Challenges For Health Technology Assessment Research Articles

Changes and challenges in health technology assessment.

Changes and challenges in health technology assessment.

Key considerations for modelling the long-term costs and benefits of treatments for ANCA-associated vasculitis.

Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a group of severe and chronic autoimmune diseases. Patients undergo two treatment phases: inducing remission and maintaining remission to prevent organ damage. Immunosuppressants, including glucocorticoids (GCs) are used as first-line treatment, but long-term GC use is associated with toxic effects. Novel treatments reduce or replace the need for long-term GC, and therefore can reduce GC-related toxicity. The evolving treatment landscape has presented new challenges for health technology assessment (HTA) of new treatments in AAV and long-term modelling of costs and outcomes in this disease. Using the appraisal of avacopan in England (NICE) as a case study, this paper aims to identify the key challenges involved in the economic evaluation of new treatments for AAV, with a particular focus on the long-term modelling of the treatment costs and benefits for the purpose of HTA. The outcome of this study is a set of recommendations for modelling the cost-effectiveness of new treatments for AAV from the HTA perspective. The discussion focuses on the appropriate model structure, approach to modelling end-stage renal disease (ESRD) as a key determinant of costeffectiveness, capturing the impact of GC-related adverse events, and estimation of short and long-term costs of AAV. Economic evaluation of new treatments for AAV needs to capture all relevant downstream effects. ESRD is a key driver of cost-effectiveness but is associated with major uncertainty. Future observational studies need to offer sufficient detail to allow for differentiation in event rates across treatment options.

HTA83 Economic Evaluations in Relapsed or Refractory (R/R) Follicular Lymphoma (FL): Challenges for Health Technology Assessment (HTA) Submissions

HTA83 Economic Evaluations in Relapsed or Refractory (R/R) Follicular Lymphoma (FL): Challenges for Health Technology Assessment (HTA) Submissions

HPR161 International Comparisons of Health Technology Assessment and Reimbursement Outcomes for Oncology Drugs with Regulatory Review through Project Orbis

Project Orbis is an international regulatory collaboration led by the US Food and Drug Administration (FDA) Oncology Center of Excellence. It represents a framework for concurrent submission and review of oncology products, with the aim to give patients faster access to promising cancer treatments. However, products with limited clinical evidence can pose challenges for health technology assessment (HTA) and public-payer decisions. We examined HTA and reimbursement outcomes for Project Orbis drugs reviewed in Canada and other jurisdictions globally from program inception in 2019 to end of 2022.

Challenges of Health Technology Assessment in Pluralistic Healthcare Systems: An ISPOR Council Report

Health technology assessment (HTA) has been growing in use over the past 40 years, especially in its impact on decisions regarding the reimbursement, adoption, and use of new drugs, devices, and procedures. In countries or jurisdictions with “pluralistic” healthcare systems, there are multiple payers or sectors, each of which could potentially benefit from HTA. Nevertheless, a single HTA, conducted centrally, may not meet the needs of these different actors, who may have different budgets, current standards of care, populations to serve, or decision-making processes.This article reports on the research conducted by an ISPOR Health Technology Assessment Council Working Group established to examine the specific challenges of conducting and using HTA in countries with pluralistic healthcare systems. The Group used its own knowledge and expertise, supplemented by a narrative literature review and survey of US payers, to identify existing challenges and any initiatives taken to address them. We recommend that countries with pluralistic healthcare systems establish a national focus for HTA, develop a uniform set of HTA methods guidelines, ensure that HTAs are produced in a timely fashion, facilitate the use of HTA in the local setting, and develop a framework to encourage transparency in HTA. These efforts can be enhanced by the development of good practice guidance from ISPOR or similar groups and increased training to facilitate local use of HTA.

Current Issues in Health Technology Assessment of Cancer Therapies: A Survey of Stakeholders and Opinion Leaders in Australia.

