bluuebunny/crossref_metadata_2025 huggingface dataset:
OP151 Weathering The Development To Adoption Storm: NICE Safe Harbors
Authors: McCracken Fay , Deakin Carla
Published: January 2019

Introduction:

Getting technologies adopted in the UK healthcare system can be time-consuming and complex. The National Institute for Health and Care Excellence Office for Market Access (NICE OMA) has developed a novel approach to enable greater and more coordinated dialogue between life sciences companies and healthcare system stakeholders on market access issues.

Methods:

When establishing NICE OMA, interactions were carried out with life sciences trade associations and key healthcare system stakeholders to explore challenges in market access landscape. Feedback highlighted that dialogue with NICE and other stakeholders is often limited and occurs in high-risk situations; indicating a need for greater and more coordinated dialogue between industry and multiple healthcare system stakeholders outside of formal processes.

Results:

The approach developed is a safe harbor engagement framework which enables NICE OMA to facilitate interaction between life sciences companies and key healthcare system stakeholders; this collaborative approach promotes shared understanding of aspects that will allow innovative technologies to reach patients faster. It brings together multiple organizations in a safe environment where ideas can be exchanged between participants, allowing organizations to think beyond their own area of interest and to work collaboratively. Companies have used the engagement framework flexibly to engage at different stages along the development to adoption journey. Feedback indicates that companies have benefitted from channeling discussions through NICE to bring together key leaders from different organizations, as well as the neutral facilitation of discussions. Healthcare system partners have gained insights/knowledge that hadn't been apparent beforehand. Patient and clinical representatives have appreciated the opportunity to provide views to a broad range of stakeholders often early in the development of the technology.

Conclusions:

The NICE OMA safe harbor engagement framework has been well-received to date. Further feedback will be sought to understand the impact in helping to optimize the market access journey.
OP154 Industry And Clinician Views Of Medtech Innovation Briefings
Authors: Burton Alexia Campbell , Unsworth Harriet
Published: January 2019

Introduction:

The National Institute for Health and Care Excellence Medtech Innovation Briefings (MIBs) are commissioned by the National Health Service (NHS) England and designed to support NHS and social care commissioners and staff who are considering using new medical devices and other medical, digital or diagnostic technologies. MIBs are fast flexible summaries of single technologies that are intended to be responsive to commissioners needs for information about innovative technologies. MIBs include a description of the technology, how it is used and its potential role in the treatment pathway. They also include a review of relevant published evidence and likely costs. As a relatively new product, the format of MIBs continues to evolve and in 2016 a more streamlined evaluation template was introduced. To ensure MIBs continue to meet users’ needs, a study was conducted to understand the opinions and requirements of core stakeholders and to identify key areas for future development.

Methods:

An initial cross-sectional online survey with NHS staff who were potential users of MIBs was carried out in December 2015. A second round of online and mail-out surveys were circulated between November 2016 and May 2017 to medical technology manufactures and an additional group of NHS staff. Descriptive analysis was used for all quantitative data and qualitative data was summarized using thematic analysis.

Results:

Thirty-nine medical professionals and fourty-two manufacturer representatives participated in the surveys. More than half of clinicians were aware of MIBs and thought that raising awareness and visibility should be a future priority. Manufactures regarded MIBs as having a positive or mixed impact on innovation, access, or uptake by the healthcare system.

Conclusions:

Stakeholders are using MIBs in a variety of ways and there was and a range of suggestions for their future development particularly regarding moving from single technology evaluation to simultaneous assessment of similar technologies.
OP158 Overview Of Technology Assessment For Multiple Sclerosis In The Brazilian Public Health System
Authors: Souza Kathiaja , Salomon Flavia , de Brito Artur Felipe
Published: January 2019

Introduction:

The National Committee for Health Technology Incorporation (CONITEC) has a structured process for the incorporation, disinvestment, or alteration of different health technologies in the Brazilian public health system and provides technical support for the decision-making process. Since its creation, CONITEC has received several submissions for the incorporation of medicines and the update of clinical practice guidelines for multiple sclerosis (MS). Nowadays, more than twelve different therapies are currently available to treat MS and the Brazilian clinical practice guideline, which was last updated in 2015, offers six medicines to treat MS that are divided into first, second, and third line treatments. The purpose of this study was to describe CONITEC's assessments of applications for incorporation, disinvestment, or alteration of medicines for MS.

