Health Technology Assessment Reports Research Articles

A comparative analysis of international health technology assessments for novel gene silencing therapies: patisiran and inotersen.

Using the case study of patisiran and inotersen, we conducted a narrative comparative analysis of the health technology assessment (HTA) agency appraisals of these two first-in-class transthyretin gene silencers, which represent exceptional advances in the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis, a rare and multisystemic disease. Despite the impact of each product on the treatment landscape, the majority of HTAs are only considered standard of care as a comparator, resulting in a void of information and limited comprehension of the clinical and pharmacoeconomic differences between the two treatments. A search was conducted internationally for HTA reports, and only instances where assessment decisions for both treatments were publicly available were included in the present analysis. The HTA reports were analyzed broadly for the assessment of clinical and pharmacoeconomic evidence. Only economic models considering both patisiran and inotersen were included in this analysis. A total of nine agencies with public assessment reports for both treatments were identified. HTA agency assessments for both treatments were essentially positive; however, differences were noted in the final recommendations, place in treatment or reimbursed indications, and in the narrative of the evaluations. Only the Canadian Agency for Drugs and Technologies in Health (CADTH) assessment for patisiran evaluated an economic model comparing the two treatments. The differences summarized in this comparative analysis may provide a more comprehensive overview of the two treatments.

Validity of Surrogate Endpoints and Their Impact on Coverage Recommendations: A Retrospective Analysis across International Health Technology Assessment Agencies.

BackgroundSurrogate endpoints (i.e., intermediate endpoints intended to predict for patient-centered outcomes) are increasingly common. However, little is known about how surrogate evidence is handled in the context of health technology assessment (HTA).Objectives1) To map methodologies for the validation of surrogate endpoints and 2) to determine their impact on acceptability of surrogates and coverage decisions made by HTA agencies.MethodsWe sought HTA reports where evaluation relied on a surrogate from 8 HTA agencies. We extracted data on the methods applied for surrogate validation. We assessed the level of agreement between agencies and fitted mixed-effects logistic regression models to test the impact of validation approaches on the agency’s acceptability of the surrogate endpoint and their coverage recommendation.ResultsOf the 124 included reports, 61 (49%) discussed the level of evidence to support the relationship between the surrogate and the patient-centered endpoint, 27 (22%) reported a correlation coefficient/association measure, and 40 (32%) quantified the expected effect on the patient-centered outcome. Overall, the surrogate endpoint was deemed acceptable in 49 (40%) reports (k-coefficient 0.10, P = 0.004). Any consideration of the level of evidence was associated with accepting the surrogate endpoint as valid (odds ratio [OR], 4.60; 95% confidence interval [CI], 1.60–13.18, P = 0.005). However, we did not find strong evidence of an association between accepting the surrogate endpoint and agency coverage recommendation (OR, 0.71; 95% CI, 0.23–2.20; P = 0.55).ConclusionsHandling of surrogate endpoint evidence in reports varied greatly across HTA agencies, with inconsistent consideration of the level of evidence and statistical validation. Our findings call for careful reconsideration of the issue of surrogacy and the need for harmonization of practices across international HTA agencies.

(Ultra-)long-acting insulin analogues for people with type 1 diabetes mellitus.

(Ultra-)long-acting insulin analogues for people with type 1 diabetes mellitus.

Position Document on the Use of Direct Oral Anticoagulants in Nonvalvular Atrial Fibrillation

ArgentineFederation of Cardiology have carried out a detailedanalysis of the recent Ninth Health TechnologyAssessment Report by the National Commission forHealth Technology Assessment (CONETEC) on theuse of direct oral anticoagulants in nonvalvular atrialfibrillation. (1)Atrial fibrillation (AF) represents 60% of the indicationsfor anticoagulation in Argentina and worldwide.The view of AF reality in our country shouldtake into account that AF is not systematically detected,anticoagulation is rarely performed, and when itis, the levels of time in therapeutic range (TTR) areinadequate, as we have verified in our records.We believe that this assessment has its limitations:it was carried out under economistic rather thanhealthcare criteria, which means that the conclusionsdrawn are not oriented towards the correct use of avaluable resource such as the new non-vitamin K-dependentdirect oral anticoagulants (DOACs) and theirpositive impact on AF patients, but rather to theirrestrictive use due to their high cost, without counterbalancingaspects as the correct training of physiciansin concepts such as the rational prescription ofDOACs.

