Many countries have a national antimicrobial resistance strategy. In Australia, primary care is especially important because this setting encompasses a high proportion of antibiotic use. While antibiotic use decreased during the 1990s, it began to increase again in the mid-2000s. In response to this, in 2009 NPS MedicineWise implemented a series of nationwide educational interventions for consumers, family physicians (general practitioners), and community pharmacies that aimed to reduce excessive antibiotic use.

For consumers a social marketing approach was used, including strategies that leveraged collectivism, nudge theory, celebrity endorsement, and co-creation. Channels included social, print, radio, and other media as well as practice waiting rooms and pharmacies. For health professionals, interventions included face-to-face education, audits, comparative prescribing feedback, case studies, and point-of-care materials. Surveys of consumers and family physicians were conducted periodically to evaluate changes in knowledge and behavior. National Pharmaceutical Benefits Scheme claims data were analyzed using a Bayesian structural time-series model to estimate the cumulative effect of interventions by comparing the observed and expected monthly dispensing volumes if the interventions had not occurred.

The consumer survey results indicated that more people were aware of antibiotic resistance (seventy-four percent in 2017 versus seventy percent in 2014), with the minority requesting or expecting antibiotics for upper respiratory tract infections (URTIs) (twenty-two percent in 2017). People underestimated the usual duration of symptoms for URTIs and were more inclined to expect antibiotics beyond that timeframe. Compared with non-participants, family physicians who participated in the program reported more frequent discussions about hand hygiene (ninety percent versus eighty-two percent) and proper use of antibiotics with patients (ninety-five percent versus eighty-eight percent). Between 2009 and 2015 there was an estimated fourteen percent reduction in prescriptions dispensed to concessional patients for antibiotics commonly prescribed for URTIs.

Family physicians and consumers have responded positively to national programs. Sustaining and building on these improvements will require continued education and further innovation.

The use of transcatheter aortic valve implantation (TAVI) is evolving. Our Cardiovascular Evaluation Unit is implementing a comprehensive approach to inform decision-makers on optimal use of TAVI, including the development of quality standards. We are implementing a multifaceted evaluation framework in collaboration with clinical stakeholders.

Our unit has carried out a continuous field evaluation in collaboration with the clinical teams at all six TAVI centers in Québec for the past four years (1 April 2013–31 March 2017), with regular feedback to the teams and sharing of results with each individual center. Hospital documentation was reviewed according to established national quality indicator definitions. Field evaluation data were combined with the results of systematic literature review to establish provincial standards for practice, through a deliberation process by an interdisciplinary committee of clinical experts from each center. Systematic surveillance of the literature is ongoing.

In the period 2013–2017, use of TAVI in Québec was limited to very elderly patients with significant comorbidities at high risk of operative mortality. We observed improvements in both processes of care (e.g. documentation of risk scores) and clinical outcomes (e.g. 30-day and 1-year mortality) over time. Our consensus standards recognize the potential value of TAVI for patients at moderate operative risk, identify uncertainties and recommend best practices for patient evaluation and clinical decision-making about choice of treatment.

A comprehensive, long-term evaluation process of TAVI with feedback to centers is associated with improvements in processes of care and outcomes. In the present context of expanding clinical indications, we will continue to evaluate patient selection, processes and outcomes according to the newly-established provincial quality standards. This iterative approach facilitates continued evidence generation and decision-making for optimal use of an evolving intervention. We acknowledge the contribution of the members of the expert clinical committee.

During 2014, Roche tested whether the EUnetHTA HTA Core Model© was a useful, exhaustive and relevant value framework to promote efficiencies in scoping, storing and sharing health technology assessment (HTA) evidence within a pharmaceutical company. The conclusion was positive and Roche decided to build a cloud based information technology (IT) platform to store all relevant HTA evidence to support global and regional market access activities, tagged with metadata according to the HTA Core Model©., The platform should be user-friendly and promote efficiencies and knowledge sharing across the organization. Eventually this platform may also be used by external stakeholders to access relevant HTA evidence.

