Use Of Health Technology Assessment Research Articles

SA47 Exploring the Use of Health Technology Assessment in Uganda’s Health Policy and Financing Decision-Making Processes

SA47 Exploring the Use of Health Technology Assessment in Uganda’s Health Policy and Financing Decision-Making Processes

PP109 The Use Of Health Technology Assessment In Decision Making: Evidence From The Balkan Countries

IntroductionAccording to the most recent definition health technology assessment (HTA) “is a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The purpose is to inform decision-making in order to promote an equitable, efficient, and high-quality health system”. This article aimed to evaluate implementation of HTA in decision making in the Balkan countries.MethodsA scoping review of the existing literature took place to locate relevant scientific articles, policy papers and documents released by the respective Ministries. We searched data for 6 Western Balkan countries (Albania, Bosnia and Herzegovina, Kosovo, Montenegro, North Macedonia and Serbia) and didn’t focus on those countries that are part of the European Union (EU). For the literature search key words were used, while documents only in English were included in the analyses. Additionally, the search was conducted for the period January 2010 until October 2022.ResultsThe Western Balkan countries are in process of integration to the EU and based on this they are trying to make improvement in different sectors including health services. However, the use of HTA in most of the studied countries is in its preliminary phase. Most of the countries have established HTA bodies or specific authorities but with limited resources (both human and financial). Additionally, their reports are non-binding for policy makers and healthcare decisions are taken based on experts’ opinions and not an extensive HTA analyses.ConclusionsDespite their efforts, the Western Balkan countries need to improve and considerably increase the use of HTA in decision-making. Its use can help in provision of better healthcare services as well as to decrease costs. Specific attention should be put on human and financial resources that are lacking in all settings.Western Balkan countries need to put much more efforts for harmonization of their legal framework with that of the EU countries.

Use of Health Technology Assessment for the Continued Funding of Health Technologies: The Case of Immunoglobulins for the Management of Multifocal Motor Neuropathy.

Funding decisions for many health technologies occur without undergoing health technology assessment (HTA), in particular, without assessment of cost effectiveness (CE). Immunoglobulins in Australia are an interesting case study because they have been used for a long time for various rare disorders and their price is publicly available. Undertaking an HTA enables us to assess CE for an intervention for which there is limited clinical and economic evidence. This study presents a post-market review to assess the CE of immunoglobulins for the treatment of multifocal motor neuropathy (MMN) compared with best supportive care. A Markov model was used to estimate costs and quality-adjusted life-years (QALYs). Input sources included randomised controlled trials, single-arm studies, the Australian clinical criteria for MMN, clinical guidelines, previous Medical Services Advisory Committee (MSAC) reports and inputs from clinical experts. Sensitivity analyses were conducted to assess the uncertainty and robustness of the CE results. The cost per patient of treating MMN with immunoglobulin was AU$275,853 versus AU$26,191when no treatment was provided, with accrued QALYs of 6.83 versus 6.04, respectively. The latter translated into a high incremental cost-effectiveness ratio (ICER) of AU$317,552/QALY. The ICER was most sensitive to the utility weights and the price of immunoglobulins. MSAC advised to continue funding of immunoglobulins on the grounds of efficacy, despite the high and uncertain ICER. Beyond the ICER framework, other factors were acknowledged, including the high clinical need in a patient population for which there are no other active treatments available. This case study highlights the challenges of conducting HTA for already funded interventions, and the efficiency trade-offs required to fund effective high-cost therapies in rare conditions.

Intangible assets innovation through Health Technology Assessment. The case of the Italian Healthcare Organisations

The use of Health Technology Assessment (HTA) for the evaluation of medical devices in healthcare organisations implies the creation of new knowledge to be combined with that stored in the human, relational, and structural dimensions of their Intellectual Capital (IC).Therefore, HTA can represent a trigger for innovating the IC and this process can be influenced by external factors that can be reconducted to the Institutional Theory (IT). However, to the best of our knowledge, there are no studies in the literature analysing these aspects. By using the case study methodology, this work explores how the innovation of the IC dimensions is triggered by the use of the HTA tools, considering also the moderating role of the IT pillars.

