HTA Methods Research Articles

OP39 Adapting HTA To Suit Emerging Needs - An Australian Experience

IntroductionAustralia has a well-developed HTA system for approving, funding and disinvesting in medical services, but how does it cope when it assesses new or existing services when health system frameworks, policy and legislation do not keep pace? This presentation will present a number of case studies where HTA methods have adapted to suit emerging health needs of the Australian community.MethodsAustralia's HTA system has been adapted over recent years to allow it to perform HTA on novel services that do not fit into its standard HTA pathway to public funding. There has also been an increasing number of assessments where HTA has been unusually sponsored by the funder to assess priority health care needs.ResultsMore Medicare funding for addiction and sexual health consultation services are an example of a novel HTA which led to more public funding for these services. Limited evidence for these services was available and there was difficulty in demonstrating that increased public funding would lead to better outcomes for patients. A range of techniques, such as examining real world data and stakeholder views were partnered with HTA to assess these services. Currently, HTA is being utilised to assess a novel medical treatment known as the anti-CD19 chimeric antigen receptor T cell (CAR-T) therapeutic process. This is a complex, non-standard HTA which encompasses aspects of the Australian hospital funding system, Medicare and the Pharmaceutical Benefits Scheme and requires an adapted HTA process to assess evidence across a range of funder systems.ConclusionsAustralia's well established HTA system has adapted to become more agile to suit emerging health care needs for a range of interest groups, the government, sponsors and consumers. Consultation with stakeholders and the community have assisted in developing and refining these new processes.

Preventing Human Error on Online Transaction (A Case Study of B.com)

Buying and selling transactions using internet media has advantages related to time and costs. However, buyers often feel difficult when accessing online websites. There are several types of errors that are experienced by buyers when using online buying and selling site services. It is including mistakes in selecting display menus, difficulties in finding items needed because there are too many choices available, errors in interpreting menus used, and sometimes difficulties in knowing product specifications because no relevant information is available on the site. In this study, we discuss the application of the HTA and SHERPA method to assess one of the online buying and selling sites currently used by Indonesian people, namely B.com. There are 100 respondents participated in this study. The study methods are including error identification, analyzing the error using SHERPA, and evaluating the website design. The result of the study provides some recommendation to the online buyer such as ensuring the quality of internet network, filling the data carefully, and confirming the purpose and nominal value of the transaction before it sent to the seller.

PHP135 - Is Eunethta Enough? The Remaining Challenges in Navigating the Early European Regulatory and HTA Advice

In July 2017, the European Network for Health Technology Assessment (EUnetHTA) and the European Medicines Agency (EMA) began offering manufacturers simultaneous, coordinated regulatory and payer/HTA advice. This research reviews the available early dialogue services in major European markets, providing an overview of payer/HTA advice and highlights the remaining challenges for companies seeking to incorporate early HTA input. Publically-available data on early dialogue services provided by regulatory, payer and HTA bodies in Europe were reviewed, comparing the costs, duration, processes and outputs. The EMA and some European payer/HTA agencies have provided individual scientific advice since 2004. However, individual/national agency advice varies in duration (8-24+ weeks), processes (e.g., expertise used), price (~€2,000-€80,000) and outputs (e.g., report vs. meeting minutes). Stand-alone advice also requires separate company submissions. Parallel EMA/HTA advice was set up in 2011 to simplify the process of seeking pan-European advice requiring only a single document submission to all agencies. Despite the number of parallel advice projects steadily increasing since 2011, several areas of regulator and payer/HTA misalignment have been reported. The manufacturers were also left to consolidate individual agency reports. In a move to provide more coordinated advice, EUnetHTA Consolidated-Parallel-Consultation (EUnetHTA-CPC) builds on EMA/HTA parallel advice and provides a joint report of all agency positions, highlighting key differences. Despite joint advice output, the agencies nonetheless rely on different methodologies, often also reflecting variations in treatment pathways and/or comparator preferences. Early advice options have improved significantly since 2011, with EUnetHTA-CPC procedure further supporting companies accessing the European market. However the challenges in reconciling differences in HTA methods and preferences remain. Furthermore, while EUnetHTA provides an overview of HTA opinions, it does not match the depth of advice provided by individual national advice, adding to the challenge of choosing the optimal early dialogue procedure(s).

OP90 Do Patients With Open Fractures Need Urgent Surgery?