In order to better equip global functions, regions and affiliates in a major pharmaceutical company with user-friendly and fast access to product-relevant HTA and payer evidence as well as access evidence plans, an easy-to-use IT-based platform was needed. The platform, internally called #TAg, is a central repository of information to support market access activities and promote collaboration between Affiliate, Region and Global teams. The platform uses metadata to label all types of evidence and uses the HTA Core Model© domains to categorize the evidence.

The platform #TAg was developed throughout 2016/2017 and officially launched on 1 October 2017. Within the first 30 days, the platform has been readily accepted by affiliates, regions and global functions through significant use uptake as measured by user registration and download activities. In addition, #TAg was used successfully in a pilot project for a submission to an external HTA body.

A complete knowledge management system for HTA evidence is important for driving efficiency in scoping, storing and disseminating access evidence information within a pharmaceutical company. #TAg has so far proved a good start on such a system with further development expected in the coming years.

The factors associated with opioid poisoning death are poorly understood. We performed a retrospective autopsy study of decedents (a term used for people who are deceased) of opioid poisoning in Wales in 2015. Using anonymized linked data, we describe demographic characteristics, patterns of emergency service utilization, and clinical presentation prior to death.

Decedents of opioid poisoning in Wales in 2015 were identified from the Office of National Statistics (ONS) mortality dataset. Records were linked with the Emergency Department Dataset (EDDS) by the National Welsh Informatics Service (NWIS); and held in the Secure Anonymized Information Linkage (SAIL) databank. The data were accessed and analyzed in the SAIL gateway.

Age at death ranged from eighteen to seventy-eight years, with a mean age of forty-two years. Average male age was forty-one years and average female age was forty-four and a half years. Seventy-three percent of decedents were men (n = 228/312). Eight-seven percent of decedents (n = 281/312) attended the emergency department in the three years prior to death. In total 2081 attendances were made, forty-one percent of which involved conveyance by ambulance. Attendances per individual ranged from one to 114, with over half of decedents attending more than three times. Diagnostic codes were mostly missing or non-specific, with only seven and a half percent of attendances representing eighty-two decedents, coded as drug related. Treatment codes were also mostly missing or non-specific, with sixteen percent of attendances representing 148 attendees attributed a treatment code. Thirty-nine percent of attendances (n = 822) ended in treatment and discharge, whilst twenty-seven percent (n = 562) led to hospital admission.

Matching previously published data, we found that fatal opioid poisoning is preceded by a period of high emergency health service utilization. On average decedents were in their fifth decade and more likely to be male than female. Attendances varied widely, with men less likely to attend than women.

More and more software programs, including those with medical device status, are coming into the market that aim to facilitate management of diabetic patients. In France, their coverage requires a positive opinion from the French National Health Agency (HAS) dedicated committee. To understand the utility of these products for patients, real-life experiments are in progress. Since the evaluation principles are similar for all medical devices, it was important to find out with this first connected software if specific methods or evaluation criteria are necessary.

After obtaining CE marking, the manufacturer submitted a dossier to HAS outlining the clinical data and technical performance of the software. HAS assessed the dossier and the opinion of stakeholders (i.e. professionals and patient associations) in order to determine the actual clinical benefit of this software. At the same time, HAS set technical features in particular to secure patient data and limit access to only those involved in telemonitoring. Terms of prescription and use of this connected software had also been defined.

Two feasibility studies and one randomized controlled trial were analyzed. Specific clinical data demonstrated that the risk-benefit balance was positive in type 1 diabetic patients.

In addition to the need to keep personal data confidential and to integrate the technology in the organization of healthcare, this assessment shows that randomized clinical trials are feasible and necessary to demonstrate the clinical benefit of connected software; however, specificities exist regarding data collection methods and the scope of healthcare organization that should be taken into account. A specific guide to connected medical devices for industry has been developed by HAS to help them build their application dossier for reimbursement. The second step for HAS is to develop guidelines on the specificities of the assessment of these connected devices.

Cochlear implantation (CI) is a standard technology for the management of children with sensorineural hearing loss. In the Republic of Kazakhstan (RK), CI was introduced in 2007. In 2012, a report for the Ministry of Health (MoH) of the RK considered the effectiveness of CI, age of implantation, use of clinical protocols, and availability of audiological screening and rehabilitation services. We assessed the influence of the report findings on the provision of medical care in the RK for children with sensorineural hearing loss.

