bluuebunny/crossref_metadata_2025 huggingface dataset:
OP121 Experiences With Using The GRADE-CERQual Approach In Systematic Review
Authors: Jensen Lotte Groth , Carstensen Kathrine
Published: January 2019

Introduction:

There are many approaches to synthesis of qualitative studies. The GRADE-CERQual approach (Confidence in the Evidence from Reviews of Qualitative research) provides a transparent method for assessing the confidence of evidence from reviews of qualitative research. This presentation aims at giving examples of applying CERQual, presenting and discussing its strengths and limitations.

Methods:

This presentation draws on practical experiences with the conduction of three qualitative systematic reviews using the CERQual approach. The reviews differ in aim and field of research.

Results:

The three CERQual reviews to be discussed in this presentation are: (i) Emergency departments and mental health patients - Purpose: Uncovering knowledge in a project on merging emergency departments to include both somatic and psychiatric patients. (ii) Parental responses to severe or lethal prenatal diagnosis – Purpose: Providing physicians with knowledge on a patient group from their daily clinical practice. (iii) Patients' experiences with home mechanical ventilation – Purpose: Disseminating important knowledge from a national project to an international audience.

CERQual strengths:

•

Presents complex and large amount of knowledge in a clear way

•

Pools knowledge from different studies into common outcome measures across studies

•

Presents an assessment of the quality and strength of outcome measures

•

The clear presentation makes it useful in decision making.

CERQual weaknesses:

•

Time consuming to conduct the reviews

•

Simplification of qualitative research, missing out on context and nuances.

Conclusions:

CERQual represents a useful tool to facilitate the use of qualitative evidence in clinical and political decision making. CERQual is time-consuming to learn, but a useful tool to apply when learned. CERQual may encourage more uniform reporting of qualitative research, including assessment of confidence in findings. This may increase the impact of systematic reviews of qualitative studies.
OP123 Translating Evidence To Action – The Role Of Health Research Funders
Authors: Reijmerink Wendy
Published: January 2019

Introduction:

National health research funders are accountable to the public with regard to the societal impact of the research, including health technology assessment (HTA), that they fund. Failing to do so can not only negatively affect public trust in the allocation of resources to funding agencies, but can also lead to public mistrust in science.

Methods:

We present the results of reducing research waste to ensure societal responsible research, both at an international and national level. In the Netherlands, the National Organization for Health Research and Development (ZonMw) developed an analytical framework to assess its research programs, including the national HTA program.

Results:

An evaluation of 12 national funding agencies in Australia, Europe and North America demonstrated that certain processes (e.g. how research questions are prioritized or decided) are not transparent. At the international level, health funders believe that they have a joint responsibility not just to seek to advance knowledge, but also to advance the practices of health-related research and research funding. In the Netherlands, ZonMw (HTA) research programs perform well regarding addressing societal relevance (e.g. stakeholder participation) and reasonably well on scientific quality (e.g. international cooperation and knowledge sharing). Efficiency (e.g. encouraging use of existing data and systematic reviews) appears to be less well developed, while integrity (e.g. preventing publication bias) is underexposed.

Conclusions:

Although ZonMw is doing reasonably well in terms of reducing research waste, it was concluded that more focus on societal impact assessment is needed. To do so funding agencies need to collaborate with all relevant stakeholders. This is especially relevant in the field of HTA where the ambition is to move from evidence to impact.
OP134 Pan-Canadian Oncology Drug Review Decisions And Access To Anticancer Treatments In Canada
Authors: Singh Ambrish , Hussain Salman
Published: January 2019

Introduction:

The Canadian Agency for Drugs and Technologies in Health (CADTH) pan-Canadian Oncology Drug Review (pCODR) plays an important role in public reimbursement decision-making for oncology drugs in Canada. This research studies the relation of positive pCODR decisions to new cancer treatment and their subsequent inclusion in Canada's public drug plans.

Methods:

We studied all oncology drugs that received an approval from Health Canada and were reviewed by the pCODR from inception till 26th Sep, 2017. The data was obtained from CADTH and Health Canada. Data such as indication, submission type and date, recommendation date, final recommendation, and subsequent provincial funding status was extracted and analyzed. Impact was evaluated by analyzing the percentage of drug submissions with assessment outcome (positive recommendation rate and conditional recommendation rate) and time taken for the final decision (recommendation gap). The percentage of drugs included in public formulary after positive recommendation by pCODR (coverage rate) and the gap in days from positive recommendation to subsequent coverage in provinces (coverage gap) was also assessed.