Introduction:Calling in staff and preparing the operating room for an urgent surgical procedure is a significant draw on hospital resources and disrupts care of other patients. It has been common practice to treat open fractures on an urgent basis. HTA methods can be applied to examine this prioritization of care, just like they can be applied to the acquisition of drugs and devices.Methods:Our center completed a rapid systematic review of guidelines, systematic reviews, and primary clinical evidence, on urgent surgical debridement and stabilization of open fractures of long bones (“urgent” being defined as within six hours of the injury) compared to surgical debridement and reduction performed at a later time point. Meta-analyses were performed for infection and non-union outcomes and the GRADE system was used to assess the strength of evidence for each conclusion.Results:We found no published clinical guidelines for the urgency of treating open fractures. A good systematic review on the topic was published in 2012. We found six cohort studies published since completion of the earlier review. The summary odds ratio for any infection in patients with later treatment was 0.97 (95% confidence interval (CI) 0.78–1.22, sixteen studies, 3,615 patients) and for deep or “major” infections was 1.00 (95% CI 0.74–1.34, nine studies, 2,013 patients). The summary odds ratio of non-union with later treatment was 0.95 (95% CI 0.65–1.41, six studies, 1,308 patients). There was no significant heterogeneity in any of the results (I-squared = 0 percent) and no apparent trends in the results as a function of study size or publication date. We graded the strength of each of the conclusions as very low because they were based on cohort studies where the treating physician could elect immediate treatment for patients with severe soft-tissue injuries or patients at risk of complications. This raises the risk of spectrum bias.Conclusions:Default urgent scheduling of patients with open fractures for surgical debridement and stabilization does not appear to reduce the risk of infection or fracture non-union. Based on this information, our surgery department managers no longer schedule patients with open fractures for immediate surgery unless there are specific circumstances necessitating it.

Adoption of the HPV vaccine: a case study of three emerging countries.

The human papillomavirus (HPV) vaccine has recently attracted considerable attention in emerging countries, due to its potential to reduce the impact of HPV-related diseases. This case study sheds new light about the variety of HTA arrangements, methods and processes involved in the adoption and use of HPV vaccines in a selected sample of central, eastern and southern Europe and Latin America and the Caribbean, all of them emerging in the use of HTA. A multi-country case study was designed. Mixed methods, document review, semi-structured surveys and personal communication with experts, were used for data collection and triangulation. This study shows that common elements of good practice exist in the processes and methods used, with all countries arriving at the same appraisal recommendations. However, the influence of socio-politico-economic factors appears to be determinant on the final decisions and restrictions to access made. This case study intends to draw useful lessons for policymakers in emerging settings interested in the adoption of the HPV vaccine supported by evidence-informed processes, such as those offered by institutionalized HTA. Future studies are also recommended to elucidate the specific roles that social values and uncertainties play in vaccine decision-making across different societies.

Challenges in the Assessment of Medical Devices: The MedtecHTA Project.

Assessing medical devices (MDs) raises challenges which require us to reflect on whether current methods are adequate. Major features of devices are: (i) device-operator interaction can generate learning curve effects; (ii) incremental nature of innovation needs to be addressed by careful identification of the alternatives for comparative and incremental cost-effectiveness analysis; and (iii) broader organizational impact in terms of training and infrastructure, coupled with dynamic pricing, requires a more flexible approach to costing. The objective of the MedtecHTA project was to investigate improvements in HTA methods to allow for more comprehensive evaluation of MDs. It consisted of several work packages concerning (i) the available evidence on the currently adopted approaches for regulation and HTA of medical devices; (ii) the geographical variation in access to MDs; (iii) the development of methodological frameworks for conducting comparative effectiveness research and economic evaluation of MDs; and (iv) the organizational impact of MDs. This introductory paper summarizes the main results of the project and draws out the main overarching themes. This supplement represents a comprehensive report of all the main findings of the MedtecHTA project, and it is intended to be the main source for researchers and policy makers wanting information on the project. © 2017 The Authors. Health Economics published by John Wiley & Sons, Ltd.

