medRxiv - Health Economics最新文献

{"title":"The economic effects of kidney failure treatment on the household welfare of patients on Dialysis in Buea and Bamenda–Cameroon","authors":"Therence Nnwana Dingana, Stewart Ndutard Ngasa, Neh Chang Ngasa, Leo Fosso Fozeu, Fuein V Kum, Aloysius M Njong","doi":"10.1101/2023.12.05.23299509","DOIUrl":"https://doi.org/10.1101/2023.12.05.23299509","url":null,"abstract":"Background: Noncommunicable diseases, such as kidney failure, diabetes, and cancer, are among the leading causes of death worldwide. There is a sharp increase in the incidence and prevalence of patients with kidney failure requiring replacement therapy. This has led to a very high cost, especially in resource-limited settings like Cameroon. The aim of this study is to determine the effects of direct and indirect costs of kidney failure treatment on their household income.\u0000Methods: A descriptive cross-sectional study design was used. Primary data was collected using a self-administered pre-tested questionnaire for the economic impact of chronic disease. For bivariate analysis, we used the Cochran-Mantel-Haenszel test to obtain crude Odd Ratios (OR) of factors associated with household welfare. Multivariate logistic regression, the OLS model was used to identify independent associations between kidney failure treatment and household welfare. This was presented as adjusted odd ratios along with their p-values. A p-value of <0.05 was used as a cut-off for statistical significance. Results: The mean age of participants was 44.6±15.5 years; most patients (83(62.4%)) were married. Seventy-nine (59.4%) were unemployed, and eighty-one (60.9%) had no financial support. Their annual household expenditure ranged from 300,000FCFA to 3,360,000FCFA, with a mean and standard deviation of 1,547,729FCFA and 781,882FCFA, respectively. The yearly total cost of kidney failure treatment ranged from 520,000FCFA to 10,000,000FCFA with a mean and standard deviation of 2,137,556FCFA and 1,541,163FCFA, respectively. The cost of consultation and laboratory tests had negative regression coefficients (P=0.001 and <0.001 respectively).\u0000Conclusion: kidney failure has a significant negative effect on the household welfare of patients on dialysis. Kidney disease screening and prevention programs are necessary to reduce the number of persons in need of hemodialysis. Health insurance schemes and universal health coverage should target patients on hemodialysis.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138555297","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost minimization analysis of treatments for metastatic HER2-positive breast cancer: Fixed-Dose combination of pertuzumab and trastuzumab for subcutaneous injections","authors":"Miguel Figallo, María F Delgado, Mauricio Gonzalez, Adrián Arenas","doi":"10.1101/2023.12.05.23299511","DOIUrl":"https://doi.org/10.1101/2023.12.05.23299511","url":null,"abstract":"The main objective of this study is to determine whether the employment of fixed-dose combination of pertuzumab and trastuzumab for subcutaneous injection (or Phesgo) to treat metastatic HER2-positive breast cancer patients would minimize costs compared to the traditional treatment of separate intravenous doses of pertuzumab and trastuzumab in Peru. To achieve this, we used EsSalud (the social security health insurance) data and assessed it through a mixed strategy, which consisted of a quantitative and a qualitative approach. The first one aimed to calculate the direct (non-drug consumables, drugs, and healthcare professionals) and indirect costs of both treatments to develop a comparison, whilst the second aimed to validate information and internalize the procedure in an EsSalud context.\u0000Overall, we found that the usage of Phesgo would be cost saving in EsSalud’s context. Specifically, we found three main insights. Firstly, the savings produced from switching from the traditional intravenous treatment to the subcutaneous one would allow EsSalud to afford full annual costs of 2 additional treatments, but without increasing their budget. This would cover 7% of the gap of 29 patients who do not have access to full treatment. Secondly, the result of a univariate sensitivity analysis in this work indicates that the price of Phesgo would have to increase by 16% so that both treatments cost the same. Thirdly, to increase the number of patients, the intravenous treatment would have to be totally replace by the Phesgo treatment, since the use of the latter requires less personnel and infrastructure.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138547191","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treating Rheumatoid Arthritis in Zanzibar: a cost effectiveness study comparing conventional, biologic, and targeted-synthetic disease modifying anti-rheumatic drugs","authors":"Sanaa Suleiman Said, Melf-Jakob Kühl, Bjørg-Tilde Svanes Fevang, Tone Wikene Nystad, Kjell Arne Johansson","doi":"10.1101/2023.12.05.23299489","DOIUrl":"https://doi.org/10.1101/2023.12.05.23299489","url":null,"abstract":"To evaluate the cost effectiveness of six disease modifying anti-rheumatic drug (DMARD) treat-to-target treatment strategies for patients with rheumatoid arthritis in Zanzibar.