Information was collected in a survey of all RK health regions on issues related to CI, audiological screening and rehabilitation. Administrative data relevant to the provision of CI in the RK were obtained from the MoH. Data obtained were compared with those available for preparation of the 2012 report.

The proportion of medical organizations with equipment to provide audiological screening had improved, from 29 percent in 2012 to 90 percent in 2018. The proportion of children under two receiving CI increased from 12 percent to 36 percent, while that for children over five years decreased from 48 percent to 17 percent. A clinical protocol for CI in children was developed by a center in the MoH. Progress with post-CI rehabilitation of children was limited by a lack of specialists in the health regions. The proportion of school-age children with implants who have attended general schools remains low.

The findings of the HTA report had a positive influence on availability of screening services and a protocol for CI. The average age of children receiving an implant has decreased, though it is still higher than in other countries. The need for improvements in post-CI rehabilitation and placement of children with implants in general schools is recognized but these await further resources.

The US Agency for Healthcare Research and Quality (AHRQ) Evidence-based Practice Center (EPC) program sponsors the development of systematic reviews to inform clinical policy and practice. The EPC program sought to better understand how health systems identify and use this evidence.

Representatives from eleven EPCs, the EPC Scientific Resource Center, and AHRQ developed a semi-structured interview script to query a diverse group of nine Key Informants (KIs) involved in health system quality, safety and process improvement about how they identify and use evidence. Interviews were transcribed and qualitatively summarized into key themes.

All KIs reported that their organizations have either centralized quality, safety, and process improvement functions within their system, or they have partnerships with other organizations to conduct this work. There was variation in how evidence was identified, with larger health systems having medical librarians and central bureaus to gather and disseminate information and smaller systems having local chief medical officers or individual clinicians do this work. KIs generally prefer guidelines, especially those with treatment algorithms, because they are actionable. They like systematic reviews because they efficiently condense study results and reconcile conflicting data. They prefer information from systematic reviews to be presented as short digestible summaries with the full report available on demand. KIs preferred systematic reviews from reputable entities and those without commercial bias. Some of the challenges KIs reported include how to resolve conflicting evidence, the generalizability of evidence to local needs, determining whether the evidence is up-to-date, and the length of time required to generate reviews. The topics of greatest interest included predictive analytics, high-value care, advance care planning, and care coordination. To increase awareness of AHRQ EPC reviews, KIs suggest alerting people at multiple levels in a health-system when new evidence reports are available and making reports easier to find in common search engines.

Systematic reviews are valued by health system leaders. To be most useful they should be easy to locate and available in different formats targeted to the needs of different audiences.

In Brazil, cardiovascular disease accounted for twenty-eight percent of deaths in 2013 with an estimated prevalence of five to eight in adults over forty years of age. Health care costs have quadrupled in the last decade, reaching USD 125 billion in 2013, of which forty-four percent were paid by the public system. The objective of this study was to estimate the direct costs associated with inpatient stay for myocardial infarction in a public teaching hospital from the perspective of the service provider.

We used a bottom up microcosting methodology for collecting data from computerized hospital records and patients' hospital bills. The costs included salaries of health professionals, medications, consumables, laboratory and diagnostic tests performed during hospitalization and maintenance expenses. Mean, standard-deviation, median and total costs were calculated. The costs were presented as mean and median values in Brazilian currency and converted to US dollars by the exchange rate.

A total of eighty-one patients were included in this study. The mean population age was 60 ± 10.6 years, the follow-up period were 107 ± 2.6 months; fifty-four percent were male, eighty-four percent had hypertension, thirty-six percent had diabetes, and twelve percent had previous cerebrovascular accident. During follow-up, there were 101 hospitalizations for myocardial infarction, of which fifty-seven with intensive care unit (ICU) days. The total cost with hospitalizations was USD 177,288, of which fifty-two percent were the health professionals’ costs. The average cost for hospitalization was USD 1,755 (median USD 1,221). However, the average reimbursement paid by the public system was USD 1,188 (median USD 1,044) per hospitalization, generating a deficit of thirty-two percent for the hospital.

