Increasing numbers of research papers about information retrieval for Health Technology Assessments (HTA), systematic reviews and other evidence syntheses are being published. It is time-consuming for information specialists to keep up-to-date with the latest developments in the field. To help searchers with this challenge, the Interest Group on Information Retrieval (IRG) of Health Technology Assessment International (HTAi) has compiled the best available research evidence on information retrieval aspects into an open-access web resource: Summarized Research in Information Retrieval for HTA (SuRe Info). The resource can be accessed at http://www.sure-info.org

The Sure Info team run topic-specific search strategies in selected relevant databases to identify information retrieval methods publications that fulfil the SuRe Info inclusion criteria. Eligible publications receive a structured abstract containing a brief critical appraisal. Key messages for search practice based on the appraisals and accepted best practice are summarized into topic-specific chapters.

SuRe Info currently offers fourteen chapters, with more in development. SuRe Info chapters fall into two categories: (i) chapters about general search methods that are used across all types of research, such as how to develop search strategies and the availability and use of search filters, and (ii) chapters summarizing the methods to use when searching for specific aspects of HTA (as defined in the European Network for HTA (EUnetHTA) HTA Core Model®), including searching for evidence on clinical effectiveness and safety, and identifying economic evaluations. References at the end of each chapter are linked to appraisals of publications that have been used to develop each chapter. Links to the full-text of the publications are provided when freely available. The SuRe Info chapters are reviewed every six months and updated if new evidence is identified or if resources change.

SuRe Info is a unique resource, identifying and summarizing current best research evidence on information retrieval aspects for HTA. It supports the timely uptake of potential efficiencies arising from new evidence that may be incorporated into the evidence identification processes of HTA organizations.

Budget Impact Analysis (BIA) is an integral element of a comprehensive Health Technology Assessment. Prior systematic reviews showed significant methodological dissimilarities in BIAs published from 2002 to 2015 (1,2). Aimed to improve the generalisability and transferability of outcomes, a guidance on methods was updated in 2014 (3). The objective of this study was to measure the adherence to Principles of Good Practice of BIAs published after the release of the updated guidelines.

Fifteen features representative of methodological appropriateness were identified from the Principles of Good Practice. A systematic review of the extant literature was conducted to identify BIAs published from January 2015 to December 2016. The adherence of each BIA to the Principles of Good Practice was defined by the number of representative characteristics taken into consideration as a percent of the total.

The full study protocol is available online: http://www.crd.york.ac.uk/PROSPERO/display_record.asp?ID=CRD42016049287

A sample of thirty-nine published BIAs were included in the analysis. The mean adherence of BIAs to the Principles of Good Practice was 69 percent (10.4 representative features out of 15). The highest adherence was 87 percent, while the lowest was 33 percent. The distribution of the scores was highly concentrated around the mean value, with thirty-four BIAs (87 percent of total sample) showing a level of adherence ≥ 60 percent. Only two BIAs reported an adherence < 50 percent (5 percent of total sample). Six representative features showed a level of adherence < 50 percent: off-label use (0 percent); uncertainty (26 percent); validation (33 percent); choice of computing framework (44 percent); eligible population (44 percent) and relevant features of healthcare system (49 percent).

Compared to the Principles of Good Practice, the BIAs included in the systematic review were overcomplicated and deterministic, ignoring the impact of possible scenarios relevant to budget holders. The research advocates a wider use of scenario planning as a tool to link uncertainty to the economic assessment of new interventions.

One of the challenges of large scale Health Technology Assessment (HTA) projects is managing the large volume of studies retrieved by the requisite comprehensive literature searches. At the scoping stage of the project, a pragmatic judgement needs to be made as to how sensitive the search strategy should be in order to find all the relevant papers without returning an overwhelming volume of irrelevant studies.

For this HTA (evaluating prognostic and predictive markers in rheumatoid arthritis), the research team already had prior knowledge of several key markers of interest, but wanted to ensure that no others had been missed. Advice from practising clinicians was obtained, but for additional validation, a broad scoping search was conducted for ‘rheumatoid arthritis’ using the sensitive Haynes filters for prognostic (1) and clinical prediction (2) studies. Unsurprisingly, this initial search retrieved too many studies for them all to be admitted to the full review; but once those dealing with known markers had been removed, a sample of the remaining records was loaded into a software visualization tool (3) to display “heat maps” of frequently occurring terms and phrases.