\u0000A Markov model was used to calculate the cost-effectiveness of various DMARD strategies in the treatment of rheumatoid arthritis over a three-year period. A health-provider perspective was used and only outpatient costs were considered. The Clinical Disease Activity Index (CDAI) was utilized for measurement of efficacy and values were obtained from literature. Quality Adjusted Life Years (QALYs) were obtained from 122 patients attending the rheumatology clinic at Mnazi Mmoja Hospital. Data on costs were obtained from the central medical stores and hospital administration. Treatment strategies were given in sequential approach based on treat to target goals of therapy. This included methotrexate monotherapy, methotrexate + sulfasalazine + hydroxychloroquine, methotrexate followed by one or two biologic/targeted-synthetic DMARDs (b/tsDMARDs). Probabilistic and one way sensitivity analysis were performed. Scenario analysis was undertaken comparing drug prices from India and Scandinavia.\u0000Costs of therapy/patient/three years ranged from USD 634 for methotrexate monotherapy and USD 5011 for methotrexate and two consecutive b/tsDMARDs. The highest and lowest effects were 2.209 and 2.079 QALYs gained from methotrexate therapy + two consecutive b/tsDMARDs and methotrexate monotherapy, respectively. From a healthcare perspective methotrexate monotherapy was the cost-effective option at a willingness to pay of USD 282. Pairwise comparison also favored methotrexate monotherapy as the feasible option. We found that increasing the willingness to pay led to a change in the most acceptable option from methotrexate monotherapy to methotrexate followed by b/tsDMARD.\u0000Methotrexate monotherapy is the cost-effective option for the management of rheumatoid arthritis in Zanzibar. Other options may be feasible if the willingness to pay threshold is increased or the drug prices are lowered, particularly for the b/tsDMARDs.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138547123","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The potential economic impact of the updated COVID-19 mRNA fall 2023 vaccines in Japan","authors":"Kelly Fust, Keya Joshi, Ekkehard Beck, Michael Maschio, Michele Kohli, Amy Lee, Yuriko Hagiwara, Nicolas Van de Velde, Ataru Igarashi","doi":"10.1101/2023.12.04.23299402","DOIUrl":"https://doi.org/10.1101/2023.12.04.23299402","url":null,"abstract":"This analysis estimates the economic and clinical impact of a Moderna updated COVID-19 mRNA Fall 2023 vaccine for adults at least 18 years in Japan. A previously developed Susceptible Exposed Infected Recovered (SEIR) model with a 1 year analytic time horizon (September 2023 to August 2024) and consequences decision tree were used to estimate symptomatic infections, COVID19 related hospitalizations, deaths, quality adjusted life years (QALYs), costs, and incremental cost-effectiveness ratio (ICER) for a Moderna updated Fall 2023 vaccine versus no additional vaccination, and versus a Pfizer-BioNTech updated mRNA Fall 2023 vaccine. The Moderna vaccine is predicted to prevent 7.2 million symptomatic infections, 272,100 hospitalizations and 25,600 COVID-19 related deaths versus no vaccine. In the base case (healthcare perspective), the ICER was 1,300,000 Yen/QALY gained ($9,400 USD/QALY gained). Sensitivity analyses suggest results are most affected by COVID 19 incidence, initial vaccine effectiveness (VE), and VE waning against infection. Assuming the relative VE between both bivalent vaccines apply to updated Fall 2023 vaccines, the base case suggests the Moderna version will prevent an additional 1,100,000 symptomatic infections, 27,100 hospitalizations, and 2,600 deaths compared to the Pfizer-BioNTech vaccine. The updated Moderna vaccine is expected to be highly cost effective at a 5 million Yen willingness to pay threshold across a wide range of scenarios.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138494183","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The impact of perioperative diagnostic tools on clinical outcomes and cost-effectiveness in parathyroid surgery: a health economic evaluation","authors":"Daniel Batora, Rowan Iskandar, Juerg Gertsch, Reto M. Kaderli","doi":"10.1101/2023.12.04.23299113","DOIUrl":"https://doi.org/10.1101/2023.12.04.23299113","url":null,"abstract":"Objectives\u0000Pre- and intraoperative diagnostic tools influence the surgical management of primary hyperparathyroidism (PHPT), whereby their performance of classification varies considerably for the two common causes of PHPT: solitary adenomas and multiglandular disease. A consensus on the use of such diagnostic tools for optimal perioperative management of all PHPT patients has not been reached. Design\u0000A decision tree model was constructed to estimate and compare the clinical outcomes and the cost-effectiveness of preoperative imaging modalities and intraoperative parathyroid hormone (ioPTH) monitoring criteria in a 14-year time horizon. The robustness of the model was assessed by conducting a one-way sensitivity analysis and probabilistic uncertainty analysis.