The aim of this study was to find ways of bridging the gap in opinions concerning health technology assessment (HTA) in reimbursement submission between manufacturers and payers to avoid access delays for patients of vital medicines such as oncology drugs. This was done by investigating differences and similarities of opinion among key stakeholders in Australia. The survey comprised of nine sections: background demographics, general statements on HTA, clinical claim, extrapolations, quality of life, costs and health resource utilization, agreements, decision making, and capability/capacity. Responses to each question were summarized using descriptive statistics and comparisons were made using chi-square statistics. There were ninety-seven respondents in total, thirty-seven from the public sector (academia/government) and sixty from the private sector (industry/consultancies). Private and public sector respondents had similar views on clinical claims. They were divided when it came to extrapolation of survival data and costs and health resource utilization. However, they generally agreed that rebates are useful, outcomes-based agreements are difficult to implement, managed entry schemes are required when data are limited, and willingness to pay is higher in cancer compared to other therapeutic areas. They also agreed that training mostly takes place through on the job training and that guideline updates were a least favored opportunity for continued training. Private sector respondents favor methods that reduce the incremental cost-effectiveness ratio when compared to the public sector respondents. There still exist a number of challenges for HTA in oncology and many research opportunities as a result of this study.

Reported Challenges in Health Technology Assessment of Complex Health Technologies

ObjectivesWith complex health technologies entering the market, methods for health technology assessment (HTA) may require changes. This study aimed to identify challenges in HTA of complex health technologies. MethodsA survey was sent to European HTA organizations participating in European Network for HTA (EUnetHTA). The survey contained open questions and used predefined potentially complex health technologies and 7 case studies to identify types of complex health technologies and challenges faced during HTA. The survey was validated, tested for reliability by an expert panel, and pilot tested before dissemination. ResultsA total of 22 HTA organizations completed the survey (67%). Advanced therapeutic medicinal products (ATMPs) and histology-independent therapies were considered most challenging based on the predefined complex health technologies and case studies. For the case studies, more than half of the reported challenges were “methodological,” equal in relative effectiveness assessments as in cost-effectiveness assessments. Through the open questions, we found that most of these challenges actually rooted in data unavailability. Data were reported as “absent,” “insufficient,” “immature,” or “low quality” by 18 of 20 organizations (90%), in particular data on quality of life. Policy and organizational challenges and challenges because of societal or political pressure were reported by 8 (40%) and 4 organizations (20%), respectively. Modeling issues were reported least often (n = 2, 4%). ConclusionsMost challenges in HTA of complex health technologies root in data insufficiencies rather than in the complexity of health technologies itself. As the number of complex technologies grows, the urgency for new methods and policies to guide HTA decision making increases.

Review of the Challenges for Health Technology Assessment in the World

The article's abstract is not available.

The representation of public values in health technology assessment to inform funding decisions: the case of Australia's national funding bodies.

Over the past few years, there has been an increasing recognition of the value of public involvement in health technology assessment (HTA) to ensure the legitimacy and fairness of public funding decisions [Street J, Stafinski T, Lopes E, Menon D. Defining the role of the public in Health Technology Assessment (HTA) and HTA-informed decision-making processes. Int J Technol Assess Health Care. 2020;36:87-95]. However, important challenges remain, in particular, how to reorient HTA to reflect public priorities. In a recent international survey of thirty HTA agencies conducted by the International Network of Agencies for HTA (INAHTA), public engagement in HTA was listed as one of the "Top 10" challenges for HTA agencies [O'Rourke B, Werko SS, Merlin T, Huang LY, Schuller T. The "Top 10" challenges for health technology assessment: INAHTA viewpoint. Int J Technol Assess. 2020;36:1-4].Historically, Australia has been at the forefront of the application of HTA for assessing the effectiveness and cost-effectiveness of new health technologies to inform public funding decisions. However, current HTA processes in Australia lack meaningful public inputs. Using Australia as an example, we describe this important limitation and discuss the potential impact of this gap on the health system and future directions.