Health Economic Aspects of Chimeric Antigen Receptor T-cell Therapies for Hematological Cancers: Present and Future.

Since 2018, 2 chimeric antigen receptor (CAR) T-cell therapies received approval from the European Medicine Agency, with list prices around 320 000 Euro (€) (EUR) per treatment. These high prices raise concerns for patient access and the sustainability of healthcare systems. We aimed to estimate the costs and budget impact associated with CAR T-cell therapies for current and future indications in hematological cancers from 2019 to 2029. We focused on the former France, Germany, Spain, Italy and the United Kingdom (EU-5) and the Netherlands. We conducted a review of list prices, health technology assessment reports, budget impact analysis dossiers, and published cost-effectiveness analyses. We forecasted the 10-year health expenditures on CAR T-cells for several hematological cancers in selected European Union countries. Nine cost-effectiveness studies were identified and list prices for CAR T-cell therapies ranged between 307 200 EUR and 350 000 EUR. Estimated additional costs for pre- and post-treatment were 50 359 EUR per patient, whereas the incremental costs of CAR T-cell therapy (when compared with care as usual) ranged between 276 086 EUR and 328 727 EUR. We estimated market entry of CAR T-cell therapies for chronic mantle cell lymphoma, follicular lymphoma, chronic lymphocytic leukemia, multiple myeloma, and acute myeloid leukemia in 2021, 2022, 2022, 2022, and 2025, respectively. Cumulative expenditure estimates for existing and future indications from 2019 to 2029 were on average 28.5 billion EUR, 32.8 billion EUR, and 28.9 billion EUR when considering CAR T-cell therapy costs only, CAR T-cell therapy costs including pre- and post-treatment, and incremental CAR T-cell therapy costs, respectively. CAR T-cell therapies seem to be promising treatment options for hematological cancers but the financial burden on healthcare systems in the former EU-5 and the Netherlands will contribute to a substantial rise in healthcare expenditure in the field of hematology.

Mixed claims in Health Technology Assessment: The case of Non-Invasive Prenatal Testing.

Health Technology Assessment (HTA) uses explicit methods to determine the value of a health technology. This typically results in several claims regarding the effects that are expected to follow from the use of a health technology in a particular context. These claims seem to capture conclusions based solely on facts, but they often combine empirical information with normative presuppositions. Claims that have this character reflect (implicit) value judgments and have been labelled mixed claims. Not recognizing these normative components of such claims risks value inattention and value imposition, presenting results as self-evident and not in need of any moral justification. As proposed by Anna Alexandrova, to avoid these risks of value inattention and imposition we need rules to deal with mixed claims. According to her, when producing and evaluating mixed claims we need to unearth the invoked value presuppositions and check whether these presuppositions are invariant to disagreements. By applying these rules, the robustness of mixed claims can be checked: it can be evaluated whether their truth value is independent from the way in which their components, involving normative presuppositions, are conceptualized. This paper aims to illustrate the role of mixed claims in HTA, and expand upon the work by Alexandrova, by analyzing claims and recommendations presented in an HTA report on the introduction of Non-Invasive Prenatal Testing (NIPT) in The Netherlands. Our results show that the report contains mixed claims, and that a normative analysis of these claims can help to clarify the normativity of HTA and evaluate the robustness of claims on alleged effects of a health technology.

Integration of ethical considerations into HTA reports: an analysis of integration levels using a systematic review.

To describe the type and level of ethical integration in published health technology assessment (HTA) reports and systematically identify the ethical approaches utilized. A literature search was conducted with the Google™ search engine using the keyword "ethic" between 1 January 2015 and 20 August 2019. Only HTA assessment reports with a section on ethics were retained and classified according to their level of ethical integration: no ethical analysis, ethical issues highlighted, assessments according to legal or social norms, and assessments from a moral or axiological perspective-using a qualitative methodology to distinguish such integration. This review yielded 188 reports with a section identified as being on ethics, produced by seventeen HTA agencies in eleven countries. One hundred and thirty-six reports did not develop an ethical analysis, thirty-one highlighted ethical issues, seventeen conducted a norm-based ethical assessment using a descriptive approach grounded in social norms, and four developed an assessment grounded in a moral or axiological perspective. The bioethical "four-principles" framework was used, but mainly for presenting ethical issues and not as a moral framework. The majority of reports featuring a section on ethics mention ethical considerations without ethical analysis. Ethical issues are grouped with legal, social, and organizational issues and treated as contextual considerations that decision makers should be aware of. When reports present systematic norm-based ethical assessments from a descriptive perspective or ethical assessment based on a moral or axiological perspective, there is a tendency to ground these analyses in frameworks created for the purpose and reliant on a concept of ethics supporting them.

