Goran Bencina, Edward Oliver, Anne Meiwald, Robert Hughes, Edith Morais, Georgie Weston, Karin Sundström
{"title":"Global burden and economic impact of vaccine-preventable cancer mortality.","authors":"Goran Bencina, Edward Oliver, Anne Meiwald, Robert Hughes, Edith Morais, Georgie Weston, Karin Sundström","doi":"10.1080/13696998.2024.2350877","DOIUrl":"10.1080/13696998.2024.2350877","url":null,"abstract":"<p><strong>Background: </strong>Infections are responsible for approximately 13% of cancer cases worldwide and many of these infections can be prevented by vaccination. Human papillomavirus (HPV) and hepatitis B virus (HBV) are among the most common infections that cause cancer deaths globally, despite effective prophylactic vaccines being available. This analysis aims to estimate the global burden and economic impact of vaccine-preventable cancer mortality across World Health Organization (WHO) regions.</p><p><strong>Methods: </strong>The number of deaths and years of life lost (YLL) due to five different vaccine-preventable cancer forms (oral cavity, liver, laryngeal, cervical, and oropharyngeal cancer) in each of the WHO regions (African, Eastern Mediterranean, European, the Americas, South-East Asia Pacific, and Western Pacific) were obtained from the Institute for Health Metrics Evaluation global burden of disease dataset. Vaccine-preventable mortality was estimated considering the fraction attributable to infection, to estimate the number of deaths and YLL potentially preventable through vaccination. Data from the World Bank on GDP per capita were used to estimate the value of YLL (VYLL). The robustness of these results was explored with sensitivity analysis. Given that several Epstein-Barr virus (EBV) vaccines are in development, but not yet available, the impact of a potential vaccine for EBV was evaluated in a scenario analysis.</p><p><strong>Results: </strong>In 2019, there were 465,740 potentially vaccine-preventable cancer deaths and 14,171,397 YLL across all WHO regions. The estimated economic impact due to this mortality was $106.3 billion globally. The sensitivity analysis calculated a range of 403,025-582,773 deaths and a range in productivity cost of $78.8-129.0 billion. In the scenario analysis EBV-related cancer mortality increased the global burden by 159,723 deaths and $32.4 billion.</p><p><strong>Conclusion: </strong>Overall, the findings from this analysis illustrate the high economic impact of premature cancer mortality that could be potentially preventable by vaccination which may assist decision-makers in allocating limited resources among competing priorities. Improved implementation and increased vaccination coverage of HPV and HBV should be prioritized to decrease this burden.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"9-19"},"PeriodicalIF":2.4,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140891709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Goran Bencina, Ugne Sabale, Edith Morais, Olga Ovcinnikova, Edward Oliver, Hayley Shoel, Anne Meiwald, Robert Hughes, Georgie Weston, Karin Sundström
{"title":"Burden and indirect cost of vaccine-preventable cancer mortality in Europe.","authors":"Goran Bencina, Ugne Sabale, Edith Morais, Olga Ovcinnikova, Edward Oliver, Hayley Shoel, Anne Meiwald, Robert Hughes, Georgie Weston, Karin Sundström","doi":"10.1080/13696998.2024.2374684","DOIUrl":"https://doi.org/10.1080/13696998.2024.2374684","url":null,"abstract":"<p><strong>Background: </strong>The economic and mortality burden of cancer is high worldwide. In Europe, cancer was responsible for 1.3 million deaths in 2020 and incurred an estimated cost of €50 billion from premature mortality. Human papillomavirus (HPV) and hepatitis B virus (HBV) are among the leading causes of infection-related cancers despite the availability of effective vaccines against these infections. This analysis estimated the mortality and productivity loss of HBV- and HPV-associated cancers that could be preventable through vaccination across European regions.</p><p><strong>Materials and methods: </strong>Institute for Health Metrics Evaluation (IHME) data were used to estimate mortality, years of life lost (YLL), and the value of years of life lost (VYLL) from five HBV- and HPV-related cancers (oral cavity, oropharynx, larynx, cervical, and liver cancers) across 40 European countries in 2019. Preventable deaths and YLL were estimated based on fractions attributable to infections. Data from the World Bank on GDP per capita were used to estimate the VYLL. The robustness of these results was explored with sensitivity and scenario analyses.