Health Technology Assessment Reports Research Articles

Evolving Evidence-Based Value Assessment of One-Time Therapies: Tisagenlecleucel as a Case Study.

Economic evaluation of one-time therapies during reimbursement decision-making is challenging due to uncertain long-term outcomes. The availability of 5-year outcome data from the ELIANA trial and real-world evidence of tisagenlecleucel, the first chimeric antigen receptor T-cell (CAR-T) therapy, presents an opportunity to re-evaluate the predictions of prior cost-effectiveness analyses (CEAs). To conduct a systematic literature review (SLR) of prior CEAs of tisagenlecleucel for pediatric/young adult relapsed or refractory acute lymphoblastic leukemia (r/r ALL) and evaluate the impact of recently available 5-year efficacy data from ELIANA and advances in CAR-T manufacturing in an updated CEA model. OVID MEDLINE/Embase and health technology assessment (HTA) databases were searched for full-text economic evaluations in English reporting cost-effectiveness results for tisagenlecleucel for r/r ALL. Evaluations with publicly reported incremental cost-effectiveness ratios (ICERs) were included in the SLR. Study screening and data abstraction were conducted following PRISMA guidelines. Data extracted included the country/currency, perspective, clinical trial evidence, model structures, long-term efficacy extrapolation approaches (i.e., overall survival [OS]), time horizon, discount rates, and outcomes (i.e., life years [LY], quality-adjusted LY [QALY], and ICERs). The CEA model reported in Wakase et al. was updated using 5-year OS data from ELIANA and the CAR-T infusion rate informed by real-world practice. Sixteen records corresponding to 15 unique studies were included in the SLR (11 publications and 5 HTA reports); all were conducted from the health care system perspective of the respective countries. Most studies found tisagenlecleucel to be cost effective, but all studies' projected 3- and 5-year OS rates for tisagenlecleucel were lower than the observed 3- and 5-year rates, respectively, derived from 5-year ELIANA data. When applying updated OS projections from the most recent ELIANA data cut and higher infusion rates of 92.5% (per the real-world infusion rate)-96.0% (per the manufacturer success rate) to theCEA of Wakase et al., the associated QALYs for tisagenlecleucel increased from 11.6 to 14.6-15.0, andLYs increased from 13.3 to 17.0-17.5. Accordingly, the ICERs for tisagenlecleucel decreased from ¥2,035,071 to ¥1,787,988-¥1,789,048 versus blinatumomab and from ¥2,644,702 to ¥2,257,837-¥2,275,181 versus clofarabine combination therapy in the updated CEA model. Projections at launch of the likely cost effectiveness of tisagenlecleucel appear to have underestimated its ultimate economic value given more recent trial and real-world data. To balance uncertainty in initial valuation with the need to provide access to novel oncology therapies, payers can consider flexible reimbursement policies alongside ongoing assessments as new data emerge.

Costs and benefits in patients with NYHA class III heart failure treated with CardioMEMS in Italy.

CardioMEMS is a device suitable for telemedicine that is currently being evaluated by the Regional Health Technology Assessment (HTA) Committee of Tuscany. Two detailed HTA reports are available in the specialized literature, the results of which need to be transferred to our regional setting. These decisions in Tuscany are made by the so-called Centro Operativo HTA. To validate, with local cost-effectiveness data, the decision on CardioMEMS that will be made in the Tuscany region. Two detailed international HTA reports were rearranged and adapted to our regional setting to generate a simplified analysis that could form the basis of our decision. Two willingness-to-pay (WTP) thresholds of €20,000/quality-adjusted life year (QALY) and €50,000/QALY were considered. Based on epidemiological and regulatory information, the target population in Tuscany for this device is 166 cases. The value-based price of CardioMEMS is estimated to be €4,332 and €16,662 at WTP thresholds of 20,000/QALY and 50,000/QALY, respectively. Its current price in Italy is €12,000. In our region, the introduction of CardioMEMS is likely to be gradual, around 50 patients/year (or €0.60 million/year at current price). This example highlights the need to adapt the information published in the international literature to the local context in which the approval decision is made. In this context, simplified analyses are easier to apply than complex Markov models.

Application of natural language processing to predict final recommendation of Brazilian health technology assessment reports.