Methods:

A case study method was used to evaluate information, retrieved from CONITEC's database, about the health technology reports developed by CONITEC's Executive Secretariat in response to applications received in the period from 2012 to 2017.

Results:

Ten technical reports on health technologies for MS were produced by CONITEC during the study period. This number represented four percent of the external submissions for incorporation of technologies for several clinical conditions in the public health system. Six medicines were evaluated. The highest number of submissions were for incorporation (n = 6), followed by alteration of treatment lines (n = 3), and disinvestment (n = 1); fifty percent of the submissions were not recommended. The main reasons for rejection were low or unproven efficacy, high budget impact, and inadequacy of the proposal based on the evidence presented. CONITEC's favorable recommendations caused a profound change in the current clinical practice guideline and had a significant impact on the health system.

Conclusions:

MS is considered a rare disease in Brazil, but there is significant pressure from society to provide better treatment options that will impact the MS scenario in the health system. The recent CONITEC assessments have led to a revolution in the treatment of MS in Brazil, which is now in the process of being updated.
OP161 Relationship Between Appropriateness And Arthroplasty Recommendation
Authors: Marshall Deborah , Faris Peter , Jones Allyson , Noseworthy Tom , Dick Don , Howden Jane Squire , Woodhouse Linda , Reczek Tania , Bohm Eric , Dunbar Michael , Hawker Gillian
Published: January 2019

Introduction:

We examined relationships between measures of total knee arthroplasty (TKA) “appropriateness” constructs and surgeon TKA recommendations in people with knee osteoarthritis (OA). Although TKA is highly effective, fifteen to thirty percent of recipients report dissatisfaction and/or little or no symptom improvement. More appropriate selection of surgical candidates may improve both patient outcomes and healthcare resource use, but no validated appropriateness criteria exist currently in Canada.

Methods:

Patients 30 years of age or older with knee OA referred for surgical consultation at two large joint arthroplasty centres in Alberta, Canada were invited to participate. Participants completed a standardized pre-consult questionnaire, which included the following sociodemographics and validated measures of appropriateness constructs for TKA: knee symptoms; non-surgical management; patient readiness for and expectations of TKA; and net patient benefit. Post-consultation, surgeons were asked to confirm knee OA and their recommendation. We used multivariable logistic regression to examine the relationship between measures of appropriateness constructs and receipt of surgeon TKA recommendation.

Results:

Of 3,009 patients approached, 2,360 completed the questionnaire and 2,064 (sixty-nine percent) were eligible at surgical consultation (mean age 65.7 years, standard deviation 9.1; fifty-nine percent were women); 1,495 (seventy-two percent) were recommended for TKA. The likelihood of receiving a TKA recommendation was independently associated with: knee symptoms (odds ratio [OR] per unit increase in pain intensity, 1.19 (95% confidence interval [CI]: 1.11–1.27)); prior non-surgical OA management (OR for prior knee injection, 1.53 (95% CI: 1.21–1.94)); readiness for surgery (OR if definitely/probably willing to undergo TKA, 3.03 (95% CI: 1.99–4.59)); and TKA expectations (OR outcome “very important”: ability to perform daily activities, 1.40 (95% CI: 1.04–1.88); straighten the knee/leg 1.42 (95% CI: 1.13–1.80); participate in exercise/sports 0.75 (95% CI: 0.58–0.98)).

