PharmacoEconomics Open最新文献

{"title":"Budget Impact Analysis of Risperidone Use and Adverse Event Monitoring in Autism Spectrum Disorder in Brazil: Assessment of Theoretical Versus Real Data.","authors":"Luis Phillipe Nagem Lopes, Alexander Itria, Luciane Cruz Lopes","doi":"10.1007/s41669-023-00436-9","DOIUrl":"10.1007/s41669-023-00436-9","url":null,"abstract":"<p><strong>Background and aims: </strong>Risperidone is used in autism spectrum disorder (ASD) to manage aggressive behavior. Budget impact analysis (BIA) assists managers in promoting more sustainable health systems; however, it is unclear whether BIAs underestimate or overestimate the estimates derived from real-world data. This study aimed to compare the estimated BIA values of risperidone use and the monitoring of adverse events in ASD using theoretical and real data.</p><p><strong>Methods: </strong>Analyses were conducted based on the clinical protocol and the Brazilian therapeutic guidelines for ASD. The perspective adopted was that of the Unified Health System (SUS), considering a time horizon of 5 years. Three possible scenarios were considered based on the maximum daily dose of risperidone. Expenses related to the acquisition of risperidone and the monitoring of adverse events were taken into account using health databases in Brazil. For the calculation based on theoretical data, the prevalence of ASD was estimated using information from the scientific literature and the Brazilian demographic census. The model calculated from real data was obtained by analyzing the linear trend of the number of users assisted in the SUS from 2017 to 2021.</p><p><strong>Results: </strong>The population estimated by the theoretical model compared with the model calculated from the real data was higher. Likewise, the 5-year budgetary impact of the theoretical model versus the model calculated from the real data was higher, with statistical significance in all scenarios (p < 0.001). In the real data model, the most economically advantageous scenarios were Scenario 1 for children (International dollars [I$] 7,630,040.73) and Scenario 3 for adults (I$60,329,288.17). Estimated expenditures for monitoring adverse events ranged from 17 to 74% in children and from 50% to 63% in adults.</p><p><strong>Conclusions: </strong>The data revealed significant differences in population and cost estimation between theoretical data and real-world data. The expenses associated with monitoring adverse events represented a substantial expenditure estimate for the SUS.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721756/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10581446","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":"Model and Empirical Data-Based Cost-Utility Studies of Continuous Glucose Monitoring in Individuals with Type 1 Diabetes: A Protocol of a Systematic Review on Methodology and Quality.","authors":"L A de Jong, X Li, S Emamipour, S van der Werf, M J Postma, P R van Dijk, T L Feenstra","doi":"10.1007/s41669-023-00428-9","DOIUrl":"10.1007/s41669-023-00428-9","url":null,"abstract":"<p><strong>Introduction: </strong>This review aims to critically appraise differences in methodology and quality of model-based and empirical-data-based cost-utility studies to address key limitations, opportunities, and challenges to inform future cost-utility analyses of continuous glucose monitoring (CGM) in type 1 diabetes. This protocol is registered at PROSPERO (CRD42023391284).</p><p><strong>Methods: </strong>The review will be conducted in accordance with the PRISMA guideline for systematic reviews. Searches will be conducted in MEDLINE, Embase, Web of Science, Cochrane Library, and Econlit from 2000 to January 2023. Model and empirical data-based studies evaluating the cost-utility of any CGM system in type 1 diabetes will be considered for inclusion. Studies that only report on cost per life year or any other clinical outcome, or reporting only costs or only clinical outcomes studies in type 2 diabetes populations, and studies on bi-hormonal closed loops and do-it-yourself hybrid closed loop devices will be excluded. Two reviewers will independently screen each study for inclusion. Data on the intervention, population, model settings (such as perspective, time horizon), model type and structure, clinical outcomes used to populate the model, validation, and uncertainty will be extracted and qualitatively synthesised. Quality will be assessed using the Philips et al. 2006 (model-based studies) or Consensus Health Economic Criteria (empirical data-based studies) checklists. Model validation will be assessed using the AdViSHE checklist.