Results:

Among 119 drugs reviewed by pCODR, the positive recommendation rate was eight percent. Nine applications comprising seven drugs for six indications received positive recommendations, and genitourinary treatments received maximum positive recommendations. The conditional recommendation rate was 52 percent; 62 applications of 45 drugs for 46 indications received conditional recommendation. Lymphoma and myeloma treatments received maximum conditional recommendations. The average recommendation gap for positive and conditional recommendations was 180 and 172 days, respectively. The coverage rate for drugs with positive recommendation was 100 percent for all provinces except 89 percent for Newfoundland and Labrador, and 67 percent for Prince Edward Island. Among the provinces, British Columbia had a maximum of 433 days and Saskatchewan has the minimum of 165 days coverage gap.

Conclusions:

Despite Health Canada's approval, only a fraction of oncology drugs receive positive pCODR recommendation; furthermore, provincial drug plans take time to include these in the reimbursement formularies. While health technology assessment is crucial for appropriate allocation of limited resources, efforts should also be made to reduce access barriers, particularly to positively recommended oncology drugs inclusion in provincial formularies.
OP164 Hospital Budget Impact Of High-Cost Drugs: The Case Of Nusinersen
Authors: Demirdjian Graciela
Published: January 2019

Introduction:

Nusinersen is an orphan drug for spinal muscular atrophy (SMA) recently approved for marketing. Its high cost, striking but limited evidence of efficacy, and strong demand by media and patient organizations have generated a health policy conflict. We analyze the flaws of available evidence on nusinersen and its budget impact at a pediatric hospital, and report a collaborative strategy for drug procurement and financing.

Methods:

Nusinersen is the highest-cost drug assessed by our hospital-based health technology assessment (HB-HTA) program so far. At the time of our assessment, only interim-analysis data of the pivotal randomized trial submitted to Federal Drug Administration (FDA) for approval and the European Medicines Agency (EMA) report containing unpublished final results were available. These secondary sources and other published phase II results were appraised. As a referral hospital, we concentrate most of the 300 SMA patients in our country. Hospital budget impact estimation included drug and hospitalization costs for the first and following years. The HTA report was submitted to the Ministry of Health to address this financing issue.

Results:

The available evidence of efficacy raised serious methodological and clinical uncertainties. First-year treatment cost per patient was estimated in ARS 13,008,688 (USD 752,000, 10 percent of pharmacy annual drug budget). Hospital budget impact (70 eligible patients) was ARS 910,608,160 (USD 52,000,000; 18 percent of total annual hospital budget). Our recommendation was to contact central level authorities to resolve both drug financing and patient access by negotiating a shared-risk approach for an expanded access program, allowing further data collection for reassessment after 12 months. This, in turn, fostered mutual collaboration and consensus within the health system where several lawsuits were demanding drug coverage. Negotiation with the industry was initiated by the Ministry.

Conclusions:

This case is a clear example of forthcoming ultra-high-cost drugs unaffordable by hospital budgets. Their acquisition opportunity cost is a health policy matter requiring to display collaborative coping strategies with Ministries and other stakeholders including industry.
OP169 Usability Evaluation Of A Portable Dry-Electrode Electrocardiography Device In Vietnam
Authors: Kilimo Precious , Phan Ngoc , Le Tai , Hoang Thai , Han Dung , Dang Linh , Vu Nguyen , Pham Nga , Nguyen Tuan , Cao Hung , Nguyen Cuong
Published: January 2019

Introduction:

According to the Vietnamese Cardiovascular Association, one-fifth of Vietnam's population is suffering from cardiovascular disease (CVD) – now the leading cause of death in the country that accounts for about one-third of total deaths every year. Yet affordable and convenient solutions to monitor and detect CVDs remain limited and not available nation-wide. This study aimed to investigate the usability of a portable dry-electrode electrocardiography (ECG) device, paired with a mobile phone, in supporting ECG service delivery in Vietnam.

Methods:

An evaluation study was designed to combine a portable dry-electrode ECG device to measure and a mobile phone to receive and record ECG signals. Healthy young college students were invited to participate in the study. Three rounds of ECG measurement were administered for each of the participants. Usability of the device was assessed through the reliability of the measures and feasibility of use during intervention. Standard error of measurement (SEM) and intra-class correlation coefficient (ICC) estimations were used for reliability, while structured questionnaire administered before and after measures was used for feasibility assessments.