Health technology assessment (HTA) plays a vital role in healthcare decision-making globally, necessitating the identification of key factors impacting evaluation outcomes due to the significant workload faced by HTA agencies. The aim of this study was to predict the approval status of evaluations conducted by the Brazilian Committee for Health Technology Incorporation (CONITEC) using natural language processing (NLP). Data encompassing CONITEC's official report summaries from 2012 to 2022. Textual data was tokenized for NLP analysis. Least Absolute Shrinkage and Selection Operator, logistic regression, support vector machine, random forest, neural network, and extreme gradient boosting (XGBoost), were evaluated for accuracy, area under the receiver operating characteristic curve (ROC AUC) score, precision, and recall. Cluster analysis using the k-modes algorithm categorized entries into two clusters (approved, rejected). The neural network model exhibited the highest accuracy metrics (precision at 0.815, accuracy at 0.769, ROC AUC at 0.871, and recall at 0.746), followed by XGBoost model. The lexical analysis uncovered linguistic markers, like references to international HTA agencies' experiences and government as demandant, potentially influencing CONITEC's decisions. Cluster and XGBoost analyses emphasized that approved evaluations mainly concerned drug assessments, often government-initiated, while non-approved ones frequently evaluated drugs, with the industry as the requester. NLP model can predict health technology incorporation outcomes, opening avenues for future research using HTA reports from other agencies. This model has the potential to enhance HTA system efficiency by offering initial insights and decision-making criteria, thereby benefiting healthcare experts.

Health technology assessment in traditional and complementary medicine: a scoping review of international activity and examples of acupuncture.

Traditional therapies are crucial in maintaining and improving human well-being. China's healthcare policymakers are attempting to use health technology assessment (HTA) as a decision-making supportive tool. The value assessment framework for Chinese patent medicine (CPM) has been developed and is being adopted and validated widely by research institutions. Subsequently, the healthcare decision-makers particularly hanker for the value framework of traditional non-pharmacological therapies. To construct a practical value framework for traditional non-pharmacological therapies, a scoping review methodology was adopted to identify the evaluation domains and obstacles. A search, screening, and analysis process was conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR). Evidence was retrieved from scientific databases and HTA agencies' websites. The search strategy identified 5 guidelines records and 17 acupuncture HTA reports. By synthesizing the valuable reports of CPM and acupuncture evaluation in representative countries, this study found that Mainland China was promoting the comprehensive value assessment of CPM, whereas the United Kingdom, Singapore, Canada, the United States, and Malaysia had carried out the HTA evaluation of acupuncture for various conditions among which chronic pain was the most common. UK and Singapore applied the HTA results to support acupuncture reimbursement decisions. Three domains, including safety, effectiveness, and economy, were commonly adopted. The identified biggest challenge of evaluating traditional non-pharmacological therapies is the scarce high-quality clinical evidence. This study identified value domains and issues of traditional therapies, and pointed out future research implications, to promote the development value framework of traditional therapies.

Research gaps identified in Iran’s health technology assessment reports

IntroductionIdentifying gaps in the evidence is a useful byproduct of conducting a health technology assessment (HTA). This study aims to identify research gaps in Iran’s HTA reports.MethodWe reviewed the HTA reports published between 2014 and 2016. Then, we developed two separate questionnaires for principal investigators (PIs) and independent HTA researchers. The questionnaire for independent HTA researchers consisted of four main parts. However, the PIs’ questionnaire consisted of two main parts. We also conducted a literature search in the PubMed database in November 2017 to find frameworks for prioritizing research gaps. We also conducted a semi-structured interview with the head of the Iran’s HTA Office at that time and sought feedback based on his expert opinion about questionnaires, the priority-setting tool and our process for extracting research gaps.ResultsA total of 11 HTA reports published between 2014 and 2016 by Iran’s HTA Office were selected for the study. Of these 11 reports, 5 involved technologies related to medical equipment, while 6 involved medical and surgical interventions. Assessing the outcomes of technology use in various indications and updating HTAs when new evidence arises; evaluating the viewpoints of patients, clinicians and key technology users; conducting post-marketing evaluations of technology; comparing the impact of the technology in question to other treatments for the same condition; and requesting long-term clinical and cost–effectiveness data for technologies with limited follow-up periods were identified as the main gaps by independent HTA researchers and PIs.ConclusionsThe research gaps identified from Iran’s HTAs could be utilized by research funding agencies.

