PharmacoEconomics Open最新文献

{"title":"Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany.","authors":"Niklaus Meier, Hendrik Fuchs, Katya Galactionova, Cedric Hermans, Mark Pletscher, Matthias Schwenkglenks","doi":"10.1007/s41669-024-00480-z","DOIUrl":"10.1007/s41669-024-00480-z","url":null,"abstract":"<p><strong>Background and objective: </strong>Haemophilia B is a rare genetic disease that is caused by a deficiency of coagulation factor IX (FIX) in the blood and leads to internal and external bleeding. Under the current standard of care, haemophilia is treated either prophylactically or on-demand via intravenous infusions of FIX. These treatment strategies impose a high burden on patients and health care systems as haemophilia B requires lifelong treatment, and FIX is costly. Etranacogene dezaparvovec (ED) is a gene therapy for haemophilia B that has been recently approved by the United States Food and Drug Administration and has received a recommendation for conditional marketing authorization by the European Medicines Agency. We aimed to examine the cost-effectiveness of ED versus extended half-life FIX (EHL-FIX) prophylaxis for moderate-to-severe haemophilia B from a German health care payer perspective.</p><p><strong>Methods: </strong>A microsimulation model was implemented in R. The model used data from the ED phase 3 clinical trial publication and further secondary data sources to simulate and compare patients receiving ED or EHL-FIX prophylaxis over a lifetime horizon, with the potential for ED patients to switch treatment to EHL-FIX prophylaxis when the effectiveness of ED waned. Primary outcomes of this analysis included discounted total costs, discounted quality-adjusted life years (QALYs), incremental cost-effectiveness, and the incremental net monetary benefit. The annual discount rate for costs and effects was 3%. Uncertainty was examined via probabilistic analysis and additional univariate sensitivity analyses.</p><p><strong>Results: </strong>Probabilistic analysis indicated that patients treated with ED instead of EHL-FIX prophylaxis gained 0.50 QALYs and experienced cost savings of EUR 1,179,829 at a price of EUR 1,500,000 per ED treatment. ED was the dominant treatment strategy. At a willingness to pay of EUR 50,000/QALY, the incremental net monetary benefit amounted to EUR 1,204,840.</p><p><strong>Discussion: </strong>Depending on the price, ED can save costs and improve health outcomes of haemophilia patients compared with EHL-FIX prophylaxis, making it a potentially cost-effective alternative. These results are uncertain due to a lack of evidence regarding the long-term effectiveness of ED.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"373-387"},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058170/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140194336","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":"Cost-Effectiveness Analysis of Digital Breast Tomosynthesis Added to Synthetic Mammography in Breast Cancer Screening in Brazil.","authors":"Henrique Lima Couto, Ludmila Peres Gargano, Vilmar Marques de Oliveira, Bertha Andrade Coelho, Eduardo Carvalho Pessoa, Augusto Tufi Hassan, Agnaldo Lopes Silva, Linei Augusta Brolini Delle Urban, Luciano Chala Fernandes, Nisha Sharma, Ritse Mann, Stuart A McIntosh, Fernando Zanghelini","doi":"10.1007/s41669-023-00470-7","DOIUrl":"10.1007/s41669-023-00470-7","url":null,"abstract":"<p><strong>Background: </strong>Literature meta-analysis results show that digital breast tomosynthesis (DBT) combined with synthesized two-dimensional (s2D) mammograms can reduce recalls and improve breast cancer detection. Uncertainty regarding the screening of patients with breast cancer presents a health economic challenge, both in terms of healthcare resource use and quality of life impact on patients.</p><p><strong>Objective: </strong>This study aims to estimate the cost effectiveness of DBT + s2D versus digital mammography (DM) used in a biennial breast cancer screening setting of women aged 40-69 years with scattered areas of fibroglandular breast density and heterogeneous dense breasts in the Brazilian supplementary health system.</p><p><strong>Methods: </strong>A cost-effectiveness analysis was performed on the basis of clinical data obtained from a systematic review with meta-analysis performed to evaluate the analytical validity and clinical utility of DBT + s2D compared with DM. The search was conducted in the PubMed, Cochrane Library and Embase databases, with the main descriptors of the technology, a comparator, and the clinical condition in question, on 9 June 2022. The hybrid economic model (decision tree plus Markov model) simulated costs and outcomes over a lifetime for women aged 40-69 years with scattered areas of fibroglandular breast density and heterogeneous dense breasts. We analyzed incremental cost-effectiveness ratio (ICER) to measure the incremental cost difference per quality-adjusted life year (QALY) of adding DBT + s2D to breast cancer screening.