Health Technology Assessment Methodology Research Articles

Assessing medical devices: a qualitative study from the validate perspective.

Our objective was to explore procedures and methods used at health technology assessment (HTA) agencies for assessing medical devices and the underlying views of HTA practitioners about appropriate methodology to identify challenges in adopting new methodologies for assessing devices. We focused on the role of normative commitments of HTA practitioners in the adoption of new methods. An online survey, including questions on procedures, scoping, and assessments of medical devices, was sent to members of the International Network of Agencies for Health Technology Assessment. Interviews were conducted with survey respondents and HTA practitioners involved in assessments of transcatheter aortic valve implantation to gain an in-depth understanding of choices made and views about assessing medical devices. Survey and interview questions were inspired by the "values in doing assessments of health technologies" approach towards HTA, which states that HTA addresses value-laden questions and information. The current practice of assessing medical devices at HTA agencies is predominantly based on procedures, methods, and epistemological principles developed for assessments of drugs. Both practical factors (available time, demands of decision-makers, existing legal frameworks, and HTA guidelines), as well as commitments of HTA practitioners to principles of evidence-based medicine, make the adoption of a new methodology difficult. There is a broad recognition that assessments of medical devices may need changes in HTA methodology. In order to realize this, the HTA community may require both a discussion on the role, responsibility, and goals of HTA, and resulting changes in institutional context to adopt new methodologies.

Assessment of medical technologies in the formation of government programs to assist patients with rare metabolic diseases

The aim: carrying out an assessment of the technologies of nutritional and pharmacological therapy of phenylketonuria (PKU) to justify a set of measures for the implementation of the government program to support patients with rare diseases (RD). Materials and methods: scientific publications, regulatory acts, treatment protocols, statistical data, epidemiological indicators, results of patient questionnaires, marketing information, data from the electronic procurement system "ProZorro" were used in the research process. The research was conducted using the methodology of health technology assessment (HTA), methods of marketing analysis, questionnaire survey, document analysis, comparison, systematization and generalization of data. Research results. An analysis of the evaluation of modern approaches to the treatment of hereditary rare metabolic disease (PKU) was carried out. According to clinical protocols, the main technology for the treatment of PKU is nutritional therapy - a diet (diet-for-life) with restriction of the use of phenylalanine (Phe) and the use of food products for special medical purposes (Special low protein foods for phenylketonuria - SLPF-PKU). Innovative drugs "Kuvan" and "Palynziq" are recommended in the case of atypical PKU. For RD, specific and innovative cost-effective medical technologies (MT) are usually used, which have insufficient evidence due to limited experience, low availability, and small patient populations. Centralized procurement and managed entry agreements (MEA) make it possible to expand the availability of MT to patients and obtain real data on their safety and effectiveness. Integration into the global information space, participation in international projects, joint clinical assessment (JCA) in accordance with Regulation (EU) 2021/2282 on HTA, cooperation with Orphanet, EURORDIS, other professional and patient organizations are extremely important. The key components of HTA for RD are socio-economic and organizational and legal aspects, in particular the special status of MT, which provides certain preferences. The foreign experience of providing orphan patients (in particular, reimbursement) is summarized. The legal framework for RD is systematized. Based on the results of the analysis of PKU prevalence indicators, modelling and budget impact calculations were carried out, considering that SLPF-PKU products are purchased from local budgets. An analysis of prescriptions, assortment, and prices of SLPF-PKU was carried out using data from the ProZorro procurement system. A survey of 156 patients with PKU made it possible to identify unmet needs and formulate recommendations for expanding the SLPF-PKU food basket. Conclusions: Conducting the HTA made it possible to identify key problems, as well as to justify a set of measures for the development and implementation of the government program to support patients with rare diseases, based on the obtained results

HTA challenges for personalized medicine: the case of liquid biopsy for lung cancer