PP149 Reengineering Of Processes For The Elaboration Of Health Technology Assessment Reports In Catalonia

IntroductionIn order to increase the value of its services and activities, the Agency for Health Quality and Assessment of Catalonia (AQuAS) has incorporated in its strategic plan the commitment to improve the processes, quality and people, while ensuring transparency, independence, rigor and efficiency following the guidelines of the European Foundation for Quality Management. We aim to present the standardization processes to improve the efficiency in elaborating health technology assessment (HTA) reports at AQuAS.MethodsProcess standardization has been developed in seven stages: (i) definition of scope, objectives and creation of working groups; (ii) mapping and analysis of all ongoing processes to determine whether they needed improvement or were already optimal; (iii) creation of new processes by evaluating the inclusion of automatic tools and their possible digitization; (iv) creation of the process map; (v) communication to the team for its implementation after training; (vi) dissemination on corporate website; (vii) monitoring and evaluation of their impact.ResultsThe creation of AQuAS’ HTA report development process map has involved 14 people over the past two years. After an initial two-day workshop, the team was organized in working groups of two to three people, with regular monitoring and the creation of a specific knowledge management unit led by the AQuAS’ evaluation manager. The process map was configured based on three axes. The strategic axis contains legal frameworks, ethical principles, good practices and methodological frameworks (29 specific for HTA reports). The key axis presents in an integrated way the process and methodology followed from the request of the HTA report to the final product’s dissemination. The support axis includes, among other, the management of 31 requests, information specialist processes (37 searches), training (51 courses) and 6 internal procedures with their corresponding 26 templates and 27 tools.ConclusionsWith a process reengineering approach, HTA reporting has become more efficient. We believe this approach can help other agencies improve their internal processes and subsequently improve team and customer satisfaction.

PP01 Health Technology Assessment Of Cervical Artificial Disc Replacement: Highlighting The Need For A Consistent International Approach

IntroductionCervical artificial disc replacement (C-ADR) is not a new technology but one that has seen many technological advances in the past 10 years. Indeed, a recent review described total disc arthroplasty as the most innovative development in the history of spinal surgery. The primary goals of C-ADR are to reduce or eliminate pain, and restore normal segmental motion. The aim of this analysis was to identify, extract and examine key health outcomes and economic data from published health technology assessment (HTA) reports on C-ADR, with the aim of understanding how the evolution of this technology has influenced assessments internationally.MethodsA comprehensive search of over 90 HTA organization websites and the INAHTA HTA database using key terms for C-ADR surgical procedures was coupled with a literature search of recent systematic reviews. No language restrictions were applied.ResultsTwenty HTA reports of C-ADR surgery published from 2005 to 2022 were included for review. Several HTAs (4/20) were updates or reassessments by the same agency and one was an update across agencies (Italy update of Belgian HTA). While many of the HTAs concluded C-ADR is as effective as standard care and superior in certain outcomes, there was no pattern or consistency in the conclusions or recommendations from these assessments, even as the evidence base expanded over time. Our analysis found this was largely due to variations in HTA approaches among agencies including: differences in research questions asked, PICO (Population, Intervention, Comparator, Outcomes) criteria and methods performed, such as: rapid versus full systematic reviews; inclusion of economic evaluations and/or budget impact analyses.Indeed, one of the only predictive factors for a positive HTA was a favorable cost-effectiveness analysis.ConclusionsC-ADR is an established technology with extensive HTA investigation internationally. The lack of a consistent approach taken by HTA bodies made prediction of successful HTA outcomes difficult. Future alignment of key evaluation processes and methods may help address current international variations and support consistent decision making on patient access.

OP23 Early Detection Tools For Emotional Distress In Adult Cancer Patients In Spain: A Health Technology Assessment Report