Results:

A total of 234 participants enrolled in the study. No major difference was found in SEMs between trials one and two (4.96 percent, 90% CI: 4.61 − 5.37) and two and three (4.14 percent, 90% CI: 3.85 − 4.48). A slight improvement was observed in ICC of trials two and three (0.95, 90% CI 0.94 − 0.96) in comparison to one of trials one and two (0.94, 90% CI: 0.92 − 0.95). The SEM and average ICC of all trials were 3.41 (90% CI: 3.17 − 3.69) and 0.96 (90% CI: 0.95 − 0.96) respectively. Forty-five percent of participants thought the device would be suitable for their parents while 69 percent thought the device would benefit their grandparents the most.

Conclusions:

High consistency of measures demonstrated that the device is reliable to provide ECG service delivery. The study also showed great potential of device usage in primary health care of Vietnam.
PP14 Development Of The European Network For Health Technology Assessment Standards Tool For Registries In Health Technology Assessment
Authors: Garcia Emmanuel Gimenez , Espallargues Mireia , Long Jae , Valentic Maja , Guzina Irena , Rodrigues Jorge , Varela-Lema Leonor , Dedeu Toni
Published: January 2019

Introduction:

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.

Methods:

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

Results:

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.

Conclusions:

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.
PP17 Comprehensive Evaluation Of A Technology With Expanding Indications
Authors: Lambert Laurie , Désy François , Azzi Leila , Vutcovici Maria , Brière Anabèle , Boothroyd Lucy , Bogaty Peter , Racine Normand , de Guise Michèle
Published: January 2019

Introduction:

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.

Methods:

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.

Results:

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.

Conclusions:

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.
PP25 Direct Costs Of Ischemic Heart Disease: Real World Data From Brazil
Authors: Schlatter Rosane , Hirakata Vania , Etges Ana , Polanczyk Carisi
Published: January 2019

Introduction:

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.

Methods:

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.

Results:

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.

Conclusions:

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.
PP34 A Cost-Utility Analysis Of The Syncope: Pacing Or Recording Trial
Authors: Hofmeister Mark , Sheldon Robert , Spackman Eldon , Raj Satish , Talajic Mario , Becker Giuliano , Essebag Vidal , Sarah Rose M. , Ritchie Deborah , Morillo Carlos , Krahn Andrew , Safdar Shahana , Maxey Connor , Clement Fiona
Published: January 2019

Introduction:

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.

Methods:

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.

Results:

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.

Conclusions:

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.
PP57 Grading The Quality Of Evidences In HTA Process
Authors: Andellini Martina , di Mauro Roxana , Faggiano Francesco , Derrico Pietro , Ritrovato Matteo
Published: January 2019

Introduction:

In decision-making processes, health technology assessment (HTA) plays an important role ensuring the adoption of effective technologies and translating scientific evidence into decisions. Bambino Gesù Children's Hospital developed a new method which integrates EUnetHTA Core Model with multi-criteria decision analysis (MCDA) enabling decision makers to make a more informed decision between different alternatives. This approach quantifies assessment parameters, which are defined by literature evidence, or by expert opinion when lacking such evidence. MCDA results (i.e. decision tree of assessment elements, weighting systems and numerical values of technology’ performance) are derived from expert judgement. This means that indicators are weighed by the same weight system; either they are supported by strong literature evidence or otherwise based on expert opinion. The objective of this work is to use the GRADE approach to weight the relevance of each indicator starting from its source of information because different level of evidence should result in different weights.

Methods:

A GRADE level was associated with each judgement value of performance indicators and a normal probability function was built with the standard deviation inversely proportional to GRADE level to describe the possible dispersion of the judgement due to the different levels of evidence that support each indicator. The higher the GRADE value, the lower the associated standard deviation. A Monte Carlo simulation was carried out to evaluate the expected value of technology’ performance modulated by GRADE level.

Results:

Four Gaussian distributions were built and associated to four GRADE levels. When an indicator has a low GRADE level, its performance value will vary in a broader way according to the linked Gaussian distribution.