Abstract Background Personalized medicine and related health technologies are growing rapidly. In this context, health technology assessment methodologies must be prepared and adapted. Evaluating liquid biopsy (LB) for the diagnosis, treatment indication and prognosis of lung cancer is an example of the challenges in the personalized medicine assessment. Methods A systematic review and metanalysis based on EUnetHTA CoreModel. Review question: What is the effectiveness of LB for the diagnosis, therapeutic adequacy and prognosis of lung cancer versus tissue biopsy? The Covidence software was used for the systematic review and MetaDiSC software for the meta-analysis. The quality of the included systematic reviews was assessed with AMSTAR-2 and the quality of the clinical studies with QUADAS-II. Results 69 systematic reviews corresponding to different therapeutic purposes: 12 for treatment adequacy, 47 for diagnosis and 13 for prognosis. Also 24 clinical trials were included. The omics alterations analysed were: punctual mutations in EGFR, BRAF, KRAS and ALK, methylation patterns, lncRNA expression, detection of circulating tumour cells, quantification of circulating DNA, NGS analysis of point mutations, miRNA expression and analysis of exosome content. Conclusions Use of LB tests at different disease stages and need of individual validation for the biomolecules and mutations analysed, are the main challenges. An evaluation framework that includes the specificities of these technologies is necessary for their proper assessment. Key messages • Liquid biopsy may be an alternative to tissue biopsy. The evaluation of the biomolecules analysed should be disaggregated. • The particularities of personalised medicine technologies require assessment methodologies adapted to them.

PP59 Multidimensional Evaluation Of The Reducer Device In Patients With Refractory Angina

IntroductionTreatments for coronary heart disease patients have had major developments in recent decades, both in the pharmacological and interventional fields, and this has helped to prolong the survival of these patients. However, the growing number of patients who show persistent and disabling symptoms of angina proves that at the same time their quality of life has not been equally improved.MethodsWe conducted a multidimensional assessment coherent with health technology assessment methodology on the Coronary Sinus Reducer System (CSRS). CSRS is the latest line of therapy for patients with coronary artery disease who are ineligible for revascularization, demonstrate reversible ischemia, and have refractory angina pectoris (AP) despite optimal standard medical therapy. We performed a literature review in order to gather evidence on efficacy and safety of the device and on the economic and organizational impact of the procedure. In the economic domain we developed a cost-utility model based on a decision tree and a five-year time horizon budget impact model.ResultsSeveral studies in the literature have shown that this therapy is related to an increase in quality of life and an improvement in symptoms of refractory angina. The economic evaluations conducted show how the therapy, despite an increase in the resources absorbed in the first years of implementation, reaches a cost saving profile in the medium term due to positive outcomes, while leading to an increase in the quality of life in patients suffering from refractory angina.ConclusionsThe treatment of refractory angina remains a challenge for today’s medicine. Patients suffering from this condition are often described as “no option” patients. Thus, despite there is a need of further evidence to establish even more robustly the economic sustainability of the device, especially on its effectiveness in the medium-long term, the device should be taking into account in those patients who could benefit from it in terms of relieving the symptoms of angina and improving their quality of life.

Accounting for vaccines specificities in the Joint Clinical Assessment (JCA): a proposal for guiding principles

Abstract Issue/problem Vaccines are an important public health intervention protecting the population against infectious diseases. The value of vaccines is broad ranging from individual to societal as achieving community immunity protects the unvaccinated, minimizes the risk of outbreaks, reduces the emergence of antimicrobial resistance, and leads to broader societal benefits. Description of the problem Vaccines’ market access processes are characterized by the development of recommendations by NITAGs followed by the assessment of health technology assessment (HTA) bodies in less than half of 27 EU member states. Despite that HTA for therapeutic drugs is well established, there is very limited experience in applying HTA methodologies to vaccines, especially for clinical assessments, as HTA methods and frameworks are traditionally geared toward therapeutics. However, following the adoption of the EU regulation on HTA, Joint Clinical Assessments (JCAs) of vaccines are expected. Results To support a discussion on how to account for vaccine specificities in the JCA, Vaccines Europe has performed a project which aimed at developing a proposal for high-level guiding principles on processes and methodologies for clinical HTA for vaccines. The proposal is informed by findings of literature reviews on currently applied processes, methods, and clinical assessment frameworks of vaccines as well as the outcomes of an advisory board with scientific experts. Lessons A proposal for high-level guiding principles for clinical HTA for vaccines is being developed based on both evidence and the advice from scientific experts which focuses on processes (e.g., horizon scanning, early advice, consideration of vaccine-specific expertise) and methods (e.g., unmet need, safety, efficacy/effectiveness, real-world evidence and technical characteristics of the technology). Lastly, the implementation of vaccines specificities in JCA represents a call for action.