On this occasion, no additional markers were identified, however this provided reassurance that the advice obtained from clinicians was comprehensive, enabling the HTA team to proceed confidently with its evaluation of the selected markers.

Visualization offers an alternative means of exploring and interrogating large text archives, and has the potential to complement the role of traditional search methods in identifying literature for systematic reviews and health technology assessments. As processing power increases and more and more full-text papers become available open access, it may provide a solution to some of the limitations associated with comprehensive searching.

A predictive risk stratification tool (PRISM) to estimate a patient's risk of an emergency hospital admission in the following year was trialled in general practice in an area of the United Kingdom. PRISM's introduction coincided with a new incentive payment (‘QOF’) in the regional contract for family doctors to identify and manage the care of people at high risk of emergency hospital admission.

Alongside the trial, we carried out a complementary qualitative study of processes of change associated with PRISM's implementation. We aimed to describe how PRISM was understood, communicated, adopted, and used by practitioners, managers, local commissioners and policy makers. We gathered data through focus groups, interviews and questionnaires at three time points (baseline, mid-trial and end-trial). We analyzed data thematically, informed by Normalisation Process Theory (1).

All groups showed high awareness of PRISM, but raised concerns about whether it could identify patients not yet known, and about whether there were sufficient community-based services to respond to care needs identified. All practices reported using PRISM to fulfil their QOF targets, but after the QOF reporting period ended, only two practices continued to use it. Family doctors said PRISM changed their awareness of patients and focused them on targeting the highest-risk patients, though they were uncertain about the potential for positive impact on this group.

Though external factors supported its uptake in the short term, with a focus on the highest risk patients, PRISM did not become a sustained part of normal practice for primary care practitioners.

Europe's population is aging rapidly. Europeans aged 60 years and over formed only 16 percent of Europe's total population in 1980, but they now constitute 24 percent and will grow to 34 percent by 2050 (1). These challenges may be expected in the form of tighter labor markets, lower savings rates, and slower economic growth, as well as fiscal stress from lower earnings and tax revenue and increased pension and healthcare spending.

We may, however, overestimate the magnitude of these challenges and make poorer policy choices if we underestimate the productive contributions that older adults make to society. The literature measuring these productive contributions is regrettably underdeveloped, as is the literature on what policies can enhance such contributions.

This study focuses on the market and non-market productive contributions of older adults in Europe and addresses three questions:

1. (i) What is the nature and magnitude of the contributions made by older adults in Europe?

2. (ii) How do those contributions vary by country, time, and age, and how are they likely to evolve as the relative size of older cohorts swells?

3. (iii) How might changes in policy, institutions, behavior, and health likely influence the economic effects of population aging in Europe?

These research questions are explored using multivariate statistical tools to analyze rich data from multiple countries and waves of the Survey of Health, Ageing, and Retirement in Europe (SHARE) and the English Longitudinal Study of Ageing (ELSA).

Older adults in Europe make significant productive contributions in the form of labor force participation, caregiving for family and friends, and volunteering. These contributions vary widely by country and are correlated with age, health status, official retirement age, and population age structure.

The economic effects of population aging in Europe can be significantly moderated by effective retirement and healthcare policy.

Clinicians, epidemiologists, economists and other non-medical professions are involved in the production of Health Technology Assessment (HTA) reports. In addition, patients or representatives from patient organizations, as well as the general public, are increasingly involved. In 2015 a new proposal process of topic selection for HTA reports was initiated by German legislation with the aim of more closely involving patients and the general public. The new process has been implemented by the Institute for Quality and Efficiency in Health Care (IQWiG) under the project title “ThemenCheck Medizin” (Topic Check Medicine).