</p><p><strong>Discussion: </strong>Now that CGM is being used more broadly in practice, critical appraisal of existing cost-utility methodology and quality is important to inform future cost-utility analyses of CGM in type 1 diabetes in various settings.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721749/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10052181","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":"Trade-Offs between Vaccine Effectiveness and Vaccine Safety: Personal versus Policy Decisions.","authors":"Semra Ozdemir, Sean Ng, Vinh Anh Huynh, Axel Mühlbacher, Hiang Khoon Tan, Eric Andrew Finkelstein","doi":"10.1007/s41669-023-00442-x","DOIUrl":"10.1007/s41669-023-00442-x","url":null,"abstract":"<p><strong>Objective: </strong>We aimed to investigate whether individuals' trade-offs between vaccine effectiveness and vaccine safety vary if they are asked to consider the perspective of a policymaker making decisions for others compared with the decisions they would make for themselves.</p><p><strong>Method: </strong>A web-enabled discrete choice experiment survey was administered between 1 April and 1 May 2022 to participants recruited from the general population of two Southeast Asian countries (Indonesia and Vietnam). In each country, 500 participants were randomly assigned to make decisions regarding coronavirus disease 2019 (COVID-19) vaccines for others as a policymaker or in a personal capacity for their own use. Vaccines were characterized by three attributes: (1) effectiveness of the vaccine in reducing infection rate; (2) effectiveness of the vaccine in reducing hospitalization among those infected; and (3) risk of death from vaccine-related serious adverse events. A mixed logit model was utilized for analyses.</p><p><strong>Results: </strong>Based on the attributes and levels used in this study, the most important vaccine attribute was the risk of death from vaccine-related adverse events, followed by effectiveness in reducing infection rate and hospitalizations. Compared with personal decisions, the mean probability of choosing a vaccine was (1) lower, and (2) more sensitive to the changes in risk of death from adverse events in policy decisions (p ≤ 0.01).</p><p><strong>Conclusions and relevance: </strong>Our results suggest that, in the face of an infectious disease pandemic, individuals are likely to be more risk-averse to vaccine-related deaths when making decisions for others as a policymaker than they would for themselves.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721762/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41208227","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 EQ-5D-3L Valuation Study in Pakistan.","authors":"Madeeha Malik, Ning Yan Gu, Azhar Hussain, Bram Roudijk, Fredrick Dermawan Purba","doi":"10.1007/s41669-023-00437-8","DOIUrl":"10.1007/s41669-023-00437-8","url":null,"abstract":"<p><strong>Background: </strong>To utilize EQ-5D in economic evaluations, a societal-based value set is needed. To date, no value sets exist for any EQ-5D instrument in Pakistan. Previous EQ-5D studies conducted in Pakistan 'borrowed' health preferences developed in other countries. However, for a value set to be valid for Pakistani population, it should represent the preferences of the Pakistani population, and culture and living standards of Pakistan.</p><p><strong>Objective: </strong>The aim of this study was to derive a Pakistani EQ-5D-3L value set.</p><p><strong>Methods: </strong>A moderately representative sample aged 18 years and over was recruited from the Pakistani general population. A multi-stage stratified quota method with respect to ethnicity, gender, age and religion was utilized. Two elicitation techniques, the composite time trade-off (cTTO) and discrete choice experiments (DCE) were applied. Interviews were undertaken by trained interviewers using computer-assisted face-to-face interviews with the EuroQol Portable Valuation Technology (EQ-PVT) platform. To estimate the value set, a hybrid regression model combining cTTO and DCE data was used.</p><p><strong>Results: </strong>A total of 289 respondents who completed the interviews were included for the analysis. The hybrid model correcting for heteroskedasticity without a constant was selected as the final model for the value set. It is shown that being unable to do usual activities (level 3) was assigned the largest weight, followed by mobility level 3, self-care level 3, pain/discomfort level 3 and anxiety/depression level 3. The worst health state was assigned the value - 0.171 in the final model.