Decision-making about replacement or modification of an implantable cardioverter defibrillator (ICD) must be patient-centered and clinically appropriate. We engaged both patients and health care professionals in a multi-method approach in order to recommend structures and processes that facilitate informed and shared decision-making.

A systematic literature review (2000 to 2017) was performed focusing on the patient's perspective and the optimal organization of structures and processes for decision-making. A province-wide field evaluation based on medical chart review was carried out to provide ‘real world’ evidence in Québec's six ICD implanting centers (1 July to 31 December, 2016; N = 418). Patients and health care professionals reviewed the findings of the review and field evaluation, and deliberated recommendations in an anonymous manner by electronic mail. A joint meeting focused on proposed recommendations concerning shared decision-making.

The patients provided feedback on the literature review based on their ICD experience, and highlighted the need for better and more interactive decision aids, clinical information and time, and a private space for sensitive discussions. The field evaluation underlined the variability of treatment choices at the time of replacement and that more than one in ten patients had undergone ICD deactivation. Proposed recommendations focus on multi-disciplinary, integrated follow-up of patients and outline best practice for incorporating patient wishes and life objectives when discussing treatment options. The multi-round consultation process allowed both patients and professionals to co-construct recommendations with our evaluation team.

This multi-method approach enriched our interpretation of literature and ‘real world’ data and facilitated identification and prioritization of important themes. Partnership with both patients and clinicians added a new and energizing dynamic to our evaluation and recommendation processes. We acknowledge the contribution of the members of the patient committee and the clinical experts committee.

Clinical background: Until recently, populations living in remote areas did not have access to specialist care and quality diagnostic services and thus depended on the low response capacity of their local health system. Subsequently, there were equity issues between urban and rural populations. Therefore it was considered telediagnosis applications should be directed towards developing better equity in the provision of services in remote locations without access to specialists. This study has evaluated the results of a new telemedicine system in remote public hospitals in Paraguay, in order to show how the response capacity of the local integrated health service delivery networks has been improved by providing access to tertiary level diagnostic services by specialists. Objective: This study aims to evaluate the utility of telemedicine as a tool for developing better equity in the provision of services in remote locations.

This was a descriptive study, where the results of using telemedicine for diagnosis in remote public hospitals were evaluated as a tool to improve access to diagnostic services countrywide between 2014–2017. For these purposes, type and frequency of pathology diagnosed was determined.

A total of 311,562 telediagnoses were performed in fifty-seven hospitals. The 191,435 electrocardiogram diagnosis performed in the fifty-five hospitals were mainly normal (62.1%), unspecified arrhythmias (12.5%), and sinus bradycardia (10.4%). Also 115,924 teletomography tests were performed in twelve hospitals, where 54.4 percent corresponded to head as a consequence of accidents (motorcycles) and cerebrovascular diseases, 13.8 percent to chest, and the rest the other anatomical regions. Regarding the 4,184 electroencephalogram tests performed, antecedents of seizure (54.3%), evolutionary controls (14.0%), and headache (11.5%), were mainly diagnosed. The nineteen ultrasound studies corresponded to prenatal controls.

Despite the results of the telediagnosis implemented in the public health to develop better equity in the provision of services in remote locations, a widespread use-assessment should be analyzed before this tool is adopted.

Molecular techniques play a critical role in identifying breast cancer patients with overexpressed human epidermal growth factor receptor-2 (HER2). New bright field techniques such as chromogenic in-situ hybridization (CISH) and silver in-situ hybridization (SISH) have emerged to overcome some of the challenges associated with the reference standard, fluorescence in-situ hybridization (FISH). We conducted a literature review and synthesis to characterize the accuracy of HER2 tests, and inform decisions about test selection.

We searched MEDLINE and EMBASE databases using these eligibility criteria: studies evaluating invasive breast cancer samples which examined agreement between CISH or SISH, and FISH, and reported sensitivity, specificity, or concordance. We performed a bivariate meta-analysis of sensitivity and specificity using a generalized linear mixed model in Stata. We used likelihood ratio tests from meta-regression to compare accuracy between HER2 tests.

The search identified 4,475 articles, of which thirty-one were included. A total of thirteen studies (43%) evaluated dual-color SISH, twelve single-color CISH, and six dual-color CISH. The summary estimates for sensitivity and specificity were, respectively, 0.97 (95%CI 0.83–0.99) and 0.99 (95%CI 0.96–1.00) for single-color CISH, 0.98 (95%CI 0.92–0.99) and 0.98 (95%CI 0.91–0.99) for dual-color CISH; 0.92 (95%CI 0.86–0.95), and 0.96 (95%CI 0.91–0.98) for SISH. Significantly higher specificity was reported for single-color CISH than SISH (chi-square 4.12; p = 0.04), while dual-CISH had higher sensitivity than SISH (chi-square: 4.63; p = 0.03). These differences were not maintained when studies with cohorts enriched with equivocal samples were excluded.

The agreement between new bright field tests (SISH and CISH) and FISH is high (>92 percent). Indirect comparison of HER2 tests indicated that overall CISH performance exceeds that of SISH. However, low agreement between SISH and FISH in equivocal cases affects these comparative estimates. The pooled estimates from this meta-analysis can help inform future HER2 test selection decisions.

Microfracture (MF) has been the main intervention in symptomatic articular cartilage knee defects. Autologous chondrocyte implantation (ACI) has looked promising, but was not recommended by the UK National Institute for Health and Care Excellence (NICE) in 2015 due to the short-term follow-up data from trials.