HTA supporting clinical practice: the case of surgical repair of thoracoabdominal aortic aneurysms

Abstract Background Thoracoabdominal aortic aneurysms (TAAAs) are defined as those aortic aneurysms involving renovisceral arteries. They account for around 10% of aortic aneurysms, and their treatment is burdened by considerable mortality and morbidity. Open surgical repair has been practised as the standard of care since the 1950s. In 2001 endovascular repair was introduced to reduce treatment invasiveness, and the technology is still evolving. The potential benefits of endovascular repair over open surgery should be carefully weighed in a multidimensional framework. Methods We applied the Health Technology Assessment (HTA) framework (EUnetHTA core model with 9 dimensions) to conduct a report comparing the two technologies. A multidisciplinary working group was established. We derived and pooled: i) secondary data derived from systematic literature reviews, and ii) original data from IRCCS San Raffaele Hospital, Milan, a national referral centre for TAAA (qualitative and economic data). Results Endovascular repair resulted superior to the traditional open surgery in terms of efficacy and safety, as justified by the meta-analysis we performed. Despite the higher costs, a significant impact on budget and slightly lower cost-effectiveness, the endovascular protheses’ adoption could provide conspicuous benefits in terms of social and ethical dimensions without affecting long-term organisational and legal aspects. Conclusions The multi-criteria decision analysis carried out from a hospital point of view shows that there is no significant difference (final score endovascular repair 0.68 vs open surgery 0.63) between the two procedures considering all the dimensions. Still, the endovascular repair is slightly superior to the open surgery in terms of safety, effectiveness, social, ethical, legal, and organisational impact. From the policy maker’s point of view, technologies with a score superior to 0.6 are equally valuable, and the final decision should be left to the clinicians. Key messages • Further research is needed to compare endovascular prostheses and open surgery’s long-term population benefits, balancing clinical, economic, organisational and patient-related outcomes. • HTA methodology offers substantial support to compare in-use technologies, informing clinicians’ and decision-makers’ choices to strengthen healthcare provision equity and preparedness.

3rd Annual International Pharmacoeconomic Forum Highlights.

3rd Annual International Pharmacoeconomic Forum Highlights.

Health Technology Assessment in India: Necessity, Progress and Future Plans

The implementation of a universal healthcare coverage by the Indian government has forced a relook into the existing systems of healthcare decision-making. Health technology assessment (HTA) is being looked at to provide efficient decision-making and helping in priority setting for the policymakers. The Ministry of Health and Family Welfare has, therefore established a Medical Technology Assessment Board as a way of institutionalising the HTA framework and methodology in India. Despite some challenges, the future looks bright for HTA in India.

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Health technology assessment (HTA) is intended to determine the value of health technologies and, once a technology is recommended for funding, bridge clinical research and practice. Understanding the values and beliefs expressed by patients and health professionals can help guide this knowledge transfer and work toward managing the expectations of end users. We gathered patient and patient group leader experiences to gain insights into the roles that patients and patient advocacy groups are playing. We argue that through partnerships and co-creation between HTA professionals, researchers and patient advocates we can strengthen the HTA process and better align with service delivery where person-centered care and shared decision making are key elements. Patient experiences and knowledge are important to the democratization of evidence and the legitimacy of HTAs. Patient preference studies are used to balance benefits with potential harms of technologies, and patient-reported outcomes (PROs) can measure what matters to patients over time. A change in culture in HTA bodies is occurring and with further transformative thinking patients can be involved in every step of the HTA process. Patients have a right to be involved in HTAs, with patients' values central to HTA deliberations on a technology and where patients can provide valuable insights to inform HTA decision-making; and in ensuring that HTA methodologies evolve. By evaluating the implementation of HTA recommendations we can determine how HTA benefits patients and their communities. Our shared commitment can positively effect the common good and provide benefits to individual patients and their communities.

