bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP40 HTA Evaluations Of Combination Drugs: Positive Reimbursement Solutions
Authors: Solaman Dilani Angela , Walker Andrew , Alba Salus , Edoo Hanim , Jommi Claudio , Ratcliffe Mark 
Published: January 2019

Introduction:Health technology assessments (HTA) for combination drug therapies in oncology are increasingly common. Companies face multiple challenges when determining their economic value due to their complexity and high cost, while payers must balance the need for these vital innovations with sensitivity to rising costs. The study objective was to evaluate the current HTA frameworks in Europe and identify the potential barriers/solutions to reimbursement of brand-on-brand (BoB) combination therapy.Methods:A targeted literature review of HTA agency websites was undertaken to identify any literature/guidance relating to HTA decision-making for combination oncology therapies in France, Germany, Sweden, and the UK.Results:In France and the UK, BoB HTA decisions reflect clinical- and cost-effectiveness. Combination therapies have been accepted for use in France and the UK, for example, dabrafenib plus trametinib, are assessed through standard HTA processes, exemplifying that positive reimbursement is not unattainable where there is an unmet need and high clinical value. Despite this flexibility, many therapies will fail to prove their cost-effectiveness, resulting in delays or arbitrary pricing decisions. Potential solutions are the use of the ‘efficiency frontier’, as typified by the German HTA system, giving more ‘scope‘ to expensive innovations; or the Swedish HTA approach, which applies variable cost-effectiveness thresholds according to therapeutic area, disease severity, and social criteria. Other possibilities include indication-specific pricing, multiple-criteria decision analysis, and net monetary benefit with willingness-to-trade weights. One likely issue to arise is when different companies are involved, necessitating co-operation. In this scenario, a simplistic solution would be arbitration of the division of the combined price, circumventing the need for HTA agencies to make changes to decision-making criteria.Conclusions:Constructive debates and collaboration between industry and decision-makers are vital to achieve a harmonized HTA process for high-cost combination therapies which offer advanced benefits and improved safety outcomes, whilst satisfying HTA bodies and providing better access for patients.
PP46 When Composite Measures Or Indices Fail: Data Processing Lessons
Authors: Chao Yi-Sheng , Wu Chao-Jung 
Published: January 2019

Introduction:Index mining is a new discipline that aims to search for the composite measures or indices most relevant to the contexts or outcomes. After reviewing three frailty indices and principal component (PC)-based indices, we hereby show certain occasions that can lead to ineffective indices, which consist of bias or fail to represent the theories.Methods:We reproduced and reviewed the three frailty indices and the 134,689 PC (principal component) -based indices from previous publications. The impact of aggregating the input variables on the final indices was analyzed using forward stepwise regression.Results:Several methods to combine the input variables were related to ineffective projection of information onto the indices. The most common causes leading to ineffective summation of input variables were shown in three examples involving different types of input variables, which were positively or negatively correlated or uncorrelated to the outcome. Ineffective indices were created often because of the summation of redundant information or uncorrelated variables.Conclusions:The creation of ineffective indices can be avoided if the relationships between input variables and outcomes are properly scrutinized. The creation of composite measures and indices is still a discipline under active development. The three examples we identified are the mistakes that may be repeated unintentionally and need to be addressed with explicit rules. A reporting guide for the creation of composite measures has been proposed. A proper review of index objectives, data characteristics, and data limitations before creating composite measures or indices is recommended.
PP60 Producing Qualitative Syntheses In Health Technology Assessment: Challenges From The Canary Islands
Authors: Toledo-Chávarri Ana , Booth Andrew 
Published: January 2019

Introduction:With heightened awareness of the value of patient and provider perspectives to decision making, Qualitative Evidence Synthesis (QES) is increasingly used within a health technology assessment (HTA) context. Acceptability, feasibility and implementation can all be addressed by synthesis of qualitative research. Concerns have been raised about the quality of the synthesis product, especially when conducted within a constrained time window. How can we test the validity of qualitative studies and assess confidence in synthesized qualitative findings, particularly when time is tight?Methods:A brief examination of issues relating to production and use of QES identified from within the Canary Islands HTA agency will identify practical and methodological challenges. How can existing approaches address wider patient, social, organizational and ethical considerations that inform HTA? The potential for use of Evidence To Decision frameworks and approaches such as GRADE CERQual (a transparent method for assessing the confidence of evidence from reviews of qualitative research) will be briefly examined.Results:This presentation will identify potential gaps between the needs of a small HTA agency and the methodological support and tools required to address these gaps, based on experience of conducting QES to date. Issues identified are particularly relevant to other small HTA agencies but are also generalizable to larger agencies and guideline producers worldwide. Pragmatic solutions are suggested. A future research agenda for potential methodological and applied research is outlined and current GRADE-CERQual development initiatives briefly shared.Conclusions:Despite significant progress in developing methodologies for integrating QES within HTA decision making, substantive challenges remain. Observations derived from this small HTA agency can inform further developments across all HTA organizations. Research is required to examine the impact of potential dissemination bias, application of tools across a wider HTA decision making framework and use of rigorous approaches within a time-limited evaluation window.
PP62 A Guide To Report And Review Innovative Indices Or Composite Measures
Authors: Chao Yi-Sheng , Wu Chao-Jung 
Published: January 2019