Conclusions:

This study showed the importance of applying the GRADE system to indicators’ sources of information because this can modify the overall computation of parameter weights and performance, proportionally to their robustness.
PP71 Long-Term Evaluation Of Broad Mental Health Interventions: A Review
Authors: Canaway Alastair , Sampson Christopher , Meacock Rachel
Published: January 2019

Introduction:

Interventions and services for people with mental health problems can have broad remits: they are often designed to treat people with a variety of diagnoses. Furthermore, addressing mental health problems can have long-term implications for economic, social, and health outcomes. This represents a challenge for health technology assessment, for which long-term trial data can be lacking. In this review, we sought to identify how analysts have tackled this problem. We reviewed the methods used to extrapolate costs and outcomes for the purpose of economic evaluation where long-term trial data are not available.

Methods:

We conducted a systematic review of the medical and economic literature evaluating long-term costs and outcomes for mental health interventions and services designed to treat or prevent more than two mental health conditions. We searched key databases including MEDLINE, Embase, PsycINFO, CINAHL, and EconLit. Two authors independently screened citations. Articles were excluded if they reported within-trial analyses or employed a time horizon of less than 5 years.

Results:

The search identified 829 unique records. No papers could be included in the review.

Conclusions:

This review highlights the lack of research and understanding available to inform the appraisal of broad mental health interventions. In light of our findings, we consider the reasons for this lack of information and review relevant literature on the subject. Potential barriers to research in this context include: (i) challenges in understanding the value of broad mental health services, such as the mental and physical health nexus, intersectoral costs and benefits, and interpersonal impacts, (ii) methodological difficulties, such as data availability, patient heterogeneity, and the challenge of extrapolation, and (iii) parity of esteem. We make recommendations for resolving this problem with regard to funding, data collection, modelling methods, and outcome measurement.
PP88 Intravenous Medication Delivery System Cost-Effectiveness Analysis
Authors: Peregrino Antonio , Silva Roberto , Itria Alexander , Silva Carlos , Tolentino Ana , Marta Cristiano , Schutz Vivian , Scavarda Annibal , Oliveira Cristiane De
Published: January 2019

Introduction:

Medication delivery is one of the most common interventions in clinical practice. It requires the direct involvement of nurses and high precision infusion pumps in order to increase the infusion accuracy. Any mistake in the medication delivery process can lead to a medication error, resulting in adverse events with considerable increases in hospital length of stay and cost. Research studies should analyze this area more in emerging countries, as their realities differ from the realities of developed countries, where most of the literature of this area has been developed. This research study analyses this area in Brazil, a leading emerging country. The incorporation of these technologies in health services have caused two major problems: uncertainty around its effectiveness in reducing adverse drug event rates related to infusion dose errors, and the high cost of their inputs. The objective of this study was to analyze the cost-effectiveness of intelligent drug library infusion pumps to reduce adverse drug events during intravenous medication delivery in pediatric and neonatal patients.

Methods:

Cost-effectiveness was evaluated using a decision-tree framework, considering two scenarios as the base case: the reference one, which uses conventional infusion pumps for intravenous medication delivery with a volume greater than 60 mL, and an alternative one, which uses the drug library infusion pumps. The analysis is with the Unified Health System (Brazil's publicly funded health care system) perspective. The Monte Carlo simulations addressed the uncertainties of the framework. The effectiveness measure was avoidance of adverse drug events.

Results:

The probabilistic analysis showed the drug library infusion pumps to be more cost-effective than conventional pumps. This ratified what had already been revealed by acceptance curve, which demonstrated that the drug library infusion pumps are more likely to be cost-effective compared to the conventional infusion pumps (with a minimum of the incremental cost-effectiveness of USD 1,501.28).

Conclusions:

The study demonstrated that the use of the drug library infusion pumps in the pediatric and the neonatal intensive care unit can improve the results of the adverse drug event reduction strategy.
PP93 HTA Role In CoreHEM, A Multi-Stakeholder Core Outcome Set Project
Authors: Clearfield Elizabeth , Skinner Mark , Iorio Alfonso , Tunis Sean , Naber Jennifer Al , Messner Donna
Published: January 2019

Introduction:

Hemophilia gene therapy trials demonstrate a “cure” could be achievable, thereby changing disease management. CoreHEM aims to develop multi-stakeholder consensus around a clearly defined, core outcome set (COS) - a minimum set of outcomes that should be measured and reported in all clinical trials of a specific condition - that will demonstrate and allow differentiation of the effectiveness and value of gene therapy relative to the current standard of care. Health technology assessment (HTA) frequently suffers from a lack of relevant, consistently reported outcomes. When uniformly implemented, COS increase the predictability and consistency of appraisals, coverage, and reimbursement decisions by payers and HTA agencies.