Mandatory health insurance for the informal sector in Tanzania-has it worked anywhere!

Many countries in sub-Saharan Africa are struggling to expand voluntary health insurance schemes to raise finances toward achieving universal health coverage. With more than three-quarters of the population without any insurance, the government of Tanzania has unsuccessfully tried to pass a Bill proposing a mandatory, nationwide scheme to cover the large and diverse informal sector. The Bill proposed an annual premium of ∼150 USD for a household of six or 65 USD per person. Studies in Tanzania and Kenya have shown that the majority of people in the informal sector are unwilling and unable to pay premiums as low as 4 USD, mostly due to poverty. Mandatory health insurance for the informal sector is not common in this region, mostly because it is difficult to enforce. Successful insurance schemes have included significant subsidies from tax revenues. Tanzania should not seek to raise funds for health through an unenforceable insurance scheme but rather should consider a largely tax-funded scheme for the informal sector. Contributions through low-cost voluntary schemes can enhance social contracts, reduce out-of-pocket expenditure, and promote efficient utilization. In addition, progressive health taxes should be imposed on harmful products (tobacco, alcohol, sugary drinks, etc.) to raise more funds while addressing the increasing burden of non-communicable diseases. Furthermore, efficiency in the use of scarce health resources should be promoted through realistic prioritization of public services, the use of Health Technology Assessment, and strategic purchasing.

Evolving Use of Health Technology Assessment in Medical Device Procurement—Global Systematic Review: An ISPOR Special Interest Group Report

ObjectivesTo review the current academic evidence describing how data from health technology assessments (HTAs) informs procurement decisions for medical devices. MethodsA systematic literature review was performed to identify relevant studies and criteria used in medical device purchasing or procurement decisions. Included articles were screened for relevancy and risk of bias. The included studies were summarized qualitatively. ResultsA total of 292 studies were screened, of which 11 matched the inclusion criteria. Included studies’ geographies and HTA maturity varied. Some studies described hospital-level HTA processes, whereas others focused on national-level recommendations. Criteria for procurement decisions included standard HTA factors, such as efficacy, cost, cost-effectiveness, and budget impact; broader issues were also noted, including impact on the organization, ethical aspects, staff workload, and volume. There was little consideration of device-specific characteristics, such as life cycle, learning curve, or incremental technical innovation. Few decisions referred to HTA reports as part of the procurement decision; similarly, few HTA reports included a procurement perspective to help guide the procurement bodies. ConclusionsThere is minimal evidence that notes HTA influencing medical device procurement. Procurement bodies and hospitals may not be incentivized to publish their work and transparency could be improved; further research would better describe the link between HTA and procurement. Such research would enable the HTA agencies to meaningfully assess devices to target procurement bodies and allow device sponsors to prioritize evidence. This could limit redundancy, improve evidence, and ultimately promote savings to healthcare systems and expand access.

Data Governance for Real-World Data Management: A Proposal for a Checklist to Support Decision Making

ObjectivesReal-world data (RWD) and real-world evidence (RWE) can provide extensive information on healthcare for use in health technology assessment and decision making. Nevertheless, there is a lack of consensus surrounding the appropriate data governance (DG) practices for RWD/RWE. Data sharing is also a large concern, especially considering evolving data protection regulations. Our objective is to propose recommendations for international standards of evaluating the acceptability of RWD governance practices. MethodsAfter reviewing the literature, we created a checklist targeting DG practices for RWD/RWE. We then carried out a 3-round Delphi panel, including European policy makers, health technology assessment experts, and hospital managers. The consensus for each statement was measured and the checklist adjusted accordingly. ResultsThe literature review identified the main topics regarding RWD/RWE DG practices: data privacy and security, data management and linkage, data access management, and the generation and use of RWE. Members of the Delphi panel (21 experts/25 invited) were presented a total of 24 statements related to each of the topics. Experts demonstrated a progressive level of consensus and importance ratings in all topics and to most statements. We suggest a refined checklist in which the statements rated less important or with less consensus have been removed. ConclusionsThis study suggests how the DG of RWD/RWE could be qualitatively evaluated. We propose checklists that could be used by all RWD/RWE users to help ensure the quality and integrity of RWD/RWE governance and complement data protection law.