\u0000Setting\u0000The United States healthcare system.\u0000Population\u0000A hypothetical population consisting of 5,000 patients with sporadic, symptomatic, or asymptomatic PHPT.\u0000Interventions\u0000Pre- and intraoperative diagnostic modalities for parathyroidectomy.\u0000Main outcome measures\u0000Costs, quality-adjusted life years (QALYs), net monetary benefits (NMB), clinical outcomes.\u0000Results\u0000In the base-case analysis, four-dimensional (4D)-computed tomography (CT) was the least expensive strategy with $10,289 and 13.93 QALYs. Ultrasound and 99mTc-Sestamibi single-photon-emission computed tomography/CT were both dominated strategies, while 18F-fluorocholine positron emission tomography was cost-effective with a net monetary benefit of $264 considering a willingness to pay threshold of $95,958. The application of ioPTH monitoring with the Vienna criterion decreased the rate of reoperations from 10.50 to 0.58 per 1,000 patients. Due to an increased rate of bilateral neck explorations from 257.45 to 347.45 per 1,000 patients, it was not cost-effective.\u0000Conclusions\u00004D-CT is the most cost-effective instrument for the preoperative localization of parathyroid adenomas. Due to an excessive increase of bilateral neck explorations, the use of ioPTH monitoring is not cost-effective in PHPT but leads to a significant reduction of reoperations.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138494184","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tracking the impact of government response to COVID-19 epidemic: Evidence from India","authors":"Kaibalyapati Mishra","doi":"10.1101/2023.11.27.23299097","DOIUrl":"https://doi.org/10.1101/2023.11.27.23299097","url":null,"abstract":"This paper tries to quantify the impact of government policy intervention on the death due to COVID-19 in India at national, regional and sub-national levels. The data used for this study are collected from the Oxford COVID-19 Government Response Tracker (OxCGRT), a longitudinal database of daily government response from Jan 28th, 2020, when the first COVID case was diagnosed in India till December 31st, 2022. Here, <em>stringency</em> measures, which gauge the severity of interventions such as lock-downs and travel restrictions, indicative of government control; and <em>containment</em> measures, representing a spectrum of actions aimed at preventing or limiting virus transmission and the <em>overall government support</em>, providing a holistic assessment of the government’s efforts in mitigating the virus’s spread. Using the Panel Corrected Standard Error (PCSE) method, this paper finds out that the stringency and overall government support interventions by the government have been successful in reducing the death counts by 25% and 23% respectively however the containment intervention alone has failed to reduce the death at all levels. Exploring regional variations, event study plots reveal nuanced temporal dynamics. The daily and 24-day lagged dependent variables, representing overall government response and stringency measures, reveal a consistent impact post-intervention at the all-India level. Both current and lagged variables show a reduction in COVID-19 deaths, with a more pronounced effect emerging after a four-day lag. Event-study plots with a 24-day lagged dependent variable confirm the anticipated negative impact of overall government response on deaths. However, the pattern diverges for stringency and overall government interventions compared to daily death counts.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138503865","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Association between Multimorbidity and Out-of-Pocket Expenditures for Prescription Medicines among Adults in Denmark: A Population-Based Register Study","authors":"James Larkin, Susan M. Smith, Line Due Christensen, Thomas Schmidt Voss, Claus Høstrup Vestergaard, Amanda Paust, Anders Prior","doi":"10.1101/2023.11.21.23298458","DOIUrl":"https://doi.org/10.1101/2023.11.21.23298458","url":null,"abstract":"Background Multimorbidity, defined as two or more chronic conditions in an individual, is increasing in prevalence and is associated with polypharmacy. Polypharmacy can lead to increased out-of-pocket payments for prescription medicines. This, in turn, can be associated with cost-related non-adherence and impoverishment. Healthcare in Denmark is mostly free at the point-of-use; prescription medicines are one of the only exceptions. Objective To examine the association between multimorbidity and annual out-of-pocket prescription medicine expenditure for adults in Denmark. Methods A