involvement can impact HTAs by studying stories of impact.

In a multi-stakeholder workshop, experts leading patient involvement in four HTA bodies shared examples of HTAs where they believed patient involvement made a difference, then they reflected on these impact stories within the wider context of impact evaluation.

The HTA bodies drew on patient input and patient-based evidence to inform their HTAs. The patient involvement was observed to elucidate patients’ experiences, needs and preferences which, in turn, was observed to influence the HTA recommendations about optimal use of technologies, including taking account of issues for sub-groups, outcomes that matter to patients and educational needs.

Personal stories of patient involvement may enable a wider understanding of different approaches to and impact of patient involvement. The examples relate to both patient input and patient-based evidence and highlight the role that patient involvement can play in reducing uncertainties and complementing the clinical and economic evidence in HTA. They suggest that impact can be seen in recommendations about how and when a technology is used.

Ethics has been considered among the core domains of health technology assessment (HTA), but there are still disputes regarding ethical analysis. This study aimed to examine full final reports of the European Network for Health Technology Assessment (EUnetHTA) in terms of their compliance with the ethical methodology and ethical perspective of the HTA Core Model®.

The study examines seven full final HTA reports of EUnetHTA written based on the methodology proposed in the HTA Core Model®. The reports were analyzed using the following parameters: competency of the person/group who conducted ethical analysis, assessment elements, and the methodology of ethical analysis.

The results show that, although the HTA Core Model® helped to standardize the final reports of the assessment, there are still concerns regarding the competency of the ethical analysis team, the perspectives on the purpose of ethical analysis, data sources and viewpoints of various stakeholders, use of ethical analysis methodology, and the evaluation of the ethical appropriateness of the entire HTA process.

The HTA Core Model® helped to standardize the final reports on the HTA; however, not all issues with the content and outcomes were solved. The lack of expertise in ethics and insufficiency of the teams regarding ethical analysis are other existing problems. This study also demonstrated that stakeholder viewpoints in general and patient perspectives, in particular, have been overlooked in the HTA process.

The aim of this study was to provide an overview of the methodological characteristics and compare the assessment methods applied in health technology assessments (HTAs) of public health interventions (PHIs).

We defined a PHI as a population-based intervention on health promotion or for primary prevention of chronic or nonchronic diseases. HTAs on PHIs were identified by systematically searching the Web pages of members of international HTA networks. We included only full HTA reports published between 2012 and 2016. Two reviewers extracted data on the methods used to assess effectiveness/safety, as well as on economic, social, cultural, ethical, and legal aspects using a-priori standardized tables.

We included ten HTAs provided by four different organizations. Of these, all reports assessed the effectiveness of the interventions and conducted economic evaluations, seven investigated social/cultural aspects, and four each considered legal and ethical aspects, respectively. Some reports addressed applicability, context/setting, and intervention fidelity issues in different ways. We found that most HTAs adapted their methods to some extent, for example, by including nonrandomized studies, expanding the search strategy, involving stakeholders, or applying a framework to guide the HTA process.

Our analysis provides a comprehensive overview of methods applied in HTAs on public health interventions. We found that a heterogeneous set of approaches is used to deal with the challenges of evaluating complex public health interventions.

Traditional decision rules have limitations when a new technology is less effective and less costly than a comparator. We propose a new probabilistic decision framework to examine non-inferiority in effectiveness and net monetary benefit (NMB) simultaneously. We illustrate this framework using the example of repetitive transcranial magnetic stimulation (rTMS) and electroconvulsive therapy (ECT) for treatment-resistant depression.

