Technology Assessment Reports Research Articles

Is genomic testing properly evaluated? A systematic review of health technology assessment reports

Abstract Background The last decade has witnessed a steady embrace of Personalized Medicine. However, the evaluation of genetic/genomic tests is not straightforward. The purpose of this systematic review is to identify health technology assessment (HTA) reports assessing genetic and genomic tests, to summarize the methodologies used, the maturity level of the evidence included in it and the highlighted gaps in research. Methods PubMed, Scopus, and Web of Science were searched. Additionally, a desk research was performed on the main HTA reports repositories. HTA reports expressly created to assess genetic/genomic technologies including at least three core evaluation components (analytic validity, clinical validity, clinical utility, economic evaluation, organizational aspects, ethical, legal, and social implications) were included. This study was supported by the EC and MUR under PNRR - M4C2-I1.3 Project PE_00000019 ‘HEAL ITALIA’. Results Overall, 27331 unique records were retrieved, 55 of which were included in the systematic review. The reports were mainly from Australia (29%), Canada (27%) and UK (25%), regarded pharmacogenomics (36%) and oncology (35%), and analysed test use for treatment choice (29%) and diagnosis (13%). The most reported evaluation components were economic evaluation (87%), clinical utility (76%), and clinical validity (67%). Personal utility (7%), ethical (15%), legal (11%) and social (24%) implications and the patient’s perspective (27%) were poorly represented. Analytical validity, safety, and organizational aspects were included in about half of the reports. Discussion Although these are only preliminary results, the substantial lack of a shared standard in the evaluation of genetic/genomic applications appears clear, given the heterogeneity of the dimensions addressed between reports, as well as the need to strengthen the evaluation of the neglected dimensions, often of primary importance in the definition of the value and risks of personalized medicine. Key messages • The findings highlight the lack of a standard in evaluating genetic/genomic applications. • A common methodology should be developed to comprehensively evaluate genetic/genomic applications.

Cost Effectiveness of Sequencing Vedolizumab as First-Line Biologic in Ulcerative Colitis and Crohn's Disease in Canada: An Analysis Using Real-World Evidence from the EVOLVE Study.

Vedolizumab is a gut-selective anti-lymphocyte trafficking biologic indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) and Crohn's disease (CD) in Canada. The objective of this study was to evaluate the cost effectiveness of treatment sequencing for UC and CD from a public healthcare payer perspective, leveraging new real-world evidence from the literature and the EVOLVE study, a retrospective chart review. Using separate decision tree/Markov models to assess cost effectiveness for UC and CD, two sequencing approaches were estimated for adult patients (≥ 18years) diagnosed with UC or CD who were biologic-naïve: vedolizumab as first-line biologic followed by anti-tumor necrosis factor (TNF)-α versus first-line anti-TNFα followed by vedolizumab. Treatment effectiveness (response and remission), surgery rates, dose escalation and regain of response and safety inputs were estimated from EVOLVE, a retrospective chart review of real-world data, and evidence synthesis from the literature, whereas costs and utilities were estimated from health technology assessment reports, clinical trials, and the literature. Biosimilar costs were used for anti-TNFα. Both models simulated a 5-year time horizon and discounted costs and outcomes at 1.5%. Probabilistic base-case analyses (n=10,000) reported total costs (2023 Canadian dollars) and quality-adjusted life-years (QALYs). Several scenario analyses were conducted to explore robustness of results. In UC, vedolizumab as a first-line biologic followed by anti-TNFα resulted in an incremental gain of 0.09 QALYs (2.46 vs. 2.55) and saved $7179 ($134,028 vs. $126,848), making this a dominant strategy compared with first-line anti-TNFα followed by vedolizumab. In CD, use of vedolizumab as a first-line biologic resulted in an incremental gain of 0.04 QALYs (3.35 vs. 3.39) at an incremental cost of $50,631 ($89,850 vs. $140,381) versus first-line anti-TNFα followed by vedolizumab (incremental cost-effectiveness ratio of $1,265,775 per QALY). Based on this analysis, sequencing vedolizumab as a first-line biologic prior to anti-TNFα in UC and CD provided additional clinical benefit to patients. In UC, vedolizumab as a first-line biologic also saved healthcare system costs compared with anti-TNFα, whereas in CD, vedolizumab provided incremental benefit at an incremental cost, which was not considered cost effective at a threshold of $50,000/QALY.