IntroductionPatient-reported outcome measures are being increasingly considered both in clinical practice and in the field of health technology assessment. Although emotional distress is currently recognized as the sixth vital sign in cancer care, its early detection and screening is not yet included in routine clinical practice in Spain. The main objective of this study was to assess the psychometric properties and diagnostic accuracy of validated tools for the early detection of distress among adults with cancer in the Spanish context, at the request of the Spanish National Health System (NHS) Cancer Strategy.MethodsA systematic review was carried out to analyze development and validation studies. The Quality Assessment of Diagnostic Accuracy Studies tool (QUADAS-2) was used for the risk of bias assessment, and a multicriteria global assessment was used for the tests. Ethical and organizational aspects were also addressed.ResultsFifteen validation studies were included, corresponding to seven tests. The tools considered were the Distress Thermometer (DT), the Brief Symptom Inventory-18 (BSI-18), the Edmonton Symptom Assessment System-revised (ESAS-r), the Hospital Anxiety and Depression Scale (HADS), the Visual Analog Scale for Anxiety and Depression (VAS-AD), the Detection of Emotional Distress (DED) scale, and the Psychosocial and Spiritual Needs Evaluation (ENP-E) scale. Evidence of validity, reliability (internal consistency), and diagnostic accuracy (sensitivity, specificity, and area under the receiver operating characteristic curve) were summarized. Three scales were rated as poor (VAS-AD, BSI-18, and ESAS-r), the ENP-E scale was rated as acceptable, and three scales were rated as moderate (DT, DED, and HADS).ConclusionsThe DT (single-item measure) stands out as an appropriate tool for early detection of emotional distress in the Spanish NHS. The use of this scale could be considered a first stage, to be combined later with a longer scale to improve screening specificity. The HADS scale could be utilized for this purpose. The use of these tools should be framed within a structured screening program that ensures further evaluation and subsequent psychological care when needed.

PP27 The Private Healthcare Responses Against COVID-19 Emergencies In Brazil

IntroductionThe Corona Virus Disease 2019 (COVID-19) pandemic is the biggest public health crisis of all time. Private health care plays a major role in health globally. We conducted a study to document the engagement of a Brazilian private health care organization to influence decisions to private and public health emergencies of COVID-19.MethodsThis retrospective study evaluated the outputs of the health technology assessment (HTA) group of Unimed-BH, a private Health Maintenance Organization (HMO) with 1.5 million participants in Belo Horizonte, Minas Gerais state. The study evaluated the impacts on the overview of health local decisions in the municipality and the national supplementary health agency (ANS) during the period from March 2020 to December 2022.ResultsDuring the pandemic, Unimed-BH made all its sanitary decisions based on scientific research, such as the use of masks and appropriate medications. Even though some medicines, such as regdanvimab, were authorized for emergency use by the Brazilian Health Regulatory Agency (ANVISA), Unimed-BH did not recommend their use due to uncertain evidence, and months later, ANVISA withdrew the registration.Unimed-BH also conducted a systematic review of ivermectin for COVID-19 treatment, which showed no effect, and therefore did not recommend its use. Additionally, Unimed-BH provided weekly updates on COVID-19 data, including suspected and confirmed cases, hospitalizations, and deaths in their customer portfolio. The organization also actively supported the decisions made by ANS and municipal managers using evidence and statistics on the pandemic. The Unimed-BH HTA group produced a total of 167 reports from March 2020 to December 2022.ConclusionsBelo Horizonte had the lowest in-hospital mortality rates with COVID-19 in Brazil. Unimed-BH’s HTA reports provided evidence-based assessments for decision-making, proposed partnership with policymakers, fomented information transparency, and strict follow-up on pandemic numbers, which may have contributed to the lower fatality rate in our city. These findings underscore the importance of private healthcare organizations in responding to COVID-19 emergencies, and their potential to support evidence-based decision-making and minimize the impact of the pandemic.

PP14 The Role Of Health Technology Assessment In The Public Hospitals Of The National Health Service Of Catalonia

IntroductionHospitals play an essential role to facilitate the appropriate introduction of health technologies into the National Health System. We aimed to analyze if the decision-making at the hospital level is based on health technology assessment (HTA).MethodsWe developed a seventeen-question survey to assess the decision-making mechanisms for the introduction of health innovation into the public hospitals of the National Health Service (NHS) of Catalonia. All questions were related to the field of HTA and health innovation. We asked about the process of new health technology acquisition, the existence of HTA units, and the impact those units might have within the center. We invited the three main public healthcare providers (n= 68 centers). We performed a quantitative analysis grouping the results by type of healthcare provider.ResultsThirty-five different health providers (51.5%) remitted forty-two responses. We identified two differential mechanisms for the introduction of health innovation in those centers: 51.4 percent used a directive committee for decision-making, whereas 37.1 percent employed a technical commission. Some centers (8.6%) used both options. Both the directive committees and technical commissions predominantly considered the professional experience or scientific evidence provided by the healthcare professionals of the center (80%) or external HTA reports or clinical practice guidelines (65.7%) to guide their decision-making. Using HTA products developed within the center was less frequent (34.3%). Only 57.1 percent of the healthcare providers had an HTA unit, but 75 percent of them declared that HTA products had a direct and quantifiable impact on their decision-making regarding the introduction of innovation. Healthcare providers without HTA units manifested their willingness to adopt HTA practices into their decision-making processes.ConclusionsHTA at the hospital level is not a common practice when deciding to introduce innovative health technologies in the NHS of Catalonia. However, the main healthcare providers agreed that the introduction of HTA is necessary for improving their decision-making and that the HTA agency, Agency for Health Quality and Assessment of Catalonia (AQuAS) will play a key role (training, promoting, and assessing).

