Cost-Effectiveness and Value-Based Pricing of Aducanumab for Patients With Early Alzheimer Disease.

BACKGROUND AND OBJECTIVES: Aducanumab was granted accelerated approval with a conflicting evidence base, near-unanimous Food and Drug Administration Advisory Committee vote to reject approval, and a widely criticized launch price of $56,000 per year. The objective of this analysis was to estimate its cost-effectiveness. METHODS: We developed a Markov model to compare aducanumab in addition to supportive care to supportive care alone over a lifetime horizon. Results were presented from both the health system and modified societal perspective. The model tracked the severity of disease and the care setting. Incremental cost-effectiveness ratios were calculated and a threshold analysis was conducted to estimate at what price aducanumab would meet commonly used cost-effectiveness thresholds. RESULTS: Using estimates of effectiveness based on pooling of data from both pivotal trials, patients treated with aducanumab spent 4 more months in earlier stages of Alzheimer disease. Over the lifetime time horizon, treating a patient with aducanumab results in 0.154 more quality-adjusted life-years (QALYs) gained per patient and 0.201 equal value of life-years gained (evLYG) per patient from the health care system perspective, with additional costs of approximately $204,000 per patient. The incremental outcomes were similar for the modified societal perspective. At the launch price of $56,000 per year, the cost-effectiveness ranged from $1.02 million per evLYG to $1.33 million per QALY gained from the health care system perspective and from $938,000 per evLYG to $1.27 million per QALY gained in the modified societal perspective. The annual price to meet commonly used cost-effectiveness thresholds ranged from $2,950 to $8,360, which represents a discount of 85%-95% off from the annual launch price set by the manufacturer. Using estimates of effectiveness based only on the trial that suggested a benefit, the mean incremental cost was greater than $400,000 per QALY gained. CONCLUSION: Patients treated with aducanumab received minimal improvements in health outcomes at considerable cost. This resulted in incremental cost-effectiveness ratios that far exceeded commonly used value thresholds, even under optimistic treatment effectiveness assumptions. These findings are subject to the substantial uncertainty regarding whether aducanumab provides any true net health benefit, but evidence available currently suggests that an annual price of aducanumab of $56,000 is not in reasonable alignment with its clinical benefits.

The effectiveness and value of tezepelumab for severe asthma.

DISCLOSURES: Drs Rind, Campbell, Pearson, Ms Herce-Hagiwara, Ms Fluetsch, and Ms Herron-Smith report grants from Arnold Ventures; Kaiser Foundation Health Plan, Inc; The Patrick and Catherine Donaghue Medical Research Foundation; Blue Cross Blue Shield of Massachusetts; and California Healthcare Foundation during the course of this study.

Anti B-cell maturation antigen CAR T-cell and antibody drug conjugate therapy for heavily pretreated relapsed and refractory multiple myeloma.

DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, California Health Care Foundation, The Donaghue Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from AbbVie, Aetna, America's Health Insurance Plans, Anthem, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Evolve Pharmacy, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Humana, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, uniQure, and United Healthcare. Beinfeld, Fluetsch, and Pearson are employed by ICER. Ollendorf received funding from ICER for work on this summary and reports consulting and other personal fees from EMD Serono, Amgen, Analysis Group, Aspen Institute/University of Southern California, GalbraithWight, Cytokinetics, Executive Insight, Sunovion, University of Colorado, World Health Organization, and Eli Lilly, unrelated to this work. Lee and McQueen received funding from ICER for work on this summary.

The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy.

DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, Commonwealth Fund, California Health Care Foundation, National Institute for Health Care Management (NIHCM), New England States Consortium Systems Organization, Blue Cross Blue Shield of Massachusetts, Harvard Pilgrim Health Care, Kaiser Foundation Health Plan, and Partners HealthCare to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, America's Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare. Agboola, Fluetsch, Rind, and Pearson are employed by ICER. Lin reports support from ICER during work on this economic model and grants from Mount Zion Health Fund, National Institutes of Health (National Cancer Institute and National Heart, Lung, and Blood Institute), and the Tobacco-Related Diseases Research Program, unrelated to this work. Walton reports support from ICER for work on this economic model and unrelated consulting fees from Baxter.

Long-Term Opioid Prescriptions After Spine Surgery: A Meta-Analysis of Prevalence and Risk Factors.

BACKGROUND: Opioids are frequently prescribed for back pain, but the prevalence of and risk factors for long-term opioid use after spine surgery were not clearly reported. We conducted a systematic review and meta-analysis to summarize the evidence for long-term opioid use (>90 days) among adults who underwent spine surgery. METHODS: PubMed, EMBASE, and Cochrane indexing databases were searched until November 9, 2018 for studies reporting the prevalence of and risk factors for long-term opioid use after spine surgery. Separate meta-analyses were conducted for commercial claims databases or registries (claims/registries) and nonclaims observational studies using the random-effects model to estimate the pooled odds ratio (OR). Prevalence meta-analysis was performed in a clinically homogeneous subset of these patients who underwent lumbar spine surgery. RESULTS: Eight claims and 5 nonclaims were meta-analyzed to avoid double-counting participants. The meta-analysis showed that preoperative opioid users (OR, 5.59; 95% confidence interval [CI], 3.37-9.27 vs. OR 4.21; 95% CI, 2.72-6.51) and participants with preexisting depression and/or anxiety (OR, 1.86, 95% CI, 1.43-2.42 and OR, 1.20; 95% CI, 0.83-1.74, respectively) had a statistically significantly higher odds of long-term postoperative opioids, compared with their peers. Males showed lower odds of long-term postoperative opioid use in the claims group (OR, 0.85; 95% CI, 0.79-0.92), but not in the nonclaims group (OR, 0.99; 95% CI, 0.71-1.39). The pooled prevalence of post-lumbar spine surgery long-term opioid use was 63% (95% CI, 50%-74%) in claims and 47% (95% CI, 38%-56%) in nonclaims. CONCLUSIONS: Patients undergoing spine surgery represent a high-risk surgical population requiring special attention and targeted interventions, with the strongest evidence for those treated with opioids before surgery and those with psychiatric comorbidities.

The relationship of physical activity to mental health: A 2015 behavioral risk factor surveillance system data analysis.

INTRODUCTION: Available literature has repeatedly ascertained the inverse relationship of physical activity to mental health. Engaging in regular physical activity decreases symptoms of anxiety, depression, and stress in a dose-responsive manner. METHODS: Cross-sectional, national data from the 2015 Behavioral Risk Factor Surveillance System (BRFSS) were analyzed to establish the relationship between physical activity and days of poor mental health. The association between physical activity and days of poor mental health was assessed by multiple linear regression using SAS. RESULTS: Findings support the assumption of an inverse dose-response relationship between physical activity levels and self-reported days of poor mental health. Individuals who engage in higher levels of physical activity reported significantly fewer days of poor mental health when compared to those who reported low or no physical activity (e.g. -2.23, 95%CI (-2.31, -2.15)) for highly active vs. inactive individuals (unadjusted model)). Furthermore, individuals who only met the guidelines for aerobic exercise reported significantly fewer days of poor mental health than those who only met the muscle strengthening guidelines (-0.85 (95% CI: -0.98, -0.73)). LIMITATIONS: Due to the cross-sectional study design, the direction of the relationship is unknown and reverse causation is possible. CONCLUSION: Higher physical activity levels were associated with fewer self-reported days of poor mental health, which reiterates the important association between physical activity and mental health with national data. The findings of this study could prove to be valuable for developing interventions aimed at improving mental health. Nonetheless, several research gaps persist that warrant further empirical inquiry.