Conclusions:

In our cohort of patients with confirmed knee OA who consulted a surgeon for TKA, appropriateness constructs were significantly associated with receipt of a TKA recommendation. Research is ongoing to evaluate the predictive validity of these measures for patient-reported outcomes associated with TKA.
OP163 European Network for Health Technology Assessment Joint Action 3 Relative Effectiveness Pilots: Pharma Company Experience
Authors: Hebborn Ansgar , Oberdiek André , Birkmose Jennifer Cain , Gyldmark Marlene , Ducournau Pierre , Bahar Nasim , Mowbray Kate
Published: January 2019

Introduction:

More than 50 HTA agencies evaluate the clinical value of medicines in Europe, resulting in duplication of work for HTA agencies and manufacturers, and lengthy and variable time to reimbursement for patients across Europe. A consistent, single European relative clinical scientific benefit assessment of medicines could become a key element in ensuring patients get equitable and timely access across Europe. The European Network for Health Technology Assessment (EUnetHTA) is responsible under Joint Action 3 (JA3, 2016–2020) to pilot more than 30 Relative Effectiveness Assessments (REAs) of medicines. The first EUnetHTA JA3 REA pilots are now being completed and Roche, with its participation in the REA pilot for alectinib, has gathered relevant experience.

Methods:

The goal of this analysis is to summarize and reflect upon the experience with one of the first EUnetHTA REA assessments in JA3. The authors also propose potential process improvements.

Results:

The experience with the alectenib REA shows that EUnetHTA processes have improved compared to JA2. The timing of the assessment has been aligned with the EU regulatory marketing authorization process by shortening the duration of the scoping phase. More EUnetHTA members than in JA2 seem to be committed to use the reports in national HTA, pricing and reimbursement processes. At the same time, the REA pilots have identified other areas that could benefit from further refinement, for example the active engagement of patient group representatives and clinical experts, rules and principles related to the handling of confidential information.

Conclusions:

Based on the limited number of REA pilots for medicines it is too early to draw final conclusions on the state of EU-level collaboration. But first signals indicate a positive development compared to REA pilots conducted in JA2. Interim evaluations are recommended to assess progress, and capture learnings for future pilots.
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 services

Results:

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.
PP55 HTA And High Cost Innovative Therapies - Focus On Cancer Drugs
Authors: Raycheva Ralitsa , Stefanov Rumen
Published: January 2019

Introduction:

High-cost innovative therapies are considered as high-risk investments in the reality of limited health care budgets. Health technology assessment (HTA) facilitates evidence-based decision making that relies on robust assessment of the clinical cost-effectiveness of the technology that is often not available for expensive cancer therapies (CT).

Methods:

The objective of the study is to undertake a systematic comparison between HTA agencies worldwide in order to identify similarities and differences in the methods and processes in HTA of CT. A cross-sectional web-based survey was conducted between September 2013 and May 2015. We received responses from 161 HTA organizations based in thirty-nine countries.

Results:

HTA of CT is mainly performed by agencies in South America (38.46 percent), Australia (37.05 percent) and Europe (36.07 percent), followed by agencies in North America (20.00 percent) and Asia (16.67 percent). Logically, the agencies in high income countries produce more assessments of CT (40.23 percent), which in 34.43 percent they determine as innovative technologies compared with 10.00 percent of the units based in middle income countries and active in CT assessment (11.11 percent). We prove association (p < 0.05) between (i) the type of HTA and income per capita; the level at which the organization operates; its main activity; and the level of recommendation dissemination; (ii) the main target group and consumers of the final HTA product; the stage of evolution of the technology, on which it is likely to be assessed; and approaches to identify innovative technologies. The most active in the preparation of HTA reports are biomedical companies (50.00 percent), government agencies (42.11 percent) and professional organizations (40.00 percent). HTA bodies that assess CT distribute recommendations (37.50 percent) nationally and they are mainly addressed to private health care providers (66.67 percent).