</p><p><strong>Results: </strong>DBT + s2D incurred a cost saving of € 954.02 per patient, in the time horizon of 30 years, compared with DM, and gained 5.1989 QALYs, which would be considered a dominant intervention. These results were confirmed in sensitivity analyses.</p><p><strong>Conclusion: </strong>Switching from DM to biennial DBT + s2D was cost effective. Furthermore, reductions in false-positive recall rates should also be considered in decision making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"403-416"},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058155/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139486292","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-Related Quality of Life and Economic Burden Among Hospitalized Children with Hand, Foot, and Mouth Disease: A Multiregional Study in China.","authors":"Ting Zhou, Hongfei Hu, Junyang Gao, Hongjie Yu, Mark Jit, Pei Wang","doi":"10.1007/s41669-023-00468-1","DOIUrl":"10.1007/s41669-023-00468-1","url":null,"abstract":"<p><strong>Background: </strong>Hand, foot, and mouth disease (HFMD) is an infectious disease with high morbidity and mortality rates among children under 5 years old. This study aimed to explore the health-related quality of life (HRQOL), economic burden, and related influencing factors among Chinese HFMD patients.</p><p><strong>Methods: </strong>From January to October 2019, a longitudinal cohort study of 296 hospitalized patients (≤ 5 years old) with HFMD and their guardians was conducted using the proxy version of the 5-level EQ-5D-Y (EQ-5D-Y-5L, Y-5L) in face-to-face interviews in Shanghai, Zhengzhou, and Kunming, representing three regions with different economic development levels. Multiple linear regression was used to explore the factors associated with HRQOL and costs.</p><p><strong>Results: </strong>The mean Y-5L health utility score (HUS) (standard deviation, SD), and visual analogue scale (VAS) score (SD) were 0.730 (0.140) and 60.33 (16.52) at admission and increased to 0.920 (0.120) and 89.95 (11.88) at discharge, respectively. The children from Shanghai had the lowest HUSs at admission and had the best health improvement. The mean hospitalization cost and total cost were 4037 CNY and 5157 CNY, respectively. The children from Shanghai had the highest hospitalization cost (4559 CNY) and total cost (5491 CNY). Multiple regression analysis suggested that medical insurance status, type of employment, residence type, and religious status were significantly associated with the baseline HUS and improvement in the HUS after treatment. Region, loss of work time, and length of stay had a significant impact on the hospitalization cost and total cost.</p><p><strong>Conclusion: </strong>Our findings demonstrate that HFMD could lead to poor HRQOL and the economic burden varies in different regions in China. Many pediatric patients still have physical or mental health problems shortly after treatment.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"459-469"},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058149/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139403930","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":"Economic Evaluation of a Personalized Nutrition Plan Based on Omic Sciences Versus a General Nutrition Plan in Adults with Overweight and Obesity: A Modeling Study Based on Trial Data in Denmark.","authors":"Milanne Maria Johanna Galekop, Carin Uyl-de Groot, William Ken Redekop","doi":"10.1007/s41669-023-00461-8","DOIUrl":"10.1007/s41669-023-00461-8","url":null,"abstract":"<p><strong>Background: </strong>Since there is no diet that is perfect for everyone, personalized nutrition approaches are gaining popularity to achieve goals such as the prevention of obesity-related diseases. However, appropriate choices about funding and encouraging personalized nutrition approaches should be based on sufficient evidence of their effectiveness and cost-effectiveness. In this study, we assessed whether a newly developed personalized plan (PP) could be cost-effective relative to a non-personalized plan in Denmark.</p><p><strong>Methods: </strong>Results of a 10-week randomized controlled trial were combined with a validated obesity economic model to estimate lifetime cost-effectiveness. In the trial, the intervention group (PP) received personalized home-delivered meals based on metabolic biomarkers and personalized behavioral change messages. In the control group these meals and messages were not personalized. Effects were measured in body mass index (BMI) and quality of life (EQ-5D-5L). Costs [euros (€), 2020] were considered from a societal perspective. Lifetime cost-effectiveness was assessed using a multi-state Markov model. Univariate, probabilistic sensitivity, and scenario analyses were performed.