</p><p><strong>Conclusions: </strong>A Pakistani country-specific EQ-5D-3L value set is now available. The availability of this value set may help promote and facilitate health economic evaluations and health-related quality-of-life (HRQoL) research in Pakistan.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721573/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10222388","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 of Non-pharmacological Interventions for Mild Cognitive Impairment and Dementia: A Systematic Review of Economic Evaluations and a Review of Reviews.","authors":"Gillian Eaglestone, Evdoxia Gkaintatzi, Harmony Jiang, Charlotte Stoner, Rosana Pacella, Paul McCrone","doi":"10.1007/s41669-023-00440-z","DOIUrl":"10.1007/s41669-023-00440-z","url":null,"abstract":"<p><strong>Background: </strong>Dementia prevalence is increasing, with no cure at present. Drug therapies have potential side effects and risk of mortality. People with dementia are frequently offered non-pharmacological interventions to improve quality of life and relieve symptoms. Identifying which interventions are cost-effective is important due to finite resources in healthcare services.</p><p><strong>Aims: </strong>The aims were to review published economic evaluations of community and nursing home non-pharmacological interventions for people with mild cognitive impairment or dementia and assess the usefulness of these evaluations for decision making in health services, for use by policy and local and national decision makers.</p><p><strong>Methods: </strong>We conducted a systematic review (PROSPERO CRD42021252999) of economic evaluations of non-pharmacological interventions for dementia or mild cognitive impairment with a narrative approach to data synthesis.</p><p><strong>Exclusions: </strong>interventions for dementia prevention/early detection/end of life care. Databases searched: Academic Search Premier, MEDLINE, Web of Science, EMBASE, Google Scholar, Cumulative Index to Nursing and Allied Health Literature (CINAHL), PsycInfo, Psychology and Behavioural Sciences Collection, PsycArticles, Cochrane Database of Systematic Reviews, Business Source Premier and Regional Business News; timeframe 1 January 2011-11 May 2023. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS).</p><p><strong>Results: </strong>The review included 37 economic evaluations and four reviews worldwide across several distinct forms of care: physical activity, cognition, training, multicomponent, assistive technology and other (specialist dementia care, group living, home care vs care home). The intervention with the strongest evidence of cost-effectiveness was maintenance cognitive stimulation therapy. Case management, occupational therapy and dementia care management also showed good evidence of cost-effectiveness.</p><p><strong>Conclusion: </strong>More economic evidence on the cost-effectiveness of specific dementia care interventions is needed, with consistency of methods and outcome measures. This could improve local and national decision makers' confidence to promote future cost-effective dementia interventions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721583/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41129759","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":"Characteristics and Quality Appraisal of the Economic Evaluations Done in Ethiopia: A Systematic Review.","authors":"Bereket Bahiru Tefera, Desalegn Getnet Demsie, Adane Yehualaw, Chernet Tafere, Kebede Feyisa, Malede Berihun Yismaw, Belayneh Kefale, Zewdu Yilma","doi":"10.1007/s41669-023-00433-y","DOIUrl":"10.1007/s41669-023-00433-y","url":null,"abstract":"<p><strong>Background: </strong>Health economic evaluation identifies, measures, values, and compares alternative strategies to efficiently allocate scarce resources. The validity, methodological quality, and generalizability of economic evaluations must be assessed, as poorly designed studies can lead to incorrect conclusions. Therefore, this study aimed to evaluate the quality and characteristics of published economic evaluations done in Ethiopia, using the Quality of Health Economics Studies (QHES) and Consolidated Health Economic Evaluation Reporting Standards (CHEERS) instruments.</p><p><strong>Methodology: </strong>Various electronic databases were searched using different keywords. We included only original studies conducted in Ethiopia that evaluated the cost and consequences of at least two health interventions. Abstracts, treatment guidelines, reviews, expert opinions, and studies that included other countries were excluded. Two reviewers independently evaluated each study using the QHES and CHEERS instruments and any disagreements were then resolved by a third reviewer.