These results may indicate the necessity of reviewing the public reimbursement policies for the service providers in Brazil. Besides that, these data may also serve as input for the economic evaluation in coronary artery disease.

In the United Kingdom (UK), 23,000 people annually are diagnosed with facial palsy (acute onset facial paralysis). For nearly one third this will result in a permanent disability, including in some the inability to smile. In addition to initial pharmacological therapy, guidelines recommend tailored facial exercise (TFE) therapy repeated every day. However, not all patients are currently able to access such specialist physical therapy. ‘Smart specs’ (using miniaturized sensors in the frames to measure facial movement) are currently being developed. Linked to a smartphone, these could allow people to practice TFEs discreetly, provide immediate feedback, and supply data on outcomes to the patient and their clinician.

Modelling of introduction of Facial Remote Activity Monitoring Eyewear (FRAME) into treatment pathways for patients with facial palsy. This included: (i) review on effectiveness of TFE therapy; (ii) national surveys (medical staff, facial therapy specialists and patients) to gather data on access to TFE therapy; (iii) Delphi Exercise to identify consensus on key outcome measures; and, (iv) economic modelling to estimate cost-effectiveness and determine a range of acceptable costs for the technology. In parallel, research to examine target markets to inform product development, and production of integral commercialization plan.

Searches short-listed ten studies to add to the three included in the 2011 Cochrane review. Surveys indicate approximately thirteen percent of eligible UK patients access personalized TFE therapy. Estimated annual expenditure on hospital treatments for facial palsy patients is currently >GBP 80 million (>USD 106 million) compared with <GBP 0.5 million (<USD 0.66 million) on TFE therapy. Patients with permanent defects can suffer a loss of up to two quality-adjusted life years (QALYs).

Findings from this study, particularly in relation to costs and benefits, will inform the design of a subsequent randomized controlled trial. A novel wearable technology could make a major difference to people's lives, as well as generating potential efficiencies for healthcare.

Patient advocates need to process vast amounts of information to accurately and effectively represent heterogeneous patient groups and make meaningful contributions to HTA decisions. Although a wealth of data is available from a variety of sources, it is not often curated in user-friendly ways. Patient representatives have frequently requested tailored resources that allow them to mine the existing literature in preparation for their engagements. Developing such resources constitutes a complex challenge that requires contributions and scrutiny from multiple stakeholders.

We previously developed the Continuous Innovation Indicators™ (CII), an evidence-based tool to assess treatments for twelve solid tumors (freely available at www.scoringprogress.com). The foundation of the CII is a rigorous assessment of published evidence for increased overall survival. Based on feedback from patient advocates, we are expanding the framework to include information on adverse events and other patient-centered outcomes for selected prototype indications.

We present a novel, flexible framework that combines evidence of efficacy with published results on other outcomes that matter to patients. Menus and outputs are designed to facilitate dialogue between advocates, clinicians, and HTA professionals. By allowing the user to adjust settings based on known heterogeneity among subpopulations, the tool's output can be used to inform discussions about the value of new interventions for defined patient segments.

Patient representatives must frequently identify knowledge gaps in the literature before their HTA engagements and leverage this information to conduct surveys among their constituents. Our new patient advocate decision aid can support this process and facilitate a better understanding of the value of new innovations for diverse subgroups. A better definition of target populations will help to achieve balance between patient access and budget impact of new treatments. We seek feedback on our prototype from all stakeholders to further improve and maximize utility of this tool.

In a recently published review of supplementary search methods, we proposed that researchers could usefully record time taken to search and present outcome values in similar way to existing studies, to facilitate generalisability of outcomes, where appropriate. We also discuss the idea of linking literature search effectiveness to study value. In this vignette, we discuss which outcomes we believe are important to measure and why. We discuss this in the context of the review of supplementary search methods and using a recently submitted evaluation of contacting study authors for context.