VP147 Implementing Electronic Health Record In A Children's Hospital

INTRODUCTION:Since the adoption of electronic health record (EHR) systems, which contain large volumes of aggregated longitudinal clinical data, promises a number of substantial benefits including better care, improved safety issues and decreased healthcare costs (1). It is also associated with significant costs and large technical and organizational impacts, therefore it is important to conduct comprehensive evaluations of healthcare delivery outcomes. The purpose of the study is to gather evidence on safety and overall effectiveness of EHR implementation in Bambino Gesù Children's Hospital (OPBG).METHODS:Decision-oriented HTA (DoHTA) method (2) was applied to assess the technology on clinical, technical, organizational, economic, legal, ethical and safety domains. It's a new implementation of the European Network for Health Technology Assessment (EUnetHTA) CoreModel integrated with the Analytic Hierarchy Process. It allows defining an evaluation structure represented by a hierarchical decision tree filled by indicators of technology's performances, each of which was given a weight proportional to the impact that this criterion provides to achieve the purpose of the decision problem; finally, the alternatives’ ranking was defined.RESULTS:The multidisciplinary assessment took into consideration all of the aspects and recommendations about the benefits and disadvantages of EHR (3). The synthesis of scientific evidence integrated with results of the specific context analysis, resulted in the definition of components of the decisional hierarchy structure. In particular, EHR seems to offer many benefits in terms of safety and clinical effectiveness such as improved continuity and quality of care, and increased accessibility of the data. The implementation of EHR resulted in important organizational outcome such as EHR configuration, learning curve and training. For these reasons, the usability was the main technical characteristics of the technology taken into account. Finally, legal aspects on privacy and security of data, covered a key role in the assessment.CONCLUSIONS:A thorough evaluation of the EHR before its implementation has permitted hospital's decision makers to choose knowingly.

PHP244 - Cost Minimisation Analysis: When And Where? A Review Of Hta Guidance On Cost Minimisation Analysis

Cost minimisation analysis (CMA) is a form of economic evaluation advocated in cases where products have equal efficacy. Whilst there is consensus on this point, the criteria for establishing equal efficacy varies by source. Additionally, the availability of guidelines on the acceptability of CMA is not always available from HTA bodies. This study reviewed HTA guidelines to determine: 1) if guidelines on the use of CMA are available; and 2) how consistent requirements are between HTA bodies. Searches of 10 HTA body websites were undertaken. The following terms were applied: ‘cost-minimisation’ or ‘cost minimisation’ or ‘cost-minimization’ or ‘cost minimization’. Additionally, available guidelines on HTA methods were reviewed. Results were hand searched to identify those reporting methods for CMA. Where guidelines was reported the definition of equal efficacy was extracted. Of the 10 HTA websites reviewed seven had readily accessible details on CMA. In two instances this was simply a definition of CMA whilst the remaining five provided details of where CMA is considered an appropriate methodology. Of these there were two common definitions, those that focused purely on clinical efficacy and those that broadened the definition to include patient outcomes in addition to clinical outcomes. Only the guidelines published by PBAC in Australia reported detailed criteria for meeting clinical equivalence. This review aimed to identify availability of guidelines on CMA methodology. Three bodies did not report any information, although in the case of some this may be driven by the nature of the products assessed. Where details were given there was a lack of consistency in the criteria for CMA, with the issue of whether only considering clinical efficacy or also incorporating broader patient outcomes needing consistency. Finally, the exact criteria for equivalence were only stated by PBAC.

Human error identification and risk prioritization in overhead crane operations using HTA, SHERPA and fuzzy VIKOR method

Human error identification and subsequent prioritization are the foremost tasks involved in HRA. In this study a methodology is developed for performing these tasks with an application to overhead crane operations. The application of the present methodology will help to understand how the risk associated with the human errors propagates through different hierarchy levels. The methodology provides a framework for quantifying the risk of different human errors using the experts’ subjective opinions only. The incorporation of fuzzy VIKOR technique enables us develop a ranking mechanism for the failure modes where the individual constituent components are non-commensurable in nature. The developed ranking mechanism helps the decision makers in optimal allocation of safety critical resources, used for risk mitigation purposes.

PSY106 - To What Extent Do Disease and Treatment Characteristics Influence Hta-Based Recommendations for A Sample of Orphan Drugs In Three Countries, and Could These Indicate Whether Orphan Drugs Have A “Special Status”?

Routine HTA methods may not adequately capture all the important considerations of a treatment’s value and the impact of the condition on the patient given that evidence is often incomplete. This study aims to explore the influence of broader considerations of scientific and social value judgments on reimbursement decisions for a sample of orphan drugs. To identify and compare the extent to which these broader considerations not captured by the incremental cost-effectiveness ratio (ICER) influenced HTA decision-making process in three countries; and, on this basis, explore whether orphan drugs have a “special status”. Countries included were England, Scotland and Sweden. Ten drug-indication pairs with EMA orphan designation and all appraised by NICE were selected. Publicly available HTA reports were coded using thematic analysis to systematically identify and compare these broader considerations across countries using an existing analytical framework. 108 different other considerations were identified and grouped into 15 clusters based on the information provided. The most common related to the nature of the disease, and considerations based on rarity or unmet need. 52% were one of the main reasons for the decision, and in some cases, were also a pivotal factor in accepting high and uncertain ICERs. Categorising these as social or scientific value judgments was done to identify areas where further elicitation of societal preferences, and where more consistency and transparency in their use are needed, respectively. Each of these was then compared to determine whether they pertained specifically to orphan drug or rare disease characteristics. Considerable variation was seen in the application of these broader considerations. Identifying these is a way forward to highlight areas where more research, or consistency and transparency are needed. Some of these other considerations may also favour orphan drugs, furthering the debate around whether orphan drugs deserve special status.