</p><p><strong>Results: </strong>In the trial, no significant differences were found in the effectiveness of PP compared with control, but wide confidence intervals (CIs) were seen [e.g., BMI (-0.07, 95% CI -0.51, 0.38)]. Lifetime estimates showed that PP increased costs (€520,102 versus €518,366, difference: €1736) and quality-adjusted life years (QALYs) (15.117 versus 15.106, difference: 0.011); the incremental cost-utility ratio (ICUR) was therefore high (€158,798 to gain one QALY). However, a 20% decrease in intervention costs would reduce the ICUR (€23,668 per QALY gained) below an unofficial gross domestic product (GDP)-based willingness-to-pay threshold (€47,817 per QALY gained).</p><p><strong>Conclusion: </strong>On the basis of the willingness-to-pay threshold and the non-significant differences in short-term effectiveness, PP may not be cost-effective. However, scaling up the intervention would reduce the intervention costs. Future studies should be larger and/or longer to reduce uncertainty about short-term effectiveness.</p><p><strong>Trial registration number: </strong>ClinicalTrials.gov registry (NCT04590989).</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"313-331"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10883904/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138808413","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":"Cost-Utility Analysis of Total Ankle Replacement Compared with Ankle Arthrodesis for Patients Aged 50-85 Years with End-Stage Ankle Osteoarthritis: The TARVA Study.","authors":"Andrew J Goldberg, Ekaterina Bordea, Kashfia Chowdhury, Iva Hauptmannova, James Blackstone, Deirdre Brooking, Elizabeth L Deane, Stephen Bendall, Andrew Bing, Chris Blundell, Sunil Dhar, Andrew Molloy, Steve Milner, Mike Karski, Steve Hepple, Malik Siddique, David T Loveday, Viren Mishra, Paul Cooke, Paul Halliwell, David Townshend, Simon S Skene, Caroline J Doré","doi":"10.1007/s41669-023-00449-4","DOIUrl":"10.1007/s41669-023-00449-4","url":null,"abstract":"<p><strong>Background: </strong>Patients with end-stage ankle osteoarthritis suffer from reduced mobility and quality of life and the main surgical treatments are total ankle replacement (TAR) and ankle fusion (AF).</p><p><strong>Objectives: </strong>Our aim was to calculate the mean incremental cost per quality-adjusted life-year (QALY) of TAR compared with AF in patients with end-stage ankle osteoarthritis, over 52 weeks and over the patients' lifetime.</p><p><strong>Method: </strong>We conducted a cost-utility analysis of 282 participants from 17 UK centres recruited to a randomised controlled trial (TARVA). QALYs were calculated using index values from EQ-5D-5L. Resource use information was collected from case report forms and self-completed questionnaires. Primary analysis was within-trial analysis from the National Health Service (NHS) and Personal Social Services (PSS) perspective, while secondary analyses were within-trial analysis from wider perspective and long-term economic modelling. Adjustments were made for baseline resource use and index values.</p><p><strong>Results: </strong>Total cost at 52 weeks was higher in the TAR group compared with the AF group, from the NHS and PSS perspective (mean adjusted difference £2539, 95% confidence interval [CI] £1142, £3897). The difference became very small from the wider perspective (£155, 95% CI -  £1947, £2331). There was no significant difference between TAR and AF in terms of QALYs (mean adjusted difference 0.02, 95% CI -  0.015, 0.05) at 52 weeks post-operation. The incremental cost-effectiveness ratio (ICER) was £131,999 per QALY gained 52 weeks post-operation. Long-term economic modelling resulted in an ICER of £4200 per QALY gained, and there is a 69% probability of TAR being cost effective at a cost-effectiveness threshold of £20,000 per QALY gained.</p><p><strong>Conclusion: </strong>TAR does not appear to be cost effective over AF 52 weeks post-operation. A decision model suggests that TAR can be cost effective over the patients' lifetime but there is a need for longer-term prospectively collected data. Clinical trial registration ISRCTN60672307 and ClinicalTrials.gov NCT02128555.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"235-249"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884388/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139378108","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 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}