Methods:

A COS was developed using a modified Delphi process, including online surveys and an in-person consensus meeting. A literature review and key informant interviews were used to create an initial list of outcomes for voting. Participants (patients, including representatives from the National Hemophilia Foundation and the World Federation of Hemophilia, healthcare providers, payers, HTA agencies, regulators and industry representatives) condensed and prioritized the list by rating each outcome on a scale of 1 (not important to include) to 9 (essential). Participants could also suggest outcomes for voting. Outcomes were eliminated from consideration if <70 percent rated the outcome from 7–9, unless the patient stakeholder group average score was ≥7.

Results:

After two Delphi rounds, there was consensus on three outcomes: frequency of bleeds, factor activity level, and duration of expression. Additional outcomes included after an in-person consensus meeting were chronic pain, mental health status, and utilization of the healthcare system (direct costs). Adverse events of interest were evaluated and separately reported.

Conclusions:

Including the coreHEM COS in clinical development programs will ensure that relevant, consistent outcomes are available for decisions by HTA agencies, clinicians, and patients. This should result in faster access to novel, high-value therapies for appropriate patients.
PP108 Novel Approaches For Fair And Reasonable Value-Based Recommendations
Authors: Plamondon Geneviève , Auclair Yannick , de Guise Michèle , Beha Sara , Arbour Sylvie , Goetghebeur Mireille , Jobin Nathalie , Nieminen Julie
Published: January 2019

Introduction:

Stakeholders from the innovation field in Québec (Canada) have collectively stressed the need to formalize the process for evaluating innovative technologies in the province. In the context of innovation, and more so for non-pharmaceutical technologies where the pace of development is rapid and the lifecycle short, evidence supporting the added value can be limited and uncertainties are common. Therefore, pragmatic approaches are needed to guide recommendations and to assure that the process is rigorous, transparent and fair.

Methods:

Inspired by international experiences, the Institut national d'excellence en santé et services sociaux (INESSS) has developed a novel framework, where four types of recommendations are possible (introduction, refusal, limited or conditional introduction). The starting point is an evaluation of the technology's added value, for the patient, the population and the healthcare system, and the identification of uncertainties. The value of addressing uncertainty with further research is assessed, based on the value-of-information theory, and the distinct characteristics of medical devices are taken into account (e.g. learning curve effect, irrecoverable costs and incremental innovation). Those elements interact to support the formulation of recommendations by INESSS’ advisory committee.

Results:

The development of the framework was an iterative process supported by the use of the preliminary framework for the assessment of several innovative technologies. Challenges with its use were identified, and led to methodological and operational improvements. So far, the experience with the framework is positive and stakeholders confirm its relevance to support fair and reasonable recommendations for innovations.

Conclusions:

In the rapidly changing landscape of innovation, HTA has to adapt to the challenges of assessing technologies in a context of promise and uncertainties. The framework developed by INESSS is a tool for supporting timely and fair value-based decision-making, which will benefit the healthcare system, and the patients and population it serves.
PP111 Toward Healthy Coagulation In Hemophilia
Authors: Dolan Gerry , Berntorp Erik , Nugent Diane , Skinner Mark
Published: January 2019

Introduction:

Healthcare advances in hemophilia have led to near-normal life expectancies in a disorder previously associated with early death. Unlike other disorders where the therapeutic goal is to restore deficiencies to normal levels, prophylaxis in hemophilia is used to achieve a plasma level of FVIII >1%, such that severe hemophilia may be reduced to a moderate/mild phenotype. With the development of new therapies, treatment goals are evolving from on-demand treatment or prevention of bleeds to one where the risk of bleeding is minimal/absent. To accelerate this development, a new treatment paradigm is needed, with consensus from key stakeholder communities, to facilitate a shared vision for the future of hemophilia healthcare.

Methods:

A panel of hemophilia providers, patient advocates, and industry representatives convened to develop a new treatment model that establishes specific treatment milestones and target outcomes in a stepwise fashion, culminating in a progressive definition of cure.