We modeled the quality-adjusted life-years (QALYs) associated with the new intervention (rTMS), an active control (ECT), and a placebo control, and we estimated the fraction of effectiveness preserved by the new intervention through probabilistic sensitivity analysis (PSA). We then assessed the probability of cost-effectiveness using a traditional cost-effectiveness acceptability curve (CEAC) and our new decision-making framework. In our new framework, we considered the new intervention cost-effective in each simulation of the PSA if it preserved at least 75 percent of the effectiveness of the active control (thus demonstrating non-inferiority) and had a positive NMB at a given willingness-to-pay threshold (WTP).

rTMS was less effective (i.e., associated with fewer QALYs) and less costly than ECT. The traditional CEAC approach showed that the probabilities of rTMS being cost-effective were 100 percent, 39 percent, and 14 percent at WTPs of $0, $50,000, and $100,000 per QALY gained, respectively. In the new decision framework, the probabilities of rTMS being cost-effective were reduced to 23 percent, 21 percent, and 13 percent at WTPs of $0, $50,000, and $100,000 per QALY, respectively.

This new framework provides a different perspective for decision making with considerations of both non-inferiority and WTP thresholds.

Heated humidified high-flow nasal cannula (HHHFNC) is gaining popularity as a mode of respiratory support. We updated a systematic review and meta-analyses examining the efficacy and safety of HHHFNC compared with standard treatments for preterm infants. The primary outcome was the need for reintubation for preterm infants following mechanical ventilation (post-extubation analysis) or need for intubation for preterm infants not previously intubated (analysis of primary respiratory support)

We searched PubMed, MEDLINE, Embase, and the Cochrane Library for randomized controlled trials (RCTs) of HHHFNC versus standard treatments. Meta-analysis was conducted using Review Manager 5.3.

The post-extubation analysis included ten RCTs (n = 1,201), and the analysis of primary respiratory support included ten RCTs (n = 1,676). There were no statistically significant differences for outcomes measuring efficacy, including the primary outcome. There were statistically significant differences favoring HHHFNC versus nasal cannula positive airway pressure (NCPAP) for air leak (post-extubation, risk ratio [RR] 0.29, 95 percent confidence interval [CI] 0.11 to 0.76, I2 = 0) and nasal trauma (post-extubation: 0.35, 95 percent CI 0.27 to 0.46, I2 = 5 percent; primary respiratory support: RR 0.52, 95 percent CI 0.37 to 0.74; I2 = 27 percent). Studies, particularly those of primary respiratory support, included very few preterm infants with gestational age (GA) <28 weeks.

HHHFNC may offer an efficacious and safe alternative to NCPAP for some infants but evidence is lacking for preterm infants with GA ≤28 weeks.

To assess the societal cost-effectiveness of the Transmural Trauma Care Model (TTCM), a multidisciplinary transmural rehabilitation model for trauma patients, compared with regular care.

The economic evaluation was performed alongside a before-and-after study, with a convenience control group measured only afterward, and a 9-month follow-up. Control group patients received regular care and were measured before implementation of the TTCM. Intervention group patients received the TTCM and were measured after its implementation. The primary outcome was generic health-related quality of life (HR-QOL). Secondary outcomes included disease-specific HR-QOL, pain, functional status, and perceived recovery.

Eighty-three trauma patients were included in the intervention group and fifty-seven in the control group. Total societal costs were lower in the intervention group than in the control group, but not statistically significantly so (EUR-267; 95 percent confidence interval [CI], EUR-4,175–3011). At 9 months, there was no statistically significant between-group differences in generic HR-QOL (0.05;95 percent CI, −0.02–0.12) and perceived recovery (0.09;95 percent CI, −0.09–0.28). However, mean between-group differences were statistically significantly in favor of the intervention group for disease-specific HR-QOL (−8.2;95 percent CI, −15.0–−1.4), pain (−0.84;95CI, −1.42–−0.26), and functional status (−20.1;95 percent CI, −29.6–−10.7). Cost-effectiveness acceptability curves indicated that if decision makers are not willing to pay anything per unit of effect gained, the TTCM has a 0.54–0.58 probability of being cost-effective compared with regular care. For all outcomes, this probability increased with increasing values of willingness-to-pay.

The TTCM may be cost-effective compared with regular care, depending on the decision-makers willingness to pay and the probability of cost-effectiveness that they perceive as acceptable.