MSR53 A Review of Methods for Estimating Individual Treatment Effect From Real World Data for Use in Health Technology Assessment: Separating Hype From Reality

MSR53 A Review of Methods for Estimating Individual Treatment Effect From Real World Data for Use in Health Technology Assessment: Separating Hype From Reality

OP41 Facilitating Dialogue Of Real-World Evidence Use In Health Technology Assessment: Taxonomy Of Question/Data Source Pairings To Support A Registry Of Studies

IntroductionThis paper reports the results of the collaboration within the European initiative of new Reimbursement and aCCess Approaches (EUreccA) which is concerned with the use of real-world evidence (RWE) in health technology assessment (HTA) decision-making. The work grew from the observation of a large, very experienced group of HTA practitioners which found that the use of RWE varied depending on the type of question asked and the particulars of the data source(s) used. We set out to examine how RWE is used in HTA decision-making and to make proposals on its facilitation.MethodsLiterature reviews covering earlier reviews of RWE use, academic papers, and HTA agency websites were combined with case studies involving interviews with decision-makers in four countries (England, France, Italy, Sweden) to identify the circumstances of breakdown of RWE use and to build a categorization of the uses of RWE and associated difficulties. This evidence supported the creation of a taxonomy of pairings of data sources and the questions they were used to address. The face validity of the approach was tested at an advisory board of senior HTA practitioners.ResultsIn total, 27 questions were identified and 10 types of data source, giving 270 pairings. These pairings were linked to relevant methods guidance and to examples of their use, itemizing HTA issues and decisions made. Reports are being prepared for publication, covering the detail of the methods of the literature searches; methods of the country case studies; a description of the taxonomy; and guidance on governance.ConclusionsWhen using RWE in HTA decision-making, the detail of the particular data sources and question addressed matter. Recently, both the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the Real-World Transparency Initiative have argued for a registry of the uses of RWE. The work described here offers a starting classification of the material that should be held in such a registry, and which in itself could be developed by the stakeholders, both agencies and companies, that use it, furthering trust and confidence.

PD03 Uptake Of Health Technology Assessment In Hospitals: A Scoping Review

IntroductionThe use of Health Technology Assessment (HTA) in hospitals contributes to decision-making, professional training, greater interaction with technical to scientific knowledge, resource-saving, and partnerships. Hospitals are strategic for the field of clinical management and quality of care and are open to partnerships with national and international agencies and groups. Despite the hospital-based (HB)-HTA movement, the incipient application of HTA to the hospital decision-making process is related to incipient planning, and there is still much room for progress worldwide. The aim of this study was to analyze the uptake level of HB-HTA in diverse contexts.MethodsA scoping review was conducted according to the methodology of the Joanna Briggs Institute, whose data analysis model consisted of the combination of Donabedian’s structure, process, and outcome categories and the dimensions of the project Adopting Hospital Based Health Technology Assessment in European Union (AdHopHTA).ResultsWe identified 270 studies, and after removing duplicates and reading full texts, 36 references met the eligibility criteria. Thirty-six hospitals were identified, of which there were 24 largescale hospitals with extra bed capacity. Twenty-three hospitals were affiliated with universities. Canada stood out with five university hospitals, four of which with public funding. Half of the identified hospitals had HB-HTA units (18/36). Hospitals with full uptake level of HTA corresponded to 75 percent of the sample (27/36), and the remainder had partially uptake level of HTA, or 25 percent of the hospitals in the review (9/36). There were no hospitals with incipient uptake level of HTA.ConclusionsMeasuring the uptake level of HTA in hospitals contributed to understanding how their participation has occurred in the field of HB-HTA. This study revealed the importance of identifying factors such as sustainability, growth, and evolution of HB-HTA in countries with and without a tradition in this field.