PNS167 Challenges in Health Technology Assessment of Complex Health Technologies

PNS167 Challenges in Health Technology Assessment of Complex Health Technologies

Health technology assessment in traditional Chinese medicine in China: current status, opportunities, and challenges

Abstract Objectives The widespread usage of traditional Chinese medicine (TCM) and the government's emphasis on TCM in China have created a positive macro-environment and great opportunities to develop and strengthen health technology assessment (HTA) in TCM. Over the past ten years, a series of policies have been issued by the government to promote the application of HTA and the development of TCM in China. This study reviews the concepts and characteristics of TCM, summarizes the current status of HTA in TCM, and analyzes the opportunities and challenges faced by HTA development in TCM with respect to deepening of healthcare reforms. Methods Literature review and website searches were used to acquire information regarding the basic theories of TCM and the current status, opportunities, and challenges for HTA in TCM. Results The number of articles on HTA in TCM rose substantially, especially those published in China National Knowledge Infrastructure (CNKI). With the optimistic policy environment and huge market demand for TCM, the development of HTA in TCM is expected to flourish in China. However, HTA in TCM also faces numerous challenges such as the absence of a clinical efficacy evaluation system for TCM and insufficient basic research evidence and qualified personnel. Conclusions TCM has unique characteristic that distinguishes it from Western medicine. Currently, it is important to take all measures to address the existing main challenges and thereby take advantage of the present opportunities to develop and improve HTA in TCM.

The 'Top 10' Challenges for Health Technology Assessment: INAHTA Viewpoint.

The International Network of Agencies for Health Technology Assessment (INAHTA) spans the globe as a network of 50 publicly-funded health technology assessment (HTA) agencies supporting health system decision making for 1.4 billion people in thirty countries. Agency members are non-profit HTA organizations that are part of, or directly support, regional or national governments. Recently, INAHTA surveyed its members to gather perspectives from agency leadership on the most important issues in HTA today. This paper describes the top 10 challenges identified by INAHTA members. Addressing these challenges requires a call for action from INAHTA member agencies and the many other actors involved in the HTA ecosystem. In opening this call for action, INAHTA will lead the way; however, a comprehensive undertaking from all players is needed to effectively address these challenges and to continue to evolve HTA in its role as a strong and effective contributor to health systems.

Allocating healthcare resources to genomic testing in Canada: latest evidence and current challenges.

Precision medicine (PM) informed by next-generation sequencing (NGS) poses challenges for health technology assessment (HTA). To date, there has been limited reimbursement of genomic testing with NGS in Canada, particularly for whole-genome and whole-exome sequencing (WGS/WES). Through a structured literature review, we examine Canadian economic evidence and evidentiary challenges for the adoption of genomic testing. We searched Medline (PubMed) for published Canadian studies generating economic evidence for PM informed by NGS. Our search focused on studies examining the costs and/or value of NGS. We reviewed included studies and summarized results according to evaluation type, clinical context, NGS technology, and test strategy. We then grouped HTA challenges encountered by authors when evaluating NGS. Our review included twenty-five studies. To determine the economic impacts of NGS-informed PM in Canada, studies applied cost-effectiveness analysis (52%, n = 13), stated preference analysis (20%, n = 5), cost-consequence analysis (16%, n = 4), and healthcare resource utilization or costing analysis (12%, n = 3). NGS panels were the most common technology evaluated (n = 13), followed by WGS and/or WES (n = 8). The included studies highlighted multiple challenges when generating economic evidence, many of which remain unaddressed. Challenges were broadly related to (1) accounting for all NGS outcomes; (2) addressing uncertainty; and (3) improving consistency of economic approaches. Canadian studies are beginning to produce estimates of the economic impacts of NGS-informed PM, yet challenges for HTA remain. While solutions and real-world evidence are generated, lifecycle health technology management methods can be designed to better support resource allocation decisions for genomic testing in Canada.

PP08 Evaluation Of The Brazilian Health Technology Assessment Network

IntroductionThe Brazilian Network for Health Technology Assessment (REBRATS) is a network of collaborating centers and teaching and research institutions, focused on the generation and synthesis of scientific evidence in the field of health technology assessment. Currently, the network is composed of 119 member institutions and mobilizes approximately 1,094 researchers and 4,998 relations. The objective of this study was to evaluate the member institutions of REBRATS.MethodsThe evaluation process was developed in seven stages, including the identification of the objectives of the Network; identification of evaluation criteria; selection of performance indicators for each criterion; identification of the measures appropriate to each indicator; data collection and analysis; classification of the institutions and production of the final report.ResultsThe evaluation of the member institutions of REBRATS mapped the capacity of these institutions to produce health technology assessment activities. The evaluation also provided information on the advances and challenges of health technology assessment in the country. In the long term, the initiative will contribute to the strengthening of the evaluation of health technology in Brazil, since the weaknesses of these institutions in the development of activities were mapped.ConclusionsThe production of this study will contribute to the dissemination of the evaluation methodology at the national and international level. This study is one of the few initiatives that exist in the world on the evaluation of networks and will contribute to the strengthening of the evaluation of health technology in Brazil.