In a recently completed systematic review, we contacted eighty-two study authors to ask three questions. We aimed to measure the following outcomes when contacting study authors: Effectiveness - determined as number of contacts compared to number of replies; Efficiency - i) time to make contact and ii) time between contact and reply. We determined this in hours, minutes and seconds, in line with other studies; Cost - determined by comparing the efficiency of contacting authors with the effectiveness; and Value - determined by reading and comparing the published studies with the replies received to see if any unique data were identified.

Effectiveness: thirty-eight answers were received from eighty-two possible contacts. Efficiency: In total, author contact took six hours, fifty-four minutes and twenty-five seconds across thirty-nine weeks. Replies were received across zero to thirty-nine days (median fourteen days). Cost: Cost for staff time was GBP 80.33 (EUR 91.20) or GBP 2.11 (EUR 2.40) per e-mail reply received. Value: We were able to identify value in author replies for each of the questions asked.

In a recently published review of supplementary search methods, and a linked evaluation of the effectiveness of contacting study authors, we suggest outcomes that should be measured to determine effectiveness of literature search methods. We conclude that measuring these outcomes demonstrate both effectiveness and value.

In Brazil, the National Pharmaceutical Assistance Policy was published in 2004. Pharmaceutical assistance at the primary health care level in Brazil is understood as a broad set of activities comprising regulation, planning, distribution and dispensation of essential medicines in primary health care facilities. Considering governance capacity as a key requirement for the success of a public health policy, this article aims to demonstrate the adaptation and operationalization processes of using a national survey database for a national evaluation of the pharmaceutical assistance governance in public primary health care in Brazil.

This is a systematic study of an evaluation model on pharmaceutical assistance governance at the municipal level, and of the data collection instruments and databases used in the recent National Survey on Access, Use and Promotion of Rational Use of Medicines, in addition to the preparation of indicators protocol validated for application throughout the country. The study steps were as follows: selection of data from the survey and their adaptation to evaluation indicators nationwide; validation of an evaluation matrix adapted in a workshop with actors in the field; database construction; data analysis; and, issue of value judgment.

The adaptation of the evaluation matrix caused seventeen indicators to be reformulated. In six of these indicators, the changes referred only to the data source. As the recommended measures could not be implemented due to lack of information, sixteen indicators were excluded from the original protocol. Ultimately, the proposed protocol comprised thirty indicators presented in three dimensions (organizational, operational and sustainability).

The methodology enabled the redesign of the evaluation matrix according to the specific national characteristics by crossing the data provided by the reference survey and evaluation model. The participatory process, the use of data from all actors involved in pharmaceutical assistance at the municipal level, and the use of the principles of the national health policy as the basis for selection and construction of a fitted evaluation protocol are important strengths of the new protocol proposed. The absence of international studies on evaluations using the same model is a major weakness.

For patients with bifascicular block and syncope of unknown origin, different American Heart Association guidelines give Class 2A recommendations for two treatments: the implantable loop recorder (ILR) and empiric pacemaker insertion (PM). Equipoise reflected in guidelines may contribute to uncertainty in management and inefficient resource use. The objective of this analysis is to determine the cost-effectiveness of ILR compared to PM in the management of older adults (age>50 years) with bifascicular block and syncope over two years, from the perspective of a Canadian publicly funded health care system, in the Syncope: Pacing or Recording In ThE Later Years (SPRITELY) trial.

Resource utilization data was collected throughout the trial, and unit costs were assigned (2017 Canadian dollars). Utility was measured at baseline and annually with the EQ-5D-3L. Quality adjusted life years (QALYs) were calculated as area-under-the-curve, and adjusted for baseline imbalances in utility. Confidence intervals for the incremental cost effectiveness ratio were generated with non-parametric bootstrapping.

Mean cost in participants randomized to PM was CAD 9,759 (USD 7,400), compared to CAD 13,453 (USD 10,200) in participants randomized to ILR. The ILR strategy resulted in 0.020 QALYs more than the PM strategy. The incremental cost effectiveness ratio was CAD 186,553 (95% CI: −831,950–1,191,816) (USD 141,900, 95% CI: −632,740–906,440) per additional QALY. In 1,000 bootstrapped replicates, the cost of the ILR strategy was always greater than that of the PM strategy. At the threshold of CAD 50,000 (USD 38,000) per additional QALY, the probability that the ILR strategy is the cost effective option is 0.504.

