The Impact of Eliminating Out-of-Pocket Payments on Asthma Medication Use.

BACKGROUND: High costs of controller therapies may be a barrier to guideline-recommended asthma treatment. We determined whether eliminating out-of-pocket (OOP) payments among low-income patients with asthma impacted controller medication use. METHODS: We applied a controlled interrupted time series design to administrative claims data in British Columbia, Canada from 2017-2020. Cases were individuals with an annual household income <$13,750 in whom copays were eliminated on January 2019; there was no change in public coverage for the control group with annual income >$45,000. We evaluated trends in asthma medication costs, use, the ratio of inhaled corticosteroid (ICS)-containing medications to all asthma medications, excessive use of short-acting ß-agonists (SABA) (>1 canister/month), and the proportion of days (PDC) covered by controller therapies. RESULTS: There were 12,940 cases (62% female, mean age 30.3 years, SD 14.9), and 71,331 controls (55% female, mean age of 31.3 years, SD 16.3). Removal of OOP payments increased monthly mean medication costs by $3.32 (95% CI $0.08 - $6.56, 2020 Canadian dollars), days supply of controller medications by 1.50 days (95% CI 0.61 - 2.40), and the ratio of ICS-containing medications to total medications by 4.20% (95% CI 0.73% - 7.66%) compared to the control group. The policy had no effect on PDC by controller therapies (0.01, 95% CI -0.01 - 0.04), but non-significantly decreased the percentage of patients with excessive SABA use (-6.37%; 95% CI -12.90% - 0.16%). INTERPRETATION: Removal of OOP payments increased the dispensation of controller therapies, suggesting cost-related non-adherence could impair optimal asthma management.

Benefit-Harm Analysis of Earlier Initiation of Triple Therapy for Prevention of Acute Exacerbation in Patients with Chronic Obstructive Pulmonary Disease.

Rationale: Reducing the risk of exacerbation is a fundamental goal in managing stable chronic obstructive pulmonary disease (COPD). Guidelines recommend triple therapy (inhaled corticosteroids, long-acting muscarinic antagonists, and long-acting ß-agonists) only as a stepup from dual therapy (long-acting muscarinic antagonists and long-acting ß-agonists) for patients at continued high risk of exacerbation, because of the trade-off of an increased risk of pneumonia associated with inhaled corticosteroid-containing therapies. However, there is little evidence on the optimum timing of initiating triple therapy. Objectives: To perform a benefit-harm analysis to evaluate the net benefit of earlier initiation of triple therapy for the prevention of acute exacerbations in patients with COPD compared with standard timing recommended in current guidelines. Methods: We used a validated whole-disease microsimulation model of COPD in the Canadian general population aged ⩾40 years to determine the benefit versus harm of earlier initiation of triple therapy over a 20-year time horizon compared with standard care. We assessed net change in quality-adjusted life-years (QALYs) from the reduction in risk of acute exacerbations and the increased risk of treatment-related pneumonia in subgroups of patients with COPD defined by exacerbation history, symptoms, and disease severity. Model parameters were determined from clinical trials and other published literature. Key parameters were varied in one-way sensitivity analysis. Results: In patients at high risk of acute exacerbation (54.7% female; mean age, 74.0 yr; 68% Global Initiative for Chronic Obstructive Lung Disease grades I and II), earlier initiation of triple therapy was associated with a net QALY gain of 4.8 per 100 patients with COPD over 20 years compared with standard care. The net QALY gain increased to 5.9 per 100 patients in the subgroup of patients with a high symptom burden (modified Medical Research Council dyspnea scale score, >1). Earlier initiation remained net beneficial in all subgroup and sensitivity analysis scenarios. Conclusions: Modeling suggests that earlier initiation of triple therapy is likely to be net beneficial for patients at high risk of acute exacerbation, with an even greater benefit to patients with a high symptom burden. Further clinical research is needed to verify these findings in empirical studies.

Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis.

BACKGROUND: Sickle cell disease (SCD) and its complications contribute to high rates of morbidity and early mortality and high cost in the United States and African heritage community. OBJECTIVE: To evaluate the cost-effectiveness of gene therapy for SCD and its value-based prices (VBPs). DESIGN: Comparative modeling analysis across 2 independently developed simulation models (University of Washington Model for Economic Analysis of Sickle Cell Cure [UW-MEASURE] and Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model [FH-HISCORE]) using the same databases. DATA SOURCES: Centers for Medicare & Medicaid Services claims data, 2008 to 2016; published literature. TARGET POPULATION: Persons eligible for gene therapy. TIME HORIZON: Lifetime. PERSPECTIVE: U.S. health care sector and societal. INTERVENTION: Gene therapy versus common care. OUTCOME MEASURES: Incremental cost-effectiveness ratios (ICERs), equity-informed VBPs, and price acceptability curves. RESULTS OF BASE-CASE ANALYSIS: At an assumed $2 million price for gene therapy, UW-MEASURE and FH-HISCORE estimated ICERs of $193 000 per QALY and $427 000 per QALY, respectively, under the health care sector perspective. Corresponding estimates from the societal perspective were $126 000 per QALY and $281 000 per QALY. The difference in results between models stemmed primarily from considering a slightly different target population and incorporating the quality-of-life (QOL) effects of splenic sequestration, priapism, and acute chest syndrome in the UW model. From a societal perspective, acceptable (>90% confidence) VBPs ranged from $1 million to $2.5 million depending on the use of alternative effective metrics or equity-informed threshold values. RESULTS OF SENSITIVITY ANALYSIS: Results were sensitive to the costs of myeloablative conditioning before gene therapy, effect on caregiver QOL, and effect of gene therapy on long-term survival. LIMITATION: The short-term effects of gene therapy on vaso-occlusive events were extrapolated from 1 study. CONCLUSION: Gene therapy for SCD below a $2 million price tag is likely to be cost-effective when applying a societal perspective at an equity-informed threshold for cost-effectiveness analysis. PRIMARY FUNDING SOURCE: National Heart, Lung, and Blood Institute.