Aims Haemophilia A patients with factor VIII inhibitors (HAPI) experience frequent spontaneous bleeding, approximately once a week, and require expensive bypassing agent (BPA) treatments to control bleeding over their lifetime. According to the HAVEN 1 trial, weekly emicizumab (Hemlibra®) prophylaxis injection reduces annualized bleeding rates (ABR) by 87% compared with BPA on‐demand treatment (BPA‐OD) administered at the time of bleeding. Our study aimed to assess the cost‐effectiveness of emicizumab prophylaxis in HAPI in Korea. Methods Using a lifetime Markov model with health states of ‘alive with bleeds’ and ‘dead’, we simulated the experience of HAPI receiving emicizumab prophylaxis (treatment arm) or BPA‐OD (control arm) and estimated expected clinical and economic outcomes under each treatment arm. Model parameters included comparative effectiveness, clinical and epidemiologic characteristics of Korean HAPI, costs of drug treatment and medical events and utility for ‘alive with bleeds’ state under each treatment. We utilized local data, including National Health Insurance claims data, national statistics, literature and expert surveys with haematologists. Results Base‐case analysis results showed that compared with BPA‐OD, lifetime emicizumab prophylaxis prevented 807 bleedings, extended 3.04 quality‐adjusted life‐years and reduced costs by 2.6 million US dollars. Thus, emicizumab prophylaxis is a dominant treatment option with better effectiveness and lower costs than BPA‐OD. A series of one‐way sensitivity analyses consistently showed dominant results, confirming that lifetime emicizumab prophylaxis is a cost‐saving intervention for HAPI. Conclusion Emicizumab prophylaxis is an excellent treatment choice reducing ABR, improving quality of life and reducing costs.
Background The concurrent use of anticholinergics and acetylcholinesterase inhibitors (ACHEIs) in Parkinson’s disease (PD) patients with dementia should be avoided because the opposing pharmacological actions of both drugs reduce the treatment efficacy. We aimed to investigate the prevalence of the concurrent use of these two types of drugs in Korean patients. Methods In the 2017 Health Insurance Review and Assessment Service–National Aged Patient Sample data, comprising insurance claims records for a 10% random sample of patients aged ≥ 65 years in Korea, “concurrent use” was defined as the overlapping of anticholinergic and ACHEI doses for at least 2 months. Results Among 8,845 PD patients with dementia, 847 (9.58%) were co-administered anticholinergics, used to treat the motor symptoms of PD, and ACHEIs for a mean duration of 7.7 months. A total of 286 (33.77% of all co-administered) patients used both drug types concurrently all year. About 80% of concurrent users were prescribed each drug by the same prescriber, indicating that coadministration may not be due to a lack of information sharing between providers. Logistic regression analysis showed that patients mainly treated at clinics (odds ratio (OR), 1.541; 95% confidence interval (CI), 1.158–2.059), hospitals (OR, 2.135; 95% CI, 1.586–2.883), and general hospitals (OR, 1.568; 95% CI, 1.221–2.028) were more likely to be co-prescribed anticholinergics and ACHEIs than those mainly treated at tertiary-care hospitals. PD patients with dementia treated at healthcare organizations located in areas other than the capital city had an approximately 22% higher risk of concurrent use (OR: 1.227, 95% CI: 1.046–1.441). Conclusions The concurrent use of anticholinergics for the motor symptoms of PD and ACHEIs in elderly Korean PD patients with dementia cannot be ignored, and strategies that mitigate potentially inappropriate concurrent drug use are required.
European countries. Methods: This was a cross-sectional study carried out in the United Kingdom, France and Germany. Data was collected from July 2015 to November 2015. Healthcare costs (hospitalizations, emergencies, medical tests, drugs consumption, visits to GPs and specialists, health material and healthcare transport) and non-healthcare costs (social services and informal care) were identified and valued (reference year: 2014). EQ-5D instrument, Barthel index and Zarit score were also used to reflect the burden and the social impact of the disease beyond the healthcare cost. Results: 86 children with SMA were included, 26.7% of them were Type I and 73.3% were Type II or III. The annual average costs associated with SMA reached 54,295V in UK, 32,042V in France and 52,985V in Germany. Direct non-healthcare costs ranged between 65%-80% of the total cost associated with SMA disease in the three countries analysed. More precisely, the cost associated with informal care was 40,526V (74% of the total cost) for UK, 26,619V (80% of the total cost) for France and 39,926V (77% of the total cost) for Germany. Regarding HRQOL, it was observed that in France and United Kingdom children have a very poor quality of life (0.11 and 0.16, respectively, using time trade-off (TTO) utility score). On contrary, German children had a significantly better quality of life with a TTO of 0.53. Conclusions: SMA represents a considerably high socioeconomic impact both in terms of healthcare and social costs and in reduced HRQOL of children affected. Our analysis may help to design more efficient and equitable policies, with special emphasis on the need to increase the resources and the support provided to the families.
Objectives: The objective of this analysis was to examine patient-reported productivity losses for Charcot-Marie-Tooth disease (CMT) in EU and US real-world practice. Methods: Adults with CMT were recruited to a two-year international observational study exploring the real-world impact of the disease. Data were collected via CMT&Me, a 'bring your own device' app specifically developed for this study, through which participants were asked questions about demographic and employment variables. This interim analysis examined the impact of CMT on ability to work and study in participants from Germany, Italy, Spain, the UK and the US. Results: Of participants who responded, just over half (52%) reported that they were currently working for pay. Twenty-one percent of all participants reported not working due to a disabled status. A further 7% were unemployed, of whom 62% reported that CMT was a contributing factor. Of those working for pay, 77% reported that their work life was affected by CMT. The highest rates were in the UK (81%), while the lowest were in Italy and Spain (both 74%). The most frequently reported ways in which work life was affected by CMT were type of job (58%), number of days taken as sick leave (31%), and working part-time (28%). Participants reported missing a mean of 1.08 days of work in the past two weeks due to their condition. Of the 5% of participants who reported that they were studying, 74% reported that their study life was affected by CMT. The most frequently reported ways in which study life was affected were choice of school/college/university, and Other (both 52%). Conclusions: CMT is associated with substantial productivity losses, which are comparable across EU countries and the US. Further research is needed to explore the specific indirect costs associated with these productivity losses, and to better manage CMT's impact on patients' work/ study lives.
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