Oral vitamin D supplementation for adults with obesity undergoing bariatric surgery.

Vitamin D deficiency following bariatric surgery is common and is expected to be associated with a deleterious impact on the skeleton. However, the benefits of vitamin D supplementation and the optimal dose in this population is currently unknown. The available guidelines on the topic are derived from experts' opinions, and are not evidence based. To compare the effects of different doses of vitamin D supplementation (low dose (less than 600 international units (IU)/day), moderate dose (600 IU/day to 3500 IU/day), high dose (greater than 3500 IU/day)) to each other or to placebo in adults living with obesity undergoing bariatric surgery. We searched CENTRAL, MEDLINE, Embase, LILACS, two trial registries, and the reference lists of systematic reviews, articles, and health technology assessment reports without language restrictions. The last search of all databases was 27 June 2023, except Embase, which we searched on 14 August 2015. We included randomised controlled trials or controlled clinical trials on vitamin D supplementation comparing different doses or comparing vitamin D to placebo in people undergoing bariatric surgery. We used standard Cochrane methods. Primary outcomes were fractures and adverse events. Secondary outcomes were vitamin D status, all-cause mortality, bone mineral change, secondary hyperparathyroidism, health-related quality of life, and muscle strength. We used GRADE to assess the certainty of the evidence for each outcome in each comparison. We identified five trials with 314 participants. We included three trials in the quantitative analysis. Moderate-dose vitamin D compared to placebo One trial compared moderate-dose vitamin D (3200 IU/day) to placebo. Moderate-dose vitamin D, compared to placebo, may improve vitamin D status and may result in little to no difference in the achieved parathyroid hormone level (achieved 25-hydroxyvitamin D level: mean difference (MD) 13.60 ng/mL, 95% confidence interval (CI) 7.94 to 19.26; achieved parathyroid hormone level: -6.60 pg/mL, 95% CI -17.12 to 3.92; 1 study, 79 participants; low-certainty evidence). The trial reported no adverse events in the moderate-dose vitamin D arm, but did not provide any information on adverse events in the placebo arm. There were no data on fractures, all-cause mortality, bone density change, health-related quality of life, and muscle strength. High-dose vitamin D compared to moderate-dose vitamin D Two trials in Roux-en-Y gastric bypass compared moderate-dose (equivalent dose 800 IU/day to 2000 IU/day) to high-dose (equivalent dose 5000 IU/day to 7943 IU/day) vitamin D. The evidence of high-dose vitamin D on adverse events is very uncertain (risk ratio (RR) 5.18, 95% CI 0.23 to 116.56; 2 studies, 81 participants; very low-certainty evidence). High-dose vitamin D may increase 25-hydroxyvitamin D levels compared to a moderate dose at 12 months, but the evidence is very uncertain (MD 15.55 ng/mL, 95% CI 3.50 to 27.61; I2 = 62%; 2 studies, 73 participants; very low-certainty evidence). High-dose vitamin D may have little to no effect on parathyroid hormone levels compared to a moderate dose at 12 months, but the evidence is very uncertain (MD 2.15 pg/mL, 95% CI -21.31 to 17.01; I2 = 0%; 2 studies, 72 participants; very low-certainty evidence). High-dose vitamin D may have little to no effect on mortality and bone mineral density at the lumbar spine, hip, and forearm, but the evidence is very uncertain. There were no data on fractures, health-related quality of life, or muscle strength. No trials reported on fractures and the evidence available on adverse events is scarce. Moderate-dose vitamin D may improve vitamin D status and may result in little to no improvement in parathyroid hormone levels compared with placebo. High-dose vitamin D supplementation (greater than 3500 IU/day) may increase 25-hydroxyvitamin D levels, and may have little to no effect on parathyroid hormone levels, compared to a moderate dose, but the evidence for both is very uncertain. The currently available limited evidence may not have a significant impact on practice. Further studies are needed to explore the impact of vitamin D supplementation on fractures, adverse events, and musculoskeletal parameters in people undergoing bariatric surgery.