</p><p><strong>Result: </strong>The study included 21 studies published between 2002 and 2021. HIV was the most frequently evaluated medical condition, examined in four (19.06%) of the 21 studies. Seventeen of the studies (80.95%) compared healthcare services or programs, while the other four examined pharmaceutical products. Cost-utility analysis was the economic evaluation technique used in 14 studies (66.67%). Of the studies that disclosed their funding sources, foreign institutions were involved in funding 71.43% of them. Disability-adjusted life-year (DALY) was an outcome metric used in nine (42.86%) studies. The average QHES score of the studies was 82%. Fourteen studies had QHES scores of ≥75% and two had scores of <50%. The studies evaluated using the CHEERS instrument ranged in quality from 42.9% to 92.9%, with an average of 78.23%.</p><p><strong>Conclusion: </strong>Our study revealed that Ethiopia lacks health economic evaluations, particularly on non-communicable diseases. This indicates that the economic evaluation of healthcare interventions in Ethiopia is still in its early stages. Additionally, Ethiopian institutions have played a very limited role in funding research, highlighting the importance of active participation from local institutions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721755/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10058613","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":"Estimating Cumulative Health Care Costs of Childhood and Adolescence Autism Spectrum Disorder in Ontario, Canada: A Population-Based Incident Cohort Study.","authors":"Claire de Oliveira, Bryan Tanner","doi":"10.1007/s41669-023-00441-y","DOIUrl":"10.1007/s41669-023-00441-y","url":null,"abstract":"<p><strong>Background: </strong>Few studies have estimated cumulative health care costs post-diagnosis for individuals with autism spectrum disorder (ASD).</p><p><strong>Objectives: </strong>Using an incidence-based approach, the objective of this analysis was to estimate cumulative costs of ASD to the Ontario health care system of children and adolescents.</p><p><strong>Methods: </strong>Using administrative health records from Ontario, Canada's most populous province, a retrospective, population-based, incident cohort study of children and adolescents aged 0-19 years old diagnosed with ASD was undertaken to estimate cumulative health care costs of ASD to the health care system from 2010 to 2019. Cumulative health care costs in 2021 Canadian dollars (CAD) from diagnosis to death or end of observation period were estimated using a consistent estimator based on the inverse probability weighting technique. Cumulative health care costs (and respective 95% confidence intervals [CI]) were estimated for 1, 5 and 10 years post-diagnosis by sex, age group and health service.</p><p><strong>Results: </strong>In 2010, there were 2867 diagnosed cases of ASD; in 2019, the number of incident cases had risen to 6072. The first year (i.e., 1-year) post-diagnosis cost of ASD was $4710.18 CAD (95% CI 4560.28-4860.08); just under a third of costs were for physician services. Total cumulative 5- and 10-year discounted costs were $16,025.95 CAD (15,371.64-16,680.26) and $32,635.76 CAD (28,906.94-36,364.58), respectively. Mean costs were higher for females and older age groups.</p><p><strong>Conclusions: </strong>These results suggest that costs of ASD are high in the year of diagnosis and then increase at a steady rate thereafter. This information will help with future resource planning within the health care sector to ensure individuals with ASD are supported once their diagnosis is established.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721567/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41143731","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":"Correction: Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15.","authors":"Marty Chaplin, Rebecca Bresnahan, Nigel Fleeman, James Mahon, Rachel Houten, Sophie Beale, Angela Boland, Yenal Dundar, Ashley Marsden, Pinki Munot","doi":"10.1007/s41669-023-00462-7","DOIUrl":"10.1007/s41669-023-00462-7","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721744/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138488191","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":"Hospital Resource Utilization and Costs in Patients with Heart Failure in France.","authors":"Frank Chemouni, Tiffany Chihiro Nishikawa, Harinala Groyer, Oumou Diaby, Julien Chollet, Deborah Ittah","doi":"10.1007/s41669-023-00431-0","DOIUrl":"10.1007/s41669-023-00431-0","url":null,"abstract":"<p><strong>Background and objectives: </strong>Heart failure (HF) is one of the leading causes of morbidity and mortality, and economic burden on the healthcare system. The aim of this study was to estimate the current hospital resource utilization and costs for HF patients in France.