PharmacoEconomics Open最新文献

{"title":"The Efficiency of Increased HCV Testing and Treatment Strategies in Spain to Achieve Elimination Goals.","authors":"Jose Luis Calleja, Jaime Espin, Ankita Kaushik, Manuel Hernandez-Guerra, Rob Blissett, Alon Yehoshua, Adam Igloi-Nagy","doi":"10.1007/s41669-023-00458-3","DOIUrl":"10.1007/s41669-023-00458-3","url":null,"abstract":"<p><strong>Background: </strong>In 2015, Spain launched a national eradication strategy for hepatitis C virus (HCV), resulting in the highest treatment rate in Europe and substantial reductions in HCV prevalence. However, to achieve the goal of HCV elimination, it is necessary to scale-up the diagnosis, treatment, and management of HCV infection.</p><p><strong>Objective: </strong>Our aim was to assess the prevalence, incidence, and cost effectiveness of scaling-up compared with status quo scenarios.</p><p><strong>Methods: </strong>A compartmental dynamic transmission model was developed comprising of a cascade of care and a liver progression module. Cost and quality-of-life inputs were sourced from the literature. Key outcomes were the prevalence and incidence of HCV and the incremental cost per quality-adjusted life-year (QALY) and per life-year (LY). Outcomes for a hypothetical elimination strategy were compared with the status quo.</p><p><strong>Results: </strong>The base-case analysis found that scaling-up testing and treatment reduced both the prevalence and incidence of HCV over time, resulting in incremental costs per QALY and LY of €13,291 and €12,285 respectively, compared with the status quo. The main drivers of the cost-effectiveness results included cost of diagnosis, cost of treatment, proportion of people who are unaware, percentage of population who inject drugs, and calibration parameters related to HCV infection prevalence.</p><p><strong>Conclusions: </strong>This analysis demonstrated that scaling-up testing and treatment with direct-acting antivirals may be an efficient strategy for reducing the incidence and prevalence of HCV and may help achieve HCV elimination goals in Spain.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"221-233"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884368/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138794327","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Understanding the Natural History of Chronic Hepatitis D: Proposal of a Model for Cost-Effectiveness Studies.","authors":"Ankita Kaushik, Geoffrey Dusheiko, Chong Kim, Nathaniel J Smith, Csilla Kinyik-Merena, Gian Luca Di Tanna, Robert J Wong","doi":"10.1007/s41669-023-00466-3","DOIUrl":"10.1007/s41669-023-00466-3","url":null,"abstract":"<p><strong>Background: </strong>As new therapeutic options become available, better understanding the potential impact of emerging therapies on clinical outcomes of hepatits D virus (HDV) is critical.</p><p><strong>Objective: </strong>The aim of this study was to develop a natural history model for patients with hepatitis D virus.</p><p><strong>Methods: </strong>We developed a model (decision tree followed by a Markov cohort model) in adults with chronic HDV infection to assess the natural history and impact of novel treatments on disease progression versus best supportive care (BSC). The model time horizon was over a lifetime (up to 100 years of age); state transitions and health states were defined by responder status. Patients in fibrosis stages 0 through 4 received treatment; decompensated patients were not treated. Response was defined as the combined response endpoint of achievement of HDV-RNA undetectability/≥2-log₁₀ decline and alanine aminotransferase normalization; response rates of 50% and 75% were explored. Health events associated with advanced liver disease were modeled as the number of events per 10,000 patients. Scenario analyses of early treatment, alternate treatment response, and no fibrosis regression for treatment responders were also explored.</p><p><strong>Results: </strong>The model was able to reflect disease progression similarly to published natural history studies for patients with HBV/HDV infection. In a hypothetical cohort of patients reflecting a population enrolled in a recent clinical trial, fewer advanced liver disease events were observed with a novel HDV treatment versus BSC. Fewer liver-related deaths were observed under 50% and 75% response (900 and 1,358 fewer deaths, respectively, per 10,000 patients). Scenario analyses showed consistently fewer advanced liver disease events with HDV treatment compared with BSC, with greater reductions observed with earlier treatment.