Since July 2016, patients and the general public can propose topics for HTA reports to IQWiG, excluding topics on the assessment of drugs. The proposals are submitted via the IQWiG website www.themencheck-medizin.iqwig.de (available only in German). No specific expertize is required for the submission of a topic. On the basis of the proposals a selection committee (patient representatives appointed by patient organizations legitimized in Germany) choose up to fifteen topics deemed suitable for HTA reports. In 2017 the committe will be extended to include members of the general public. IQWiG makes the final decision on the selection of up to five topics per year for HTA reports.

In the first proposal phase thirty proposals were submitted. The topics were allocated to the following categories: two for diagnostics; four for teeth; one for skin and hair; four for head and nerves; three for muscles, bones and joints; two for heart and circulation; four for cancer; two for children and adolescents; one for reproductive health and birth; and three for mental and emotional wellbeing. Four topics could not be considered because they either addressed the assessment of drugs or could not be transferred into an HTA question.

Through “ThemenCheck Medizin” patients and the general public in Germany can actively be involved in the process of collecting proposals for HTA reports as well as in topic prioritization. The prerequisites for successful implementation were in particular the easy-to-use online form and IQWiG's support of persons submitting topics in their formulation of an HTA question. The integration of a selection committee of patient representatives was shown to be constructive and productive.

Involving patients in defining the scope of health technology assessments is recognized as a valuable strategy that ensures that patient-relevant outcomes are considered.

The aim of this project was to pilot the focus group approach with cardiac patients in a European rapid assessment on the wearable cardioverter defibrillator, to improve involvement processes, to identify neglected outcomes, and to explore the potential of this methodology for eliciting the patients views on their disease and the wearable cardioverter defibrillator therapy.

An e-mail was sent to members of the nine regional associations of the Austrian Organization for Heart and Lung Transplant Patients to identify eligible participants. Guiding questions for the discussion were developed based upon a hand search of patient involvement initiatives and a review of appropriate literature. The 4-hour meeting was moderated by a patient support expert and recorded upon approval of participants. The anonymized transcript was analyzed using framework analysis.

Ten eligible patients responded, of which five men, aged between 55 and 73 years (mean 65 years) from Austria and Germany, were able to participate. All respondents experienced heart transplantation, and four had received an implantable cardioverter defibrillator before.

Participants reported that experiencing a sense of security was crucial to them and that they expected to do sports and live a life with few limitations, despite receiving a therapy. A wearable cardioverter defibrillator was hence not considered a long-term solution due to expected restrictions in living a ‘normal’ life.

Challenges included the identification of participants representative of this patient group and the complexity of patient histories.

The focus group approach proved useful in the wearable cardioverter defibrillator assessment. Gathered results informed the inclusion of outcomes relevant to the target group and revealed patients views on health-related quality of life. Lessons learned guide us in further improving patient involvement processes within the European Network for HTA (EUnetHTA) project.

The Health Technology Assessment International (HTAi) community recognises the importance of including patients’ views from published research and systematically obtaining input from patient organizations (POs). The HTAi's Patients and Public Involvement Subgroup has elaborated the Patient Group Submission Template for HTA (HTA Template) to facilitate the collection of evidence from patients via POs involvement. In 2015 AGENAS outlined a procedure to involve POs and tested the HTA Template within a Health Technology Assessment (HTA) report on dialysis.

The HTAi template was translated into Italian and adapted to the HTA report's specific information needs: to understand patients’ experiences with different dialysis modalities and any delivery problems at the regional level. Some questions were reformulated, others were cut and two different versions of the template were used. One was tailored to POs representatives and the other to individual patients selected with a purposive sampling procedure. We provided the HTA Template to POs appointed by an umbrella organization, Cittadinanzattiva, for their input and to identify other relevant POs to be involved. We identified a list of four associations, based on geographical location and typology of patients. Each POs representative completed the first template and administered the second one to, at least one patient for each five dialysis modalities. AGENAS staff provided support on a cascade basis POs collected and returned all templates.

Researchers performed a thematic analysis of the answers received and this input was introduced in the HTA report within the chapter on Patients Aspects. Patients’ experiences closely corresponded to the ones in our qualitative literature's systematic review. However, PO representatives templates revealed an important problem of equity in access to different dialysis modalities across regions that we highlighted in the HTA report's recommendations.