ILR costs were greater than PM costs, with little difference in QALY outcomes over two-years. Findings are generalizable to patients similar to SPRITELY participants, from the perspective of the Canadian health care system. However, practice pattern variation and payment systems inhibit generalizability to other countries. Future analysis will explore cost and QALY outcomes in countries that participated in the SPRITELY trial.

The National Oral Health Policy (NOHP) “Smiling Brazil” was launched in 2004, with the goal of reorienting the model of oral health care in the Unified Health System. Up to then, this area was impaired by limited access and curative procedures. The NOHP aims to reorganize Primary Health Care in Oral Health, expand and qualify Specialized Care and add fluoride in the public water supply. This review will bring a reflective view of NOHP evaluation.

This review work searched for evidence on the Bireme and Google Academic databases, with the keywords “Evaluation” and “National Oral Health Policy” in October 2017. The search was limited to full texts in Portuguese, English and Spanish. After reading the titles, the abstracts and finally the complete texts, the articles that did not correspond to the evaluation objective of the NOHP were excluded.

Of the 381 initial articles found, fifteen were selected for inclusion in this study. The majority reported advances in the quality and scope of oral health care with expanded access and provision of services, such as preventive actions, health education, fluoridation of the public water supply and an increase in population coverage. There was also an improvement in the main indicators, in resolution, financial investments and epidemiological surveys. Moreover, few studies showed improvement in user satisfaction. Conversely, difficulties were identified in overcoming the traditional care model, in training and professional appreciation. Challenges included the need to expand access to fluoridated water, increase coverage, build a more comprehensive care network and reduce regional disparities.

After 13 years, advances and challenges can be observed in the quality and comprehensiveness of oral health care in Brazil. There is evident improvement in indicators; however there remains a lack of access and resolution in the actions, with a large number of regional discrepancies.

Screening for psoriatic arthritis (PsA) is expected to identify patients at earlier stages of the disease. Early treatment is expected to slow disease progression and delay the need for biologic therapy. This study estimated the cost-effectiveness of screening tools for PsA in Canada.

A Markov model was built to estimate the associated costs and quality-adjusted life-years (QALYs) of screening tools for PsA in patients using topical treatment for psoriasis. The screening tools included: the Toronto Psoriatic Arthritis Screening (ToPAS) questionnaire; the Psoriasis Epidemiology Screening Tool (PEST); the Psoriatic Arthritis Screening and Evaluation (PASE) questionnaire; and the Early ARthritis for Psoriatic patients (EARP) questionnaire. Health states were defined by disability levels, as measured by the Health Assessment Questionnaire (HAQ), and state transition was modeled according to annual disease progression. Screening was assumed to be effective during a 2-year sojourn period. Incremental cost-effectiveness ratios (ICERs) were estimated based on health-state specific costs and utilities. A probabilistic analysis was undertaken to account for parameter uncertainty. All results were compared with the commonly cited cost-effectiveness threshold of CAD 50,000 (USD 37, 600) per additional QALY.

Screening with the ToPAS questionnaire resulted in cost savings compared with no screening or the EARP questionnaire, with a total cost of CAD 30,706 (USD 23,090) and 17.29 QALYs. The PEST dominated the PASE questionnaire and was more costly and more effective than the ToPAS questionnaire, with an ICER of CAD 312,398 (USD 234,909). The results were most sensitive to test sensitivity and specificity, HAQ progression, and average HAQ score at diagnosis and the start of biologic therapy. A scenario analysis tested screening efficacy for a 1-year period before diagnosis, with the ToPAS questionnaire remaining the most cost-effective option.

Screening was cost-effective compared with no screening at the commonly used cost-effectiveness threshold of CAD 50,000 (USD 37, 600). Value of information analyses will be useful for determining the need to collect further information around test accuracy parameters.