Proposed amendments to NICE's HTA methods criticised

Proposed amendments to NICE's HTA methods criticised

European network for Health Technology Assessment Joint Action (EUnetHTA JA): a process evaluation performed by questionnaires and documentary analysis.

The European network for Health Technology Assessment Joint Action (EUnetHTA JA) project's overarching objective was to 'establish an effective and sustainable HTA [Health technology assessment] collaboration in Europe that brings added value at the regional, national and European level'. Specific objectives were to develop a strategy and business model for sustainable European collaboration on HTA, develop HTA tools and methods and promote good practice in HTA methods and processes. We describe activities performed on behalf of the National Institute for Health Research HTA programme; evaluating the project processes and developing a data set for a registry of planned clinical studies of relevance to public funders. Annual self-completion online questionnaires were sent to project participants and external stakeholders to identify their views about the project processes. Documentary review was undertaken at the project end on the final technical reports from the work packages to examine whether or not their deliverables had been achieved. The project's impact was assessed by whether or not the deliverables were produced, the objectives met and additional 'added value' generated. The project's effectiveness was evaluated by its processes, communication, administration, workings of individual work packages and involvement of external stakeholders. A two-stage Delphi exercise was undertaken to identify the data elements that should be included in a registry of planned clinical studies of relevance to public funders. The data set was validated by an efficacy testing exercise. High response rates were achieved for the questionnaires sent to project participants and this was attributed to the evidence-based strategy implemented. Response rates to questionnaires sent to external stakeholders were disappointingly lower. Most of the high-level objectives were achieved, although applying the developed tools in practice will be implemented in the European network for Health Technology Assessment Joint Action 2 (EUnetHTA JA2). Most work packages produced their planned deliverables. Networking emerged as one of the main benefits of the project and face-to-face meetings were important. However, the overarching objective did not appear to have been met because there will be a follow-up EUnetHTA JA2 project (reliant on project funding) before the establishment of any permanent network. Twelve organisations from three continents participated in the Delphi exercise to develop the data set. It was demonstrated that a registry for matching pragmatic clinical studies under consideration by funders could be built on a very small data set. This would include 10 unique items, of which five are required to describe a study and the rest are metadata. In the test sample the data set with an appropriate matching rule was able to deliver a sensitivity of between 50% and 100% and a specificity of between 43% and 86% for matching different elements. A number of recommendations have been made for the next EUnetHTA JA2 project and its evaluation. This included that the evaluation of the EUnetHTA JA2 project should extend beyond the end of the project to allow assessment of its impact; that the quality, usability and cost-effectiveness of tools in 'real-world HTA practice' should be assessed and tangible benefits of international networking should be evaluated. It is worth proceeding to develop a database registry aimed at identifying trials in development based on the data set developed. The study was funded by the National Institute for Health Research Health Technology Assessment programme (50%) and the European Union Commission (50%).

PCN85 - The Analysis of Costs and Reimbursements for Lung Cancer Treatment in the Czech Republic

Acknowledgement The authors acknowledge financial support for the development of HTA methods by the Ministry of Health of the Czech Republic under Grant No. NT11532 „Medical Technology Assessment“.

HT1 - Inter-Country Variability In Coverage Decisions For Orphan Drugs: Criteria Driving Hta Recommendations In Six Countries

Inter-country variability in access to orphan drugs across countries has been highlighted in a number of studies. Understanding the reasons driving coverage decisions is a way forward in identifying areas where HTA methods may be improved. Objectives are three-fold: a) to establish a methodological framework enabling to systematically compare HTA processes across countries; b) to identify the criteria driving HTA recommendations for a sample of orphan drugs, and; c) to understand the reasons for diverging recommendations and propose ways to minimize these. All common orphan drug-indication pairs appraised in six countries (England, Scotland, France, Sweden, Canada and Australia) between 2001 and 2012 were selected. Agreement levels in HTA outcomes between countries were measured using Cohen’s kappa scores. Thematic analysis, by creating an NVivo-9 coding manual, was conducted to systematically compare each compound. Reasons for diverging HTA outcomes were differentiated based on whether they are a consequence of country-specific considerations or of the HTA process, and ranked by frequency of occurrences. Fourteen orphan drug-indication pairs were retrieved. Agreement in HTA outcomes was poor (k = [-0.5; 0.3]). Eight drug-indication pairs appraised by at least four HTA bodies were analyzed, five of which received diverging outcomes. Preliminary results suggest that in four of five cases, reasons for diverging recommendations were a consequence of the HTA process. Examples of non-homogeneous assessments include: lack of appropriate primary endpoint, lack of long-term data, evidence not reflecting clinical practice, orphan status or unmet clinical need. Preliminary results identify the criteria driving the assessments and reasons why they result in diverging HTA outcomes, enabling a better understanding of these processes by elucidating the expectations and value judgments from HTA bodies, particularly on the orphan status, and identifying areas where more consensus on what constitutes appropriate HTA methodologies is needed. Final results will quantify these criteria in a systematic manner.