population-based register study was conducted. The study population included all adults residing in Denmark in 2020. Frequencies and descriptive statistics were used and regression analyses were conducted to assess the association between multimorbidity and annual out-of-pocket prescription medicine expenditure, while controlling for demographic and socioeconomic covariates. Results Overall, 1,212,033 (24.2%) individuals had multimorbidity. Individuals with five or more conditions spent, on average, 320 euro in out-of-pocket prescription medicines expenditure compared to 187 euro for those with two conditions and 44 euro for those with no conditions. Amongst those with any out-of-pocket prescription medicine expenditure, having multimorbidity was associated with 2-4 times greater out-of-pocket prescription medicine expenditure than those with zero conditions. Amongst those in the quantile with the highest expenditure, those with five or more conditions spent 408 euro more than those with no conditions, and those with two conditions spent 185 euro more than those with no conditions. Conclusions For adults in Denmark, multimorbidity was associated with significantly higher out-of-pocket prescription medicine expenditure, even after controlling for demographic and socioeconomic covariates. This is similar to patterns in other countries and likely affects those with lowest income the most, given the known socioeconomic patterning of multimorbidity and raises concerns about cost related non-adherence. Potential protective mechanisms could include subsidies for certain vulnerable patient groups (e.g. those with severe mental illness) and low-income groups.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138517328","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of State Telehealth Parity Laws for Private Payers on Hypertension Management before and during the COVID-19 Pandemic","authors":"Donglan Zhang, Jun Soo Lee, Adebola Popoola, Sarah Lee, Sandra L. Jackson, Lisa M Pollack, Xiaobei Dong, Feijun Luo, Nicole Leigh Therrien","doi":"10.1101/2023.11.16.23298658","DOIUrl":"https://doi.org/10.1101/2023.11.16.23298658","url":null,"abstract":"BACKGROUND: Telehealth has emerged as an effective tool for managing common chronic conditions such as hypertension, especially during the COVID-19 pandemic. However, the impact of state telehealth payment and coverage parity laws on hypertension management remains uncertain. METHODS: Data from the MerativeTM MarketScan(R) Commercial Claims and Encounters Database from January 1, 2016 to December 31, 2021 were used to construct the study cohort. The sample included non-pregnant individuals aged 25?64 years with hypertension. We reviewed and coded telehealth parity laws related to hypertension management in all 50 states and the District of Columbia, distinguishing between payment parity laws and coverage parity laws. The primary outcomes were antihypertension medication use, measured by the average medication possession ratio (MPR), medication adherence (MPR ?80%), and average number of days of drug supply. We used a generalized difference-in-difference (DID) design to examine the impact of these laws. Results were presented as marginal effects and 95% confidence intervals (CI). RESULTS: Among 353,220 individuals, states with payment parity laws were significantly linked to increased average MPR by 0.43 percentage point (95% CI: 0.07 - 0.79), and an increase of 0.46 percentage point (95% CI: 0.06 - 0.92) in the probability of medication adherence. Payment parity laws also led to an average increase of 2.14 days (95% CI: 0.11 - 4.17) in antihypertensive drug supply, after controlling for state-fixed effects, year-fixed effects, individual sociodemographic characteristics and state time-varying covariates including unemployment rates, GDP per capita, and poverty rates. In contrast, coverage parity laws were associated with a 2.13-day increase (95% CI: 0.19 - 4.07) in days of drug supply, but did not significantly increase the average MPR or probability of medication adherence. In addition, telehealth payment or coverage parity laws were positively associated with the number of hypertension-related telehealth visits, but this effect did not reach statistical significance. These findings were consistent in sensitivity analyses. CONCLUSIONS: State telehealth payment parity laws were significantly associated with greater medication adherence, whereas coverage parity laws were not. With the increasing adoption of telehealth parity laws across states, these findings may support policymakers in understanding potential implications on management of hypertension.