European collaboration on health technology assessment: looking backward and forward

The establishment of health technology assessment (HTA) has been an important topic in Europe for many years. There have been a series of activities starting with first projects in 1994 leading to joint actions from the European Network of HTA (EUnetHTA) ending in 2021. This long interval of engagement with HTA structures, methodology, and processes by all member states led to a reliable basis for European collaboration in HTA. This article shows milestones and developments from EUR-ASSESS in 1994 through the progress of EUnetHTA and the accompanying EU-HTA-Network up to the recent elaboration of the EU-HTA-Regulation. With the EU-HTA-Regulation HTA collaboration is taken out of the trial phase of more than 15 years. Through the previous EU HTA collaboration, the appreciation and understanding of the differences and complexities behind the HTA processes in the EU healthcare systems have improved. It is now necessary to make the final steps toward a sustainable European Network for HTA.

Patient Perspectives: An Integral Part of Health Technology Assessment Methodology.

Patient Perspectives: An Integral Part of Health Technology Assessment Methodology.

Expectations for the Development of Health Technology Assessment in Brazil.

The implementation of health technology assessment (HTA) in emerging countries depends on the characteristics of the health care system and the needs of public health care. The objective of this survey was to investigate experts’ expectations for the development of HTA in Brazil and to derive measures to strengthen the impact of HTA in Brazil on health care decisions. Based on a scoping literature review, a questionnaire was developed proposing eight theses for seven domains of HTA: (i) capacity building, (ii) public involvement, (iii) role of cost-effectiveness analysis (CEA), (iv) institutional framework, (v) scope of HTA studies, (vi) methodology of HTA, and (vii) HTA as the basis for jurisdiction. Thirty experts responded in full to the survey and agreed to five of the eight theses proposed. Experts suggested several measures to promote HTA within the scope of each domain, thus addressing capacity building related to HTA, availability, and reliability of population data, and legal endowment of the HTA system. Finally, HTA processes in Brazil should also address public health issues (e.g., appraisal of interventions directed at chronic diseases).

HTA of novel biocontainment systems for ambulance transport: public health implications

BackgroundBiocontainment systems in healthcare settings are intended to ensure isolation of contagious or potentially contagious patients so as to protect healthcare providers and control infection spread. The need for safe and effective biocontainment systems has emerged during the COVID-19 outbreak. In particular, transport of potentially contagious patients poses technical, logistical and medical challenges that are to be addressed to protect population health and enable safe healthcare services delivery.MethodsAs novel biocontainment systems become available it is important to assess their operational effectiveness and safety and to define their organizational impact. We applied the Health Technology Assessment (HTA) framework to evaluate a novel rigid bio-containment system (Billy-Cab) integrated with the stretcher support, for ambulance transport of contagious patients.ResultsWe applied the EUnetHTA core model to conduct an HTA report of Billy-Cab. A multi-disciplinary working group was established. We assessed available biocontainment under the nine dimensions of the EUnetHTA core model. We derived and pooled: 1) original data derived from clinical practice, 2) secondary data derived from systematic reviews of the literature, conducted according to the PRISMA guidelines, 3) original data derived from validated questionnaires, 4) economic original data.ConclusionsPreliminary data allow to conclude selected biocontainment systems are effective in ensuring infection control during ambulance transport, with manageable organizational impact. In addition, the use biocontainment systems positively influence healthcare professionals' attitudes and performances. The HTA methodology offers substantial support to evaluate the introduction of innovative technologies during health emergencies and beyond.Key messages Selected biocontainment systems are effective in ensuring infection control during ambulance transport.HTA is to be applied to evaluate the introduction of innovative technologies during health emergencies and beyond.

Investigation of Factors Considered by Health Technology Assessment Agencies in Eight Countries.