</p><p><strong>Conclusion: </strong>This HDV disease progression model replicated findings from natural history studies. Furthermore, it found that a hypothetical HDV treatment results in better clinical outcomes for patients versus BSC, with greater benefit observed when starting treatment early. This validated natural history model for HBV/HDV infection can serve as a foundation for future clinical and economic analyses of novel HDV treatments that can support healthcare stakeholders in the management of patients with chronic HDV.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"333-343"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884366/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139087982","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Artificial Intelligence to Automate Network Meta-Analyses: Four Case Studies to Evaluate the Potential Application of Large Language Models.","authors":"Tim Reason, Emma Benbow, Julia Langham, Andy Gimblett, Sven L Klijn, Bill Malcolm","doi":"10.1007/s41669-024-00476-9","DOIUrl":"10.1007/s41669-024-00476-9","url":null,"abstract":"<p><strong>Background: </strong>The emergence of artificial intelligence, capable of human-level performance on some tasks, presents an opportunity to revolutionise development of systematic reviews and network meta-analyses (NMAs). In this pilot study, we aim to assess use of a large-language model (LLM, Generative Pre-trained Transformer 4 [GPT-4]) to automatically extract data from publications, write an R script to conduct an NMA and interpret the results.</p><p><strong>Methods: </strong>We considered four case studies involving binary and time-to-event outcomes in two disease areas, for which an NMA had previously been conducted manually. For each case study, a Python script was developed that communicated with the LLM via application programming interface (API) calls. The LLM was prompted to extract relevant data from publications, to create an R script to be used to run the NMA and then to produce a small report describing the analysis.</p><p><strong>Results: </strong>The LLM had a > 99% success rate of accurately extracting data across 20 runs for each case study and could generate R scripts that could be run end-to-end without human input. It also produced good quality reports describing the disease area, analysis conducted, results obtained and a correct interpretation of the results.</p><p><strong>Conclusions: </strong>This study provides a promising indication of the feasibility of using current generation LLMs to automate data extraction, code generation and NMA result interpretation, which could result in significant time savings and reduce human error. This is provided that routine technical checks are performed, as recommend for human-conducted analyses. Whilst not currently 100% consistent, LLMs are likely to improve with time.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"205-220"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884375/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139716304","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An Analysis of Uncertainties and Data Collection Agreements in the Cancer Drugs Fund.","authors":"Laura A Trigg, Maxwell S Barnish, Samuel Hayward, Naomi Shaw, Louise Crathorne, Brad Groves, John Spoors, Thomas Strong, G J Melendez-Torres, Caroline Farmer","doi":"10.1007/s41669-023-00460-9","DOIUrl":"10.1007/s41669-023-00460-9","url":null,"abstract":"<p><strong>Background: </strong>Managed Access Agreements (MAAs) are a commercial arrangement that provide patients earlier access to innovative health technologies while uncertainties in the evidence base are resolved through data collection. In the UK, data collection agreements (DCAs) outline the evidence that will be collected during the MAA period and are intended to resolve uncertainties in the clinical- and cost-effectiveness of a technology sufficient for the National Institute of Health and Care Excellence (NICE) committee to make a final decision on reimbursement.</p><p><strong>Objective: </strong>The aim of this study was to identify the primary uncertainties leading to a recommendation for entry to the Cancer Drugs Fund (CDF) and evaluate how the corresponding DCAs attempt to address these.</p><p><strong>Methods: </strong>A database of MAAs agreed within the CDF was compiled with coverage between July 2016 and December 2020 (the time during which evidence generation was routinely collected within the CDF up until the time of analysis). Uncertainties in the evidence base for technologies entering the CDF were analysed alongside the outcomes planned for data collection during the MAA. These data provide an overview of the key uncertainties surrounding health technologies in the CDF on entry and the types of evidence targeted by DCAs.</p><p><strong>Results: </strong>In the assessment of 39 Cancer Drugs Fund (CDF) cases, NICE committees identified a total of 108 key uncertainties in cost-effectiveness estimates. Overall survival was the most commonly identified uncertainty, followed by generalisability of the evidence to the target population. DCAs specified a range of outcomes relevant to understanding the clinical effectiveness of the technology, though fewer than half (43.6%) of the DCAs addressed all the key uncertainties identified by the NICE committee.