Examining the impact of different country processes for appraising rare disease treatments: a case study analysis.

Conventional appraisal and reimbursement processes are being challenged by the increasing number of rare disease treatments (RDTs) with a small evidence base and often a high price. Processes to appraise RDTs vary across countries; some use standard processes, others have separate processes or adapted processes that explicitly deal with rare disease specificities. The objective of this study was to examine the impacts of different appraisal processes for two RDTs. A case study analysis was conducted using countries with different forms of appraisal processes for RDTs for which public health technology assessment (HTA) reports were available. Two contrasting RDTs were chosen according to the criteria: rare versus ultra-rare treatment, affecting child versus adult, life-threatening versus disabling. Information from public HTA reports for each country's RDT appraisal was extracted into templates, allowing a systematic comparison of the appraisals across countries and identification of the impact of the different processes in practice. Reports from Belgium, England, France, Germany, Italy, Netherlands, Norway, Scotland, Sweden, and the USA were selected for nusinersen (for spinal muscular atrophy) and voretigene neparvovec (for inherited retinal disorders). Countries with separate or adapted processes had more consistent approaches for managing RDT-related issues during appraisal, such as stakeholder involvement and criteria to address the specificities of RDTs, creating more transparency in decision-making. Findings suggest that separate or adapted approaches for RDT appraisal may facilitate more structured, consistent decision-making and better management of RDT specificities.

The contribution of French patient and consumer groups to health technology assessments over a 2-year period: an observational retrospective study.

In 2017, The French National Authority for Health (HAS) created an open, online, systematic contribution process to enable patient and consumer groups (PCGs) to contribute to health technology assessment (HTA) carried out to aid public authorities in reimbursement and pricing decision making. This retrospective study analyzes how French PCGs contributed to the HTA process within the HAS for the first 2 years of this new mechanism. PCG contributions received between 01 January 2017 and 31 December 2018 and the recording of deliberations leading to reports of the corresponding HTAs were included. Analysis grids were designed by the investigators with 5 rounds of refinement tests on 10 random PCG contributions and the reports. Systematic data extraction was then performed separately by two investigators. PCG answers to the open-question templates and the related final HTA report published by the HAS were analyzed. Seventy-nine contributions from 44 PCGs were received and analyzed by the HAS for 78 out of the 592 HTAs performed for drugs or medical devices during the 2-year period. Twenty-five percent of the HTAs performed for drugs received at least one contribution. The contributions covered quality-of-life aspects, access to care, and personal and family impact. Membership and budget of the contributing PCGs varied greatly. The experience gained in the first 2 years demonstrates the feasibility of the process and the fact that PCG contribution actually provides relevant input on the patient perspective for HTAs used for reimbursement decisions. The challenges identified on the side of PCGs were time constraints and human resources.

Development of a decisional flowchart for meaningful patient involvement in Health Technology Assessment.

This paper aims to describe the development of a flowchart to guide the decisions of researchers in the Spanish Network for Health Technology Assessment of the National Health System (RedETS) regarding patient involvement (PI) in Health Technology Assessment (HTA). By doing so, it reflects on current methodological challenges in PI in the HTA field: how best to combine PI methods and what is the role of patient-based evidence. A decisional flowchart for PI in HTA was developed between March and April 2019 following an iterative process, reviewed by the members of the PI Interest Group and other RedETS members and validated during an online deliberative meeting. The development of the flowchart was based on a previous methodological framework assessed in a pilot study. The guidelines on how to involve patients in HTA in the RedETS were graphically represented in a flowchart. PI must be included in all HTA reports, except those that assess technologies with no relevant impact on patients' experiences, values, and preferences. Patient organizations or expert patients related to the topic of the HTA report must be identified and invited. These patients can participate in protocol development, outcomes' identification, assessment process, and report review. When the technology assessed affects in a relevant way patient experiences, values, and preferences, patient-based evidence should be included through a systematic literature review or a primary study. The decisional flowchart for PI in HTA contributes to the current methodological challenges by proposing a combination of direct involvement and patient-based evidence.

