The aim of this study was to investigate how innovation is defined with respect to new medicines.

MEDLINE, Embase, and EconLit databases were searched for articles published between January 1, 2010 and May 25, 2016 that described a relevant definition of innovation. Identified definitions were analyzed by mapping the concepts described onto a set of ten dimensions of innovation.

In total, thirty-six articles were included, and described a total of twenty-five different definitions of innovation. The most commonly occurring dimension was therapeutic benefit, with novelty and the availability of existing treatments the second and third most common dimensions. Overall, there was little agreement in the published literature on what characteristics of new medicines constitute rewardable innovation.

Alignment across countries and among regulators, health technology assessment bodies and payers would help manufacturers define research policies that can drive innovation, but may be challenging, as judgements about what aspects of innovation should be rewarded vary among stakeholders, and depend on political and societal factors.

When making decisions in health care, it is essential to consider economic evidence about an intervention. The objective of this study was to analyze the methods applied for systematic reviews of health economic evaluations (SR-HEs) in HTA and to identify common challenges.

We manually searched the Web pages of HTA organizations and included HTA-reports published since 2015. Prerequisites for inclusion were the conduct of an SR-HE in at least one electronic database and the use of the English, German, French, or Spanish language. Methodological features were extracted in standardized tables. We prepared descriptive statistical (e.g., median, range) measures to describe the applied methods. Data were synthesized in a structured narrative way.

Eighty-three reports were included in the analysis. We identified inexplicable heterogeneity, particularly concerning literature search strategy, data extraction, assessment of quality, and applicability. Furthermore, process steps were often missing or reported in a nontransparent way. The use of a standardized data extraction form was indicated in one-third of reports (32 percent). Fifty-four percent of authors systematically appraised included studies. In 10 percent of reports, the applicability of included studies was assessed. Involvement of two reviewers was rarely reported for the study selection (43 percent), data extraction (28 percent), and quality assessment (39 percent).

The methods applied for SR-HEs in HTA and their reporting quality are very heterogeneous. Efforts toward a detailed, standardized guidance for the preparation of SR-HEs definitely seem necessary. A general harmonization and improvement of the applied methodology would increase the value of SR-HE for decision makers.

The Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) met to discuss the challenges of achieving universal health care (UHC) in Asia.

Group discussions and presentations at the 2017 HAPF, informed by a background paper, including a literature review and the results of pre-meeting surveys of health technology assessment (HTA) agencies and industry, formed the basis of this article.

Affordability was identified as the greatest barrier to establishing UHC; however, other impediments include the lack of political will to implement UHC, and the cultural issue of deference to expert opinion instead of evidence-based assessments. Although HTA was identified as an important prioritization tool when adding new technologies to benefit packages, it is used inconsistently in the region, resulting in a less transparent decision-making process for stakeholders. Although regional challenges exist around real-world data (RWD), including a lack of capacity to enable information and data sharing, most policy or funding decision makers in the region have access to data. However, there appears to be a disconnect with the experience of industry, whose representatives identify the lack of RWD as their primary issue. To overcome these issues, both HTA agencies and industry agree that collaboration and transparency should be fostered to support the development of robust evidence generation in the region.

There is a willingness for HTA agencies and industry to collaborate to develop HTA methodology for the prioritization of technologies in the Asia region that support healthcare systems to achieve the ultimate outcome of UHC.

This study is an attempt to demystify and clarify the idea of cost in health economics and health technology assessment (HTA).

Its method draws on standard concepts in economics. Cost is a more elusive concept than is commonly thought and can be particularly elusive in multidisciplinary territory like HTA.

The article explains that cost is more completely defined as opportunity cost, why cost is necessarily associated with a decision, and that it will always vary according to the context of that decision: whether choice is about inputs or outputs, what the alternatives are, the timing of the consequences of the decision, the nature of the commitment to which a decision maker is committed, who the decision maker is, and the constraints and discretion limiting or liberating the decision maker. Distinctions between short and long runs and between fixed and variable inputs are matters of choice, not technology, and are similarly context-dependent. Harms or negative consequences are, in general, not costs. Whether so-called “clinically unrelated” future costs and benefits should be counted in current decisions again depends on context.