Results:

To represent the collective experience of hemophilia for patients and treaters around the world, the following treatment milestones were defined based on optimized outcomes: (i) Sustain Life – prevention of premature death; (ii) Minimal Joint Impairment – improved quality of life; participation in activities of daily living; (iii) Freedom From Spontaneous Bleeds – ability to engage in low-risk activities; (iv) Attainment of ‘Normal’ Mobility – participation in work, career, and family life without restriction; (v) Able to Sustain Minor Trauma – more unrestricted lifestyle; (vi) Ability to Sustain Major Surgery or Trauma Without Additional Intervention – no dependency on specialized healthcare; (vii) Normal Hemostasis – optimal health and well-being; and (viii) Cure – health equity.

Conclusions:

With milestones for disease management leading toward normalized hemostasis, this treatment model provides a vision to improve hemophilia care for all patients. And by providing achievable outcomes, the community—patients, treaters, and their industry partners—has a clear path to achieve that goal.
PP114 Clinical Effectiveness Of Insulin Glargine; Findings And Implications
Authors: Marra Lays , Guerra Augusto , Araújo Vânia , Oliveira Gerusa , Diniz Leonardo , Acurcio Francisco , Godman Brian , Alvares Juliana
Published: January 2019

Introduction:

There is continuing controversy surrounding the funding of insulin glargine versus neutral protamine Hagedorn (NPH) insulin in Brazil, due to substantial differences in their prices, and recent meta-analyses of randomized controlled trials and independent observational studies that show no difference in effectiveness; however, sponsored observational studies show greater effectiveness of insulin glargine. Overall, the cost-effectiveness of insulin glargine in Brazil is controversial. In view of the continuing controversy, there is a need to address this using patient level data within the public health system in Brazil.

Methods:

We conducted a retrospective historical cohort study of type 1diabetes patients receiving insulin glargine from January 2011 to January 2015, including patients in the public health system in Minas Gerais. Variables included (i) demographic variables, (ii) clinical variables e.g. time with a diagnosis of type 1 diabetes, (iii) treatment characteristics, and (iv) laboratory results of HbA1c. Individuals were compared with themselves in an analysis of HbA1c values before and after six months of using insulin glargine, with each patient acting as their own control.

Results:

Five hundred and eighty patients were included in the study. HbA1c varied from 8.80±1.98 percent in NPH insulin users to 8.54±1.88 percent after insulin glargine for six months, which is not clinically significant. The frequency of glycemic control varied from 22.6 percent with NPH insulin to 26.2 percent with insulin glargine. No statistically significant difference was observed between groups for all analyzed factors, including type and frequency of insulin use and carbohydrate counting.

Conclusions:

There were limited differences between NPH insulins and insulin analogues in this real world study. As a result, the continued appreciable cost difference in between insulin glargine and NPH insulin in Brazil cannot be justified.
PP137 Colorectal Cancer Screening In The Philippines: Cost-Utility Analysis
Authors: Wong John , Co Stephanie Anne , Bagas Joy , Reyes Ma. Sophia Graciela , Lim Hadrian , Haw Nel Jason
Published: January 2019

Introduction:

Colorectal cancer (CRC) is the fourth leading cause of cancer deaths in the Philippines. In 2014, the Philippine Health Insurance Corporation (PhilHealth) created a CRC treatment package. The study aimed to determine the cost-utility and budget impact of CRC screening strategies.

Methods:

A discrete-event microsimulation model was used to simulate four screening modalities: (i) guaiac-fecal occult blood test (gFOBT) followed by colonoscopy every 10 years; (ii) fecal immunochemical test (FIT) followed by colonoscopy every 10 years; (iii) FIT followed by flexible sigmoidoscopy; and (iv) colonoscopy screening every 10 years. These interventions were all compared to no screening. Parameter values were taken from a rapid review of the medical literature and primary data collection from a nationally representative sample of tertiary hospitals.

Results:

All screening modalities were very cost effective considering that the incremental cost-effective ratios (ICERs) were lower than the gross domestic product per capita threshold suggested by the World Health Organization. Sensitivity analysis showed that the ICERs of all screening modalities evaluated remained below this threshold. The strategy of using FIT followed by colonoscopy every 10 years had an ICER of USD 6,025, with an annual budget impact of USD 6.5 million, assuming low compliance. With moderate compliance this could increase to USD 18.7 million annually.