With the rapid increase in technologies and innovations to support a growing aging population in many countries, health technology assessment (HTA) of technologies for the aging populace warrants special consideration. Building on our efforts at Health Technology Assessment international (HTAi) conferences in 2016 and 2017, this presentation will highlight themes generated from two previous HTAi collaborations, with an aim of continuing to build interest and capacity in HTA for aging-related technologies in an international ecosystem that is responsive to local needs and global opportunities.

Researchers from Canada's technology and aging network (AGE-WELL) collaborated with international panelists at HTAi conferences in 2016 and 2017 to explore interest in HTA focused on aging. International panelists shared the current state of aging and HTA in their respective countries. At both sessions, opportunities were provided for participants to rate the importance of themes identified by the panelists.

At the 2016 session, the two most highly ranked themes were: (i) how HTA can help identify the unmet needs of older adults in society that could be met by technology; and (ii) engagement of older adults and caregivers. These two themes became the starting point for the panel discussion in 2017. At this session, the highest ranked themes were: (i) identification of challenges in HTA and aging; (ii) approaches to advancing the effectiveness of HTA in addressing technology and aging; and (iii) development of an aging-related interest group in HTAi.

International collaborations have identified a number of recommendations to consider for HTA and aging-related work including: developing a good mutual awareness and understanding of barriers and opportunities; the importance of co-creating solutions with patients, healthcare providers, researchers, innovators, and funders; and the identification of a suite of methods and tools that can help accelerate technological innovation in care delivery.

Health technology assessments (HTA) for combination drug therapies in oncology are increasingly common. Companies face multiple challenges when determining their economic value due to their complexity and high cost, while payers must balance the need for these vital innovations with sensitivity to rising costs. The study objective was to evaluate the current HTA frameworks in Europe and identify the potential barriers/solutions to reimbursement of brand-on-brand (BoB) combination therapy.

A targeted literature review of HTA agency websites was undertaken to identify any literature/guidance relating to HTA decision-making for combination oncology therapies in France, Germany, Sweden, and the UK.

In France and the UK, BoB HTA decisions reflect clinical- and cost-effectiveness. Combination therapies have been accepted for use in France and the UK, for example, dabrafenib plus trametinib, are assessed through standard HTA processes, exemplifying that positive reimbursement is not unattainable where there is an unmet need and high clinical value. Despite this flexibility, many therapies will fail to prove their cost-effectiveness, resulting in delays or arbitrary pricing decisions. Potential solutions are the use of the ‘efficiency frontier’, as typified by the German HTA system, giving more ‘scope‘ to expensive innovations; or the Swedish HTA approach, which applies variable cost-effectiveness thresholds according to therapeutic area, disease severity, and social criteria. Other possibilities include indication-specific pricing, multiple-criteria decision analysis, and net monetary benefit with willingness-to-trade weights. One likely issue to arise is when different companies are involved, necessitating co-operation. In this scenario, a simplistic solution would be arbitration of the division of the combined price, circumventing the need for HTA agencies to make changes to decision-making criteria.

Constructive debates and collaboration between industry and decision-makers are vital to achieve a harmonized HTA process for high-cost combination therapies which offer advanced benefits and improved safety outcomes, whilst satisfying HTA bodies and providing better access for patients.

Health services users must participate in health technology assessment (HTA) activities. Users, caregivers, and citizens have the practical experience of healthcare and social services. HTA outputs are more useful when values and preferences of patients, caregivers, and citizens are taken into account. Despite this, the best methods of stakeholders’ involvement, timing for doing so, selection of participants, and the type of users to recruit depending of methods and contexts remain unspecified. Herein, an involvement policy has been developed to formalize the participation of users, caregivers and citizens in the services offering of a regional HTA unit.

A steering committee composed of stakeholders (i.e. user, caregiver, citizen, User Experience Service representative, manager, provincial HTA body representative, HTA unit members) was constituted to discuss user involvement in a regional HTA unit. A preliminary vision statement emerged from this committee, and included objectives and principles for users, caregivers, and citizens participation. This statement was deliberated using a Delphi consensus method. Three rounds of deliberations were needed to reach a strong consensus.