</p><p><strong>Methods: </strong>This retrospective cohort study included adult HF patients hospitalized in France between January 1, 2019 and December 31, 2019. Data related to sociodemographic characteristics, number and duration of hospital stays, use of medical procedures or expensive and innovative drugs/medical devices included in the \"liste-en-sus\", and comorbidities were retrieved from the French national hospital discharge database. Data were further stratified based on the presence or absence of cardiac decompensation, comorbidities, ejection fraction (EF) status, and incident/prevalent patients.</p><p><strong>Results: </strong>In 2019, a total of 430,544 patients were hospitalized in France with HF as a primary or associated diagnosis, with 51.9% male and 48.1% female and a mean age of 79.0 years. More than 75% of the study population was composed of prevalent HF patients. About 3.1% of patients were diagnosed with at least one event of cardiac decompensation during follow-up. Also, 20.2% and 9.9% of patients were identified with preserved and reduced EFs, respectively. The average number and length of hospital stays were 1.7 per patient and 10.4 days per patient, respectively. The annual cost of hospitalization for HF was €8341.3 per patient. Presence of cardiac decompensation at index date or during follow-up, reduced EF, and comorbidities were associated with numerically higher frequency and length of hospitalization, and hospitalization cost. For hospitalization and 'liste-en-sus' medical devices, higher cost was observed in incident than prevalent HF patients, while for 'liste-en-sus' drugs, higher cost was reported in prevalent than incident HF patients.</p><p><strong>Conclusion: </strong>This study highlighted the high economic hospital burden of HF in France. More studies investigating different HF patient profiles must be conducted to help determine the main factors of hospital cost for HF.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721763/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10592236","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":"Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15.","authors":"Marty Chaplin, Rebecca Bresnahan, Nigel Fleeman, James Mahon, Rachel Houten, Sophie Beale, Angela Boland, Yenal Dundar, Ashley Marsden, Pinki Munot","doi":"10.1007/s41669-023-00439-6","DOIUrl":"10.1007/s41669-023-00439-6","url":null,"abstract":"<p><p>As part of the National Institute for Health and Care Excellence (NICE) highly specialised technology (HST) evaluation programme, Novartis submitted evidence to support the use of onasemnogene abeparvovec as a treatment option for patients with pre-symptomatic 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival of motor neuron (SMN) 1 gene and up to three copies of the SMN2 gene. The Liverpool Reviews and Implementation Group at the University of Liverpool was commissioned to act as the External Assessment Group (EAG). This article summarises the EAG's review of the evidence submitted by the company and provides an overview of the NICE Evaluation Committee's final decision, published in April 2023. The primary source of evidence for this evaluation was the SPR1NT trial, a single-arm trial including 29 babies. The EAG and committee considered that the SPR1NT trial results suggested that onasemnogene abeparvovec is effective in treating pre-symptomatic SMA; however, long-term efficacy data were unavailable and efficacy in babies aged over 6 weeks remained uncertain. Cost-effectiveness analyses conducted by the company and the EAG (using a discounted price for onasemnogene abeparvovec) explored various assumptions; all analyses generated incremental cost-effectiveness ratios (ICERs) that were less than £100,000 per quality-adjusted life-year (QALY) gained. The committee recommended onasemnogene abeparvovec as an option for treating pre-symptomatic 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene in babies aged ≤ 12 months only if the company provides it according to the commercial arrangement (i.e. simple discount patient access scheme).</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10721753/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41145248","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}