BackgroundHealth technology assessment (HTA) organizations play a crucial role in optimizing healthcare resources, but the factors influencing decision making vary by country.ObjectiveHTAs of cancer and hepatitis C drugs were evaluated across developed countries to understand differences in decision processes and criteria.MethodsThe HTA organizations evaluated are from France, Germany, Italy, Spain, the United Kingdom (UK), Australia, Canada and Japan. Economic evaluation types and 28 factors in the following categories were evaluated: clinical uncertainties/issues; disease/population/treatment consideration factors including National Institute for Health and Care Excellence’s (NICE) special circumstances factors (e.g. end-of-life and innovation); and International Society for Pharmacoeconomics and Outcomes Research (ISPOR) additional value elements. Qualitative and correspondence analyses were conducted to assess the differences across organizations.ResultsIncremental cost-effectiveness ratio (ICER) using quality-adjusted life-year (QALY) was evaluated in Canada, the UK, Australia and Japan. The highest observed clinical uncertainties were clinical benefits and comparator. For cancer drugs, correspondence analysis showed France, Australia, Canada and the UK to have common attributes observed, such as unmet needs and stakeholder persuasion. In addition, the UK reported end-of-life, issues around current treatment and innovation, whereas Germany reported manageable/insignificant adverse events more frequently. Finally, fear of contagion, equity and scientific spillover value elements were only observed in Australia.ConclusionAlthough clinical factors play a predominant role in the decision to reimburse medicine, HTA organizations consider additional aspects as well. If the methodology of HTA was clearly outlined, there would be more transparency in HTA systems leading to better understanding amongst stakeholders about decision making.Electronic supplementary materialThe online version of this article (10.1007/s41669-020-00235-6) contains supplementary material, which is available to authorized users.

Health technology assessment methodology in metastatic renal cell carcinoma.

Cancer care is currently experiencing rapid development in novel therapeutics, with an associated rise in treatment costs. These changes not only create a critical challenge for therapeutic decision-making but also highlight the need to prioritize therapies of high clinical and economic value. Health technology assessment methodology is a novel approach that could help guide value-based decision-making.

Identification of a strategy for disinvestment in the Italian National Health Service (NHS)

Abstract The disinvestment of ineffective or inappropriate technologies or interventions with an inadequate value for money ratio is a growing priority in order to contribute to the objectives of maintaining high quality and appropriate care and allocation of resources that guarantee the sustainability of the National Health System (NHS).The possibility of reallocating resources from obsolete and no longer appropriate technologies, allows for recovered resources to be allocated to technological innovation and therefore to the overall sustainability of the system.In order to define criteria concerning disinvestment techniques of health technologies and strategies of obsolete technologies to be applied within the Italian reference framework, we started an ongoing research project. A systematic review of the scientific literature of the last 10 years was carried out on 5 different databases with the purpose of analyzing the international disinvestment experiences of health technologies using the Health Technology Assessment (HTA) method and focusing on orthopedic prosthesis procedure and cardiac stenting procedure.By means of National Dataset of the Italian National Institute of Health, real consumption and estimated consumption have been identified,using the Italian NHS perspective. The preliminary systematic review retrieved a total of 548 records. Nine results were finally included. In the development of disinvestment strategies, particular attention must be paid to the identification of an integrated model between the different levels of the NHS (national,regional,local). In Italy there are no prior example of broad and complex disinvestment experiences using HTA methodologies. Effective disinvestment strategies require a systematic approach coordinated by a national body and implemented at regional level,to be extended to cascade to the organization and provision of services and services by health companies,and required to actively involve health professionals and citizens. Key messages Identifying effective strategies for disinvestment of health technologies is crucial, considering continuous health innovation and improvement and development opportunities. HTA methodologies, such as reassessment, could support disinvestment processes and represent a systematic and objective approach to be implemented and used at different levels of Italian NHS.

Multiple Criteria Decision Analysis for HTA across four EU Member States: Piloting the Advance Value Framework

Multiple Criteria Decision Analysis (MCDA) has emerged as a methodology for Health Technology Assessment (HTA). However, limited empirical evidence is available on its use by decision-makers; where available, it only comes from single-setting exercises, while cross-country comparative studies are unavailable. This study applies the Advance Value Framework (AVF), an MCDA methodology for HTA based on multi-attribute value theory, through a series of case studies with decision-makers in four countries, to explore its feasibility and compare decision-makers' value preferences and results.The AVF was applied in the evaluation of three drugs for metastatic, castrate resistant, prostate cancer (abiraterone, cabazitaxel and enzalutamide) in the post-chemotherapy indication. Decision conferences were organised in four European countries in collaboration with their HTA or health insurance organisations by involving relevant assessors and experts: Sweden (TLV), Andalusia/Spain (AETSA), Poland (AOTMiT) and Belgium (INAMI-RIZIV). Participants' value preferences, including performance scoring and criteria weighting, were elicited through a facilitated decision-analysis modelling approach using the MACBETH technique.Between 6 and 11 criteria were included in each jurisdiction's value model, allocated across four criteria domains; Therapeutic Benefit criteria consistently ranked first in relative importance across all countries. Consistent drug rankings were observed in all settings, with enzalutamide generating the highest overall weighted preference value (WPV) score, followed by abiraterone and cabazitaxel. Dividing drugs' overall WPV scores by their costs produced the lowest “cost per unit of value” for enzalutamide, followed by abiraterone and cabazitaxel. These results come in contrast with the actual country HTA recommendations and pricing decisions.Overall, although some differences in value preferences were observed between countries, drug rankings remained the same. The MCDA methodology employed could act as a decision support tool in HTA, due to the transparency in the construction of value preferences in a collaborative manner.