The costs of entire health programs are context-dependent, relating to planned rates of activity, volumes, and timings. The implications for the methods of HTA are different in the contexts of low- and middle-income countries compared with high-income countries, and further differ contextually according to the budget constraints (fixed or variable) facing decision makers.

This study investigated which databases and which combinations of databases should be used to identify economic evaluations (EEs) to inform systematic reviews. It also investigated the characteristics of studies not identified in database searches and evaluated the success of MEDLINE search strategies used within typical reviews in retrieving EEs in MEDLINE.

A quasi-gold standard (QGS) set of EEs was collected from reviews of EEs. The number of QGS records found in nine databases was calculated and the most efficient combination of databases was determined. The number and characteristics of QGS records not retrieved from the databases were collected. Reproducible MEDLINE strategies from the reviews were rerun to calculate the sensitivity and precision for each strategy in finding QGS records.

The QGS comprised 351 records. Across all databases, 337/351 (96 percent) QGS records were identified. Embase yielded the most records (314; 89 percent). Four databases were needed to retrieve all 337 references: Embase + Health Technology Assessment database + (MEDLINE or PubMed) + Scopus. Four percent (14/351) of records could not be found in any database. Twenty-nine of forty-one (71 percent) reviews reported a reproducible MEDLINE strategy. Ten of twenty-nine (34.5 percent) of the strategies missed at least one QGS record in MEDLINE. Across all twenty-nine MEDLINE searches, 25/143 records were missed (17.5 percent). Mean sensitivity was 89 percent and mean precision was 1.6 percent.

Searching beyond key databases for published EEs may be inefficient, providing the search strategies in those key databases are adequately sensitive. Additional search approaches should be used to identify unpublished evidence (grey literature).

The aim of this study was to identify good practice principles for health technology assessment (HTA) that are the most relevant and of highest priority for application in Latin America and to identify potential barriers to their implementation in the region.

HTA good practice principles proposed at the international level were identified and then explored during a deliberative process in a forum of assessors, funders, and product manufacturers.

Forty-two representatives from ten Latin American countries participated. Good practice principles proposed at the international level were considered valid and potentially relevant to Latin America. Five principles were identified as priority and with the greatest potential to be strengthened at this time: transparency in the production of HTA, involvement of relevant stakeholders in the HTA process, mechanisms to appeal decisions, clear priority-setting processes in HTA, and a clear link between HTA and decision making. The main challenge identified was to find a balance between the application of these principles and the available resources in a way that would not detract from the production of reports and adaptation to the needs of decision makers.

The main recommendation was to progress gradually in strengthening HTA and its link to decision making by developing appropriate processes for each country, without trying to impose, in the short-term, standards taken from examples at the international level without adequate adaptation of these to local contexts.

The aim of this study was to identify guidelines and assessment tools used by health technology agencies for quality assurance of registries and investigate the current use of registry data by HTA organizations worldwide.

As part of a European Network for Health Technology Assessment Joint Action work package, we undertook a literature search and sent a questionnaire to all partner organizations on the work package and all organizations listed in the International Society for Pharmaco-economics and Outcomes Research directory.

We identified thirteen relevant documents relating to quality assurance of registries. We received fifty-five responses from organizations representing twenty-one different countries, a response rate of 40.5 percent (43/110). Many agencies, particularly in Europe, are already drawing on a range of registries to provide data for their HTA. Less than half, however, use criteria or standards to assess the quality of registry data. Nearly all criteria or standards in use have been internally defined by organizations rather than referring to those produced by an external body. A comparison of internal and external standards identified consistency in several quality dimensions, which can be used as a starting point for the development of a standardized tool.

The use of registry data is more prevalent than expected, strengthening the need for a standardized registry quality assessment tool. A user-friendly tool developed in conjunction with stakeholders will support the consistent application of approved quality standards, and reassure critics who have traditionally considered registry data to be unreliable.