Four objectives and four principles that should underlie the development of an involvement policy reached consensus. Participants agreed that users, caregivers, and citizens should: i) propose principles of involvement for each HTA projects; ii) co-realize evaluations with HTA professionals; iii) contribute to evaluation processes; and, iv) be involved in some management decisions of regional HTA units. Four principles to formalize users, caregivers and citizens’ involvement in regional HTA units also emerged. These principles were about utility and feasibility of involvement as well as ethical and methodological considerations.

Users, caregivers, and citizens must participate in the activities of regional HTA units. Each of them have different roles and can contribute to evaluation processes. Their involvement in HTA activities is warranted for co-producing better evaluation more adapted to users’ needs in healthcare and social services.

Instituto Nacional de Traumato-Ortopedia (INTO) administrates the Enoxaparin drug to prevent deep vein thrombosis (DVT) after extensive orthopedic surgeries. Nevertheless, new oral anticoagulants that offer more comfort and efficacy, but present higher risk of bleeding, have been putting in question the use of Enoxaparin. Making use of the MACBETH method, this study develops a Multicriteria Value Measurement model to evaluate such drugs.

MACBETH was applied in helping INTO to evaluate two drugs (Rivaroxaban and Enoxaparin), taking into account drug benefits and risks, through a series of interviews and decision conferences attended by INTO stakeholders that acted as evaluators in the model-building process, supported by M-MACBETH DSS (www.m-macbeth.com). Following MACBETH preference elicitation process, the evaluators were asked to make qualitative pairwise comparison judgements of difference in value between stimuli for constructing quantitative value and weighting scales. These scales allow measuring the relative value of the drugs on each evaluation criterion, separately and globally. The value measurement process was informed by a literature review and meta-analysis of randomized clinical trials with a critical appraisal of the evidence.

We report a model-structure with eight criteria, hereafter presented by decreasing order of their weighting: Death from any cause, Clinically significant bleeding, Proximal DVT, Distal DVT, Existence of antidote, Thrombocytopenia, Costs, and Comfort. From the value model developed and after performing sensitivity and robustness analyses, Rivaroxaban was considered a robust option for thrombosis prophylaxis, under the MACBETH value framework and at the light of a simple additive aggregation of those eight criteria.

This study shows how a value measurement socio-technical framework, combining MACBETH with scientific evidence within a participatory group evaluation process, can support health technology assessment in a user-friendly and effective way. MACBETH facilitates transparent and robust decision-making in the face of complex evaluation problems that the hospital often faces.

The use of long-acting insulin analogues have been reported in patients with type 1 diabetes mellitus who exhibit important oscillations of their daily blood glucose, although the therapeutic benefits are lacking. The aim of this study was to evaluate the effectiveness and safety of the insulin analogue glargine compared detemir to support health decision-making.

We performed a systematic review with meta-analysis of observational studies (cohort and registry), available in the MEDLINE (Pubmed), Latin American and Caribbean Health Sciences (LILACS), EMBASE and Cochrane Library databases (accessed August 2017), including research in the electronic journal Diabetes Care and gray literature. Several combinations of terms were used, including disease terms, interventions and type of study. The results evaluated were: glycated hemoglobin; weight gain; occurrence of severe hypoglycemia; total insulin dose; and, fasting capillary glycemia. Methodological quality was assessed using the Newcastle scale. The meta-analyses were performed in Review Manager® 5.2 software using a random effects model. Protocol number CRD42017054925 (International Prospective Register of Ongoing Systematic Reviews).

A total of 705 publications, eight cohort studies were included. The quality of included studies was classified as high. In the meta-analysis, the results for episodes of severe hypoglycemia (p = 0.002), measurements of fasting capillary glycemia (p = 0.01), and weight gain (p = 0.001) were favorable for detemir. The glycated hemoglobin endpoint (p = 0.49, heterogeneity = 89 percent) revealed high heterogeneity and no statistically significant difference between groups, showing no difference between the interventions for glycemic control.

Although some results are favorable to detemir, it was not possible to identify significant differences in effectiveness and safety between the two analogues evaluated, requiring new long term studies and better quality of methodological studies.