bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP06 HER2 Evaluation By CISH And SISH In Breast Cancer: A Meta-Analysis
Authors: Lago Luiza Grazziotin , Dada Bukky , Yang Hua , Marshall Deborah 
Published: January 2019

Introduction: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.Methods: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.Results: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.Conclusions: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.
PP11 Would A Highly Specialized Technology Be Approved In England Under The New NICE Guidance?
Authors: Turkstra Erika , Mardiguian Silvy , Budhia Sangeeta 
Published: January 2019

Introduction: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.Methods: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.Results: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.Conclusions: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.
PP22 How Do Health System Leaders Use Evidence To Inform Action?
Authors: Mitchell Matthew D , White C Michael , Guise Jeanne-Marie , Bañez Lionel , Umscheid Craig , Hartling Lisa , Lyer Suchitra , Borsky Amanda , Berkman Nancy , Robinson Karen , Newberry Sydne , Fiordalisi Celia , Henderson Jillian , Sandlers-Schmidler Gillian 
Published: January 2019

Introduction: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.Methods: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.Results: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.Conclusions: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.
PP32 Protocol For Evaluation Of Pharmaceutical Assistance Governance
Authors: Faraco Emilia , Rover Marina , Farias Mareni , Contezini Silvana 
Published: January 2019

Introduction: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.Methods: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.Results: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).Conclusions: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.
PP39 Health Technology Assessment And Aging: Moving Evidence To Action
Authors: McNeil Heather , Juzwishin Don , Stolee Paul , Ahn Jeonghoon , Chen Yingyao , Cicchetti Americo , Kume Naoto 
Published: January 2019

Introduction: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.Methods: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.Results: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.Conclusions: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.
PP43 MACBETH In Brazilian Hospital-Based HTA: Thrombosis Prophylaxis
Authors: Santos Marisa , Morais Quenia Cristina Dias , Rodrigues Teresa , Bana e Costa Carlos 
Published: January 2019

Introduction: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.Methods: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.Results: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.Conclusions: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.
PP47 Defining The Needs And Preferences Of Patients With Dry Eye Disease
Authors: Cook Nigel , Sloesen Brigitte , Panapurina Anna , Johnson Gary , Prince Clementine , Medi Sharath 
Published: January 2019

Introduction:Dry eye disease (DED) is a common condition that significantly impacts patients’ quality of life. Previous studies have explored the impact of DED on patients’ lives qualitatively; however, patients’ preference structures have not been thoroughly explored quantitatively.Methods:A targeted literature review and social media listening project guided design of a discussion guide for in-depth patient interviews (n=12). These, in turn, guided construction of a quantitative questionnaire administered to moderate to severe DED patients, 40 per country in Australia, Germany, United States and United Kingdom (total n=160). Patients’ preference structures were explored through an online survey using a self-explicated conjoint methodology, because of its high respondent-friendliness. Additionally, we administered the EQ5D-5L instrument to determine the health states/utilities of patients. Reaction to a hypothetical novel treatment was further obtained to check for convergent validity with the self-explicated conjoint. Finally, we asked respondents to rate the ease and relevance of the questionnaire to them.Results:Qualitative research uncovered important patient perspectives that were built into the quantitative survey. For example, patients seek medical advice when their symptoms are not improving. Patients’ lives are most affected by sensitivity to light, itchy and tired eyes and an inability to perform computer/screen work; however, of most concern/worrying to them is that their DED will get worse and they go blind. Results from the quantitative preference research will also be shared and its implications for future clinical trials in DED outlined. The results of the patient research and preference study are to be shared with health technology assessment (HTA) bodies and regulators through the early dialogue scientific advice process.Conclusions:A process of using qualitative research to determine what matters to patients and then quantification through respondent-friendly preference research can identify outcomes that are most patient-relevant, to inform future drug development strategies.
PP48 Caregiver Perceptions And Experiences Of Diagnostic Genome-Wide Sequencing
Authors: Hong Jenny , Conklin Annalijn , Dragojlovic Nick , Elliott Alison , Friedman Jan , Lynd Larry 
Published: January 2019