OP380 A Review Of The Methodology Used To Synthesize Continuous And Time-To-Event Outcomes For Clinical And Cost-Effectiveness

IntroductionSynthesis of continuous and time-to-event outcomes is often complicated by the use of multiple outcome scales and heterogeneous reporting of outcomes across trials. Simple methods of evidence synthesis for clinical effectiveness can fail to account for these issues and result in a reduction of the evidence base, which can be further reduced at the cost-effectiveness stage as common outcome measures, such as standardized mean differences, cannot easily be incorporated into the economic decision model. Recent methodological advances for synthesizing continuous and time-to-event outcomes aim to include a greater proportion of the available evidence base within a single coherent analysis.MethodsTo assess the statistical methods commonly used in health technology assessment (HTA) and establish whether recent advances in synthesis methods have been adopted in practice, we conducted a review of HTA reports and guidelines published in the United Kingdom (UK) between 1 April 2018 and 31 March 2019 reporting a quantitative meta-analysis (MA), network meta-analysis (NMA) or indirect treatment comparison (ITC) of at least one continuous or time-to-event outcome.ResultsForty-seven articles were considered eligible for this review. Fifty-one percent of eligible articles reported at least one continuous outcome and 55 percent at least one time-to-event outcome. Twenty-nine articles reported NMA or ITC and twenty-seven reported MA of a continuous or time-to-event outcome. Forty articles included a decision model, of which twenty-seven incorporated evidence from a synthesis of a continuous or time-to-event outcome with eleven informed by a single trial (despite synthesis being conducted).ConclusionsUptake of methods to include a greater proportion of the available evidence base within a single coherent analysis in UK HTA reports has been slow. Evaluating health technologies using an evidence-based approach often results in better outcomes for patients. Therefore, HTA analysts and decision modelers must be aware of the expanding literature for synthesis of continuous and time-to-event outcomes and appreciate the limitations of simpler approaches.

PRO98 Country Specific Approaches to Appraising RARE Disease Treatments: A Case Study Analysis of the IMPACT of Different Processes

Rare disease treatments (RDTs) often pose challenges to standard appraisal and reimbursement processes. Countries vary in their approaches to appraising RDTs; some use different ‘supplemental’ processes, others use standard processes. This study examines the impacts of these different appraisal processes for two RDTs in selected countries. Literature Review and Synthesis. A case study analysis was conducted on countries with different types of appraisal processes. Country selection was based on availability of public health technology assessment (HTA) reports and representative countries with various processes for appraising RDTs. Two contrasting RDTs were chosen according to the criteria rare/ultra-rare treatment, affecting child/adult, and life-threatening/disabling. Information for each country's RDT appraisal was extracted into templates to enable comparison of appraisals across countries, and evaluation of the impact of the different processes in practice. Reports from Belgium, England, France, Germany, U.S., Italy, Netherlands, Norway, Scotland, Sweden and the U.S. were selected for Nusinersen and Voretigene. Characteristics of each country's process were identified, including information related to evidence submitted and committees’ interpretation of it. Uncertainties raised did not vary between the two treatments and did not seem to depend on the type of appraisal process. Additional considerations beyond clinical and cost effectiveness were seen for both drugs. For Nusinersen, more countries with supplemental processes took into account the impact on QoL than countries with standard processes, while for Voretigene, more countries with supplemental processes took into account severity, rarity and unmet need than countries with standard processes. While acceptance of uncertainty and additional considerations existed within both standard and supplemental processes, countries with supplemental processes more often referred to formal criteria for these considerations. The formal criteria for RDT appraisal within supplemental approaches may make them more appropriate to facilitate consistent decision-making and management of RDT challenges.

The use of real-world evidence in ICER's scoping process and clinical evidence assessments.