Conclusions:

PhilHealth may introduce a benefit package for outpatient screening of colorectal cancer using the screening modality of annual FIT followed by colonoscopy every 10 years.
PP142 Is Insulin Therapy Important For The Quality Of Life Of Diabetics?
Authors: Almeida Paulo Henrique Ribeiro Fernandes , Silva Thales Brendon Castano , de Lemos Lívia Lovato Pires , de Assis Acúrcio Francisco , Júnior Augusto Afonso Guerra , de Araújo Vânia Eloísa , Diniz Leonardo Mauricio , Godman Brian , Bennie Marion , Almeida Alessandra Maciel , Álvares Juliana
Published: January 2019

Introduction:

Quality of life (QoL) is an important health measure and is widely used to assess the difference between treatments for Type 1 Diabetes Mellitus (T1DM) since the desirable glycemic control and the minimization of episodes of hypoglycemia are fundamental aspects for a better QoL. This study aims to identify the factors associated with QoL in patients with T1DM.

Methods:

A cross-sectional study (approved by ethics committee) was carried out in the state of Minas Gerais with 401 T1DM patients who used insulin glargine (GLA) selected in March 2017, and 179 patients who used insulin-neutral protamine (NPH) selected between January and February 2014, and both groups were treated by Brazilian National Health System (SUS). A questionnaire with three blocks was used: A) sociodemographic data; B) clinical data and access to the service; and C) QoL by Euroqol (EQ-5D-3L). We used multiple linear regression model by the forward stepwise method to access the correlation between the utilities of the EQ-5D-3L and all the explanatory variables (blocks A and B). We adopted the significance level and confidence interval of 95 percent (95% CI).

Results:

Of the 580 patients evaluated, 54 percent were women, 47 percent were in the age group between 18–40 years, 53 percent reported to be non-black. The EQ-5D-3L analysis showed patients treated with insulin analogue GLA had an average utility of 0.849 and those treated with NPH insulin 0.722 (p < 0.000). Individuals young, very good/good health self-perception, having not been bedridden in the last 15 days, zero to three medical appointments in the last year, no hospitalization in the last year, regular physical activity in the last 15 days to practice physical exercise, having between zero and three comorbidities and no severe hypoglycemia in the last 30 days were explained 41.3 percent of QoL. The type of insulin therapy, GLA or NPH, did not enter into the final multiple regression model.

Conclusions:

The findings of this study pointed to a lack of correlation between insulin therapy and QoL of patients with T1DM. Sociodemographic and clinical factors were more important to explain the QoL of diabetics. In addition, the evidence pointed to the importance of episodes of hypoglycemia for Qol. Of the 191 episodes of hypoglycemia (non-severe and severe) reported, 66 percent were from patients treated with GLA.
PP145 Using Health Technology Assessment To Drive Guideline Development
Authors: Scott Ann , Moga Carmen , Harstall Christa
Published: January 2019

Introduction:

Clinical practice guidelines (CPGs) are a key vehicle for converting evidence into action. CPGs can be produced by various methods: de novo, adaptation, adoption, or a combination of these. Deciding whether and how to develop a guideline can be challenging. Health technology assessment (HTA) researchers from the Institute of Health Economics developed a multi-step decisional algorithm highlighting the decision nodes in the initial phase of guideline development where HTA products and expertise can be valuable in demystifying these decisions.

Methods:

A literature search was conducted for articles comparing methods of developing CPGs, with particular focus on finding a priori criteria for deciding when to use one method versus another.

Results:

The published literature is sparse and there are no specific criteria available for deciding when to use one method of development versus another. The proposed multi-step algorithm identifies similar steps in the production of all types of CPGs: the set-up phase; establishing the need for a new CPG in consultation with a guideline development group and local stakeholders; developing research question(s); conducting searches for suitable existing guidelines; and finalizing the guideline. HTA can help set the health question(s) and identify and screen existing CPGs. When CPGs are not available, HTA methods are implemented to update the evidence in a blend of de novo and adaptation processes by reviewing umbrella reviews, systematic reviews, and primary studies. Quality appraisal of existing guidelines and syntheses of evidence in a rapid review fashion help determine whether there are enough studies to support the guideline scope.