PP39 Health Technology Assessment And Aging: Moving Evidence To Action

Introduction:With the rapid increase in technologies and innovations to support a growing aging population in many countries, health technology assessment (HTA) of technologies for the aging populace warrants special consideration. Building on our efforts at Health Technology Assessment international (HTAi) conferences in 2016 and 2017, this presentation will highlight themes generated from two previous HTAi collaborations, with an aim of continuing to build interest and capacity in HTA for aging-related technologies in an international ecosystem that is responsive to local needs and global opportunities.Methods:Researchers from Canada's technology and aging network (AGE-WELL) collaborated with international panelists at HTAi conferences in 2016 and 2017 to explore interest in HTA focused on aging. International panelists shared the current state of aging and HTA in their respective countries. At both sessions, opportunities were provided for participants to rate the importance of themes identified by the panelists.Results:At the 2016 session, the two most highly ranked themes were: (i) how HTA can help identify the unmet needs of older adults in society that could be met by technology; and (ii) engagement of older adults and caregivers. These two themes became the starting point for the panel discussion in 2017. At this session, the highest ranked themes were: (i) identification of challenges in HTA and aging; (ii) approaches to advancing the effectiveness of HTA in addressing technology and aging; and (iii) development of an aging-related interest group in HTAi.Conclusions:International collaborations have identified a number of recommendations to consider for HTA and aging-related work including: developing a good mutual awareness and understanding of barriers and opportunities; the importance of co-creating solutions with patients, healthcare providers, researchers, innovators, and funders; and the identification of a suite of methods and tools that can help accelerate technological innovation in care delivery.

Key Recommendations from the MedtecHTA Project

There are particular characteristics of Medical Devices, such as the device-user interaction, the incremental nature of innovation and the broader organizational impact that lead to additional challenges for health technology assessment (HTA). The project explored key aspects of the conduct and methods of HTA for MDs. Systematic reviews and original research studies were conducted to determine improvements in processes and methods that could enhance the potential for HTA and optimize the diffusion of MDs. Regulatory processes for MDs should be more closely aligned, the HTA evaluative framework should be harmonized and processes for conditional coverage and evidence development should be used. The methods for HTA should consider MDs as complex interventions, require the establishment of high quality registries, consider an iterative approach to the evaluation over time, recognize and allow for the particular characteristics of devices and use appropriate approaches for confounder adjustment in comparative effectiveness studies. To optimize the diffusion, a common classification should be developed across countries in order to facilitate international comparisons, factors driving diffusion should be explored in HTA reports and physicians' personal goals and motivation should be better understood. The key recommendations of the MedtecHTA project should improve the conduct and use of HTA for MDs. © 2017 The Authors. Health Economics published by John Wiley & Sons, Ltd.

VP71 Health Technology Assessment In Japan: Current Issues And Challenges

INTRODUCTION:Japan plans to introduce Health Technology Assessment (HTA) in 2018 after a two-year trial period. Japan currently requires HTA for certain innovative products which may have a large budget impact. Through this trial implementation, the government can examine the criteria of applicable products, the necessary infrastructure to conduct and evaluate HTA, the quality of data content, and localization to meet the current Japanese reimbursement and pricing scheme. However, the pharmaceutical industry in Japan is still puzzled by this introduction. The aim of this study is to visualize the issues and implementation challenges of HTA in Japan through a survey of the pharmaceutical industry.METHODS:A semi-structured nineteen-item questionnaire was designed and the survey was conducted through face-to face or phone interviews. Answers were summarized after the interview and confirmed with the respondents via e-mail. The survey focused on pharmaceutical companies which develop new innovative products.RESULTS:The differences between Japanese and non-Japanese pharmaceutical companies were observed in terms of HTA staff expertise and experience, the source of HTA data, and relationships with external vendors. Many respondents stated that a sufficient number of HTA professionals in Japan is critical to implement HTA, and raised a concern that the same public experts who are involved in HTA preparation may also review HTA submissions. Although companies are generally pessimistic about HTA for pharmaceutical pricing, they also have some positive views that HTA may be used as an indicator to enable stakeholders to understand product value. Many are unsure about the link between HTA and pharmaceutical prices.CONCLUSIONS:If HTA is implemented for an extended number of products, a shortage of experts may cause delays of HTA review and appraisal processes. Consequently, product launch and patient access will be delayed. Practical timing of HTA review and appraisal after product launch could affect the results of re-pricing.