Introduction:The objective of this qualitative analysis was to explore caregiver perceptions and experiences of receiving or not receiving a genetic diagnosis following genome-wide sequencing (GWS) in children with suspected genetic disorders.Methods:One caregiver for each child completed an online survey two weeks after enrolling in the Clinical Assessment of the Utility of Sequencing and Evaluation as a Service (CAUSES) study, beginning in January 2016, and again about six months after receiving the GWS results. The survey covered the caregivers' experiences and quality of life and children's healthcare resource utilization, and provided open-ended questions for comments. The follow-up survey was completed by twenty families who had received a diagnosis with their GWS results and by twenty-two families who had not received a diagnosis. A thematic analysis of the free-text comments from both groups was performed using NVivo 11.4.2.Results:Caregivers from both groups expressed similar experiences of negative socioeconomic effects of caregiving, particularly related to employment and time burden. Caregivers who did not receive a diagnosis with the GWS results were generally hopeful of receiving a diagnosis in the future and reported expectations of a positive benefit from receiving a diagnosis, both in terms of access to additional resources and of positive psychological effects. The absence of a diagnosis was a source of anxiety for many caregivers. By contrast, caregivers who had received a diagnosis reported positive, neutral, and negative psychological effects from the knowledge gained; no participants commented on the consequences for access to additional services or other socioeconomic effects.Conclusions:Our findings suggest that caregivers may have high expectations for what a diagnosis can provide to them and their families, which may not be fully met once a diagnosis is obtained. The study underpins the importance of patient-centered communication of genomic testing results so that families can set realistic expectations of what having a diagnosis will achieve.
PP49 Assessing Values In National And Regional Governance Of E-health
Authors: Ekeland Anne , Linstad Line , Holm Inger Marie 
Published: January 2019

Introduction:Globally, countries are investing substantially in e-health. Failures of programs to achieve valuable economic, clinical and societal outcome are increasingly reported. Unsuitable governance models may be one explanation. Research on governance models’ usefulness for realization of valuable outcomes is incomplete and scattered. Our goal is to fill this gap by producing knowledge on e-governance in Norway. Our hypotheses are: i) Co-governance and Relational Coordination will positively impact the realization of valuable outcome; and, ii) Multilateral stakeholder dialogue and collaboration, including health service delivery perspective, have been proposed to innovate health technology assessment (HTA). This will improve the relevance of HTA e-governance research.Methods:We undertook the following: i) Systematic Review of e-governance in healthcare ii) Participatory observations, in depth interviews/focus groups iii) Document retrieval and analyses iv) Creation and support of arenas for dialogue between stakeholders on values and governance v) Analyses of co-produced value adjustments vi) Analyses of the usefulness of the Scientific Dialogue Approach for changing HTA paradigms. The study populations were: i) Governmental bodies responsible for innovation of the electronic health record (EHR) in Norway; ii) Regional and municipal authorities and management responsible for implementation of her; and, iii) The leaders of different levels at a municipal “Health House” established as a hybrid between primary and specialist health servicesResults:The project runs between January 2018–2022. Expected findings are: i) Diverging and common values; ii) Diverging governance models; iii) Diverging attitudes towards “best governance practices”; iv) Diverging levels of trust; v) Different world views, belief-systems and individual values; vi) Attitudes towards consensus building or conflict; and, vii) Experiences to feed into the discussion of stakeholder dialogue as an HTA approach.Conclusions:We expect: i) To present results from the systematic review and preliminary findings from the first phases of participatory observations; ii) That results from the overall project will have high impact on the Norwegian governance models of e-health; and, iii) Publications in high impact scientific journals.
PP54 Effectiveness And Costs Analysis Of Smartphone Apps in Health Care
Authors: Picoli Renato , Braga Carlos 
Published: January 2019

Introduction:Smartphones have been one of the success stories of the last decade. Recently apps have been used to promote, manage, and provide medical and healthcare education. Since smartphones are used to support healthcare and public health interventions, they can also provide a useful and easy method for collecting data for healthcare research. In addition, smartphone apps have been successfully used to support telemedicine and remote healthcare in developing nations. This study aimed to assess the effectiveness and analyze costs before and after the inclusion of type 2 diabetic patients in a smartphone app healthcare program.Methods:The smartphone app healthcare program is available for Android and IOS systems, and is used to manage behavior changes and to improve patient adherence to pharmacotherapy. Patient follow up is done through a specialized telephone monitoring center made up of a physician, nurses, nutritionists, and psychologists who provide constant monitoring and guidance to patients. A retrospective study was conducted of twenty-nine patients before (year 2016) and 12 months after they were included in the smartphone app healthcare program. Data on physician visits, hospitalizations, and medical and laboratory exams were collected from medical records. The cost analysis was conducted from the private healthcare group perspective and was performed using the micro-costing method.Results:Ninety-eight percent of patients had reduction or maintenance of glycosylated hemoglobin levels, reaching the therapeutic goal (glycosylated hemoglobin of less than 7%). The cost analysis showed a twenty-five percent total cost saving due to a twenty-three percent reduction in the number of physician visits, a thirty-three percent decrease in hospitalizations, and a thirty-five percent cutback in medical and laboratory exams.Conclusions:The smartphone app healthcare program can facilitate and improve diabetes care, especially with respect to controlling and managing the use of health resources.