Conclusions:

Making coverage decisions based on HTA recommendations control the technologies introduction into the healthcare system that is why it's very important this tool to be properly adjusted to the specific needs of CT assessment.
PP66 Hospital Cleaning: Detergent Or Disinfectant-Detergent? A Rapid Review
Authors: Harkin Kathleen , Dee Anne
Published: January 2019

Introduction:

Healthcare-associated infections (HAIs) are an important, potentially preventable reason to maintain a clean healthcare environment. However, guidelines from Europe and North America do not concur—European guidelines recommend using neutral detergent (followed by chlorine-based disinfection (CBD) if required), whilst North American guidelines recommend using detergent or hospital-grade disinfectant-detergents for routine cleaning or decontamination of noncritical healthcare environmental surfaces. The objective of this study was to compare the effectiveness on rates of HAIs of: (i) disinfectant-detergents versus detergents; and (ii) the active ingredient of many disinfectant-detergents—quaternary ammonium compounds (QAC)—versus CBD.

Methods:

A rapid review of systematic reviews was conducted using the following search terms: keywords and controlled vocabulary terms for the concepts of “healthcare environmental surfaces” AND (“QAC-based disinfectants” OR “disinfectant-detergents” OR “decontamination”) AND (“environmental contamination” OR “colonization” OR “HAIs”). The search filters included systematic reviews, guidelines, and technology reports. The following databases were searched: The Cochrane Library; PubMed; and health technology assessment and guideline websites for gray literature. Systematic reviews of studies comparing the effects of disinfectant-detergents with detergent, or comparing QAC with CBD, on rates of HAIs in the healthcare environment were included. Reviews on the cleaning or disinfection of body surfaces or disinfection of invasive medical devices were excluded. Quality assessment was not conducted. Data extraction was performed using a pro forma.

Results:

The literature search resulted in 356 titles. From ninety-four potentially relevant abstracts, fifty-seven full-texts were evaluated: fifty-one were excluded (eight non-English) and six were included. All review authors cautioned that the evidence was low level, methodologically poor, subject to confounding, and didn't address adverse outcomes. The reviews identified eight relevant primary studies, three of which compared disinfectant-detergents with detergent and found no difference in rates of HAI. Five studies compared QAC with CBD. All five demonstrated that CBD was superior to QAC and reduced Clostridium difficile infection rates in outbreak contexts. Furthermore, QAC may induce sporulation and microbial resistance.

Conclusions:

Low-level evidence suggested that: there is no advantage in using disinfectant-detergents for routine cleaning of noncritical surfaces; CBD is superior to QAC-based disinfection in reducing clostridial infections; and QAC agents may induce sporulation or microbial resistance.
PP75 Genetic Testing For Bladder And Kidney Cancer: An Interactive Evidence Map
Authors: Treadwell Jonathan , Tsou Amy
Published: January 2019

Introduction:

Recently, voluminous research and commentary have touted genetic and molecular testing to improve the management of urologic cancer. The purposes of such testing include screening, risk assessment, diagnosis, prognosis, pharmacogenetics, and monitoring (for example, recurrence, predicting treatment response). An interactive graphical tool (“evidence map”) would help policy makers examine the current state of research, identify prevailing trends, and prioritize research efforts.

Methods:

A professional information specialist searched MEDLINE/EMBASE for articles published in 2010 or later that primarily focused on genetic/molecular testing and either kidney or bladder/urothelial cancer. Two research analysts classified all relevant abstracts regarding to cancer type, genetic marker(s), clinical purpose(s), assay methods, publication type, and author country/region. We created an interactive map using HTML5 and JavaScript.

Results:

We identified 4,731 articles, 828 (18 percent) of which met our inclusion criteria. Our map has interactive filters which allow flexible selection of articles and automatic updating of the counts. For example, one can quickly redraw the map to focus only on U.S./European systematic reviews and meta-analyses. Research on bladder/urothelial cancer focuses on both diagnosis and prognosis, with some interest in monitoring. In kidney cancer, research on prognosis outweighs research on diagnosis. Overall, research on genetic/molecular markers is in an exploratory phase, e.g. for kidney cancer prognosis alone, 173 empirical studies considered hundreds of different markers.