Estimating health system opportunity costs: the role of non-linearities and inefficiency

BackgroundEmpirical estimates of health system opportunity costs have been suggested as a basis for the cost-effectiveness threshold to use in Health Technology Assessment. Econometric methods have been used to estimate these in several countries based on data on spending and mortality. This study examines empirical evidence on four issues: non-linearity of the relationship between spending and mortality; the inclusion of outcomes other than mortality; variation in the efficiency with which expenditures generate health outcomes; and the relationship among efficiency, mortality rates and outcome elasticities.MethodsQuantile Regression is used to examine non-linearities in the relationship between mortality and health expenditures along the mortality distribution. Data Envelopment Analysis extends the approach, using multiple measures of health outcomes to measure efficiency. These are applied to health expenditure data from 151 geographical units (Primary Care Trusts) of the National Health Service in England, across eight different clinical areas (Programme Budget Categories), for 3 fiscal years from 2010/11 to 2012/13.ResultsThe results suggest differences in efficiency levels across geographical units and clinical areas as to how health resources generate outcomes, which indicates the capacity to adjust to a decrease in health expenditure without affecting health outcomes. Moreover, efficient units have lower absolute levels of mortality elasticity to health expenditure than inefficient ones.ConclusionsThe policy of adopting thresholds based on estimates of a single system-wide cost-effectiveness threshold assumes a relationship between expenditure and health outcomes that generates an opportunity cost estimate which applies to the whole system. Our evidence of variations in that relationship and therefore in opportunity costs suggests that adopting a single threshold may exacerbate the efficiency and equity concerns that such thresholds are designed to counter. In most health care systems, many decisions about provision are not made centrally. Our analytical approach to understanding variability in opportunity cost can help policy makers target efficiency improvements and set realistic targets for local and clinical area health improvements from increased expenditure.

Spotlight commentary: Navigating between financial risks and improved treatment outcomes with immune checkpoint inhibitors in cancer patients: The need for biomarker identification and dose optimization.

Spotlight commentary: Navigating between financial risks and improved treatment outcomes with immune checkpoint inhibitors in cancer patients: The need for biomarker identification and dose optimization.

Value-Based Care in Systemic Therapy: The Way Forward.

The rising cost of cancer care has shed light on an important aspect of healthcare delivery. Financial toxicity of therapy must be considered in clinical practice and policy-making. One way to mitigate the impact of financial toxicity of cancer care is by focusing on an approach of healthcare delivery that aims to deliver value to the patient. Should value of therapy be one of the most important determinants of cancer care? If so, how do we measure it? How can we implement it in routine clinical practice? In this viewpoint, we discuss value-based care in systemic therapy in oncology. Strategies to improve the quality of care by incorporating value-based approaches are discussed: use of composite tools to assess the value of drugs, alternative dosing strategies, and the use of Health Technology Assessment in regulatory procedures. We propose that there must be a greater emphasis on value of therapy in determining its use and its cost.

Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries.