OP147 Measuring Health Technology Assessment Impact On The Introduction Of Transcatheter Aortic Valve Replacement In A Private Healthcare System

IntroductionAortic stenosis is an insidious disease that has a high mortality rate when it becomes symptomatic. Surgical valve replacement is the treatment of choice and has predictable risks. Transcatheter aortic valve replacement (TAVR) is a less invasive alternative to surgery, which is indicated for high-risk patients.Complications after TAVR include paravalvular leak, cerebrovascular events, and the need for pacemaker implantation. A health technology assessment report carried out by the Health Technology Assessment Unimed-BH group in 2018, two years before it became part of the National Supplementary Health Agency, recommended the introduction of TAVR with the following criteria: indications provided by a group of specialists; forwarding of a report with detailed clinical data; results of imaging exams; and follow-up results for up to one year after the procedure. After the introduction of TAVR with the agreed criteria, it was possible to access TAVR results from the private healthcare system of Unimed-BH.MethodsAdministrative data were collected from the Unimed-BH database. All patients who received a TAVR implant from 2013 to 2017 were included by virtue of a court injunction, and after 2018 by operator concession and within agreed criteria.ResultsFrom July 2013 to June 2019, 83 patients underwent TAVR implantation by Unimed BH. The median age of patients was 83.4 years (interquartile range 66.5 to 97.9), most of whom were women (56%). There was a predominance of patients in New York Heart Association classification III (50%) and IV (29%). There were 36 patients who underwent TAVR before 2018 and 47 patients within the agreed criteria. In the period prior to the agreed criteria, 28 percent needed a pacemaker, compared with 23 percent after 2018. During the follow-up period, 39 patients died: 18 (50%) before 2018 and 11 (23%) after 2018.ConclusionsThe agreement made with the providers, which included the obligation of having a team of specialists responsible for the indication and access to clinical data through the report, improved patient outcomes. This may be due to having a better indication for the procedure or to the greater experience of the professionals involved in its delivery.

PP19 Health Technology Assessment Reports In Brazilian Unified Health System: Number Of Potential Beneficiaries in 2022

IntroductionUniversal access to health public services was established in Brazil 32 years ago. However, health technology assessment (HTA) as a requirement for the decision-making process in the Unified Health System (SUS) was defined only in 2011 with the National Committee for Health Technology Incorporation of the SUS (Conitec), which advises the Ministry of Health on the decision to incorporate health technologies into the SUS. All of Conitec’s recommendations are based on the best available scientific evidence regarding efficacy, effectiveness, and safety of a technology, but also include economic evaluation studies of these technologies, developed from the perspective of the SUS. Considering this an estimate of the population eligible for use of the technology under evaluation, if it is incorporated into the SUS, it is considered in the decision-making process.MethodsThis descriptive study, based on open access data from Conitec’s website, aimed at identifying the estimated number of patients potentially benefitting by the recommendations published in 2022, from January to November. Since each report includes an estimate for the first five years of incorporation of the technologies, all data were collected to check whether population growth was considered.ResultsFinally, 38 recommendation reports were identified, with 28 reports on incorporation and 10 reports for expansion of technology use. Among them, they related to 31 medicines and seven procedures or products. In the first year after incorporating the listed technologies, a total of 7,767,321 potential beneficiary patients were estimated, while over five years the total number would increase to approximately 7,967,874 patients. There were recommended drugs for rare diseases whose benefited population did not exceed the estimate of 10 patients per year; as an example is cerliponase alfa for treating neuronal ceroid lipofuscinosis. In another example, an estimate of 4,494,539 women would benefit by incorporated contraceptives into the SUS.ConclusionsThis study describes how accessible public health is becoming, meeting more health policies, showing potential to benefit more patients every day and showing what trends can be expected in the future.