One of the template's limitations was related to self-administration. In some cases, a lack of familiarity with communicating one's views in writing may have affected the survey's informative power. This pilot also demonstrated the need for a more inclusive involvement procedure, as some important POs were not initially represented by the umbrella organization.

Priority setting in health care has been long recognized as an intrinsically complex and value-laden process. Yet, Health Technology Assessment (HTA) agencies presently employ value assessment frameworks that are ill-fitted to capture the range and diversity of stakeholder values, and thereby risk to compromise the legitimacy of their recommendations. We propose ‘evidence-informed deliberative processes’ as an alternative framework with the aim to enhance this legitimacy.

The framework is based on an integration of two increasingly popular and complementary frameworks for priority setting: multi-criteria decision analysis (MCDA) and accountability for reasonableness (A4R), Evidence-informed deliberative processes are, on the one hand, based on early, continued stakeholder deliberation to learn about the importance of relevant social values. On the other hand, they are based on rational decision-making – through evidence-informed evaluation of the identified values.

The framework has important implications for how HTA agencies should ideally organize their processes. Firstly, HTA agencies should take the responsibility to organize stakeholder involvement. Second, agencies are advised to integrate their assessment and appraisal phase, allowing for the timely collection of evidence on values that are considered relevant. Third, HTA agencies should subject their specification of decision-making criteria to public scrutiny. Fourth, agencies are advised to use a checklist of potentially relevant criteria, and to provide argumentation how each criterion affected the recommendation. Fifth, HTA agencies must publish their argumentation and install options for appeal.

Adopting ‘evidence-informed deliberative processes’ as a value assessment framework could be an important step forward for HTA agencies to optimize the legitimacy of their priority setting decisions. Agencies can incorporate elements according to their needs and affordances.

Patient Blood Management (PBM) describes a multidisciplinary approach that strives to optimize patients own blood and has been reported to reduce blood components utilization while achieving improved patient outcomes and reduced healthcare costs. The aim of this study was to evaluate the public health and economic impact related to the implementation of a nationwide PBM program in Portugal.

A decision-model comparing two scenarios (“current clinical practice” and “with PBM implementation”) was used to estimate the PBM impact including hospital-assisted patients from the following therapeutic areas: surgery (orthopaedic, cardiac and urologic), cardiology, oncology, gastrointestinal bleeding, abnormal uterine bleeding, hemodialysis, inflammatory bowel disease and pregnancy. Model inputs were obtained from Portuguese national health databases and literature review. The public health impact was measured in life years (LY) gained, disability-adjusted life years (DALY) reduction, hospital length of stay (LOS) and 30-day readmission rate reduction. The economic value was expressed in total and hospitalization costs savings.

A total of 384,704 patients were eligible for PBM strategies. We estimated that a one year nationwide PBM implementation could avoid 594 premature deaths, representing a gain of 1,481 LY and a reduction of 3,660 DALYs relative to the current paradigm. An 8.4 percent and 37.3 percent reduction in length of stay and 30-day readmission rate are expected, respectively. This corresponds to EUR70.4 million savings in hospitalization costs. Although PBM closer monitoring would imply additional physician visits and medicines use, leading to EUR24.1 million in additional expenditure, in this population the overall PBM implementation can generate net savings of more than EUR67.7 million per year (6.3 percent reduction of public expenditure).

The implementation of a nationwide PBM in Portugal may represent a great public health impact, especially in decreased mortality and disability, with substantial public expenditure reduction.

There is an increasing interest in divesting activities, giving rise to several initiatives both academic and governmental to identify and address one of the problems of health systems. In 2013 the Spanish Atlas of Variability in Clinical Practice (VPM) in collaboration with the Spanish Network of Health Technology Assessment (HTA) Agencies started a project with the purpose of providing elements to support a national strategy aimed at minimizing the use of doubtful procedures in the Spanish National Health System (1).