</p><p><strong>Results: </strong>In 2019, 31,906 cancer deaths resulted in an economic burden of €18,521,614,725 due to productivity losses across Europe. HPV-related cervical cancer had the highest mortality (19,473 deaths) and economic burden (€10,706,253,185). HBV-related liver cancer and HPV-related larynx, oral cavity, and oropharynx cancers also had a substantial burden, particularly in males. Eastern Europe had the highest YLL (308,179; 39%) and Western Europe was responsible for the greatest VYLL (€8,281,306,504; 45%), although the highest VYLL per death was in Northern Europe (€923,638). HPV-related oropharynx cancer had the highest VYLL per death (€656,607).</p><p><strong>Conclusion: </strong>HPV- and HBV-related cancer deaths are associated with substantial mortality and productivity losses in Europe, which could be reduced by the continued prioritization and implementation of prophylactic public health measures including systematic awareness, vaccination, and screening efforts.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"27 sup2","pages":"30-40"},"PeriodicalIF":2.9,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141620183","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kayla Engelbrecht, Olga Ovcinnikova, Dionysios Ntais, Hayley Shoel, Anne Meiwald, Robert Hughes, Georgie Weston, Edith Morais, Goran Bencina
{"title":"Productivity costs due to human papillomavirus-related cancer mortality in the United Kingdom.","authors":"Kayla Engelbrecht, Olga Ovcinnikova, Dionysios Ntais, Hayley Shoel, Anne Meiwald, Robert Hughes, Georgie Weston, Edith Morais, Goran Bencina","doi":"10.1080/13696998.2024.2368986","DOIUrl":"10.1080/13696998.2024.2368986","url":null,"abstract":"<p><strong>Background: </strong>Human papillomavirus (HPV) causes several cancers such as cervical cancer and some head and neck (oral cavity, pharynx, and larynx), vulval, vaginal, anal, and penile cancers. As HPV vaccination is available, there is potential to prevent these cancers attributed to HPV and consequently the burden associated with them. The aim of this analysis was to estimate the number of HPV-related cancer deaths and the productivity costs due to years of life lost (YLL) in the United Kingdom (UK).</p><p><strong>Method: </strong>A model was developed utilizing UK 2019 mortality data sourced from country-specific databases for England, Scotland, Wales, and Northern Ireland for the following HPV-related cancers: head and neck (ICD-10 C00-14 and C32), cervix uteri (C53), vaginal (C51), vulval (C52), anal (C21), and penile (C60). The proportion of deaths and years of life lost (YLL) due to HPV were estimated using HPV attributable fractions for each anatomic location from the published literature. Labor force participation, retirement ages, and mean annual earnings, discounted at 3.5% annually, were applied to YLL to calculate the present value of future lost productivity (PVFLP).</p><p><strong>Results: </strong>A total of 1817 deaths due to HPV-related cancers were reported in the UK in 2019 resulting in 31,804 YLL. Restricting to only YLL that occurred prior to retirement age yielded a total YPLL of 11,765 and a total PVFLP of £187,764,978.</p><p><strong>Conclusions: </strong>There is a high disease burden in the UK for HPV-related cancers, with a large economic impact on the wider economy due to productivity losses. Implementing and reinforcing public health measures to maintain high HPV vaccination coverage in both males and females may further facilitate reduction of this burden.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"20-29"},"PeriodicalIF":2.9,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141419467","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Siguroli Teitsson, Thor-Henrik Brodtkorb, Murat Kurt, Miraj Y. Patel, Tayla Poretta, Christopher Knight, Farzam Kamgar, Stephen Palmer