Introduction:Composite measures and indices are used in medical research to represent certain concepts that cannot be measured with one variable. They can be used to predict outcomes or serve as outcomes in trials. The creation of innovative indices is important to increase publications and secure research funding. However, some assumptions and problems are prevalent among indices. We aim to develop a reporting guide and an appraisal tool for indices based on the issues we identified.Methods:We reproduced the three frailty indices from a previous publication and 134,689 principal component-based indices. We reviewed the index assumptions, bias introduced by data processing, relationships between input variables. We interpreted the indices with input variables.Results:We identified four major issues to be addressed in a reporting guide: constraints imposed by index creation on the input variables; data processing without evidence base; indices poorly linked to input variables; and, relatively inferior predictive power. We demonstrated a flow diagram and a checklist to report and review these four issues related to innovative indices.Conclusions:A reporting and critical appraisal tool for innovative indices is lacking and needed. These four issues that need to be explicitly considered are previously neglected. This guide is the first attempt to improve the quality and generalizability of innovative indices. This guide can be used to lead further discussion with other experts and review committees.
PP70 Health Technology Assessment Evaluations Of Combination Drugs: The Italian Case Study
Authors: Solaman Dilani Angela , Jommi Claudio , Walker Andrew , Edoo Hanim , Ratcliffe Mark 
Published: January 2019

Introduction:Health technology assessment (HTA) must adapt to support the changing health system landscapes and improve access to valuable innovation under budgetary constraints. This is exemplified by the pricing and reimbursement of high-cost combination therapies increasingly used in oncology. Variability exists in current HTA practices across different countries, resulting in discrepancies in reimbursement outcomes and patient access. Using Italy as a case study, the objective was to assess the challenges faced by HTA agencies in the negotiation of pricing and reimbursement of combination therapies.Methods:A targeted literature review of Italian HTA agency websites was undertaken to identify any literature/guidance relating to HTA decision-making for combination oncology therapies.Results:In Italy, there is no fixed cost-effectiveness threshold and decisions are based on multiple criteria. Managed market entry agreements are extensively used; price-volume agreements and drug registries are common. While this framework allows flexibility and avoids the rigidity of incremental cost-effectiveness ratio thresholds, it has raised concerns about transparency and budget impact. Combination therapies are not given specific concessions; however, market access for a combination of a new high-cost drug with an existing one is complex, particularly if the drugs are manufactured by different companies. The added value provided by the new drug in the combination should be rewarded while the older product benefits from the increased volume of use. The price of the older drug cannot be lowered unless the pricing and reimbursement contract is expiring or a new indication/formulation is pending, presenting a challenge to both pharmaceutical companies and HTA agencies.Conclusions:Combination therapies pose a challenge for HTA agencies. In the Italian system this is partially mitigated by the use of multiple criteria for decision-making and managed access agreements. However, these approaches have also led to concerns about a lack of transparency in decision-making.
PP86 Impact Of Health Technology Assessment On Drug Price Negotiations: Canada
Authors: Rocchi Angela , Mills Ferg 
Published: January 2019