The identification, selection and definition of low added value procedures and the determination of the most cost-effective alternatives were carried out jointly between the AtlasVPM group and the HTA agencies of Andalusia (AETSA), Catalonia (AQUAS), Galicia (Avalia-t), Basque Country (Osteba), Madrid (UETS) and Aragon (IACS). The process consisted of the following phases: (i) Literature review; (ii) Preliminary list of procedures of dubious value; (iii) Analysis of feasibility and construction of the indicators (variability); and (iv) Empirical validation of the defined indicators. Different lists and sources of evidence were used to identify the procedures and evidence that support their low-value.

The synthesis of the evidence gave rise to an initial list of fifty-nine procedures of doubtful value that could be classified as: obsolete or outdated procedures in comparison to more effective / cost-effective alternatives (n = 31), procedures of doubtful value when used outside their main indication (n = 17) and procedures for which the evidence around effectiveness was still insufficient (n = 11). With the advice of clinical experts and coders, the original list was reduced to seventeen procedures and after some adjustments to thirteen.

Identifying procedures of low-added value is a complex task and is context dependent. Literature could be useful to identify a preliminary list but the analysis of the clinical practice, its variability and reasons that justify it are required to determine which procedures are good candidates for disinvestment.

Malaria remains the number one cause of morbidity and mortality in Ghana. Since 1961, several malaria control strategies have been adopted, some of which were discontinued due to funding. In spite of the numerous malaria control strategies in place, its prevalence continues to rise. Priority setting using economic evidence has been proven to ensure efficient use of resources in a cost-effective manner (1). This study, therefore, sought to examine economic evaluation studies conducted on malaria in Ghana and their influence on malaria control policies.

A systematic search was conducted in databases including Medline and Embase to identify relevant Malaria economic evaluation studies conducted in Ghana up to December 2016. Malaria control policies formulated in Ghana over the years were also reviewed. The economic studies were examined alongside the policies to establish their influence on them.

A total of eight studies were identified, all of which were conducted in response to a global directive on malaria control and funded by international agencies. All studies were cost-effective; five evaluating preventive measures and the remaining evaluating treatment. The studies used different methodological approaches, rendering the comparison between alternatives impossible.

Most malaria control initiatives are funded by international agencies, hence its abandonment when funding ceases. Although the majority of economic studies addressed some of these policies, none of them directly influenced their adoption. These policies were rather influenced by global malaria control initiatives. Also, malaria chemoprophylaxis; demonstrated as cost-effective by three studies, is not on the Ghana malaria control policy (2,3).

To ensure sustainability of malaria control strategies and subsequently reduce its prevalence, Ghana must invest financially into economic analysis for formulating and implementation of these policies. Also, the use of economic evidence by policy makers can be promoted, should researchers adopt a methodological guideline for its conduct that ensures comparability of results.

The Da Vinci surgical system is classified as a type “A” medical device in China; the procurement plan of which is regulated by the National Health and Family Planning Commission (NHFP). Between 2010 to 2015, there were thirty-four Da Vinci surgical robots purchased, and installed in thirty tertiary public hospitals across the country. In order to generate context-specific evidence and support further capital funding decisions, the NHFP commissioned a Health Technology Assessment (HTA) of Da Vinci surgical robots, with a focus on real use of the technology in those tertiary public hospitals.

Nine hospitals were selected to collect real word data between 2013 to 2015. Using a cross-sectional survey, data of all robotic surgical cases were collected and described. The unit costs of the robotic surgery were estimated from activity based costing. We also collected cases of prostatectomy (427 versus 421) and hysterectomy (247 versus 105) using the robotic system and laparoscope respectively, and then compared hospital fees and effectiveness during hospitalization. Simulation of the budget impact on health insurance in Shanghai City over the next 5 years was also performed.

A full HTA was conducted based on real data from nine public hospitals in the central and eastern region. Based on a systematic review methodology, we appraised evidence on safety, effectiveness and cost-effectiveness of the Da Vinci surgical robot. Data on technology use, clinical management, and pricing and payment were collected through a cross-sectional survey and interviews of hospital managers, surgeons and nurses. We designed a cohort study on cost-effectiveness of Da Vinci-assisted prostatectomy and hysterectomy, comparing Da Vinci-assisted and laparoscopic prostatectomy (427 vs 421) and Da Vinci-assisted and laparoscopic hysterectomy (247 versus 105). Ethics and inequity issues were discussed based on patient interviews. A budget impact analysis was performed based on scenario mapping of promoting Da Vinci-assisted prostatectomy in Shanghai City over a 5-year timeline.