Yoga as Potential Therapy for Burnout: Health Technology Assessment Report on Efficacy, Safety, Economic, Social, Ethical, Legal and Organizational Aspects.

PURPOSEOF REVIEW: This health technology assessment aimed to systematically assess the efficacy and safety of yoga as therapy for burnout. Economic, ethical, legal, social and organizational aspects were considered as well. RECENT FINDINGS: Yoga as a therapy has been shown to have positive effects on a range of symptoms, including stress, anxiety and depression. Regarding work-related stress and burnout, the effects of yoga have mainly been examined in a preventative context. Meta-analyses revealed no effects on burnout severity comparing yoga with passive controls in general. Compared with passive controls, yoga had a positive effect on subjective stress. Compared to active control, yoga had an effect on the burnout subscale depersonalization on individual study level. Yoga may have positive effects on burnout, but the results are mixed. Common definitions and standardized diagnostic tools are necessary to improve research and further assess yoga as therapy for burnout. TRIAL REGISTRATION: The HTA is registered with PROSPERO, CRD42022299405, on 6th February 2022.

Utilising Health Technology Assessment to Develop Managed Access Protocols to Facilitate Drug Reimbursement in Ireland.

The Health Service Executive, responsible for operating the Irish health service, has introduced health technology management (HTM) initiatives to manage expenditure on medicines. One such approach is managed access protocols (MAPs) to support access to high-cost medicines, while providing oversight, governance and budgetary certainty to the payer. Herein we describe the development and operation of MAPs, using case studies of liraglutide (Saxenda®), dupilumab (Dupixent®) and calcitonin gene-related peptide monoclonal antibodies. A MAP imposes the eligibility criteria attached to reimbursement support of a medicine. Criteria applied include controls on prescribing authority, clinical diagnostic and severity criteria, previous lines of treatment, concomitant treatments, outcome data collection, and validations within the reimbursement claims system. The choice of criteria are specific to each medicine, dictated by the areas of uncertainty highlighted in the health technology assessment report, such as the place in treatment, population, duration of treatment, etc., the commercial arrangements reached with the marketing authorisation holder, and specific recommendations made by the decision maker. By December 2023, there were28 medicines reimbursed subject to a MAP in Ireland. Across the three case studies outlined, over 3000 patients were accessing novel treatments for chronic illnesses in September 2023. Managed access protocols can provide some cost certainty for the payer by aligning utilisation and expenditure with committed funds, while enabling access where unmet need is highest. Managed access protocols are now established in the drug reimbursement process in Ireland, meeting the needs of both payers, patients and industry, and are likely to remain a feature of the reimbursement landscape.

Health Technology Assessment Reports for Non-Oncology Medications in Canada from 2018 to 2022: Methodological Critiques on Manufacturers' Submissions and a Comparison between Manufacturer and Canadian Agency for Drugs and Technologies in Health (CADTH) Analyses.

Identifying key differences between manufacturers' submitted analysis and economic reanalysis by the Canadian Agency for Drugs and Technologies in Health (CADTH) is an important step toward understanding reimbursement recommendations. We compared economic values reported in manufacturers' analysis with the CADTH reanalysis and also assessed methodological critiques. Two reviewers extracted data from the clinical and economic reports in publicly available CADTH reports from 2018 to 2022. We used the Wilcoxon rank-sum test to assess the difference between mean economic values, and the Chi-square test to assess the association between the CADTH critique final recommendations. Of the total submissions, 99.4% included effectiveness critiques, 88.8% included model structure critiques, 69.1% included utility score critiques, and 78.7% included cost critiques. The median incremental cost-utility ratio (ICUR) in the manufacturers' analyses was $138,658/quality-adjusted life-year (QALY), 2.5-fold lower than the CADTH's reanalysis at $380,251/QALY (p<0.001). The median CADTH reanalysis for 3-year budget impact analysis (BIA) was $4,575,102, which was 27% higher than the manufacturers' submitted 3-year BIA (p<0.001). CADTH requested a price reduction for 95% of all submissions, and the median price reduction request was 63.5%. In 2021 and 2022, the willingness-to-pay threshold identified in CADTH reports remained constant at $50,000 per QALY gained for all medications. There was high frequency of CADTH critiques on manufacturers' submissions in all four aspects of economic submissions: effectiveness, cost, utility score and structure. We observed a higher median incremental cost and lower median incremental QALYs in the CADTH reanalysis compared with the manufacturers' submissions. The resulting higher ICUR in the CADTH reanalysis often leads to a recommendation that the manufacturer needs to reduce its price. The 3-year budget impact was higher in the CADTH reanalyses compared with manufacturers' submissions.