This review aims to assess the cost-effectiveness of psychological interventions for schizophrenia/bipolar disorder (BD), to determine the robustness of current evidence and identify gaps in the available evidence.

Electronic searches (PsycINFO, MEDLINE, Embase) identified economic evaluations relating incremental cost to outcomes in the form of an incremental cost-effectiveness ratio published in English since 2000. Searches were concluded in November 2018. Inclusion criteria were: adults with schizophrenia/BD; any psychological/psychosocial intervention (e.g., psychological therapy and integrated/collaborative care); probability of cost-effectiveness at explicitly defined thresholds reported. Comparators could be routine practice, no intervention, or alternative psychological therapies. Screening, data extraction, and critical appraisal were performed using pre-specified criteria and forms. Results were summarized qualitatively. The protocol was registered on the PROSPERO database (CRD42017056579).

Of 3,864 studies identified, 12 met the criteria for data extraction. All were integrated clinical and economic randomized controlled trials. The most common intervention was cognitive behavioral therapy (CBT, 6/12 studies). The most common measure of health benefit was the quality-adjusted life-year (6/12). Follow-up ranged from 6 months to 5 years. Interventions were found to be cost-effective in most studies (9/12): the probability of cost-effectiveness ranged from 35-99.5 percent. All studies had limitations and demonstrated uncertainty (particularly related to incremental costs).

Most studies concluded psychological interventions for schizophrenia/BD are cost-effective, including CBT, although there was notable uncertainty. Heterogeneity across studies makes it difficult to reach strong conclusions. There is a particular need for more evidence in the population with BD and for longer-term evidence across both populations.

The cost-effectiveness of molecular pathology testing is highly context dependent. The field is fast-moving, and national health technology assessment may not be relevant or timely for local decision makers. This study illustrates a method of context-specific economic evaluation that can be carried out in a limited timescale without extensive resources.

We established a multi-disciplinary group including an oncologist, pathologists and a health economist. We set out diagnostic and treatment pathways and costs using registry data, health technology assessments, guidelines, audit data, and estimates from the group. Sensitivity analysis varied input parameters across plausible ranges. The evaluation setting was the West of Scotland and UK NHS perspective was adopted. The evaluation was assessed against the AdHopHTA checklist for hospital-based health technology assessment.

A context-specific economic evaluation could be carried out on a timely basis using limited resources. The evaluation met all relevant criteria in the AdHopHTA checklist. Health outcomes were expected to be at least equal to the current strategy. Annual cost savings of £637,000 were estimated resulting primarily from a reduction in the proportion of patients receiving intravenous infusional chemotherapy regimens. The result was not sensitive to any parameter. The data driving the main cost saving came from a small clinical audit. We recommended this finding was confirmed in a larger population.

The method could be used to evaluate testing changes elsewhere. The results of the case study may be transferable to other jurisdictions where the organization of cancer services is fragmented.

We sought to assess the presence and reporting quality of peer-reviewed literature concerning the accuracy, precision, and reliability of home monitoring technologies for vital signs and glucose determinations in older adult populations.

A narrative literature review was undertaken searching the databases Medline, Embase, and Compendex. Peer-reviewed publications with keywords related to vital signs, monitoring devices and technologies, independent living, and older adults were searched. Publications between the years 2012 and 2018 were included. Two reviewers independently conducted title and abstract screening, and four reviewers independently undertook full-text screening and data extraction with all disagreements resolved through discussion and consensus.

Two hundred nine articles were included. Our review showed limited assessment and low-quality reporting of evidence concerning the accuracy, precision, and reliability of home monitoring technologies. Of 209 articles describing a relevant device, only 45 percent (n = 95) provided a citation or some evidence to support their validation claim. Of forty-eight articles that described the use of a comparator device, 65 percent (n = 31) used low-quality statistical methods, 23 percent (n = 11) used moderate-quality statistical methods, and only 12 percent (n = 6) used high-quality statistical methods.

Our review found that current validity claims were based on low-quality assessments that do not provide the necessary confidence needed by clinicians for medical decision-making purposes. This narrative review highlights the need for standardized health technology reporting to increase health practitioner confidence in these devices, support the appropriate adoption of such devices within the healthcare system, and improve health outcomes.