PCN151 HTAs for the Deadliest Diseases: What Can We Learn from Multi-National Comparisons of Oncology and Cardiology Health Technology Assessments?

To examine the similarities and differences in the HTAs conducted in 6 countries in the last 5.5 years in the areas of cardiology and oncology, the therapeutic areas of greatest mortality. We reviewed and abstracted information from 768 cardiology and 960 oncology HTAs conducted from January 1, 2007 to June 23, 2012. Our primary focus was those made by the following public organizations: Canadian Agency for Drugs and Technology in Health, Haute Autorite de Sante, Institute for Quality and Efficiency in Health Care, National Institute for Clinical Excellence, Pharmaceutical Benefits Advisory Committee, Medical Services Advisory Committee, and the Agency for Healthcare Research and Quality. For comparative purposes and overall interest, we also studied the HTAs of the following private American organizations: BlueCross BlueShield Technology Evaluation Center, California Technology Assessment Forum, Drug Effectiveness Review Program, Healthcore/Wellpoint, Institute for Clinical and Economic Review, and the MedCo Research Institute, and the multinational Cochrane Collaboration. Finally, we looked at the American Recovery and Reinvestment Act generated CER grants recently made by the federal government to the National Institutes of Health and the Department of Health and Human Services to determine any new directions in the US. Cardiology HTAs were divided into 12 sub-therapeutic categories; oncology 18 for ease of analysis. Variables analyzed included specific subject of HTA and analytic methods, date of release, and results. Market entry of drugs and selected devices tended to affect HTA content and timing; country processes for review also affect these variables and results. HTAs of other single interventions and multiple modality comparisons were more variable as to timing, content, and results. Both the commonalities and differences found in the HTAs lend themselves to the examination of potential “economies” of evidence assessment and bases for optimal patient care. The authors provide suggestions for policy makers.

Commonalities and differences in HTA outcomes: A comparative analysis of five countries and implications for coverage decisions

ObjectiveTo identify diverging HTA recommendations across five countries, understand the rationale for decision-making in specific therapeutic categories, and suggest ways forward to minimize these inter-country differences. MethodsA comparative analysis of HTA recommendations for 287 drug-indication pairs appraised by five countries (England, Scotland, Sweden, Canada, and Australia) between 2007 and 2009, including an in-depth analysis of two case studies. Agreement levels were measured using kappa scores. Associations were explored through correspondence analysis. ResultsSignificant inter-country variability in the HTA recommendations exists: 46% of the drug-indication pairs studied received diverging recommendations across countries. The level of agreement between agencies was poor to moderate. Associations between HTA recommendations issued by each HTA body per therapy area (cancer, orphan, CNS) differed from the general pattern observed across the complete sample. Expectations from HTA bodies in terms of relative effectiveness differ depending on the drug and disease's characteristics, although agency-specific guidelines are homogeneous for all treatments. Policy implicationsDistinguishing and accounting for the specifics underpinning individual conditions and their characteristics in HTA processes may constitute a way forward to improved HTA methods, while increasing transparency in the expectations that HTA bodies have in terms of relative effectiveness of the drug depending on these characteristics.

A Modification of Extended Homoclinic Test Approach to Solve the (3+1)-Dimensional Potential-YTSF Equation

By means of the extended homoclinic test approach (EHTA) one can solve some nonlinear partial differential equations (NLPDEs) in their bilinear forms. When an NLPDE has no bilinear closed form we can not use this method. We modify the idea of EHTA to obtain some analytic solutions for the (3+1)-dimensional potential-Yu-Toda-Sasa-Fukuyama (YTSF) equation by obtaining a bilinear closed form for it. By comparison of this method and other analytic methods, like HAM, HTA and three-wave methods, we can see that the new idea is very easy and straightforward.