Conclusions:

Deciding which method of guideline development to employ requires ample methodological expertise, an intimate knowledge of the clinical practice environment, and access to detailed contextual information. The proposed multi-step algorithm shows how to successfully leverage HTA resources to support CPG production and move research evidence into practice.
PD42 Safety And Cost-Effectiveness Of Platelet-Rich Plasma For Chronic Wounds
Authors: del Pino-Sedeño Tasmania , Linertová Renata , Trujillo-Martin Maria M. , Toledo-Chávarri Ana , Girardot Sybille Káiser , De Pascual Ana M. , Andia Isabel , Sánchez Javier Aragón , Huerta Dolores Rodríguez , León-Salas Beatriz , Herrera-Ramos Estefanía
Published: January 2019

Introduction:

New therapeutic strategies have been established in chronic wound healing procedures, such as the use of platelet-rich plasma (PRP). There is currently still uncertainty about the effectiveness, cost-effectiveness and real safety of PRP in promoting chronic wound healing and what specific types of chronic wounds can benefit most from its use.

Methods:

We conducted a systematic review of available scientific literature on the effectiveness, safety and cost-effectiveness of PRP compared to placebo, standard care or alternative topical therapies for the treatment of chronic wounds in adults. Overall effect size was estimated through a meta-analysis. A cost-effectiveness analysis was conducted using a Markov model which simulates the costs and health outcomes of individuals for a 5-year horizon, from the perspective of the Spanish National Health Service (NHS) for the PRP versus standard treatment in patients with diabetic foot ulcers. The effectiveness measure was quality-adjusted life years (QALYs). We ran extensive sensitivity analyses, including a probabilistic sensitivity analysis.

Results:

Sixteen RCTs and four observational studies were included for the effectiveness and safety meta-analysis. The primary outcome was the proportion of chronic wounds completely healed: 143 patients out of 334 (42.8 percent) were cured in the standard treatment arm and 251 patients out of 375 (66.9 percent) in the PRP arm, relative risk (RR) 1.68 (95% CI: 1.22–2.31). It was unclear whether there was a difference in the risk of infection (RR 0.53, 95% CI: 0.10–2.71) or adverse events (RR 1.05, 95% CI: 0.29–3.88) between PRP and standard care. Three studies were considered for the cost-effectiveness analysis. In the base case analysis, PRP led to higher QALYs and healthcare costs with an estimated incremental cost-effectiveness ratio (ICER) of EUR 41,767 (USD 48,323)/QALY.

Conclusions:

PRP treatment is more expensive and more effective than standard treatment. The estimated ICER is above the acceptability threshold in Spain.
PD47 Implanted Hypoglossal Nerve Stimulation For Obstructive Sleep Apnea
Authors: Orrego Juan-Pablo Chalco , Polo-Desantos Mar , Sánchez-Gómez Luis María , Luengo-Matos Setefilla
Published: January 2019

Introduction:

The hypoglossal nerve stimulation (HNS) produces a tongue protrusion for the treatment of mod-severe obstructive sleep apnea (OSA). It is one of the emerging health technologies prioritized to assess its possible inclusion on the Spanish National Health System. The objective of this study is to evaluate the efficacy and safety of this system in the treatment of OSA.

Methods:

An early assessment (horizon scanning) was performed. The searched databases were: PubMed, WOS, Tripdatabase, Dynamed, Cochrane Library and ICTRP. Clinical studies of OSA patients treated with HNS published until 01 March 2017 were reviewed. Outcomes considered were: AHI (Apnea Hypopnea Index) ODI (Oxygen Desaturation Index) ESS (Epworth sleepiness scale) and AE (adverse events).

Results:

Four devices of HNS were founded: Inspire™, HGNS®, Aura6000™, and Nixoah™. We found two randomized controlled trials (RCT). The Inspire™ RCT showed significant results on mean differences on AHI (−16.9, 95% CI −24.7 to −9.0); ODI (−15.1, 95% CI −22.7 to −7.5) and ESS (−4.5, 95% CI −7.5 to −1.4) in 46 patients, after one week of follow-up. The HGNS® RCT showed non-significant differences on AHI (device active 22.1 ± 5.2 vs control 29.7 ± 6.2), ODI (11.4 ± 4.1 vs 19.5 ± 5.2) and ESS (9.8 ± 1.0 vs 14.1 ± 2.5) in 21 patients at 6 months. A systematic review that included 6 cases series (3 with HGNS®, 2 with Inspire™ and 1 with Aura6000™) without device subgroup analysis and 7 cohorts studies (6 with Inspire™ and one with Aura6000™) showed significant differences comparing AHI, ODI and ESS results to before treatment values. Major AE reported from the studies varied from 4 to 4.5%. No study with Nixoah™ was found.

Conclusions:

Inspire™ seems to be an effective option for OSA patients although the evidence is scarce and of low quality for all HNS devices. It would be necessary further well-designed studies.