The Impact of Additive Population(s), Intervention, Comparator(s), and Outcomes in a European Joint Clinical Health Technology Assessment

ObjectivesTo assess the potential number of European Union (EU) population(s), intervention, comparator(s), and outcomes (PICOs) based on European Network for Health Technology Assessment 21 (EUnetHTA 21) guidance and to explore further evidence-based opportunities to produce more predictable and workable EU PICOs. MethodsThe consolidated EU PICOs of 2 future hypothetical medicines in first-line non-small cell lung cancer (1L NSCLC) and third line multiple myeloma (3L MM) were derived using published health technology assessment reports of 2 recent medicines in similar indications based on EUnetHTA 21 proposed guidance. Sensitivity analysis assessed the impact of additional PICO requests. The number of analyses requested was estimated. ResultsIn 1L NSCLC and 3L MM, 6 and 9 EU Member States (MS), respectively, had published health technology assessment reports. PICO consolidation resulted in 10 PICOs for 1L NSCLC and 16 PICOs for 3L MM, increasing to 14 and 18 PICOs, respectively, when England’s National Institute for Health and Care Excellence scope was included to proxy remaining MS. A minimum of 280 and 720 analyses would be requested, exponentially increasing as additional outcome measures and subgroups are requested. ConclusionsThe PICO approach outlined by EUnetHTA 21 results in a significant number of analysis requests and substantial resources. Use of complementary analyses alongside evidence-based methods to derive PICOs and engaging with the health technology developer throughout the process would create a workable EU PICO that is predictable and most impactful for the EU, resulting in a timely and high-quality assessment report that is more usable at a MS level.

Illustrating the Financial Consequences of Outcome-Based Payment Models From a Payers Perspective: The Case of Autologous Gene Therapy Atidarsagene Autotemcel (Libmeldy®)

ObjectivesTo illustrate the financial consequences of implementing different managed entry agreements (managed entry agreements for the Dutch healthcare system for autologous gene therapy atidarsagene autotemcel [Libmeldy]), while also providing a first systematic guidance on how to construct managed entry agreements to aid future reimbursement decision making and create patient access to high-cost, one-off potentially curative therapies. MethodsThree payment models were compared: (1) an arbitrary 60% price discount, (2) an outcome-based spread payment with discounts, and (3) an outcome-based spread payment linked to a willingness to pay model with discounts. Financial consequences were estimated for full responders (A), patients responding according to the predicted clinical pathway presented in health technology assessment reports (B), and unstable responders (C). The associated costs for an average patient during the time frame of the payment agreement, the total budget impact, and associated benefits expressed in quality-adjusted life-years of the patient population were calculated. ResultsWhen patients responded according to the predicted clinical pathway presented in health technology assessment reports (scenario B), implementing outcome-based reimbursement models (models 2 and 3) had lower associated budget impacts while gaining similar benefits compared with the discount (scenario 1, €8.9 million to €6.6 million vs €9.2 million). In the case of unstable responders (scenario C), costs for payers are lower in the outcome-based scenarios (€4.1 million and €3.0 million, scenario 2C and 3C, respectively) compared with implementing the discount (€9.2 million, scenario 1C). ConclusionsOutcome-based models can mitigate the financial risk of reimbursing atidarsagene autotemcel. This can be considerably beneficial over simple discounts when clinical performance was similar to or worse than predicted.

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.

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.

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.

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.

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.

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.

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.

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.

HTA44 Economic Modeling Insights for Ultra Orphan Drugs Based on Review of NICE Highly Specialized Technology (HST) Assessment Reports

To develop insights and lessons learned for economic modeling for ultra orphan drugs based on review of NICE’s Highly Specialized Technology (HST) assessment reports.