With the increase in technologies to support an aging population, health technology assessment (HTA) of aging-related technologies warrants special consideration. At Health Technology Assessment international (HTAi) 2016 and HTAi 2017, an international panel explored interests in HTA focused on aging.

Panelists from five countries shared the state of aging and HTA in their countries. Opportunities were provided for participants to discuss and rate the themes identified by the panelists.

In 2016, the highest ranked themes were: (i) identifying unmet needs of older adults that could be met by technology—how can HTA help?; (ii) differences in assessment of aging-related technologies—what is the scope?; and (iii) involvement of older adults and caregivers. These themes became the starting point for discussion in 2017, for which the highest ranked themes were: (i) identification of challenges in HTA and aging; and (ii) approaches to advancing effectiveness of HTA for aging.

These discussions allowed for examination of future directions for HTA and aging: engagement of older adults to inform the agenda of HTA and the broader public policy enterprise; a systems approach to thinking about needs of older persons should support the type and level of care desired by the individual rather than the health institutions, and HTA should reflect these desires when evaluating technological aides; and there is potential for health information systems and “big data” to support HTA activities that assess usability of technologies for older adults. We hope to build on the momentum of this community to continue exploring opportunities for aging and HTA.

Health technology assessment (HTA) is scientifically incorporating the demand, effectiveness and expedience of adopting innovative technologies, supported by evidence-based medicine. The rapid development of innovations led to an accelerated need to comprehend their benefit and satisfaction through actual utilization. ‘Real World’ Experience (RWE) in action is a source for collecting information through practice that has not yet been formally published. The target is to identify challenges and barriers in adoption, considerations on significant investment and local adjustment. This study aimed to assess the benefit of RWE as a tool for gathering information understanding and evaluating new technologies as candidates for public funding.

Twelve new technologies were assessed by 36 technology-assessors from the Ministry of Health (MOH) and the hospital, and were ranked using two scales: (i) the contribution of using RWE to benefit the assessment mechanism, and (ii) the perceptual “value” of the technology.

Analyzing the contribution of RWE to HTA, 67 percent of the technologies reviewed were recommended for adoption. Low RWE ranking correlated with a decision to reject technology adoption. The most important “value” among all assessors on RWE was “innovation”, and among hospital assessors, the parameter “local adjustment” was cardinal. MOH assessors highlighted RWE to increase knowledge of regulators to encourage adoption. The aspect relating to economic burden received only medium ranking.

RWE provides an essential contribution to HTA throughout all stages in the life of the technology, especially at early phases. It is an important methodology to gather evidence from the ongoing experience of clinicians, increasing awareness towards promising innovations and amplifying the confidence of decision-making. In this new era of HTA, new concepts arise on the horizon: multi-dimensional evaluations. Expert opinion and RWE are paving the way towards better understanding of beneficial technologies, even in the absence of formal published evidence.

The aim of this study was to describe patient level costing methods and develop a database of healthcare resource use and cost in patients with AHF receiving ventricular assist device (VAD) therapy.

Patient level micro-costing was used to identify documented activity in the years preceding and following VAD implantation, and preceding heart transplant for a cohort of seventy-seven consecutive patients listed for heart transplantation (2009–12). Clinician interviews verified activity, established time resource required for each activity, and added additional undocumented activities. Costs were sourced from the general ledger, salary, stock price, pharmacy formulary data, and from national medical benefits and prostheses lists. Linked administrative data analyses of activity external to the implanting institution, used National Weighted Activity Units (NWAU), 2014 efficient price, and admission complexity cost weights and were compared with micro-costed data for the implanting admission.

The database produced includes patient level activity and costs associated with the seventy-seven patients across thirteen resource areas including hospital activity external to the implanting center. The median cost of the implanting admission using linked administrative data was $246,839 (interquartile range [IQR] $246,839–$271,743), versus $270,716 (IQR $211,740–$378,482) for the institutional micro-costing (p = .08).

Linked administrative data provides a useful alternative for imputing costs external to the implanting center, and combined with institutional data can illuminate both the pathways to transplant referral and the hospital activity generated by patients experiencing the terminal phases of heart failure in the year before transplant, cf-VAD implant, or death.