OP165 Changing Decision Criteria: An Analysis Of Health Technology Assessment Reports To The Brazilian Unified Health System

IntroductionIn Brazil, the National Committee for Health Technology Incorporation of the Brazilian Public Health System (Conitec) defines the criteria for incorporating technologies into the public health system. However, all recommendations are submitted to public consultations to ensure transparency and public participation.Contributions from the public consultation are analyzed and entered into Conitec’s final report and sometimes change the preliminary recommendation.MethodsThis descriptive, retrospective exploratory study aimed to gather qualitative and quantitative data on criteria considered by healthcare decision makers from Conitec and to identify which criteria most changed after reports were submitted to public consultation. Data from 2012 to November 2022 were collected from the Conitec website, entered into a specific extraction form, and analyzed using descriptive statistics.ResultsMedicines (77%) were the most frequently evaluated type of technology, followed by procedures (15%), and products (8%). A total of 763 recommendations were analyzed. Of these, 70 percent did not change the decision criteria from the preliminary to the final published report, 9 percent did change, and 20 percent were simplified analyses of technologies of relevant public interest that were of low cost and low budget impact (these were not submitted to public consultation or public hearing). As pharmaceutical companies usually propose cost reductions or discounts during the Conitec public consultations, most of the recommendations (45%) were modified in the final report, with incremental budgetary reduction being the main criterion. This study identified that most of the changes occurred in neurology and were related to the high incremental budgetary impact of technologies for the care of rare diseases like spinal muscular atrophy.ConclusionsAlthough different criteria are considered for Conitec recommendations, some of these are regarded as crucial for decision-making. Unfortunately, as there is no definition for the weight of each of these criteria, it is difficult to understand their influence on the recommendations made. Therefore, it is crucial to establish standardized criteria for proper decision-making.

Criteria adopted in different models of public healthcare systems for the evaluation of reimbursement recommendations of orphan drugs: a scoping review.

Introdução: Access to drugs for rare diseases constitutes a challenge to healthcare systems, especially those with public funding. The difficulty of conducting robust clinical trials limits the quality of evidence and elevates the cost of development, later translated into the drug’s prices. Thus, it is necessary for the Health Technology Assessment (HTA) agencies to have differentiated criteria for the evaluation of reimbursement recommendations when dealing with such drugs. Objetivos: The objective of this research is to identify and summarize the specific criteria used when evaluating reimbursement recommendations for orphan drugs that are adopted by HTA agencies in countries with different models of public healthcare systems. Material e Método: A comprehensive literature search was performed on the databases PubMed, LILACS, Scopus and Embase up to March 2022. We included any publication type (opinion articles, commentaries, editorials, original articles and reviews) that addressed the criteria used by HTA agencies in countries with public healthcare systems when evaluating reimbursement recommendations for orphan drugs. Resultados: This scoping review summarizes the identified criteria for 18 countries and ranks them within three models of healthcare systems (NHS, NHI and SHI). We identified that NHS countries, such as the UK, Sweden, and Italy, lean towards innovation, the collection of real-world data, and the impact on organizational aspects of the system. Meanwhile, SHI countries, such as Germany, France and the Netherlands, often employ budget ceilings and expedited evaluation processes. All models shared concern over unmet need and disease nature. The 16 included studies range from 2015 to 2022 and the majority consists of reviews of HTA reports and original articles. Discussão e Conclusões: This review provides a good basis for the understanding of each model’s classification and general tendencies when creating differentiated criteria to accommodate and compensate for the lack of evidence and investment around rare diseases.

Analysis of heterogeneity of the different health technology assessment reports produced on the transcatheter aortic valve implantation in patients with severe aortic valve stenosis at low surgical risk.