BACKGROUND: There has been growing interest in using real-world evidence (RWE) for health technology assessment (HTA) in the United States. The Institute for Clinical and Economic Review (ICER) is an independent U.S.-based HTA organization that focuses primarily on pharmaceuticals. RWE is used to inform ICER's scoping and comparative clinical effectiveness (CCE) assessments, but the extent to which it is used has not been quantified. OBJECTIVE: To systematically evaluate use of RWE in the scoping and CCE assessment sections of the ICER HTA reports on pharmaceuticals. METHODS: We reviewed all ICER reports of pharmaceuticals published between January 2014 and June 2019. We examined the average number of instances and the proportion of RWE use in the scoping documents to inform the population, intervention, comparator, outcome, setting, or timing (PICOTS) elements of the appraisal. We also examined the average number of instances and the proportion of RWE use in the CCE assessments to inform effectiveness, safety, or treatment patterns. Finally, we evaluated use of RWE in clinical guidelines that were cited in the CCE assessments. RESULTS: In ICER scoping documents, the mean (SD) number of instances of RWE use was 3.8 (3.7) per document (55% for outcomes, 20% for population, 14% for comparator, 11% for intervention, and 0% for timing and setting). In ICER CCE assessments, the mean (SD) number of instances per assessment was 0.7 (0.5) per drug (53% for effectiveness, 44% for safety, and 3% for treatment patterns). In clinical guidelines used in ICER reports, the mean (SD) number of instances of RWE use was 1.6 (2.3) per drug per guideline (41% for effectiveness, 30% for safety, and 29% for treatment patterns). CONCLUSIONS: RWE was frequently used in the ICER scoping process, particularly to inform selection of outcomes. RWE was used infrequently in ICER CCE assessments, while more often used to inform effectiveness, safety, and treatment patterns in relevant clinical guidelines. There are opportunities to increase the use of RWE in U.S. HTA processes. DISCLOSURES: This study was supported by the Health Tech Fund, University of Washington School of Pharmacy, which was created through unrestricted support from several health care industry companies. Veenstra and Carlson report grant support from the Institute for Clinical and Economic Review outside the submitted work. Carlson reports personal fees from Bayer, Allergan, and Galderma outside the submitted work. Jiao, Lee, and Devine report no support outside the submitted work.

Demonstrating the influence of HTA: INAHTA member stories of HTA impact.

A central function of health technology assessment (HTA) agencies is the production of HTA reports to support evidence-informed policy and decision making. HTA agencies are interested in understanding the mechanisms of HTA impact, which can be understood as the influence or impact of HTA report findings on decision making at various levels of the health system. The members of the International Network of Agencies for HTA (INAHTA) meet at their annual Congress where impact story sharing is one important activity. This paper summarizes four stories of HTA impact that were finalists for the David Hailey Award for Best Impact Story.The methods to measure impact include: document review; claims analysis and review of reimbursement status; citation analysis; qualitative evaluation of stakeholders' views; and review of media response. HTA agency staff also observed changes in government activities and priorities based on the HTA. Impact assessment can provide information to improve the HTA process, for example, the value of patient and clinician engagement in the HTA process to better define the assessment question and literature reviews in a more holistic and balanced way.HTA reports produced by publicly funded HTA agencies are valued by health systems around the globe as they support decision making regarding the appropriate use, pricing, reimbursement, and disinvestment of health technologies. HTAs can also have a positive impact on information sharing between different levels of government and across stakeholder groups. These stories show how HTA can have a significant impact, irrespective of the health system and health technology being assessed.

Comparative characteristics of types of medical technologies assessment

This article presents the results obtained during the analysis of different types of health technology assessment (HTA). In the world HTA agencies can be organized at the national (federal), regional or local levels; they can have various levels of dependence on public authorities (independent, public, dependent), various sources of financing (state, mixed). The nature of the tasks and the range of powers of the institutions for HTA differ: regulatory, organizational or advisory. They themselves can initiate HTA (proactive), or they can conduct HTA at the suggestion of the applicants and analyze the quality of the HTA reports provided (reactively). HTA can be focused on economic assessment, comparative clinical assessment, as well as balanced, both on economic and comparative clinical assessments. HTA may be conducted on drugs, medical devices, and other medical technologies. The methods for conducting HTA significantly depend on its subject. In terms of the scope of the assessment, it is possible to single out full OMT, mini-OMT and express-OMT, as well as fast-track-OMT, proposed by the HTA agency in the UK. The final users of HTA (administration, suppliers, patients) and its perceptions (instrumental, conceptual, or formal) are specified. The impact of HTA on decision-making is progressively increasing, as the number of medical technologies that should be assessed to maintain budget discipline is increasing. The versatility of HTA indicates its universality and can contribute to the further expansion of its application at various levels of medical care around the world.