Most long-term data comes from observational studies. We provided new unpublished analyses to NICE based on survival data of these studies, with appropriate caveats. They included: a large ACI study by Nawaz with useful subgroup data by osteoarthritis Kellgren-Lawrence stage and previous repair attempts; a very large MF study by Layton, and a small RCT by Knutsen indicating MF was as ‘good’ as ACI. A Markov model explored the cost-effectiveness of ACI vs. MF. Different scenarios were explored: ACI or MF as a first procedure, followed by ACI or MF in those needing a second repair. A NHS England perspective was adopted. Health outcomes were expressed as quality-adjusted life-years (QALYs).

The revised base-case analysis, used a list price of £16,000 (EUR 17,380 in 2013 prices) for cells, used ACI failure data from Nawaz with no previous procedures for ACI, and pooled MF failure data from two studies-Saris and Knutsen. ACI was more expensive but provided more QALYs. The incremental cost-effectiveness ratio comparing ACI then MF with MF then ACI was £8,000 (EUR 8,690) per QALY. Various sensitivity analyses were conducted assuming a threshold of £20,000 (EUR 21,730) per QALY: previous repair attempts reduced success of ACI (£22,000 (EUR 23,900) per QALY); reducing cell costs, ACI improved its cost-effectiveness; and limiting intervention to patients with higher Kellgren-Lawrence score did not appear cost-effectiveness.

The final NICE guidance published in October 2017 approved the use of ACI for patients who had no previous knee repairs, for people with minimal osteoarthritic damage to the knee, and for people with articular defects of over 2cm2.

In April 2017, the National Institute for Health and Care Excellence (NICE) updated its guidance for highly specialized technology (HST) appraisals, whereby it would automatically fund technologies for very rare diseases that fall below a threshold of an incremental cost-effectiveness ratio (ICER) of GBP 100,000 (USD 133,000) per quality-adjusted life year (QALY). In addition, NICE proposed to introduce a ‘QALY modifier’, weighting QALYs gained by the size of gain, which will advantage treatments that offer greater QALY gains.

We reviewed all technologies reviewed through the NICE HST process until November 2017 and assessed whether additional QALYs may be awarded, and subsequently result in ICERs below the new NICE threshold.

Six products (eculizumab, elosulfase alfa, ataluren, migalistat, eliglustat, and asfotase alfa) have been through HST process. Within the appraisal documents, most analyses were cost consequence analyses with no ICERs reported. The estimated cost per patient per year ranged from approximately GBP 100,000 (USD 133,000) to GBP 400,000 (USD 532,000; listed prices). Of the six technologies, three resulted in at least ten incremental QALYs (eclizumab, elosulfase alfa and asfotase alfa). From the information in the public domain, it is unclear whether this would result in ICERs below GBP 100,000 (USD 133,000) per QALY.

It may become more difficult for HSTs to get recommended by NICE under the new guidance, which requires cost-effectiveness analyses, whereas previously there was no official ICER threshold. The additional weighting of QALYs may be insufficient to meet an ICER threshold of GBP 100,000 (USD 133,000) per QALY.

Bridging gaps between registry-holders, Health Technology Assessment (HTA) producers and users is one of the aims of the European Network for HTA (EUnetHTA) Joint Action 3. In this context, a post-launch evidence generation tool is being developed, including a quality standards tool for registries in HTA. The standards tool for registries in HTA will enable, among others, registry owners to consistently collect high quality registry data, and HTA agencies to use proper registry data collected by others as evidence for their assessments. The objective is to present the first draft version of the tool structure, which is going to be piloted during the forthcoming months.

A review and description of the currently available first version (November 2017) sections, items and criteria for HTA studies.

The tool is divided in three sections; “Methodological Information”, “Essential Standards” and “Additional Requirements”. The first section enables users to analyze not only the ability of the registry to answer to research questions but also to check the registry transparency. The second section encloses the essential elements of good practice and evidence quality (therefore all of them must be met before an HTA report can use the registry data). Finally, the third section includes elements of good practice and evidence quality useful to consider in planning and evaluating registries for specific purposes. Although suggestions are defined, the third section item requirements could depend on the individual HTA agency perspectives and needs.

There is a clear growing availability and requirement for real world data for health technology assessment. A piloted and robust registry standards tool for HTA can provide a relevant basis to improve both the evidence generation but also to make more trustful and excellent evaluations.

To address local workability, cross-setting variation, and clinician and patient perspectives, health technology assessment (HTA) practitioners and health system decision-makers incorporate varying forms of qualitative evidence into evaluations of novel health technologies. Employing principles and methods from long-established sociotechnical fields such as participatory design (PD) may help HTA teams in the production of formal, rigorous ‘practice-based evidence’.

We draw on a theoretical review of foundational PD literature and experiences using PD for a large-scale health information technology project to summarize principles and strategies for the effective introduction and evaluation of new technologies in healthcare.

HTA may benefit from observing some of the core commitments of PD: (i) Ensuring that technologies enhance rather than detract from the quality of working life; (ii) Fostering democratic engagement in the implementation and evaluation of technologies; and (iii) Proceeding via direct partnership with technology users. These are practical commitments stemming from the recognition that technology implementation entails re-configuring existing practices and social arrangements. The experts of this existing milieu are the people on the ground, who may reject or underutilize technologies that they perceive as impractical, ill-adapted to their needs, or having negative consequences on their work. At the same time, PD recognizes that local activities occur within larger systems and that effective technology introduction also requires attention to macro-politics (e.g. governance challenges, competing priorities). PD employs a diversity of methods (e.g. participant observation, focus groups, workshops, interviews) to develop evidence that is holistically informed.

Many of the challenges that HTA faces, both in terms of evidence production and translation, have been encountered before in PD. Given that decision-making around health technologies necessarily involves consideration of many forms of qualitative evidence, there is value in producing and evaluating such evidence in carefully designed manner – a challenge to which fields like PD can lend a wealth of experience.

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.