Cost-effectiveness models for Alzheimer's disease and related dementias: IPECAD modeling workshop cross-comparison challenge.

INTRODUCTION: The credibility of model-based economic evaluations of Alzheimer's disease (AD) interventions is central to appropriate decision-making in a policy context. We report on the International PharmacoEconomic Collaboration on Alzheimer's Disease (IPECAD) Modeling Workshop Challenge. METHODS: Two common benchmark scenarios, for the hypothetical treatment of AD mild cognitive impairment (MCI) and mild dementia, were developed jointly by 29 participants. Model outcomes were summarized, and cross-comparisons were discussed during a structured workshop. RESULTS: A broad concordance was established among participants. Mean 10-year restricted survival and time in MCI in the control group ranged across 10 MCI models from 6.7 to 9.5 years and 3.4 to 5.6 years, respectively; and across 4 mild dementia models from 5.4 to 7.9 years (survival) and 1.5 to 4.2 years (mild dementia). DISCUSSION: The model comparison increased our understanding of methods, data used, and disease progression. We established a collaboration framework to assess cost-effectiveness outcomes, an important step toward transparent and credible AD models.

Impact of sleep disturbances on employment and work productivity among midlife women in the US SWAN database: a brief report.

OBJECTIVE: Menopause is associated with an increased prevalence of sleep difficulties. We evaluated the economic burden of sleep disturbances among working midlife women. METHODS: This retrospective, longitudinal cohort study collected data from the US Study of Women's Health Across the Nation (SWAN) database of women age 42-52 years at enrollment. We assessed the association between sleep disturbances (trouble falling asleep, waking early, or nocturnal awakenings) and workplace productivity (employment [yes/no] and work hours/wk) for women who were employed at the baseline visit and had ≥1 follow-up visit. We estimated overall economic burden by multiplying changes in productivity by median age-specific hourly US wages. Each woman's data were compared from visit to visit and were excluded after the first observed unemployment. Regression analysis was used to estimate associations between changes in sleep and changes in workplace productivity while controlling for relevant characteristics that varied over time. RESULTS: The analysis included 2,489 working women (19,707 visits); 31% became unemployed during follow-up. Risk of unemployment was 31% higher for women with versus without new-onset sleep disturbances (P  = 0.0474). Onset of sleep disturbances was associated with a 0.44-0.57 hours/wk reduction in work time (not significant). Using the more conservative reduction (0.44 h), sleep problems were associated with an annual loss of $517 to $524 per woman and $2.2 billion/yr in lost productivity among women age 42-64 nationwide. CONCLUSIONS: New-onset sleep problems in midlife women are associated with significant increases in risk of unemployment and ∼$2 billion/yr in lost productivity nationwide.

Video Summary : http://links.lww.com/MENO/A798 .

A Model to Assess the Outcomes Associated With Dementia With Lewy Bodies.

BACKGROUND: Research investigating outcomes associated with dementia with Lewy bodies (DLB) disease progression is scarce. Developing models of DLB disease progression will provide information on the burden of disease and facilitate the evaluation of treatments for DLB from a clinical and cost perspective. METHODS: Longitudinal, cognitive evaluation data were utilized in order to identify distinct health states for DLB and to estimate transition probabilities across the DLB disease continuum. These probabilities were applied to a health state transition model to evaluate disease progression and associated outcomes for a closed cohort over a fixed time horizon. The effect of a reduction in the risk of disease progression on outcomes was assessed. RESULTS: Estimated transition probabilities indicate that a patient >60 years of age with mild DLB has a 54%, 30%, 4%, and 12% chance of remaining mild, progressing to severe DLB, being institutionalized, and dying after 1 year, respectively. Reducing the annual risk of transitioning from mild to severe DLB by 40% decreased time institutionalized and increased time to death. CONCLUSIONS: This study used real-world longitudinal data to create a clinically relevant DLB disease progression model. Reducing the rate of disease progression resulted in meaningful benefits with potentially significant public health implications.

Direct Medical Costs of Dementia With Lewy Bodies by Disease Complexity.

OBJECTIVES: There is currently no accurate profile of the economic burden of dementia with Lewy bodies (DLB), particularly any examination of the direct medical costs of DLB by the number of affected clinical domains. Understanding how trends in the use of healthcare resources evolve as DLB progresses presents opportunities for the development of earlier and more appropriate interventions. DESIGN: Retrospective study using claims data extracted from the IBM MarketScan Commercial and Medicare Supplemental database. SETTING AND PARTICIPANTS: In total, 536 patients with DLB from the Commercial database and 5485 patients with DLB from the Medicare Supplemental database. METHODS: Patients were grouped into disease complexity categories based on core clinical features (ie, fluctuating cognition, motor symptoms, visual hallucinations, and rapid eye movement sleep behavior disorder in addition to dementia) observed during the study period: dementia with no core features observed, dementia plus 1, 2, or ≥3 core features, respectively. Outcome measures included healthcare resource utilization and healthcare costs. RESULTS: In both databases, total healthcare resource utilization and costs increased with number of core features. Compared with patients with no core features observed, patients in all other complexity categories had significantly higher mean medical visits and costs in both adjusted and unadjusted analyses. Fluctuating cognition was associated with the highest total costs, suggesting that this clinical feature in particular is associated with a considerable economic burden. CONCLUSIONS AND IMPLICATIONS: Analyzing direct medical costs of DLB by disease complexity using claims data showed that a higher cost impact was associated with increasing number of clinical domains affected and with specific clinical domains, suggesting the need for both targeted and comprehensive interventions to improve the overall economic burden of DLB.