Quality Assessment of HTA reports: a Critical Appraisal Toolkit within an institutional HTA program

Abstract Issue With population aging, increased demand for healthcare and limited availability of economic and human resources, well-functioning and sustainable health systems have to rely on rigorous and evidence-based assessments of complex technologies' clinical effectiveness and safety. The Lombardy Region in Northern Italy has a well-established HTA program that offers technical support to its hospitals' network to produce and to review Health Technology Assessment (HTA) reports. Description of the Problem To better coordinate the HTA network and to reduce self-referencing of University and Community hospitals, Lombardy Region carried out an intense field training and distance learning from year 2017, with a project aimed at involving health care professionals in an accurate evaluation of technologies in 18 HTA reports. The regional HTA Supporting Centre developed a Toolkit for the critical appraisal of reports and supporting literature to improve the quality of hospital-based reports. Results In compliance with the regional Law DGR XI/1046 17.12.2018 and the framework proposed, during year 2019 hospitals used the Toolkit to help writing complete and good quality HTA reports on 37 different technologies. With a Public Health resident internship, the Toolkit was revised, extended and then used in 2020 by the hospital's HTA network during the double blinded peer review, mandated by the regional Law DGR XI/2672 16.12.2019, of the year 2019 HTA reports. Detailed results will be presented at the conference. Lessons We observed an increase in number of HTA reports from 2018 to 2019, a greater language's and format's homogeneity and an improvement in the quality of some reports, which will be submitted to the Regional HTA Committee for appraisal and reimbursement. Key messages The development and use of a Critical Appraisal Toolkit in an Regional HTA program can help hospitals write complete and good quality HTA reports. A centrally supported quality improvement of distributed assessment activities in a network of hospitals can enhance the production of HTA reports, relevant to the needs of a local healthcare system.

Experiences of using Cochrane systematic reviews by local HTA units

Abstract The aim of this study is to evaluate the utilisation of CSRs by Quebec's local health technology assessment (HTA) units to promote efficiency in hospital decision-making. A survey was conducted using a structured online questionnaire to examine: Characteristics of the HTA units; Knowledge about works and services from the Cochrane Collaboration; Level of satisfaction about the use of CSRs; Facilitating factors and barriers to the implementation of CSRs evidence in a local context; Suggestions to improve the use of CSRs. Data accuracy was checked by two independent evaluators (TGP and CAB). Ten HTA units participated. Mean number of staff was 6.3 [2-18], producing about 4.7 reports per year [2-7.3]. From their implementation a total of 321 HTA reports were published (49.8% included a SR). Works and services provided by the Cochrane collaboration were very well known and HTA units were highly satisfied with CSRs (80-100%). However, only 24.7% (21/85) of topics were searched with success. As regards to applicability in HTA and use of CSRs, major strengths were as follow: Useful as resource for search terms and background material; May reduce the workload (e.g. brief review instead of full SR); Use to update a current review. Major weaknesses were: Limited use since no CSRs were available for many HTA projects; Difficulty to apply findings to local context (lack of contextualisation); Focused only on efficacy and innocuity; Cannot be used as a substitute to a full HTA report. This study provided a unique context of assessment with a familiar group of producers, users and disseminators of CSRs in hospital setting. HTA units generally use other articles from the literature or produce an original systematic review in complement with CSRs. These results led to some suggestions to improve the use of CSRs by HTA units. However, the main limit for the use of CRS in local HTA will remain its lack of contextualisation. Key messages The main limit for the use of CRS in local HTA will remain its lack of contextualization. Context and scientific data are complementary.

