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.

Evaluating a Stone of Hope: ICER's 2019 Review of Treatments for Duchenne Muscular Dystrophy.

DISCLOSURES: No funding contributed to the writing of this commentary. Brandsema reports consulting for Alexion, Audentes, AveXis, Biogen, Cytokinetics, PTC Therapeutics, Sarepta, and WaVe and has received research funding as a site investigator from Alexion, AveXis, Biogen, CSL Behring, Cytokinetics, Fibrogen, Pfizer, PTC Therapeutics, Sarepta, Summit, and WaVe.

Cost-utility analysis of the inhaled steroids available in a developing country for the management of pediatric patients with persistent asthma.

INTRODUCTION: The choice among the different treatments available can have a great impact on the costs of asthma, OBJECTIVES: The objective of this study was to estimate the incremental cost-utility ratio of three inhaled corticosteroids (ICs): budesonide (BUD), fluticasone propionate (FP), and ciclesonide, compared to beclomethasone dipropionate (BDP) (the only IC included in the Compulsory Health Insurance Plan of Colombia), METHODS: A Markov-type model was developed to estimate costs and health outcomes of a simulated cohort of patients less than 18 years of age with persistent asthma treated over a 12-month period. Effectiveness parameters were obtained from a systematic review of the literature. Cost data were obtained from a hospital´s bills and from the national manual of drug prices. The study assumed the perspective of the national healthcare in Colombia. The main outcome was the variable "quality-adjusted life years" (QALY), RESULTS: While treatment with BDP was associated with the lowest cost (£106.16 average cost per patient during 12 months), treatment with FP resulted in the greatest gain in QUALYs (0.9325 QALYs). FP was associated with a greater gain in QALYs compared to BUD and ciclesonide (0.9325 vs. 0.8999 and 0.9051 QALYs, respectively) at lower costs (£231.19 vs. £309.27 and £270.15, respectively), thus leading to dominance. The incremental cost-utility ratio of FP compared to BDP was £19,835.28 per QALY, CONCLUSIONS: BDP is the most cost-effective therapy for treating pediatric patients with persistent asthma when willingness to pay (WTP) is less than £21,129.22/QALY, otherwise, FP is the most cost-effective therapy.

Ciclesonide for the treatment of seasonal allergic rhinitis.

Allergic rhinitis is considered one of the most common afflictions of humans, affecting up to 30% of the world's population, and is increasing in incidence. Primary symptoms, comorbid conditions and complications of this disorder exact a significant toll, resulting in an enormous physical, social and economic impact on society. Single-season allergic rhinitis accounts for approximately 20% of cases of allergic rhinitis with another 40% having mixed seasonal-perennial presentations. Management of this disorder encompasses several treatment options, with intranasal corticosteroids recommended as first-line treatment in moderate-to-severe seasonal allergic rhinitis in current practice parameters. Ciclesonide is the most recently approved product in this category for the management of seasonal allergic rhinitis and is the subject of this article.

Efficacy and cost effectiveness of inhaled steroids in asthma in a developing country.

Eighty six children with troublesome wheezing were studied, in a semiprospective clinical trial with the patients acting as their own controls, to assess the efficacy and cost effectiveness of inhaled steroids. Improvement in school attendance, hospitalisations, breakthrough wheezing, and acute severe attacks were used to assess clinical efficacy. Expenditure for the family, on a cost of illness framework, before and after treatment, was used to estimate cost effectiveness. Highly significant numbers of patients showed improvement in clinical parameters, confirming efficacy. Mean monthly cost before inhaled steroid treatment was Rs 2652.33 (36.33 pounds) and Rs 449.42 (6.16 pounds) after starting treatment. The mean cost per unit satisfaction (cost utility value) which was Rs 255.54 (3.50 pounds) before starting prophylaxis came down to Rs 5.42 (0.07 pound) after starting treatment. There are no previous reports of cost-benefit assessment of inhaled steroids in childhood asthma. It is concluded that, even for developing countries with financial constraints, inhaled steroid treatment for prophylaxis of asthma is a cost effective and rational form of treatment.

Budesonide. An appraisal of the basis of its pharmacoeconomic and quality-of-life benefits in asthma.

Budesonide is an inhaled corticosteroid that is used prophylactically to reduce the underlying inflammation and consequent airways narrowing associated with asthma. Widespread clinical experience has shown that inhaled budesonide is effective and well tolerated, and its use is well established in the management of adult and childhood asthma. In developed countries, asthma is a major health problem and consumes a large proportion of healthcare resources. Both the prevalence and severity of asthma appear to be increasing. Additionally, asthma-related mortality has been reported to have gradually increased since the mid- to late-1970s in many countries, possibly due to undertreatment and/or suboptimal management of the disease. Current guidelines recommend a shift away from initial treatment with oral bronchodilators, such as theophylline, or regular use of beta 2-agonist inhalers, toward the earlier use of more expensive inhalers containing corticosteroids. Inhaled bronchodilators are still used as indicated for treatment of acute attacks. Data suggest that the acquisition cost of budesonide is more than offset by decreased morbidity and reductions in costs associated with acute asthma exacerbations. Both once-daily administration and its administration in dry powder form via Turbuhaler appear to be well accepted by patients; these factors may potentially improve patient compliance with therapy. Budesonide appears to have positive effects on some quality-of-life indices, although studies using validated quality-of-life instruments are needed to confirm these conclusions. Modelling studies would be helpful in order to assess the possible economic benefits to society through reduction of the considerable direct and indirect costs of asthma and cost-effectiveness comparisons with other inhaled corticosteroids are needed to clarify its relative positioning in this regard. Until then, the available data provide an encouraging pharmacoeconomic rationale for budesonide as first-line asthma therapy, and a good basis for future pharmacoeconomic analysis of asthma management.