Background: Nusinersen is an orphan drug intended for the treatment of spinal muscular atrophy (SMA), a severe genetic neuromuscular disorder. Considering the very high costs of orphan drugs and the expected market entry of cell and gene therapies, there is increased interest in the use of health technology assessment (HTA) for orphan drugs. This study explores the role of the economic evaluation and budget impact analysis on the reimbursement of nusinersen. Methods: Appraisal reports for nusinersen were retrieved from reimbursement and HTA agencies in Belgium, Canada, France, England and Wales, Germany, Italy, Ireland, Scotland, Sweden, the Netherlands, and the United States. Detailed information was extracted on the economic evaluation, the budget impact, the overall reimbursement decision, and the managed entry agreement (MEA). Costs were adjusted for inflation and currency. Results: Overall, the reports included limited data on budget impact, excluding information on the sources of data for cost and patient estimates. Only three jurisdictions reported on total budget impact, estimated between 30 and 40 million euros per year. For early-onset SMA, the incremental cost-effectiveness threshold (ICER) ranged from €464,891 to €6,399,097 per quality-adjusted life year (QALY) gained for nusinersen versus standard of care. For later-onset SMA, the ICER varied from €493,756 to €10,611,936 per QALY. Although none of the jurisdictions found nusinersen to be cost-effective, reimbursement was granted in each jurisdiction. Remarkably, only four reports included arguments in favor of reimbursement. However, the majority of the jurisdictions set up an MEA, which may have promoted a positive reimbursement decision. Conclusion: There is a need for more transparency on the appraisal process and conditions included in the MEA. Additionally, by considering all relevant criteria explicitly during the appraisal process, decision-makers are in a better position to justify their allocation of funds among the rising number of orphan drugs that are coming to the market in the near future.

Health technology assessment of whole genome sequencing in the diagnosis of genetic disorders: a scoping review of the literature.

The aim of this scoping review is to map the available evidence about the use of health technology assessment (HTA) in the assessment of whole genome sequencing (WGS). A scoping review methodology was adopted. The population, concept, and context framework was used to build up the research question and to establish the eligibility criteria. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews was adopted to implement a comprehensive search strategy. Evidence was retrieved from scientific databases and HTA organizations Web sites. Reports were classified as full HTA, mini-HTA, rapid reviews or other. The search strategy identified seven reports. Five HTA organizations from five countries elaborated the reports: one full HTA, four rapid reviews, and two classified as others. The reports were mainly focused on the evaluation of the clinical utility and cost-effectiveness of genome-wide sequencing as well as informing policy questions by providing analyses of organizational and ethical considerations. Few HTA organizations are drafting reports for WGS. It is essential to stimulate a critical reflection during the elaboration of HTA reports for WGS to steer choices of decision makers in the establishment of priorities for research and policy and reimbursement rates.

POSA252 Assessing the Value of Nusinersen for SMA: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries

Nusinersen is an orphan drug intended for the treatment of spinal muscular athrophy (SMA), a severe neuromuscular disorder. Considering the very high costs of orphan drugs and the expected market entry of cell and gene therapies, there is increased interest in the use of health technology assessment (HTA) for orphan drugs. This study explores the role of the economic evaluation and budget impact analysis on the reimbursement of nusinersen.

Assessing progression of health technology assessment implementation in Asia: a balanced scorecard for cross comparison of selected countries in Asia.

To provide an update on the use of health technology assessment (HTA) in Asia and lessons for countries seeking to advance HTA. Build upon the research by Chootipongchaivat and the World Health Organization identifying eighteen "factors conducive to the development of HTA in Asia." These factors were used to create a balanced scorecard to assess the progress of HTA, measuring progress against each factor in China, India, Indonesia, Malaysia, Philippines, South Korea, Taiwan, Thailand, and Vietnam. A scoring system was used wherein: 1, No progress; 2, milestone at early stages, ad hoc HTA use; 3, progress on milestone but limited impact; 4, significant progress but limited remit; and 5, significant progress on milestone, routine HTA informs decisions. Total scores indicated progress of HTA while milestone scores provided contextual insights within countries. Literature reviews and expert interviews were used to complete scorecards. South Korea and Thailand scored highest with seventy-three and seventy-one points, respectively, while Vietnam scored lowest at 28.5. Advanced HTA programs have independent HTA agencies with a broad remit, explicit process and methods, network of researchers, and routine use of HTA. Taiwan and Malaysia fall in a middle tier, with established HTA programs with limited remit. The final tier with China, India, Indonesia, Philippines, and Vietnam, emerging HTA processes. Universal Health Coverage goals have catalyzed expansion of HTA. Political will, technical expertise, and sustained financing remain challenges for sustainable HTA programs. Legislation supporting HTA is helpful but political will is key. Recommendations for regional collaboration are provided.