{"title":"Challenges, considerations, and approaches for developing a cost-effectiveness model for the adjuvant treatment of muscle-invasive urothelial carcinoma: with a spotlight on nivolumab versus placebo","authors":"Siguroli Teitsson, Thor-Henrik Brodtkorb, Murat Kurt, Miraj Y. Patel, Tayla Poretta, Christopher Knight, Farzam Kamgar, Stephen Palmer","doi":"10.1080/13696998.2024.2322394","DOIUrl":"https://doi.org/10.1080/13696998.2024.2322394","url":null,"abstract":"To present challenges and considerations for the development of a decision analytic model for evaluating the cost-effectiveness of adjuvant nivolumab compared with surveillance in patients with hig...","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"1 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-02-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139924824","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Correction.","authors":"","doi":"10.1080/13696998.2024.2304418","DOIUrl":"https://doi.org/10.1080/13696998.2024.2304418","url":null,"abstract":"","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"27 1","pages":"153"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139403216","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Deborah R Kaye, Ibrahim Khilfeh, Erik Muser, Laura Morrison, Frederic Kinkead, Ana Urosevic, Patrick Lefebvre, Dominic Pilon, Daniel J George
{"title":"Real-world economic burden of metastatic castration-resistant prostate cancer before and after first-line therapy initiation.","authors":"Deborah R Kaye, Ibrahim Khilfeh, Erik Muser, Laura Morrison, Frederic Kinkead, Ana Urosevic, Patrick Lefebvre, Dominic Pilon, Daniel J George","doi":"10.1080/13696998.2024.2303890","DOIUrl":"10.1080/13696998.2024.2303890","url":null,"abstract":"<p><strong>Aims: </strong>To describe healthcare costs of patients with metastatic castration-resistant prostate cancer (mCRPC) initiating first-line (1 L) therapies from a US payer perspective.</p><p><strong>Methods: </strong>Patients initiating a Flatiron oncologist-defined 1 L mCRPC therapy (index date) on or after mCRPC diagnosis were identified from linked electronic medical records/claims data from the Flatiron Metastatic Prostate Cancer (PC) Core Registry and Komodo's Healthcare Map. Patients were excluded if they initiated a clinical trial drug in 1 L, had <12 months of insurance eligibility prior to index, or no claims in Komodo's Healthcare Map for the Flatiron oncologist-defined index therapy. All-cause and PC-related total costs per-patient-per-month (PPPM), including costs for services and procedures from medical claims (i.e. medical costs) and costs from pharmacy claims (i.e. pharmacy costs), were described in the 12-month baseline period before 1 L therapy initiation (including the baseline pre- and post- mCRPC progression periods) and during 1 L therapy (follow-up).</p><p><strong>Results: </strong>Among 459 patients with mCRPC (mean age 70 years, 57% White, 16% Black, 45% commercially-insured, 43% Medicare Advantage-insured, and 12% Medicaid-insured), average baseline all-cause total costs (PPPM) were $4,576 ($4,166 pre-mCRPC progression, $8,278 post-mCRPC progression). Average baseline PC-related total costs were $2,935 ($2,537 pre-mCRPC progression, $6,661 post-mCRPC progression). During an average 1 L duration of 8.5 months, mean total costs were $13,746 (all-cause) and $12,061 (PC-related) PPPM. The cost increase following 1 L therapy initiation was driven by higher PC-related outpatient and pharmacy costs. PC-related medical costs PPPM increased from $1,504 during baseline to $5,585 following 1 L mCRPC therapy initiation.</p><p><strong>Limitations: </strong>All analyses were descriptive; statistical testing was not performed.</p><p><strong>Conclusion: </strong>Incremental costs of progression to mCRPC are significant, with the majority of costs driven by higher PC-related costs. Using contemporary data, this study highlights the importance of utilizing effective therapies that slow progression and reduce healthcare resource demands despite the initial investment in treatment costs.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"201-214"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139417333","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Louis S Matza, Katie D Stewart, Josefine Redig, Timothy A Howell, Walter Morris, Rachel S Newson, Alexander Yasui, Jack Ishak, Kristina S Boye