</p><p><strong>Conclusion: </strong>The analysis indicated that data collection within the CDF is not sufficient to resolve all the uncertainties identified by the NICE committee, meaning that other approaches will be needed at re-appraisal to ensure that the NICE committee can reach a final decision on reimbursement.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"303-311"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10883900/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138808410","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From Indication-Based Pricing to Blended Approach: Evidence on the Price and Reimbursement Negotiation in Italy.","authors":"Elvio Emanuele Rossini, Carlotta Galeone, Chiara Lucchetti, Claudio Jommi","doi":"10.1007/s41669-023-00467-2","DOIUrl":"10.1007/s41669-023-00467-2","url":null,"abstract":"<p><strong>Background: </strong>New indications for existing medicines are increasing over time. In most countries, drug pricing and reimbursement conditions are renegotiated every time a new indication is approved. There is a growing interest in the price negotiation model for new indications, specifically comparing an indication-based versus blended approach. However, little evidence currently exists regarding the complexity of these negotiations and their impact on actual prices. Italy has recently transitioned from an indication-based approach to a blended price model. This study aims to measure the impact of price and reimbursement negotiation of new indications on discounts (i.e. actual prices) and on the negotiation duration, used as a proxy of its complexity.</p><p><strong>Methods: </strong>We considered new indications approved through a European centralized procedure from January 2013 to March 2022 for which the price and reimbursement status was approved in Italy between January 2015 and March 2022, amounting to 52 new indications. Data on the timeframe of the Italian price and reimbursement process and its phases were obtained from publicly available sources. Discounts for the first indication and their subsequent increases for new indications were estimated by comparing ex-factory prices and tendered prices. To calculate p-values, we employed the Mann-Whitney test, and multiple regression models were utilized to examine correlations between negotiation time and the characteristics of the medicines.</p><p><strong>Results: </strong>The mean time to reimbursement was 603 days, in contrast to 583 days for the first launch. Price negotiation took longer for rare diseases, cancer drugs, and in case of therapies with minor added therapeutic value. On average, the additional discount (on top of discounts for prior indications) was 13%, significantly lower than the mean discount for the first indications approved (24.9%). The discounts increment was lower, but negotiation took longer if a Managed Entry Agreement accompanied the final agreement. Additionally, discounts have increased over the years.</p><p><strong>Conclusion: </strong>The negotiation for new indications takes longer than the first one, and provides, on average, an additional discount of 13%. While our findings bear the potential for significant policy implications, they necessitate prudent interpretation due to a limited number of observations. The increasing trend in additional discounts over time applied to all indications in recent negotiations, may suggest a descending trend of value for new indications and a shift from an indication-based pricing approach to a blended model. Otherwise, budget impact considerations might have outweighed a value-based approach in the recent negotiations. If so, two potential options for restoring a value-based approach are returning to an indication-based pricing or giving explicit and higher weight to value within a blended model.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"251-261"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10883902/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139478060","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Artificial Intelligence to Automate Health Economic Modelling: A Case Study to Evaluate the Potential Application of Large Language Models.","authors":"Tim Reason, William Rawlinson, Julia Langham, Andy Gimblett, Bill Malcolm, Sven Klijn","doi":"10.1007/s41669-024-00477-8","DOIUrl":"10.1007/s41669-024-00477-8","url":null,"abstract":"<p><strong>Background: </strong>Current generation large language models (LLMs) such as Generative Pre-Trained Transformer 4 (GPT-4) have achieved human-level performance on many tasks including the generation of computer code based on textual input. This study aimed to assess whether GPT-4 could be used to automatically programme two published health economic analyses.