Introduction:Subsequent to review by Canada's two central health technology assessment (HTA) agencies, confidential drug prices are negotiated by the pan-Canadian Pharmaceutical Alliance (pCPA) on behalf of public drug plans. This analysis is the first to examine characteristics of drugs considered for negotiation, and the duration of negotiations, from inception in 2011 to August 2017. The objectives were to identify how HTA recommendations impacted price negotiations, and in particular the role of health economics in the process.Methods:The dataset contained 208 drug indications from the pCPA archives: those with a decision to negotiate (n=155) or a decision not to negotiate (n=53). Data were abstracted from the publicly-maintained websites of the respective agencies; descriptive statistics were conducted.Results:There was close but imperfect alignment between the HTA agency listing recommendation and the pCPA's decision to negotiate. The incremental cost-effectiveness ratio (ICER) of negotiated drugs (as estimated by HTA agencies) approached CAD 200,000/QALY (i.e. USD 157,000) for oncology drugs, but was closer to CAD 100,000/QALY (i.e. USD 78,000) for non-oncology drugs, revealing that negotiations would require a substantial discount to achieve conventionally ‘acceptable’ value-for-money. ICERs were influential to non-oncology drug recommendations (and were increasingly used to set pCPA negotiation targets) but did not appear to influence oncology drug HTA recommendations. The time period required to initiate negotiations was dramatically shorter for oncology versus non-oncology drugs (53 versus 263 days), and also differed markedly between therapeutic areas. The time period for pCPA activities was surprisingly similar for drugs recommended without a price condition and for those conditional on a price reduction.Conclusions:These findings revealed an implicit prioritization pattern at the pCPA, as well as the evolving role of health economics in Canada's two-stage reimbursement process.
PP89 Living Lab Concept: An Innovation Hub For Elderly Residential Care
Authors: Szczepura Ala , Collinson Mark , Moody Louise , Jing Yanguo , Ward Gill , Bul Kim , Arnab Sylvester , Asbury Christine , Russell Ed , Gibbons Charley , Dashwood Richard 
Published: January 2019

Introduction:Many countries face the challenge of an aging population. Development of suitable technologies to support frail elderly living in care homes, sheltered housing or at home remains a concern. Technology evaluation in real-life conditions is often lacking, and randomized controlled trials of ‘pre-designed’ technologies are expensive and fail to deliver. A novel alternative would be ‘living labs’-real-life test and experimentation environments where users and producers co-create innovations and large-scale data can be collected.Methods:The goal of the living labs and Data Driven Research and Innovation (DDRI) Programme is to use data driven analytics and insights to support technology development for independent living, healthy aging and more cost-effective care. This involves a cluster of long-term residential care facilities providing 24/7 living lab settings, linked to an embedded innovation hub. DDRI also encompasses private vehicles (e.g. sensors in cars) to enable elderly to drive safely for longer. Collaborations have been established with Universities in England, Scotland and Ireland and with international industry partners.Results:Several projects are underway: (i) develop machine learning algorithm from non-intrusive sensor data to build a well-being representation for individual residents/citizens; (ii) evaluate innovative interventions for good sleep environment and nutritional support; and (iii) establish ethics framework to ensure that needs of residents, families and staff are embedded in design, communication, and evaluation of future DDRI projects. In addition, fifteen interdisciplinary doctoral fellowships are in place, six universities are working closely with individual living lab settings, and an innovation hub has been established in one care home for horizon-scanning and strategic technology selection and implementation.Conclusions:Over the next five years, a national network of 20 residential living labs with over 1,500 participants will be established. Generation of new user-led technologies, blueprints for capture of individual data at significant scale, and ethical and organizational guidelines will be developed. Intelligent mobility via data capture/feedback in vehicles will be established.
PP98 Efficacy Of The Multi-Attribute Utility Instruments To Reflect Quality Of Life Of Cancer Patients
Authors: Hernandez-Villafuerte Karla , Ubels Jasper , Carreno Diego Andres Hernandez , Schlander Michael 
Published: January 2019

Introduction:Health state utilities measured by the generic multi-attribute utility instruments (MAUIs) differ. Empirical evidence suggests that some MAUIs are more sensitive than others in reflecting the quality of life (QoL) of patients in particular disease areas. Additionally, in order to estimate utilities based on cancer-specific health-related quality of life instruments (CSQoLs), a number of mapping functions have emerged. Although it is common practice to apply a CSQoL instead of a MAUI in clinical trials, CSQoL cannot be used to estimate utility values for economic evaluations. Mappings based on MAUIs that are not sensitive to changes in cancer patients’ QoL may result in misleading approximations of utilities that could affect allocation of resources. The study objective is to explore the validity and sensitivity of the major MAUIs to variation in the QoL measured by cancer-specific instruments. We aimed to investigate (i) the sensitivity of the general MAUIs scores to changes in the CSQoL, and (ii) whether particular dimensions of the general instrument are more sensitive.Methods:A two stage systematic literature review is conducted. First, an update of the review done by McTaggart-Cowan et al. (2013) on the mapping methods used to determine utilities from cancer-specific instrument. Second, an analysis of studies that measure the relationship between CSQoLs and general MAUIs.Results:The literature suggests that differences exist between MAUIs in their capacity to capture the QoL dimensions of the CSQoLs. Additionally, the main challenge to build an appropriate mapping function for deriving utilities values from CSQoL is the definition of an appropriate methodology that (i) responds to the distribution of the selected sample and (ii) can successfully be validated in additional samples.Conclusions:In the context of health technology assessment and cost effectiveness analysis, it is crucial to carefully select and report the CSQoL and MAUI involved in the estimation of the additional benefits. Policy makers need to be awarded of the sensitivity of the instruments to changes in QoL in relation to the CSQoL dimensions QoL.
PP100 Rapid Evidence Assessment In Hospital Health Technology Assessment
Authors: L'Espérance Sylvain , Rhainds Marc , Nourrisat Alice , Coulombe Martin , Bussières Sylvain 
Published: January 2019