Symptomatic severe aortic stenosis is a congenital or acquired aortic valve disease that occurs when the aortic valve of the heart narrows. It represents the most common valvular disease in adults and generally has a degenerative nature. Transcatheter aortic valve implantation (TAVI), due to its non-invasive approach, has become the standard treatment in patients who are ineligible to surgery or at high surgical risk, and it is also increasingly being performed in patients at intermediate to low surgical risk. The aim is to analyze the heterogeneity and explore the limitations of current health technology assessments (HTAs) on TAVI. For the purpose of this analysis, a review of the literature based on manual research was performed. A population, intervention, comparators, and outcome (PICO) model was used to gather the HTA reports assessing TAVI in the treatment of patients affected by symptomatic severe aortic valve stenosis at low surgical risk. Furthermore, a manual search has been developed to also include assessments from the Haute Autorité de Santé. At the end of the investigation, a certain degree of heterogeneity in the evidence factored and in the recommendations on the technology has emerged. Relative to the clinical domains, the main drivers for the disparity are found in the type of evidence considered and in the use or not of the grading of recommendations, assessment, development, and evaluation (GRADE) methodology to evaluate the quality of the clinical evidence included. Another element concerns the chosen device generation assessed within the evaluation. In order to perform the economic evaluation, a cost-utility analysis and a budget impact model were developed. Despite some elements of heterogeneity, the economic assessments demonstrate a favorable or dominant cost-effectiveness profile for TAVI compared with surgical aortic valve replacement (SAVR). Despite the presence of heterogeneity elements both in clinical and economic domains, HTA agencies reached the same recommendations on the use of TAVI. It emerged the need for a centralized vision on the "strong" domains, which means giving up freedom to local bodies to adapt to their context on the "soft" ones. This approach could have the potential to strengthen the role of HTA in Europe by ensuring faster decision-making and equity of access to health innovations and reduce the heterogeneity in the assessment methods.

Ethical soundness of health technology assessment reports in Islamic Republic of Iran.

Health technology assessment (HTA) is a conventional method for evaluating reasonable use of health technologies in many countries. To investigate the ethical soundness of HTA studies in Islamic Republic of Iran. All HTA reports published by the HTA office until 2020 were reviewed using the HTA Core Model and the Q-SEA questionnaires. We evaluated 91 reports for ethical soundness. The research question, literature search and inclusion/exclusion criteria were included in 91.2%, 83.5% and 82.4% of the HTA reports, respectively. Only 13.2% of the reports explicitly stated the objective of the analysis and 6.6% stated the ethics framework. Only 2.2%, 4.4%, 9.9%, 9.9%, 14.3%, and 2.2%, respectively, of the reports, complied with the completeness, bias, policy implications, other implications, conceptual clarification, and conflicting values. HTA reports in the Islamic Republic of Iran require coordinated and integrated framework acceptable to all stakeholders to ensure their compliance with sound ethical requirements.

Evaluating the quality of health technology assessment reports in a developing country.

No single method of health technology assessment (HTA) can meet all the policy- and decision-making needs in a country. However, there should be minimum criteria for performing HTA worldwide, and many HTA agencies have reached a consensus on this. This study aimed to assess the quality of HTA reports in the Islamic Republic of Iran. We examined all the HTA research reports published by the Iranian HTA office up to 2020, using the International Network of Agencies for Health Technology Assessment checklist for quality assessment. A total of 97 reports were examined, of which only 10.0% provided complete and appropriate contact details for further information and 5.6% clearly stated a conflict of interest. In 87.78% of the reports, the scope of assessment was clearly determined. The quality of the reports was relatively appropriate as well as details of the sources of information and text search strategies. Some 7.8%, 74.4%, 11.1%, 8.9%, and 4.4%, respectively, of the reports considered legal aspects, economic analysis, ethical implications, social implications, and other stakeholder perspectives. We recommend that minimum standards be established for the HTA process so that healthcare policy- and decision-makers can make reliable decisions on the basis of the HTA reports.

Algorithms and heuristics of health technology assessments: A retrospective analysis of factors associated with HTA outcomes for new drugs across seven OECD countries