</p><p><strong>Methods: </strong>The two analyses were partitioned survival models evaluating interventions in non-small cell lung cancer (NSCLC) and renal cell carcinoma (RCC). We developed prompts which instructed GPT-4 to programme the NSCLC and RCC models in R, and which provided descriptions of each model's methods, assumptions and parameter values. The results of the generated scripts were compared to the published values from the original, human-programmed models. The models were replicated 15 times to capture variability in GPT-4's output.</p><p><strong>Results: </strong>GPT-4 fully replicated the NSCLC model with high accuracy: 100% (15/15) of the artificial intelligence (AI)-generated NSCLC models were error-free or contained a single minor error, and 93% (14/15) were completely error-free. GPT-4 closely replicated the RCC model, although human intervention was required to simplify an element of the model design (one of the model's fifteen input calculations) because it used too many sequential steps to be implemented in a single prompt. With this simplification, 87% (13/15) of the AI-generated RCC models were error-free or contained a single minor error, and 60% (9/15) were completely error-free. Error-free model scripts replicated the published incremental cost-effectiveness ratios to within 1%.</p><p><strong>Conclusion: </strong>This study provides a promising indication that GPT-4 can have practical applications in the automation of health economic model construction. Potential benefits include accelerated model development timelines and reduced costs of development. Further research is necessary to explore the generalisability of LLM-based automation across a larger sample of models.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"191-203"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884386/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139716303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health Economic Evaluations of Hemochromatosis Screening and Treatment: A Systematic Review.","authors":"Malvina Hoxha, Visar Malaj, Bruno Zappacosta","doi":"10.1007/s41669-023-00463-6","DOIUrl":"10.1007/s41669-023-00463-6","url":null,"abstract":"<p><strong>Background: </strong>Hereditary hemochromatosis (HH) is an autosomal recessive disorder that leads to iron overload and multiorgan failure.</p><p><strong>Objectives: </strong>The aim of this systematic review was to provide up-to-date evidence of all the current data on the costs and cost effectiveness of screening and treatment for HH.</p><p><strong>Methods: </strong>We searched PubMed, Cochrane Library, National Health Service Economic Evaluation Database (NHSEED), Cost-Effectiveness Analysis Registry (CEA Registry), Health Technology Assessment Database (HTAD), Centre for Reviews and Dissemination (CRD), and Econlit until April 2023 with no date restrictions. Articles that reported cost-utility, cost-description, cost-minimization, cost-effectiveness, or cost-benefit analyses for any kind of management (drugs, screening, etc.) were included in the study. Patients with HH, their siblings, or individuals suspected of having HH were included in the study. All screening and treatment strategies were included. Two authors assessed the quality of evidence related to screening (either phenotype or genotype screening) and treatment (phlebotomy and electrophoresis). Narrative synthesis was used to analyse the similarities and differences between the respective studies.</p><p><strong>Results: </strong>Thirty-nine papers were included in this study. The majority of the studies reported both the cost of phenotype screening, including transferrin saturation (TS), serum ferritin, and liver biopsy, and the cost of genotype screening (HFE screening, C282Y mutation). Few studies reported the cost for phlebotomy and erythrocytapheresis treatment. Data revealed that either phenotype or genotype screening were cost effective compared with no screening. Treatment studies concluded that erythrocytapheresis might be a cost-effective therapy compared with phlebotomy.</p><p><strong>Conclusions: </strong>Economic studies on either the screening, or treatment strategy for HH patients should be performed in more countries. We suggest that cost-effectiveness studies on the role of deferasirox in HH should be carried out as an alternative therapy to phlebotomy.