An integrated multi-criteria decision making methodology for health technology assessment

This study presents a synthesis on the application of multi-criteria decision making (MCDM) methodologies as an innovative tool to provide a more robust and reliable selection or ranking processes for health technology assessment (HTA). HTA is described as the 'systematic' evaluation of health related technologies with respect to multiple criteria. Thus, to carry out effective HTA processes, decision-makers should consider utilising integrated or hybrid decision making processes. In this study, we develop a decision support tool called 'DEMATSEL' which serves as an integrated MCDM methodology in fuzzy environments. Our integrated approach combines four widely-used MCDM methods, viz. fuzzy AHP, fuzzy TOPSIS, fuzzy VIKOR and goal programming. We demonstrate the effectiveness of our proposed novel approach on a fuzzy HTA case study of bariatric surgery selection in Turkey. [Submitted 4 July 2017; Revised 30 December 2017; Accepted 27 February 2018]

An integrated multi-criteria decision making methodology for health technology assessment

This study presents a synthesis on the application of multi-criteria decision making (MCDM) methodologies as an innovative tool to provide a more robust and reliable selection or ranking processes for health technology assessment (HTA). HTA is described as the 'systematic' evaluation of health related technologies with respect to multiple criteria. Thus, to carry out effective HTA processes, decision-makers should consider utilising integrated or hybrid decision making processes. In this study, we develop a decision support tool called 'DEMATSEL' which serves as an integrated MCDM methodology in fuzzy environments. Our integrated approach combines four widely-used MCDM methods, viz. fuzzy AHP, fuzzy TOPSIS, fuzzy VIKOR and goal programming. We demonstrate the effectiveness of our proposed novel approach on a fuzzy HTA case study of bariatric surgery selection in Turkey. [Submitted 4 July 2017; Revised 30 December 2017; Accepted 27 February 2018]

VP24 HTA To Assess Esthetic Procedures In France: Haute Autorité de Santé (HAS) Seven Year Experience

Introduction:The Health Technology Assessment (HTA) of esthetic procedures was performed by the French National Authority for Health (HAS), at the request of the French Ministry of Health (MoH), and under a new regulatory framework enabling the government to ban esthetic procedures considered harmful or potentially harmful to patients and consumers by HAS. Objectives: Describe HAS’ seven year experience with the safety assessment of four esthetic procedures.Methods:This is an HAS review of its methods used in four HTAs for the following evaluated techniques: lipolysis; cryolipolysis; esthetic mesotherapy; and, ultraviolet (UV) radiation in tanning devices. The review aimed to describe how these assessments have been undertaken and information sources used, from 2010 to 2017, to appreciate the safety profile of these procedures.Results:A systematic literature review (SRL) and analysis was performed for all four HTAs. Since findings did not allow for thorough appreciation of safety, additional sources of information were consulted to address evidence gaps. Sources may have included any combination of the following: i) National and international health care authority data and alerts ii) Legal and ethical frameworks iii) Public consultation iv) Expert opinion v) Patient-consumer association opinion vi) Economic analysisConclusions:An adaptation of the HTA methodology was necessary to meet the specific requirements of these assessments. Despite sources accumulated and consulted within the seven year period, quantitative data were found insufficient to fully appreciate the safety profile for any one of the studied esthetic procedures. National regulatory reinforcement on the reporting of adverse events, with implementation of a centralized online tool, is expected to generate and capture reliable data on the frequency and severity of adverse events associated with esthetic procedures. Recent European Union (EU) regulatory requirements on the safety and performance of medical devices include equipment used for esthetic procedures, indicating agreement and alignment on national and EU-level monitoring efforts.