ContextPositive health technology assessment (HTA) outcomes can have important implications for equity, efficiency and timely patient access to novel therapies. Several outcomes and dimensions of benefit beyond utility feed into HTA processes. ObjectiveWe analyse a proprietary dataset of HTA outcomes in 7 countries, to (a) test whether HTA decision-making is grounded in welfarist or extra-welfarist approaches; and (b) empirically determine the factors associated with positive HTA outcomes, the time to achieve these and establish the magnitude of inter-country differences in assessment processes. MethodsData were extracted from publicly available HTA reports on drugs that received marketing authorisation between 2009 and 2018 (N = 1415). The outcomes of interest were the probability of positive HTA outcomes and the time-to-HTA outcome; these were examined with respect to clinical, regulatory, product- and disease-related, evidence uncertainty and contextual variables. Econometric models utilising survival analysis and multinomial logistic regression were specified. FindingsPositive HTA outcomes accounted for 87.3% of the sample (n = 1235), of which 71% (n = 1004) were restricted. Drugs with positive HTA outcomes were subject to clinical restrictions (n = 652, 46%), financial risk-sharing (n = 439, 31%) or had been rejected at least once (n = 282, 20%). Significant predictors of positive HTA outcomes were orphan drugs with cancer indications, high quality of evidence linked to clinical and economic evidence uncertainties which had been overcome, and contextual considerations, particularly innovativeness and unmet need. Comparative analyses revealed systematic differences between countries in their propensity to accept the same drugs, particularly oncology and orphan drugs. ConclusionsOur results are contextual and reinforce arguments in favour of explicitly accounting for social value judgements, establishing separate assessment frameworks for highly uncertain products, adopting risk mitigation strategies for novel therapies with early phase evidence, and sharing of HTA practices across settings. Lastly, HTA agencies have adopted an extra-welfarist approach to value assessment and resource allocation.

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.

Health technology assessment for cancer medicines across the G7 countries and Oceania: an international, cross-sectional study.

Criticisms have emerged that cancer medicines offer modest benefits at increasingly high prices. Reimbursement decisions made by health technology assessment (HTA) agencies have become a complex endeavour for cancer medicines. Most high-income countries (HICs) use HTA criteria to identify high-value medicines for reimbursement under public drug coverage plans. We compared HTA criteria specific for cancer medicines in economically similar HICs, to understand how these criteria contribute to reimbursement decisions. We did an international, cross-sectional analysis in collaboration with author investigators across eight HICs, from the Group of Seven (known as G7; Canada, England, France, Germany, Italy, and Japan) and Oceania (Australia and New Zealand). Publicly available data from HTA agency reports and official documentation were extracted and analysed between Aug 15, 2021, and July 31, 2022. We collected data pertaining to the decision-making criteria used by the national HTA agency; HTA reimbursement status for 34 medicine-indication pairs corresponding to 15 unique US top-selling cancer medicines; and HTA reimbursement status for 18 cancer medicine-indication pairs (13 unique medicines) with minimal clinical benefit (score of 1 on the European Society of Medical Oncology Magnitude of Clinical Benefit Scale). Descriptive statistics were used to compare HTA decision criteria and drug reimbursement recommendations (or for Germany and Japan, final reimbursement status) across the eight countries. Therapeutic impact related to clinical outcomes of the new medicine was a uniform criterion across the eight countries, whereas quality of evidence (under the remit of therapeutic impact assessment) and equity were infrequently cited criteria. Only the German HTA agency mandated that surrogate endpoints be validated in therapeutic impact assessment. All countries except Germany included formal cost-effectiveness analyses within HTA reports. England and Japan were the only countries that specified a cost-effectiveness threshold. Of the 34 medicine-indication pairs corresponding to US top-selling cancer medicines, Germany reimbursed the maximum (34 [100%]), followed by Italy (32 [94%] recommended for reimbursement), Japan (28 [82%] reimbursed), Australia, Canada, England, and France (27 [79%] recommended for reimbursement), and New Zealand (12 [35%] recommended for reimbursement). Of the 18 cancer medicine-indication pairs with marginal clinical benefit, Germany reimbursed 15 (83%) and Japan reimbursed 12 (67%). France recommended nine (50%) for reimbursement, followed by Italy (seven [39%]), Canada (five [28%]), and Australia and England (three [17%] each). New Zealand did not recommend any medicine-indications with marginal clinical benefit for reimbursement. Considering the overall cumulative proportion across the eight countries, 58 (21%) of 272 indications for the US top-selling medicines and 90 (63%) of 144 marginally beneficial medicine-indications were not recommended for reimbursement or reimbursed. Our findings indicate discordance in public reimbursement decisions across economically similar countries, despite overlapping HTA decision criteria. This suggests a need for improved transparency around the nuances of the criteria to ensure improved access to high-value cancer medicines, and deprioritisation of low-value cancer medicines. Health systems have opportunities to improve their HTA decision-making processes by learning from the systems in other countries. None.