model with incremental cost-effectiveness ratios or quality-adjusted life-years as outcome measures. Specific information on the decision-analytic models, including the modeling approach, perspective, population studied, interventions and data sources were extracted from each article. RESULTS: 1834 citations were screened and 68 full-text articles retrieved. Overall, 31 model-based studies were identified for extraction: 29 cohort-based state-transition models, one decision tree model, and one other cost-effectiveness model. Comparators included drugs such as dopamine agonists, entacapone, and rasagaline, as well as surgically implanted devices such as deep brain stimulation and levodopa-carbidopa intestinal gel. Overall, 10 modeled only early PD, 7 focused on advanced PD, 10 included early and late stage PD, and 4 did not specify stage. The most commonly modeled health outcomes were Hoehn and Yahr stage progression and % off-time. Other outcomes include motor complications, fluctuations, dyskinesia, falls, and dementia. CONCLUSIONS: The models identified typically had simple frameworks. Many projected disease progression from short-term clinical trial data alone and did not leverage real world observational data now available with longer longitudinal follow-up. Clinical efficacy was often applied by affecting only the initial distribution across health states, which likely does not fully capture the benefits of a treatment. Future studies should explore developing individual patient simulations to be able to more realistically represent the heterogeneity observed in the clinical manifestations and progression rates of the disease, as well as capture the potential benefits and risks of symptomatic or disease modifying treatments.
OBJECTIVES: To characterize the functional, emotional and social impact of migraine in Portugal. METHODS: A worldwide, cross-sectional, online survey was conducted including migraine patients recruited via online panels and patient organizations from September 2017-February 2018. Study participants were adults reporting 4 or more monthly migraine days over the 3 months previous to survey with a pre-determined quota of 90% participants having received previous preventive migraine treatment. Results from the Portuguese sample are reported. RESULTS: A total of 143 migraine patients were included, 79.7% women and mean age of 37.3 years (SD 12.6; range 18-72). About 79.0% of participants mentioned feeling very or extremely limited in completing daily activities during the migraine attack. Indeed, 71.3% participants rely on support of family/friends to perform daily activities (average 11.8 hours in previous 3 months; SD 18.9; range 1-90). 39.2% of participants have often/always "no energy to complete daily living tasks or feel fatigued". Most common negative feelings perceived by participants were lack of others' understanding about their pain (53.9%), feeling helpless (42.7%) and depressed (40.0%). Nevertheless, 37.1% mentioned having learned to cope with the disease. Most patients (62.9%) claimed that migraine has changed their relationships with relatives/friends/partner and 81.1% of participants also mentioned that migraine has affected their social life and activities (48.3% cannot take part in usual activities/hobbies, 47.4% stopped social events). In the previous month, 80.4% of participants had to cancel plans due to migraine. As an overall consequence, 39.9% of patients mentioned being very/extremely fearful regarding next migraine attack. CONCLUSIONS: This study confirms that migraine is associated with functional impairment requiring support from others and a clear emotional and social impact in patients' lives.
Background Patients with highly active relapsing-remitting multiple sclerosis inadequately responding to first-line therapies (interferon-based therapies, glatiramer acetate, dimethyl fumarate, and teriflunomide, known collectively as "BRACETD") often switch to natalizumab or fingolimod. Objective The aim was to estimate the comparative effectiveness of switching to natalizumab or fingolimod or within BRACETD using real-world data and to evaluate the cost-effectiveness of switching to natalizumab versus fingolimod using a United Kingdom (UK) third-party payer perspective. Methods Real-world data were obtained from MSBase for patients relapsing on BRACETD in the year before switching to natalizumab or fingolimod or within BRACETD. Three-way-multinomial-propensity-score-matched cohorts were identified, and comparisons between treatment groups were conducted for annualised relapse rate (ARR) and 6-month-confirmed disability worsening (CDW6M) and improvement (CDI6M). Results were applied in a cost-effectiveness model over a lifetime horizon using a published Markov structure with health states based on the Expanded Disability Status Scale. Other model parameters were obtained from the UK MS Survey 2015, published literature, and publicly available UK sources.
ResultsThe MSBase analysis found a significant reduction in ARR (rate ratio [RR] = 0.64; 95% confidence interval [CI] 0.57-0.72; p < 0.001) and an increase in CDI6M (hazard ratio [HR] = 1.67; 95% CI 1.30-2.15; p < 0.001) for switching to natalizumab compared with BRACETD. For switching to fingolimod, the reduction in ARR (RR = 0.91; 95% CI 0.81-1.03; p = 0.133) and increase in CDI6M (HR = 1.30; 95% CI 0.99-1.72; p = 0.058) compared with BRACETD were not significant. Switching to natalizumab was associated with a significant reduction in ARR (RR = 0.70; 95% CI 0.62-0.79; p < 0.001) and an increase in CDI6M (HR = 1.28; 95% CI 1.01-1.62; p = 0.040) compared to switching to fingolimod. No evidence of difference in CDW6M was found between treatment groups. Natalizumab dominated (higher quality-adjusted life-years [QALYs] and lower costs) fingolimod in the base-case cost-effectiveness analysis (0.453 higher QALYs and £20,843 lower costs per patient). Results were consistent across sensitivity analyses.
to disability progression rate. ConClusions: The incremental cost effectiveness ratio of 17.433 € is considerably below the threshold usually accepted for financing medicines in Portugal (around 30.000 € /QALY). Dimethyl Fumarate should be seen as a cost-effective therapy for the Portuguese setting.
PND57Cost-EffECtivENEss ANAlysis of PEgiNtErfEroN BEtA-1A iN itAliAN rElAPsiNg rEmittiNg multiPlE sClErosis mANAgEmENt
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