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"147-170"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884378/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139570246","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Systematic Literature Review of Health-Related Quality of Life Outcomes and Associated Utility Values in Relapsed and/or Refractory Large B Cell Lymphoma.","authors":"Fei Fei Liu, Meaghan Bartlett, Samantha Craigie","doi":"10.1007/s41669-023-00464-5","DOIUrl":"10.1007/s41669-023-00464-5","url":null,"abstract":"<p><strong>Background: </strong>In this ever-expanding treatment landscape, there is a lack of consolidated health-related quality of life (HRQOL) outcomes and utility reports in relapsed or refractory (R/R) large B cell lymphoma (LBCL) to inform health care policy and decision-maker assessments for both old and new products. These assessments can have a direct effect on what treatment options are available to patients and physicians.</p><p><strong>Objective: </strong>A systematic literature review (SLR) was performed to understand the HRQOL evidence for treatments in R/R LBCL and identify associated health utility values.</p><p><strong>Methods: </strong>The SLR searched and screened literature published from 1 January 2003 to 2 May 2022. Studies were screened based on Population, Intervention, Comparator, Outcome, Study design criteria established a priori and were assessed by two independent reviewers; quality assessments of the evidence were performed in accordance with health technology assessment recommendations from the National Institute for Health and Care Excellence. Several types of therapies were included, such as chimeric antigen receptor (CAR) T cell products (lisocabtagene maraleucel, axicabtagene ciloleucel, tisagenlecleucel), novel therapies (selinexor, nivolumab, polatuzumab vedotin, and bendamustine), salvage therapies, and rituximab.</p><p><strong>Results: </strong>The review identified 33 unique studies reporting HRQOL, including 15 economic studies that reported health state utility values, 9 clinical trials, 7 health technology assessment reports, and 1 each of a vignette-based study and a point-in-time survey. Improvements in general and/or lymphoma-specific HRQOL measures were observed with CAR T cell therapy in both the second-line and third-line or later settings. On-treatment utility values for CAR T cell therapies ranged from 0.50 to 0.74. Values for remission/progression-free survival (0.70-0.90) and for disease progression (0.39-0.59) were similar across studies. For novel therapies, utility values were 0.83 for progression-free survival and ranged from 0.39 to 0.71 for disease progression. On-treatment utility values for salvage chemotherapy ranged from 0.63 to 0.67.</p><p><strong>Conclusions: </strong>Overall, the evidence synthesized in this SLR provides a comprehensive understanding of the HRQOL evidence in R/R LBCL. This article identified several sources for utility values in the published literature showing variation in the HRQOL outcomes for patients across a variety of therapeutics. Treatment of R/R LBCL with CAR T cell therapies was associated with improvement in health utility values. Mixed results were found for novel therapies and salvage therapies. More data are needed as new therapies are used in this patient population to inform treatment decision-making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"171-190"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10883903/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139403929","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Acceptance of Indirect Treatment Comparison Methods in Oncology by Health Technology Assessment Agencies in England, France, Germany, Italy, and Spain.","authors":"Bérengère Macabeo, Théophile Rotrou, Aurélie Millier, Clément François, Philippe Laramée","doi":"10.1007/s41669-023-00455-6","DOIUrl":"10.1007/s41669-023-00455-6","url":null,"abstract":"<p><strong>Introduction: </strong>Randomized controlled trials (RCTs) are the gold standard when comparing treatment effectiveness, and Health Technology Assessment (HTA) agencies state a clear preference for such direct comparisons. When these are not available, an indirect treatment comparison (ITC) is an alternative option. The objective of this study was to assess the acceptance of ITC methods by HTA agencies across England, France, Germany, Italy, and Spain, using oncology cases for a homogeneous sample of HTA evaluations.</p><p><strong>Methods: </strong>The study was conducted on the PrismAccess database in May 2021 to retrieve HTA evaluation reports for oncology treatments for solid tumors, in which an ITC was presented. The analysis was restricted to HTA evaluation reports published between April 2018 and April 2021 in England, France, Germany, Italy, and Spain. Identified HTA evaluation reports were screened and reviewed by two independent reviewers. For each ITC presented, the methodology and its acceptance by the HTA agency were analyzed.