Due to a lack of evidence on long-term clinical effectiveness and high impact on public finances, the Da Vinci robotic robot should not be procured in large numbers in China. For equipment purchasing the government should strengthen regulations and require the public hospitals to collect more evidence.

South Africa is in the process of providing comprehensive health insurance to all its citizens, thus paving the pathway for Health Technology Assessment (HTA) to play a significant role in provision of safe and effective healthcare. The National Department of Health (DoH) has a published framework and Health Technology Act and strategies since the 1990s to improve health outcomes, and service and delivery of care. The purpose of this study is to explore challenges faced in the implementation of the framework and policies.

The study will be based on review and analysis of health technology policies and legislations introduced in South Africa since the 1990s. These documents are available from the DoH archive. The review from this grey literature was supplemented by information collected from a self-completion questionnaire, which was distributed to key stakeholders. Respondents were identified by direct contact with ministries of health and professional bodies, and included health professionals from the public and private healthcare sector, for example, practitioners, experts from hospitals, and industry representatives. The questionnaire addressed issues pertaining to decision making regarding health service delivery and the status of HTA in the country.

The framework lays out the strategy to facilitate appropriate utilization of health technologies and includes among others, an HTA section. Fragmented use of HTA or parts thereof has been observed in the public and private health care sector. Furthermore, the respondents pointed out that decisions on health technology can be political, institutional or professionally driven whereas they all agreed that a formal and institutional implementation of HTA would improve healthcare service.

The goal to achieve universal health care provides an excellent window of opportunity for formal use of HTA in policy- and decision-making. However, (i) the inadequate number of trained professionals and education and training opportunities (ii) lack of awareness and understanding of the principles of HTA and its impact on the improvement of health care are among the many challenges faced by the system. It has also been observed that national and regional champions can act as change agents and would have a snowball effect.

In 2007, the National Healthcare Institute (ZIN) initiated conditional financing (CF) of expensive hospital drugs as an example of conditional reimbursement schemes (CRS). CF is a 4-year procedure encompassing initial HTA assessment (T = 0) followed by additional data collection via outcomes research (separately assessing appropriate use & cost-effectiveness in routine practice) and re-assessment (T = 4). This study aims to review performance and experiences with CF in the Netherlands to date.

All dossiers for drugs that underwent the full CF procedure were reviewed. Using a standardized data abstraction form, two researchers independently extracted information on procedural, methodological and decision-making aspects (that is, related to implemented outcomes research, evidence assessment and appraisal). A scoring algorithm was used to assess all three aspects.

Fourty-seven candidates were nominated for CF; fourty-four underwent T = 0 assessments and eleven T = 4 assessments. The procedure extended beyond 4 years for 10/11 candidates. For the eleven candidates, applicants clearly defined study designs and data collection methods for outcomes research proposals addressing 16/22 research questions posed in T = 0 reports. ZIN provided discussion points and recommendations regarding research proposals for 18/22 research questions. Applicants implemented recommendations fully in 8/22 cases and partially in 12/22. Sufficient data was available at T = 4 to answer 15/22 research questions posed at T = 0. However, discussion points remained regarding implemented outcomes research for all eleven candidates at T = 4. ZIN advised to continue reimbursement for nine candidates and to stop reimbursement for two. For six of the nine candidates, reimbursement was continued on the basis of conditions relating to additional evidence generation beyond T = 4.

Theoretically, CF provides a valuable option for enabling quick but conditional access to medicines in the Netherlands. However, procedural, methodological and decision-making considerations related to scheme design and implementation may affect its value in decision-making practice.