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138503864","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-effective boosting allocations in the post-Omicron era of COVID-19 management","authors":"Thao P Le, Eamon Conway, Edifofon Akpan, Isobel Abell, Patrick Abraham, Christopher M Baker, Patricia T Campbell, Deborah Cromer, Michael J Lydeamore, Yasmine McDonough, Ivo Mueller, Gerard Ryan, Camelia Walker, Yingying Wang, Natalie Carvalho, Jodie McVernon","doi":"10.1101/2023.11.14.23298536","DOIUrl":"https://doi.org/10.1101/2023.11.14.23298536","url":null,"abstract":"Background Following widespread exposure to Omicron variants, COVID-19 has transitioned to endemic cir-\u0000culation. Populations now have diverse infection and vaccination histories, resulting in heterogeneous\u0000immune landscapes. Careful consideration of vaccination is required through the post-Omicron phase\u0000of COVID-19 management to minimise disease burden. We assess the impact and cost-effectiveness\u0000of targeted COVID-19 vaccination strategies to support global vaccination recommendations. Methods We integrated immunological, transmission, clinical and cost-effectiveness models, and simulated\u0000populations with different characteristics and immune landscapes. We calculated the expected number\u0000of infections, hospitalisations and deaths for different vaccine scenarios. Costs (from a healthcare\u0000perspective) were estimated for exemplar country income level groupings in the Western Pacific\u0000Region. Results are reported as incremental costs and disability-adjusted life years averted compared\u0000to no additional vaccination. Parameter and stochastic uncertainty were captured through scenario and\u0000sensitivity analysis. Findings Across different population demographics and income levels, we consistently found that annual\u0000elder-targeted boosting strategies are most likely to be cost-effective or cost-saving, while paediatric\u0000programs are unlikely to be cost-effective. Results remained consistent while accounting for uncertain-\u0000ties in the epidemiological and economic models. Half-yearly boosting may only be cost-effective in\u0000higher income settings with older population demographics and higher cost-effectiveness thresholds. Interpretation These results demonstrate the value of continued booster vaccinations to protect against severe\u0000COVID-19 disease outcomes across high and middle-income settings and show that the biggest health\u0000gains relative to vaccine costs are achieved by targeting older age-groups. Funding Funded by the World Health Organization.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138503863","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies","authors":"Ryan Fischer, Pat Furlong, Annie Kennedy, Kelly Maynard, Marissa Penrod, Debra Miller, Chamindra G Laverty, Linda Lowes, Nancy L Kuntz, Perry B Shieh, Jane Kondejewski, Peter J Neumann, Jason Shafrin, Richard J Willke","doi":"10.1101/2023.07.11.23292507","DOIUrl":"https://doi.org/10.1101/2023.07.11.23292507","url":null,"abstract":"BACKGROUND: Traditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as Duchenne muscular dystrophy (DMD). OBJECTIVE: To identify how value assessment frameworks may need to be adapted to measure the value to society of DMD therapies.\u0000METHODS: Three groups of stakeholders (patient advocates, clinicians, health economists) participated in semi-structured interviews around the International Society for Pharmacoeconomics and Outcomes Researchs Value Flower, which includes elements of value that can be considered within value assessments of healthcare technologies. RESULTS: All stakeholders agreed that traditional value assessment frameworks based on the quality-adjusted life year (QALY) are narrow and will undervalue new DMD therapies. All stakeholders expressed some level of concern that use of the QALY as a key metric of value discriminates against patients with severe progressive diseases and disabilities. Some stakeholders saw value in using the QALY for cross-disease comparisons in resource-constrained environments if the methodology was appropriate. All stakeholders recommended considering additional elements of value in decision-making around new DMD therapies. These elements reflect: the economic and humanistic costs incurred by patients, caregivers, and families with Duchenne, such as indirect out-of-pocket costs, lost productivity, and family spillovers; the attributes that are meaningful for individuals with disabilities and high unmet need, such as severity of disease, value of hope, and real option value; and factors that contribute to improvements in population health, such as insurance value, equity, and scientific spillovers. CONCLUSIONS: These findings highlight the need to expand traditional value assessment frameworks and take a holistic approach that incorporates the perspectives of individuals with Duchenne, caregivers, clinicians, and heath economists when assessing the societal value of new DMD therapies. Broadening value assessment will prevent restricted or delayed access to therapies for individuals with Duchenne.","PeriodicalId":501072,"journal":{"name":"medRxiv - Health Economics","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138516883","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}