PCN87 Key Clinical and Economic Rationales for Oncology Reviews in South Korea

To study the clinical and economic rationales for oncology reviews assessed by the Health Insurance Review Agency (HIRA) in South Korea. Health technology assessment (HTA) reports for oncological indications published by HIRA in the last 5 years (2015 - 2019) were analyzed to identify the clinical and economic factors affecting decisions. A total of 48 decisions were identified. In the evaluation of benefit assessment, 81.25% demonstrated a valid adequacy of benefit, whereas 18.75% demonstrated non-benefit. Clinical factors driving positive decisions were better outcome results (67.3%), unmet need/life-threatening disease condition (11.54%), acceptable efficacy/effectiveness (3.85%), and similar efficacy (3.85%), whereas those leading to negative decisions were identified as uncertain efficacy/effectiveness (11.54%) and insufficient efficacy (1.92%). Similarly, economic factors leading to positive decisions were applicability of risk-sharing agreement (37.5%), cost-effectiveness (4.17%), and similar cost (2.08%). Negative decisions were mainly due to uncertain cost-effectiveness (27.08%), drug was not cost-effective (22.92%), and economic evaluation was not submitted (2.08%). Some drugs were exempted from economic evaluation (4.17%). Patients were asked to pay the total cost out of pocket in 6 cases due to high cost or uncertain cost-effectiveness. In 8 out of 10 cases where drugs were registered in > 3 foreign countries, the medicinal products under review were classified as drugs which can omit the submission of economic evaluation documents. The research indicates that oncology product manufacturers should provide robust clinical data to show better clinical outcome results and robust economic data to demonstrate drug cost-effectiveness to gain market access in South Korea. Risk-sharing agreements and economic evaluation exemptions improve patient accessibility for drugs without alternatives in life-threatening disease conditions. Uncertainties in clinical efficacy/effectiveness and cost-effectiveness are the key factors correlated with non-benefit. South Korea is planning to introduce a post-list review focusing on anticancer and orphan drugs.

Health Technology Assessment Report on Vagus Nerve Stimulation in Drug-Resistant Epilepsy.

Background: Vagus nerve stimulation (VNS) is a palliative treatment for medical intractable epileptic syndromes not eligible for resective surgery. Health technology assessment (HTA) represents a modern approach to the analysis of technologies used for healthcare. The purpose of this study is to assess the clinical, organizational, financial, and economic impact of VNS therapy in drug-resistant epilepsies and to establish the congruity between costs incurred and health service reimbursement. Methods: The present study used an HTA approach. It is based on an extensive detailed bibliographic search on databases (Medline, Pubmed, Embase and Cochrane, sites of scientific societies and institutional sites). The HTA study includes the following issues: (a) social impact and costs of the disease; (b) VNS eligibility and clinical results; (c) quality of life (QoL) after VNS therapy; (d) economic impact and productivity regained after VNS; and (e) costs of VNS. Results: Literature data indicate VNS as an effective treatment with a potential positive impact on social aspects and on quality of life. The diagnosis-related group (DRG) financing, both on national and regional levels, does not cover the cost of the medical device. There was an evident insufficient coverage of the DRG compared to the full cost of implanting the device. Conclusions: VNS is a palliative treatment for reducing seizure frequency and intensity. Despite its economic cost, VNS should improve patients’ quality of life and reduce care needs.

Experiences of Using Cochrane Systematic Reviews by Local HTA Units.

This study evaluated the use of Cochrane systematic reviews (CSRs) by Quebec’s local health technology assessment (HTA) units to promote efficiency in hospital decision-making. An online survey was conducted to examine: Characteristics of the HTA units; Knowledge about works and services from the Cochrane Collaboration; Level of satisfaction about the use of CSRs; Facilitating factors and barriers to the implementation of CSRs evidence in a local context; Suggestions to improve the use of CSRs. Data accuracy was checked by 2 independent evaluators. Ten HTA units participated. From their implementation a total of 321 HTA reports were published (49.8% included a SR). Works and services provided by the Cochrane collaboration were very well-known and HTA units were highly satisfied with CSRs (80%-100%). As regards to applicability in HTA and use of CSRs, major strengths were as follow: Useful as resource for search terms and background material; May reduce the workload (eg, brief review instead of full SR); Use to update a current review. Major weaknesses were: Limited use since no CSRs were available for many HTA projects; Difficulty to apply findings to local context; Focused only on efficacy and innocuity; Cannot be used as a substitute to a full HTA report. This study provided a unique context of assessment with a familiar group of producers, users and disseminators of CSRs in hospital setting. Since they generally used other articles from the literature or produce an original SR in complement with CSRs, this led to suggestions to improve their use of CSRs. However, the main limit for the use of CRS in local HTA will remain its lack of contextualisation. As such, this study reinforces the need to consider the notion of complementarity of experimental data informing us about causality and contextual data, allowing decision-making adapted to local issues.