Integration of ethics into health technology assessment (HTA) remains challenging for HTA practitioners. We conducted a systematic review on social and methodological issues related to ethical analysis in HTA. We examined: (1) reasons for integrating ethics (social needs); (2) obstacles to ethical integration; (3) concepts and processes deployed in ethical evaluation (more specifically value judgments) and critical analyses of formal experimentations of ethical evaluation in HTA.

Search criteria included “ethic,” “technology assessment,” and “HTA”. The literature search was done in Medline/Ovid, SCOPUS, CINAHL, PsycINFO, and the international HTA Database. Screening of citations, full-text screening, and data extraction were performed by two subgroups of two independent reviewers. Data extracted from articles were grouped into categories using a general inductive method.

A list of 1,646 citations remained after the removal of duplicates. Of these, 132 were fully reviewed, yielding 67 eligible articles for analysis. The social need most often reported was to inform policy decision making. The absence of shared standard models for ethical analysis was the obstacle to integration most often mentioned. Fairness and Equity and values embedded in Principlism were the values most often mentioned in relation to ethical evaluation.

Compared with the scientific experimental paradigm, there are no settled proceedings for ethics in HTA nor consensus on the role of ethical theory and ethical expertise hindering its integration. Our findings enable us to hypothesize that there exists interdependence between the three issues studied in this work and that value judgments could be their linking concept.

Latin American countries are taking important steps to expand and strengthen universal health coverage, and health technology assessment (HTA) has an increasingly prominent role in this process. Participation of all relevant stakeholders has become a priority in this effort. Key issues in this area were discussed during the 2017 Latin American Health Technology Assessment International (HTAi) Policy Forum.

The Forum included forty-one participants from Latin American HTA agencies; public, social security, and private insurance sectors; and the pharmaceutical and medical device industry. A background paper and presentations by invited experts and Forum members supported discussions. This study presents a summary of these discussions.

Stakeholder involvement in HTA remains inconsistently implemented in the region and few countries have established formal processes. Participants agreed that stakeholder involvement is key to improve the HTA process, but the form and timing of such improvements must be adapted to local contexts. The legitimization of both HTA and decision-making processes was identified as one of the main reasons to promote stakeholder involvement; but to be successful, the entire system of assessment and decision making must be properly staffed and organized, and certain basic conditions must be met, including transparency in the HTA process and a clear link between HTA and decision making.

Participants suggested a need for establishing clear rules of participation in HTA that would protect HTA producers and decision makers from potentially distorting external influences. Such rules and mechanisms could help foster trust and credibility among stakeholders, supporting actual involvement in HTA processes.

Real world evidence (RWE) is changing the overall data landscape and it has potential to advance the evaluation of real world performance (comparative effectiveness) of healthcare technologies by providing a greater quantity and quality of evidence. However, many are concerned that non-randomized RWE may be substituted for RCT data and thus increase uncertainty about effectiveness. This presentation sets out the opportunities and challenges for use of RWE by payers and HTA bodies to evaluate health care technologies.

Current uses, opportunities and challenges were identified via a literature review and interviews with nine experts. Interim results were discussed at the 2017 ICER Policy Summit, which brought together leaders from payer and life sciences organizations, to develop specific and actionable recommendations for the use of RWE in drug coverage and policy decision-making.

RWE is utilized for multiple purposes in the US and globally, including: aiding design of drug development pathways; supporting regulatory approval decisions; monitoring safety; and informing HTA assessments and payer coverage decisions. Some stakeholders see great value in RWE and want to make greater use of these data sources, including for: drug effectiveness evaluations (including supplementing network meta-analyses); innovative study designs (including pragmatic trials); real time patient monitoring; and adaptive pathways or coverage with evidence development. However, others see numerous challenges, many of which are related to the quality and reliability of RWE sources. Acceptance of an expanded future role for RWE is not universal, and payers and developers must work together to find mutually beneficial strategies for progressing the development and use of RWE.

Specific and actionable recommendations will be presented which highlight the role that each stakeholder group can play in overcoming the challenges and realizing the potential for RWE.