OP123 Health Technology Assessment In Digital Health: A Rapid Approach To Assess Health Apps

INTRODUCTION:The Health Technology Assessment (HTA) of mobile health applications involves significant challenges including rapid product development cycles, sparse evidence and uncertainty over the economic impact. However apps also provide unique opportunities, such as their potential reach and use of real-world data, which will facilitate their contribution to healthcare delivery. The National Institute for Health and Care Excellence (NICE), alongside other agencies, has been piloting the development of a health app assessment programme. This presentation reports the results of a study about the development of the Health App Briefing (HAB) which is designed as the output from a rapid assessment of the effectiveness and cost-saving potential of apps to inform decision makers in the United Kingdom National Health Service.METHODS:The HAB is built on the success of the NICE Medtech Innovation Briefings programme because many of the HTA challenges are similar to those found with medical devices. HAB development is grounded in four principles: rapid assessment; transparent process; independence from industry or the health service and input from commentators. The content includes an evidence summary for effectiveness including comments from specialist experts and users; a summary of information relating to the cost saving potential and a summary of other user benefits (including issues of access and usability). Novel features are the presentation of a rating of the potential value of the app to the health system and working with commissioners of the app to obtain real-world information for a case study about the economic impact.RESULTS:The development of four HABs along with a review of the learning from the piloting process will be presented. The review will include stakeholder feedback from a workshop.CONCLUSIONS:We believe the evaluation of this work presented here will be of interest to other HTA agencies around the world that are deciding how to approach the issues surrounding the assessment of health apps.

Health technology assessment in the era of personalized health care

This article examines the challenges for health technology assessment (HTA) in the light of new developments of personalized health care, focusing on European HTA perspectives. Using the example of the Integrated Genome Research Network - Mutanom (IG Mutanom) project, with focus on personalized cancer diagnostics and treatment, we assess the scope of current HTA and examine it prospectively in the context of the translation of basic and clinical research into public health genomics and personalized health care. The approaches developed within the IG-Mutanom project are based on innovative technology potentially providing targeted therapies for cancer; making translation into clinical practice requires a novel course of action, however. New models of HTA are needed that can account for the unique types of evidence inherent to individualized targeted therapies. Using constructive health technology assessment (CTA) models is an option, but further suitable models should be developed. Integrative, systems biology-based approaches toward personalized medicine call for novel assessment methods. The translation of their highly innovative technologies into the practice of health care requires the development of new HTA concepts.

Pharmaceutical market access and the challenges of health technology assessment in the United Kingdom

AbstractHealth Technology Assessment (HTA) has been formally used in the United Kingdom for more than 10 years to help determine evidence‐based decisions on the access to new drugs, based on evidence of clinical and cost‐effectiveness. Separate HTA systems have developed in England and Wales (NICE, AWMSG), and Scotland (SMC). The primary clinical evidence desired for technology appraisal by the HTA bodies is the comparative randomized controlled trial because of its good internal validity, but the role and value of observational study designs for providing data on real‐world effectiveness have been recognized in the methods guidance of these bodies. To date HTA has had a fairly limited impact on drug development and clinical trial design in the United Kingdom, coupled with limited use to date of non‐RCT data for assessing treatment effect and outcomes in NICE/SMC appraisals. Nonetheless, with new initiatives such as closer regulatory and HTA coordination in Europe, and “conditional market access” schemes such as the Innovation Pass in the United Kingdom, in the future pharmaceutical companies are likely to need to adopt a more flexible approach to drug development and clinical data collection involving RCT, observational data collection, and indirect comparisons to meet the needs to provide relevant clinical and cost‐effectiveness evidence at drug launch and post launch. Drug Dev Res 71: 478–484, 2010. © 2010 Wiley‐Liss, Inc.