The Sandbox Approach and its Potential for Use in Health Technology Assessment: A Literature Review

BackgroundThe concept of the regulatory sandbox—a safe space for testing new regulatory processes—was first used within the financial technologies (FinTech) sector, but has since expanded into other sectors, including healthcare.ObjectivesThis review aims to describe the extent of use of sandboxes in healthcare and assess the potential for the sandbox approach to be used to test and develop emerging health technology assessment (HTA) methods, policies and processes for innovative technologies.MethodsA systematic literature review was undertaken to identify published papers and reports that described and/or assessed the use of sandboxes in the healthcare sector. Searches were conducted in Medline, Embase, Econlit, Social Policy and Practice, and Health Management Information Consortium databases from inception to March 2020. Free-text Google search was also conducted to identify relevant grey literature. Only papers and reports discussing or evaluating the use of sandboxes in healthcare settings and published in English were included. Included studies were qualitatively summarised using a thematic analysis approach.ResultsOverall, 46 papers and reports were included. The topics covered were classified into 4 major themes: history of the regulatory sandbox, the sandbox as a testing environment, the sandbox as a regulatory approach, examples of using sandboxes in healthcare. Findings show that the use of regulatory sandboxes in healthcare is relatively new and primarily used in high-income countries to support the adoption of new technologies, particularly those related to digital health. Recommendations are made based on these findings to guide its use in HTA policy and methods development.ConclusionsSandboxes are increasingly used within healthcare regulation. Despite its potential, this approach has not been used in HTA policy and methodological developments to date. HTA agencies should consider this approach to facilitate developing policies, methods and processes for innovative and disruptive health technologies. Transferability to low- and middle-income countries’ settings, however, should be assessed.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40258-021-00665-1.

PNS76 The Growing Demand for HTA in Sub-Saharan Africa

Several countries in Sub-Saharan Africa (SSA) have made advances or showing increased interest in the use of Health Technology Assessment (HTA). This paper charts the progress of HTA in various countries of SSA including the key facilitators and barriers, relevant stakeholders, and proposed institutional structures for HTA.

The most important facilitators and barriers to the use of Health Technology Assessment in Canada: a best–worst scaling approach

Background Health Technology Assessment (HTA), which can support public drug reimbursement decisions will play a core function in the planned national Pharmacare program in Canada. To address existing barriers to the use of HTA, these must be ranked in order of priority. The goal of this study was to access the relative importance of known facilitators and barriers to the use of HTA in the context of the Canadian health care system, with attention to differences between regions and stakeholder groups. Methods We used the best–worst scaling object case approach to elicit a quantitative ranking of a list of 20 facilitators and 22 barriers. A sample of 68 Canadian HTA stakeholders, including members of expert committees, decision/policymakers, researchers/academics, and others participated in the study. Their task was to identify the most important and the least important item in 12 sub-sets of five facilitators and 14 sub-sets of five barriers. Findings Relative Importance Scores derived via hierarchical Bayes analysis revealed relations, engagement, and contact between stakeholders as most important on both the barrier and facilitator sides. Other top-ranked facilitators included the availably of credible and relevant research. Other top-ranked barriers included inconsistencies in the evidence and limited generalizability. The availability of HTA guidelines did not rank highly on either side. The main limitation of the study was the challenge with reaching the relevant respondents; this was mitigated by involving the national HTA agency in the research. Conclusion Canadian stakeholders consider the relationships within the HTA network among the most important. Policies should focus on strengthening these relationships. Future research should focus on the connectivity and distribution of knowledge and power within the HTA network.