Whereas Health Technology Assessments (HTAs) by the National Institute for Health and Care Excellence (NICE) rely heavily on cost utility analysis, HTAs by the German Institute for Quality and Efficiency in Health Care (IQWiG) and the Federal Joint Committee (GBA) focus on an assessment of comparative effectiveness, rejecting a cost per quality-adjusted life year benchmark. The present study aimed to explore the differential impact of methodological choices by NICE and IQWiG/GBA on HTA outcomes.

We extracted data from all GBA decisions between January 2011 (when early benefit assessments were implemented) and April 2015 (cut-off date for the present study), as well as all single technology appraisals (STAs) by NICE published during the same period. We compared early benefit assessment results by IQWiG/GBA and by NICE overall, and by additional criteria including therapeutic area, clinical and incremental cost effectiveness, and patient-relevant endpoints.

During the study period, NICE issued guidance for 88 technologies (with 125 subgroups). GBA completed 105 appraisals (with 226 subgroups). We identified thirty-seven matched condition-intervention pairs; of these, twenty-four were evaluated differently by NICE and GBA. NICE recommended twenty-nine of thirty-seven interventions (78 percent), whereas GBA confirmed additional benefit for 21/37 only (57 percent; p< .05, two-tailed chi-square test). By therapeutic area, NICE was more likely to evaluate interventions for metabolic and cardiovascular disorders favorably, whereas IQWiG/GBA appraisals were more favorable for treatments of hematological and oncological diseases. Results including all HTAs were consistent with those for matched pairs.

Our results suggest that, overall, NICE tends to evaluate new interventions more favorably than IQWiG/GBA. However, our analysis revealed conspicuous differences by therapeutic area. The results are consistent with the hypothesis that different methodological choices may lead to systematic differences in decision making. It seems plausible that the observed differences reflect, at least in part, differences in underlying value judgments.

Evaluation of “value for money” is an important component of Health Technology Assessments (HTAs). It is often conceptualized as “cost effectiveness” or cost per (quality-adjusted) life year gained. Whether used in isolation or alongside further drivers of social value (such as priority for younger or more severely impaired patient groups, or for access to effective treatment, even if costly), for example within a multi-criteria decision analysis framework, any reference “value of a statistical life year” (VSLY) should be supported by empirical data capturing the preferences of the population(s) in question. Here we report results based on a systematic review of relevant European economic studies, which were published during the last two decades, that is, from 1995 to 2015.

Our literature search (using the EconBiz and EconLit databases, supplemented by an analysis of relevant reviews) identified fourty-one European studies providing original data, yielding a total of fourty-eight average estimates for the value of a statistical life (VSL, or fatality prevented). We classified studies by methodology, for example, revealed preference (RP) or stated preference (contingent valuation, CV; discrete choice experiment, DCE) approach. We transformed VSL estimates into VSLY expressed in year 2014 Euros, using the life expectancy of the populations studied, a real discount rate of 3 percent, the national Consumer Price Index (CPI) for inflating, and purchasing power parities for currency conversion. We calculated confidence intervals by means of nonparametric bootstrapping.

The median VSLY was EUR158,000 (for RP studies, EUR218,000; DCE, EUR188,000; CV, EUR143,000); we did not identify studies using the human capital approach. Our VSLY estimates showed large heterogeneity, both by methodology and regional origin; thus the differences that we observed did not reach statistical significance.

Our results suggest that the empirical willingness-to-pay for a statistical life year might be substantially higher than benchmarks currently used by the international HTA community.

Program budgeting marginal analysis (PBMA) accommodates economic analysis, multi-stakeholder inputs, values, needs and perspectives within one framework in order to determine optimal use of available resources to deliver the highest ‘health value’. Two pilot PBMA projects in two different services were conceived and completed in a Welsh Health Board (HB) as ‘proof of concept’ methodology for robust prioritization decisons and for improving quality of patient care, outcomes and experience. The pilots were essential to enable development of a ‘bespoke’ PBMA process for the HB to implement.

The PBMA methods were based on methods and criteria for successful PBMAs reported in the literature. Project teams and stakeholder communities supported the PBMAs which were executed over a 12 -18 month period between 2013–15. Group decision support methods were used to facilitate meetings and decision making. Formal interviews with project team members and informal feedback informed development of the final PBMA framework.