This article discusses how participatory technology appraisal as part of the Universal Coverage Scheme (UCS) in Thailand contributes to improving access to essential health services among vulnerable populations.

Document review was conducted on health technology appraisal approaches introduced by the UCS. The review involves health benefit proposals advanced by stakeholders and also meeting minutes of relevant working groups and committees published between 2010 and 2015.

From the establishment of the UCS participatory technology appraisal mechanism in 2010 until 2015, a total of 133 health interventions have been nominated. Some nominations highlight problems in access to care among vulnerable populations. As policy advocates continue to be involved in the latter stages of coverage decisions, they have opportunities to persuade policy makers and other stakeholders to agree to the rationales of their proposals. Some interventions were rejected because they did not meet value for money, affordability, and feasibility criteria; however, topic nominations from stakeholders as well as relevant deliberation throughout the technology appraisal processes have a potential to improve accessibility of health care among the disadvantaged.

Through participation in the UCS policy-making processes, key stakeholders are able to direct the attention of decision makers to significant gaps in access to services among vulnerable citizens, a health system problem rarely brought to discussion by policy elites and experts. The Thai experience reaffirms participatory technology appraisal as a supportive measure to providing universal health coverage.

Real-world evidence (RWE), derived from real-world data (RWD), is already used, to some extent, for health technology assessment (HTA) purposes. With the increased availability of RWD, there is potential for more widespread use but also challenges ensuring reliable RWE for HTA. Opportunities to overcome key challenges, identified at a scoping meeting, were discussed during the 2019 HTA international (HTAi) Global Policy Forum (GPF).

Reflection of discussions using Design Thinking (an interactive process aimed to solve complex problems) between seventy-three representatives from not-for-profit, for-profit organizations, and HTAi leadership. The discussions were informed by a background paper, and presentations from three invited keynote speakers and eleven GPF members.

Several options were listed for addressing the identified key challenges: quality and acceptability, governance and accountability, transferability, and informing decision making. The GPF emphasized that the HTA community should first understand what questions could be answered with RWE. Additionally, more clarity on methods, standards, streamlining RWD collection, data sharing across jurisdictions, replication of RWD, and expert analysis were mentioned as important priorities.

The HTA community is currently standing at a cross-road as it is not yet fully equipped to address these key challenges. It is, therefore, time for action. The community should start aligning on what is the best source of evidence according to purpose and how the data should be collected to create reliable evidence. It should also initiate the development of actions and guidance to properly develop and manage RWD/RWE to inform decision making across the technology lifecycle.

The aim of this study is to determine whether the approach used in Australia to regulate mobile medical applications (MMA) is consistent with international standards and is suitable to address the unique challenges of these technologies.

The policies of members of the International Medical Device Regulator's Forum (IMDRF) were analyzed, to determine whether these regulatory bodies address IMDRF recommendations for the clinical evaluation of software as a medical device (SaMD). Case-studies of varying types of regulated MMAs in Australia and the United States were also reviewed to determine how well the guidance in the IMDRF's SaMD: Clinical Evaluation (2017) document was operationalized.

All included jurisdictions evaluated the effectiveness of MMAs and addressed the majority of the key sub-categories recommended in the IMDRF guidance document. However, safety principles concerning information security (cybersecurity) and potential dangers of misinformation (risk-classification) were generally not addressed in either the case-studies or in the policy documents of international regulatory bodies. Australia's approach was consistent with MMA regulation conducted internationally. None of the approaches used by global regulatory bodies adequately addressed the risk of misinformation from apps and the potential for adverse clinical consequences.

The risks posed by MMAs are mainly through the information they provide and how this is used in clinical decision-making. Policy in Australia and elsewhere should be adjusted to follow the IMDRF risk-classification criteria to address potential harms from misinformation. Australian regulatory information should also be updated so the harm posed by cybersecurity and connectivity can be comprehensively evaluated.