</p><p><strong>Results: </strong>Five hundred and forty-three HTA evaluation reports were identified, of which 120 (22%) presented an ITC. This proportion was the highest in England (51%) and lowest in France (6%). The overall acceptance rate of ITC methods was 30%, with the highest in England (47%) and lowest in France (0%). Network meta-analysis (NMA; 23%) was the most commonly used ITC technique, with a 39% acceptance rate overall, followed by Bucher ITC (19%; 43% acceptance rate) and matching-adjusted indirect comparison (13%; 33% acceptance rate). The most common criticisms of the ITC methods from HTA agencies related to data limitations (heterogeneity and lack of data; 48% and 43%, respectively) and the statistical methods used (41%).</p><p><strong>Conclusions: </strong>The generally low acceptance rate of ITC methods by HTA agencies in oncology suggests that, whilst in the absence of a direct comparison ITCs may provide relevant evidence, this evidence is not widely considered sufficient for the purpose of HTA evaluations. The perception of ITC methods for the purpose of HTA evaluations varies substantially between countries. There is a need for further clarity on the properties of ITC techniques and the assessment of their results as ITC methods continue to evolve quickly and further techniques may become available in the future.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"5-18"},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781913/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138794311","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Public Health Impact and Cost-Effectiveness of Empagliflozin (JARDIANCE^®) in the Treatment of Patients with Heart Failure with Preserved Ejection Fraction in France, Based on the EMPEROR-Preserved Clinical Trial.","authors":"Laurent Fauchier, Nicolas Lamblin, Jean Tardu, Lucile Bellier, Harinala Groyer, Deborah Ittah, Julien Chollet, Stephan Linden, Pierre Levy","doi":"10.1007/s41669-023-00432-z","DOIUrl":"10.1007/s41669-023-00432-z","url":null,"abstract":"<p><strong>Introduction: </strong>The efficacy and safety of empagliflozin in the treatment of heart failure with preserved ejection fraction (HFpEF) were demonstrated in the EMPEROR-Preserved trial, which showed a 21% reduction in combined risks of cardiovascular death or HF hospitalization [hazard ratio (HR) 0.79; 95% confidence interval (CI) 0.69-0.90, p < 0.001] and a 27% reduction in the total number of HF hospitalizations (HR 0.73; 95% CI 0.61-0.88, p < 0.001) compared with placebo. On the basis of these results, the present study aimed to assess the cost-effectiveness of empagliflozin + standard of care (SoC) compared with SoC alone in the treatment of HFpEF.</p><p><strong>Methods: </strong>A published Markov model was adapted to compare the health and economic outcomes in France, considering a collective perspective, in patients treated with empagliflozin in addition to SoC versus patients treated by SoC alone. The model simulated the intention-to-treat (ITT) population of the trial, transitioning between four mutually exclusive health states representing the quartiles of the Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score (KCCQ-CSS). For each arm, the model estimated (over a lifetime time horizon) the economics and the health outcomes (HF hospitalizations avoided, and life years and quality-adjusted life years (QALYs) gained) to calculate the incremental cost-effectiveness ratios (ICERs). The resources used were derived by pairing the FREnch Survey on HF (FRESH) cohort data to French health insurance claims data, and the utilities were derived on the basis of the EQ-5D-5L questionnaire valued on the French tariff. Both economic and health outcomes were discounted at a 2.5% annual rate.</p><p><strong>Results: </strong>The model predicted that treatment of HFpEF patients with empagliflozin would prevent, for 1000 patients treated, 74 HF hospitalizations and 15 deaths attributable to cardiovascular events, resulting on average in a gain of 1 month in overall survival (7.24 versus 7.16 years with placebo) and 0.11 QALYs (6.14 versus 6.03 with placebo). Empagliflozin costs were partially offset by the cost savings from avoided hospitalizations. The ICERs were €18,597 per life year gained and €13,980 per QALY gained. The sensitivity analyses conducted showed that empagliflozin has a 65% probability to be cost-effective under the €25,000/QALY threshold.</p><p><strong>Conclusions: </strong>The base-case results showed that empagliflozin is a cost-effective strategy for management of HFpEF, in addition to the impact on public health by preventing HF-hospitalizations and deaths in France. Sensitivity analyses suggest that 65% of simulations are under the €25,000/QALY threshold.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"19-30"},"PeriodicalIF":2.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10781654/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10042623","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}