Identifying the costs and resources attributable to services and those that could be moved around services was challenging. Evidence of outcomes and ‘health value’ was more easily available. One PBMA pilot recommended that some modest service reorganization and quality improvement could be made within budget but no substantial improvement/decommissioning could be undertaken. The other pilot agreed a disinvestment decision on the basis of evidence and reallocated the resources to a higher value service. The HB commissioning team found the information from the PBMA ‘journey’ as useful as the recommendations. A PBMA framework for the HB was devised.

A ‘Prudent PBMA’ framework trimmed back to the critical essentials enables success criteria to be met. PBMA is to be adopted as a ‘way of working’ to operationalize resource reallocation and disinvestment in the ‘real world’ of Welsh healthcare commissioning.

Some countries have distinct pathways for drugs for rare diseases (DRDs) (1). In May 2014, the Canadian Agency for Technologies in Health (CADTH) rejected the option of a separate review pathway for DRDs, reiterating that “pharmacoeconomic analyses are critical for all types of drugs”. While the gap between positive recommendations for common and rare drugs may have narrowed, the rejection for DRDs is still proportionally much higher (2). The default has been to provincially negotiate drug access, for patient populations, subgroups or individuals. Still not wishing to create a separate pathway, in March 2016, CADTH produced a revised evaluation framework for “uncertain clinical and pharmacoeconomic evidence” and other considerations representing “significant unmet need” including rarity and difficulty to study because of small patient population”(3). This study analyzes recommendations for DRDs following the two CADTH revisions.

Methods used were: synthesis of previously conducted analyses of CADTH recommendations for rare and non-rare drugs, primary comparative analysis of CADTH recommendations for DRDs from 2004 to 2016, and qualitative analysis of two drugs submitted for both rare and non-rare conditions: everolimus (breast cancer, pancreatic neuroendocrine tumours, and tuberous sclerosis complex) and ibrutinib (chronic lymphocytic leukemia, small lymphocytic lymphoma, and Waldenström's Macroglobulinemia).

Previous analyses found that DRDs received more negative recommendations than did non-rare drugs; both clinical and economic evidence were differentiating factors. The primary analysis provided an additional understanding of reasons for negative recommendations. There is low consistency across assessments and across the two CADTH review committees. The case studies illustrated the challenges for DRDs to overcome barriers of cost-effectiveness and certainty of clinical evidence, even with the revised framework.

This research challenges the premise that Health Technology Assessment for all drugs can result in fair and equitable recommendations for DRDs. Moreover, assessments based on “significant unmet need” do not appear to provide consistent or equitable guidelines for addressing the issues specific to rare diseases.

Several health technologies used for therapy can also be used for health enhancement. Drugs stimulating cognitive abilities are but one example. Health Technology Assessment (HTA) has not been developed for assessing enhancements. This raises the question of how HTA should address the blurred distinction between therapy and enhancement. Should we (i) carve out a distinction between therapy and enhancement and limit HTA to therapy, (ii) use HTA for both therapy and enhancement (with some modifications), or (iii) should we develop a separate health enhancement assessment (HEA)?

A literature search of the medical, philosophical, and bioethical literature was conducted for debates, arguments, and suggested solutions to the issue of therapy versus enhancement.

The same improvement in health may be therapeutic in one patient, but an enhancement in another. Moreover, both therapy and enhancement share the same goal: increased health and wellbeing. A wide range of arguments try to establish a difference between therapy and enhancement. They refer to naturalness, rehabilitation, normality, species-typical functioning/potential, disease, sustainability, and responsibility. On closer scrutiny few of these arguments do the job in bolstering the therapy-enhancement distinction. We already use a wide range of means to extend human abilities. Moreover, the therapy-enhancement distinction raises a wide range of ethical issues that are relevant for the assessment of a number of emerging health technologies.

Existing HTA methodology can address a wide range of non-therapeutic health enhancements. However, a series of broader issues related to the goal of health care and responsibility for altering human evolution may not be addressed within traditional HTA frameworks. Specific HEAs may therefore be helpful.