Introduction:Systematic reviews (SRs) are the most valid and reliable scientific evidence to evaluate the effectiveness of healthcare interventions. However, substantial resources and months are required to conduct such a review. Most hospital-based health technology assessment (HB-HTA) units don't have the time and the academic team to produce SRs. Rapid evidence assessment (REA) may represent, in this local context, an interesting avenue. The aim was to evaluate characteristics of REA and their impacts on healthcare decision making.Methods:A SR was performed in several databases and grey literature to search data on REA including Mini-HTA and rapid reviews methodologies through March 2017. Data selection, extraction and quality assessment were performed by two independent researchers. Outcomes were about REA's methodology including question, search strategy, inclusion criteria, study selection, data extraction, quality assessment, critical appraisal and impacts on decision making.Results:Twelve publications on REA have been included. More similarities were found in the methodology between rapid review and SR than with Mini-HTA. Shortcuts in performing rapid reviews included evaluation scope, number of databases, gray literature websites, studies design mainly SR, reviewers number, critical appraisal and production time (3 to 6 months). Study selection and data extraction by two independent reviewers in rapid reviews were seen in thirty-four percent to thirty-eight percent and ten percent to twenty-two percent, respectively. Furthermore, assessment quality was optional. Although it is performed within a short timeframe (2 months), methodology to conduct Mini-HTA is not well defined in the literature. The scope is mainly to support decision making in the introduction of new medical devices. Impacts of REA on local health decision making process are not well documented.Conclusions:Methodology to conduct REA is quite diverse. According to the data available, rapid review is a more robust methodology for HB-HTA producers than Mini-HTA. Although impacts were not well reported, rapid reviews could be more useful to support health decision making in local context.
PP115 A Mobile Health App To Improve Knee Osteoarthritis Self-Management
Authors: Marshall Deborah , Barber Tanya , Shewchuk Brittany , Mrklas Kelly , Miller Jean , Teare Sylvia , Green Lee , Faris Peter , Cibere Jolanda , Sharif Behnam 
Published: January 2019

Introduction:A gap exists between the evidence for reducing risk of knee osteoarthritis (KOA) progression and its application in patients’ daily lives. We aimed to bridge this gap by identifying patient and family physician (FP) self-management priorities to conceptualize and develop a mobile-health application (m-health app). Our co-design approach combined priorities and concerns solicited from patients and FPs with evidence on risk of progression to design and develop a KOA self-management tool.Methods:Parallel qualitative research of patient and FP perspectives was conducted to inform the co-design process. Researchers from the Enhancing Alberta Primary Care Research Networks (EnACT) evaluated the mental models of FPs using cognitive task analysis through structured interviews with four FPs. Using grounded theory methods, patient researchers from the Patient and Community Engagement Research (PaCER) program interviewed five patients to explore their perspectives about needs and interactions within primary care. In three co-design sessions relevant stakeholders (four patients, five FPs, and thirteen researchers) participated to: (i) identify user needs with regard to KOA self-management; and (ii) conceptualize and determine design priorities and functionalities of an m-health app using a modified nominal group process.Results:Priority measures for symptoms, activities, and quality of life from the user perspective were determined in the first two sessions. The third co-design session with our industry partner resulted in finalization of priorities through interactive patient and FP feedback. The top three features were: (i) a symptoms graph and summary; (ii) information and strategies; and (iii) setting goals. These features were used to inform the development of a minimum viable product.Conclusions:The novel use of co-design created directive dialog around the needs of patients, highlighting the contrasting views that exist between patients and FPs and emphasizing how exploring these differences might lead to strong design options for patient-oriented m-health apps. Characterizing these disjunctions has important implications for operationalizing patient-centered health care.