Conclusions:

Assessing prognosis is a common purpose of genetic tests for both bladder/urothelial and kidney cancer. Increased research on the monitoring of bladder/urothelial cancer may be due to its high recurrence rates, whereas lower interest in genetic tests to diagnose kidney cancer may be due to effective imaging tests. For policy makers, evidence maps can inform decisions about the scope of commissioned systematic reviews as well as the targets for recommendation statements. Interactive features allow maps to be redrawn to align with users’ specific interests.
PP123 Triangulate, Converge, Assess, And Recommend (TCAR): Evaluation Method
Authors: Beauchamp Sylvie , Drapeau Martin , Dionne Carmen
Published: January 2019

Introduction:

Methods to assess evidence and to use that evidence to inform practices and policies are under developed in the area of social services. Although health professions have developed robust methods in recent decades to collect, analyze and synthesize scientific evidence and to inform clinical recommendations, the use of these methods often remains difficult in social services. A taskforce was implemented to address this and to propose a method that may be more appropriate for the social sciences.

Methods:

The project was comprised of four steps: (i) performing a qualitative review of discussions between experts, (ii) designing a cognitive map of the data, (iii) conducting a systematic literature search, and (iv) comparing the data from the meetings with experts with the scientific literature. These steps were completed using the grounded theory approach. In order to test the method developed, focus groups were then conducted and four case studies were used to assess the evidence and provide recommendations for youths with mental health problems and for elderly care.

Results:

Although robust scientific data remain crucial when developing recommendations for practice, results showed that these data are incomplete if considered alone, and that contextual (circumstances in which the intervention is delivered) and experiential data (how the intervention is perceived by stakeholders) must also be taken into consideration. A method to triangulate these three types of data is proposed. Using this technique, the value of the data is established by means of various measurements that converge towards the same result or that provide a consistent overall picture or some important nuances that need to be considered, as illustrated by the four case studies.

Conclusions:

The proposed method can be used to address the limitations that are inherent to the use of techniques and procedures drawn from the medical field when assessing evidence and developing recommendations for the social sciences. The case studies that the proposed method is not only a viable option to methods drawn from medicine, but also adds to the quality of the recommendations that are made and is more congruent with the epistemology of social sciences.
PP126 Alfa-Alglucosidase For Pompe Disease: Literature Review
Authors: da Costa Márcia Gisele Santos , Santos Marisa
Published: January 2019

Introduction:

Pompe disease is a rare disease, in which therapies are aimed at improving the function of the heart and skeletal muscles, and the quality of life of patients. This review aims to update and evaluate the safety and efficacy of alpha-alglucosidase therapy for treating Pompe disease.

Methods:

We performed a literature search of Medline, EMBASE, the Center for Reviews and Dissemination, the Latin American and Caribbean Health Sciences Literature, and Cochrane. Publications of the National Institute for Health and Care Excellence and national and international guidelines have been consulted. The quality of the evidence was assessed using the criteria of the Grading of Recommendations Assessment, Development and Evaluation - GRADE. We performed annual cost estimates of alpha-alglucosidase for the treatment of adult and pediatric patients.

Results:

In a randomized clinical trial comparing alpha-alglucosidase enzyme replacement therapy (20 mg / kg) with placebo for 78 weeks, the results favored alpha-alglucosidase (an increase of 28.1 ± 13.1 m in the six minute walk and an absolute increase of 3.4 ± 1.2 percent in forced vital capacity, p = 0.03 and p = 0.006, respectively). In another systematic review, it was observed that patients treated with alpha-alglucosidase had a mortality rate five times lower than untreated patients (rate ratio = 0.21, 95% CI: 0.11 − 0.41). In a pediatric population with advanced disease, biweekly infusions prolonged survival and survival free of invasive ventilation. The quality of the evidence was classified as very low. The annual treatment costs were USD 296,187.64 (adult patient with 70 kg) and USD 42,312.52 (pediatric patient with 10 kg).

Conclusions:

The limited available evidence suggests alpha-algalsidase is efficacious in Pompe disease patients with some clinical conditions who do not present negative cross-reactive immune material. The balance between the limited quality of the evidence and the demonstrated benefits is favorable, especially for clinical improvement, reduction of mortality and intangible benefits.