{"title":"Health state utilities associated with weight loss: preferences of people with type 2 diabetes and obesity in Japan.","authors":"Louis S Matza, Katie D Stewart, Josefine Redig, Timothy A Howell, Walter Morris, Rachel S Newson, Alexander Yasui, Jack Ishak, Kristina S Boye","doi":"10.1080/13696998.2024.2316400","DOIUrl":"10.1080/13696998.2024.2316400","url":null,"abstract":"<p><strong>Aims: </strong>Health state utilities associated with weight change are needed for cost-utility analyses (CUAs) examining the value of treatments for type 2 diabetes and obesity. Previous studies have estimated the utility benefits associated with various amounts of weight reduction in the US and Europe, but preferences for weight change in Asian cultures may differ from these published values. The purpose of this study was to estimate utilities associated with reductions in body weight based on preferences of individuals with type 2 diabetes and obesity in Japan.</p><p><strong>Methods: </strong>Health state vignettes represented type 2 diabetes with respondents' own current weight and weight reductions of 2.5%, 5%, 7.5%, 10%, 12.5%, 15%, and 20%. Utilities were elicited in time trade-off interviews with a sample of respondents in Japan with type 2 diabetes and body mass index (BMI) ≥25 kg/m<sup>2</sup> (the cutoff for obesity in Japan).</p><p><strong>Results: </strong>Analyses were conducted with data from 138 respondents (84.8% male; mean age = 58.0 years; mean BMI = 29.4 kg/m<sup>2</sup>) from all eight regions of Japan. Utility gains gradually increased with rising percentage of weight reductions ranging from 2.5% to 15%. Weight reductions of 2.5% to 15% resulted in utility increases of 0.013 to 0.048. The health state representing a 20% weight reduction yielded a wide range of preferences (mean utility increase of 0.044). Equations are recommended for estimating utility change based on any percentage of weight reduction (up to 20%) in Japanese people with type 2 diabetes and obesity.</p><p><strong>Limitations: </strong>This study was conducted in a sample with limited representation of patients with BMI >35 kg/m<sup>2</sup> (<i>n</i> = 13) and relatively few women (<i>n</i> = 21).</p><p><strong>Conclusion: </strong>Results may be used to provide inputs for CUAs examining the value of treatments that are associated with weight loss in patients with type 2 diabetes and obesity in Japan.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"370-380"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139722987","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Deborah R Kaye, Ibrahim Khilfeh, Erik Muser, Laura Morrison, Frederic Kinkead, Patrick Lefebvre, Dominic Pilon, Daniel George
{"title":"Characterizing the real-world economic burden of metastatic castration-sensitive prostate cancer in the United States.","authors":"Deborah R Kaye, Ibrahim Khilfeh, Erik Muser, Laura Morrison, Frederic Kinkead, Patrick Lefebvre, Dominic Pilon, Daniel George","doi":"10.1080/13696998.2024.2323901","DOIUrl":"10.1080/13696998.2024.2323901","url":null,"abstract":"<p><strong>Aims: </strong>To describe healthcare resource utilization (HRU) and costs of patients with metastatic castration-sensitive prostate cancer (mCSPC).</p><p><strong>Methods: </strong>Linked data from Flatiron Metastatic PC Core Registry and Komodo's Healthcare Map were evaluated (01/2016-12/2021). Patients with chart-confirmed diagnoses for metastatic PC without confirmed castration resistance in Flatiron who initiated androgen deprivation therapy (ADT) monotherapy or advanced therapy for mCSPC in 2017 or later (index date) with a corresponding pharmacy or medical claim in Komodo Health were included. Advanced therapies considered were androgen-receptor signaling inhibitors, chemotherapies, estrogens, immunotherapies, poly ADP-ribose polymerase inhibitors, and radiopharmaceuticals. Patients with <12 months of continuous insurance eligibility before index were excluded. Per-patient-per-month (PPPM) all-cause and PC-related HRU and costs (medical and pharmacy; from a payer's perspective in 2022 $USD) were described in the 12-month baseline period and follow-up period (from the index date to castration resistance, end of continuous insurance eligibility, end of data availability, or death).