Cost Effectiveness of Mirabegron Compared with Antimuscarinic Agents for the Treatment of Adults with Overactive Bladder in Colombia.

OBJECTIVES: The aim of this study was to evaluate the cost effectiveness of mirabegron relative to two antimuscarinics, oxybutynin extended release (ER) and tolterodine ER, in patients with overactive bladder (OAB) from the perspective of a third-party payer in Colombia. METHODS: A Markov model simulated the therapeutic management, disease course, and complications in hypothetical cohorts of OAB patients over a 5-year period. The model predicted costs and three outcomes: quality-adjusted life-years (QALYs), micturition state improvement (MSI), and incontinence state improvement (ISI). In each 1-month cycle, patients could transition between different health states reflecting symptom severity. Transition probabilities were estimated from a published mirabegron trial and mixed treatment comparison. Other inputs such as treatment discontinuation based on treatment-specific rates of persistence, resource use and costs, anticholinergic burden, comorbidity treatment, and drug acquisition were obtained from Società Italiana Scienze Mediche, Instituto de Seguros Sociales Tariff Manual, published literature, and expert opinion. Deterministic and probabilistic sensitivity analyses were conducted. Costs are presented in 2017 Colombia Pesos (COP). RESULTS: Mirabegron was cost effective for all outcome measures at a willingness-to-pay threshold of 124,919,725 COP, which is three times the per capita gross domestic product (GDP). Using QALYs as the measure of effect, mirabegron had an incremental cost-effectiveness ratio (ICER) of 85,802,036 COP/QALY (26,365 USD/QALY) and 66,360,134 COP/QALY (20,384 USD/QALY) versus oxybutynin and tolterodine, respectively. Probabilistic sensitivity analyses showed that mirabegron was cost effective in 99.5% and 100% of simulations compared with oxybutynin and tolterodine, respectively. Using MSI and ISI as the measure of effects yielded ICERs below one GDP. CONCLUSIONS: Mirabegron is a cost effective alternative to oxybutynin and tolterodine from the perspective of a third-party payer in Colombia.

The impact of persistence with mirabegron usage vs switching to onabotulinumtoxinA on healthcare costs and resource utilization in patients with overactive bladder in the United States.

AIMS: To compare healthcare costs and resource utilization in patients with overactive bladder (OAB) in the US who switch from mirabegron to onabotulinumtoxinA (onabotA) with those who persist on mirabegron. MATERIALS AND METHODS: A retrospective observational claims analysis of the OptumHealth Administrative Claims database conducted between April 1, 2012 and September 30, 2015 used medical and pharmacy claims to identify patients with at least one OAB diagnosis who switched from mirabegron to onabotA (onabotA group) or persisted on mirabegron for at least 180 days (mirabegron persisters). Propensity score weighting was used to balance baseline characteristics that were associated with increased healthcare expenditures across treatment groups. Multivariate analyses assessed the impact of switching and persistence on all-cause and OAB-related healthcare costs and resource utilization in the year following each patient's index date. RESULTS: In total, 449 patients were included in this study: 54 patients were included in the onabotA group, and 395 patients were included in the mirabegron persister group. Compared with the mirabegron persister patients, the onabotA patients observed significantly higher OAB-related total costs ($5,504 vs $1,772, p < .001), OAB-related medical costs ($5,033 vs $351, p < .001), sacral neuromodulation costs ($865 vs $60, p = .017), and outpatient costs ($17,385 vs $9,035, p = .009), and more OAB-related medical visits (6.0 vs 1.9, p < .001). OnabotA patients had lower OAB-related prescription costs ($470 vs $1,421, p < .001) and fewer OAB-related pharmacy claims (1.6 vs 5.0, p <.001). There were no significant differences in all-cause total medical or prescription costs. LIMITATIONS: This study was a retrospective analysis using claims data that only included patients with commercial health coverage or Medicare supplemental coverage. Accuracy of the diagnosis codes and the generalizability of the results to other OAB populations are limited. The study was not designed to determine the impact of OAB treatments on the economic outcomes examined. CONCLUSIONS: OAB patients who persisted on mirabegron treatment for at least 180 days had lower OAB-related healthcare costs and resource utilization compared with those who switched to onabotA.