</p><p><strong>Results: </strong>Of 871 patients included (mean age: 70.6 years), 52% initiated ADT monotherapy as their index treatment without documented advanced therapy use. During baseline, 31% of patients had a PC-related inpatient admission and 94% had a PC-related outpatient visit; mean all-cause costs were $2551 PPPM and PC-related costs were $839 PPPM with $787 PPPM attributable to medical costs. Patients had a mean follow-up of 15 months, during which 38% had a PC-related inpatient admission and 98% had a PC-related outpatient visit; mean all-cause costs were $5950 PPPM with PC-related total costs of $4363 PPPM, including medical costs of $2012 PPPM.</p><p><strong>Limitations: </strong>All analyses were descriptive; statistical testing was not performed. Treatment effectiveness and clinical outcomes were not assessed.</p><p><strong>Conclusion: </strong>This real-world study demonstrated a significant economic burden in mCSPC patients, and a propensity to use ADT monotherapy in clinical practice despite the availability and guideline recommendations of advanced life-prolonging therapies.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"381-391"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139990264","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Correction.","authors":"","doi":"10.1080/13696998.2024.2402139","DOIUrl":"https://doi.org/10.1080/13696998.2024.2402139","url":null,"abstract":"","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"27 sup3","pages":"34-35"},"PeriodicalIF":2.9,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142289326","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sara Olofsson, Sofia Löfvendahl, Julia Widén, Mattias Rudebeck, Peter Lindgren, Karolina M Stepien, Jean-Baptiste Arnoux, Maria Luz Couce Pico, Elisa Leão Teles, Lena Jacobson
{"title":"Societal costs and quality of life associated with arginase 1 deficiency in a European setting - a multinational, cross-sectional survey.","authors":"Sara Olofsson, Sofia Löfvendahl, Julia Widén, Mattias Rudebeck, Peter Lindgren, Karolina M Stepien, Jean-Baptiste Arnoux, Maria Luz Couce Pico, Elisa Leão Teles, Lena Jacobson","doi":"10.1080/13696998.2024.2400856","DOIUrl":"10.1080/13696998.2024.2400856","url":null,"abstract":"<p><strong>Background and aims: </strong>Arginase 1 deficiency (ARG1-D) is a ultrarare disease with manifestations that cause mobility and cognitive impairment that progress over time and may lead to early mortality. Diseases such as ARG1-D have a major impact also outside of the health care sector and the aim of this study was to estimate the current burden of disease associated with ARG1-D from a societal perspective.</p><p><strong>Methods: </strong>The study was performed as a web-based survey of patients with ARG1-D and their caregivers in four European countries (France, Portugal, Spain, United Kingdom). The survey was distributed at participating clinics and included questions on e.g. symptoms (including the Gross Motor Function Classification System, GMFCS, and cognitive impairment), health care use, medication, ability to work, caregiving, and impact on health-related quality-of-life (HRQoL) using the EQ-5D-5L.</p><p><strong>Results: </strong>The estimated total mean societal cost per patient and year was £63,775 (SD: £49,944). The cost varied significantly with both mobility impairment (from £49,809 for GMFCS level 1 to £103,639 for GMFCS levels 3-5) and cognitive impairment (from £43,860 for mild level to £99,162 for severe level). The mean utility score on the EQ-5D-5L for patients was 0.498 (SD: 0.352). The utility score also varied significantly with both mobility impairment (from 0.783 for GMFCS level 1 to 0.153 for GMFCS level 3-5) and cognitive impairment (from 0.738 for mild level to 0.364 for severe level).</p><p><strong>Conclusions: </strong>Similar to other studies of rare diseases, the study is based on a limited number of observations. However, the sample appear to be reasonably representative when comparing to previous studies of ARG1-D. This study shows that ARG1-D is associated with a high societal cost and significant impact on HRQoL. Earlier diagnosis and better treatment options that can postpone or withhold progression may therefore have a potential for improved HRQoL and savings for the patient, caregiver, and society.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"1146-1156"},"PeriodicalIF":2.9,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142125961","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}