Cost-Effectiveness of Biological Asthma Treatments: A Systematic Review and Recommendations for Future Economic Evaluations.

BACKGROUND: Recently developed asthma biological therapies have been shown to provide relief for severe asthma patients not controlled by inhaled treatment. Given the relatively high costs of biological therapies, cost-effectiveness analyses (CEAs) may be required as a prerequisite for coverage and reimbursement. OBJECTIVE: We aimed to systematically review published literature on the economic impact of biological asthma therapies and to identify key drivers that impact cost-effectiveness in order to provide recommendations for future economic evaluations. METHODS: We conducted a systematic literature search in PubMed and Google Scholar. We included studies that assessed the cost-effectiveness of asthma biologics and were published in English between 2000 and 2018. The Quality of Health Economic Studies (QHES) instrument was used to evaluate quality. RESULTS: Twenty asthma biological CEAs were identified. Nineteen studies analyzed the cost-effectiveness of omalizumab, and one study analyzed mepolizumab. Ten studies concluded that omalizumab was cost-effective in base-case scenarios, four studies concluded omalizumab was not cost-effective, and the remaining studies concluded omalizumab or mepolizumab was cost-effective only when targeted to specific severe subgroups or given considerable price discounts. Key drivers of cost-effectiveness included day-to-day health-related quality of life (HRQoL), asthma-related mortality, acquisition price of the biological therapy, and time horizon. CONCLUSIONS: Most studies recommended carefully targeting biological therapy to specific populations such as responders or discounting acquisition price in order to further improve value. The quality of the studies was generally satisfactory, but improved evidence is needed linking HRQoL to utilities as well as understanding interventions' impact on asthma-related mortality. Key recommendations from this review may allow for greater comparability across future cost-effectiveness studies.

Availability, use, and affordability of medicines in urban China under universal health coverage: an empirical study in Hangzhou and Baoji.

BACKGROUND: This study aimed to examine the availability, use, and affordability of medicines in urban China following the 2009 Health Care System Reform that included implementation of universal health coverage (UHC). METHODS: This longitudinal study was performed in Hangzhou (high income, eastern China) and Baoji (lower income, western China). Five yearly household surveys were conducted (one each year from 2009 to 2013) to evaluate the impact of UHC on medicines use and expenditure, and a health facility survey was conducted in 2013 to evaluate availability of medicines. A cohort of over 800 households in Hangzhou and Baoji was established in 2009, and 20 hospitals were included in the health facility survey. Medicines use was determined using data from health facility and household surveys. An average, two-week out-of-pocket medicines expenditure was calculated to assess the affordability of medicines. RESULTS: The number of medicines stocked in primary health facilities in Hangzhou decreased, while the number in Baoji increased. In Baoji, patients usually chose a pharmacy to buy medicines directly, despite the 48.2% increased availability of essential medicines in primary health care centers. The majority of survey respondents stated that their medicines need was basically met; however, medicines cost still accounted for a major part of their health expenditure. Medicines expenditure showed an increasing trend from 2009 to 2013. The average annual growth rate of household overall medical expenditure was significantly higher than that for household non-food consumption expenditure. CONCLUSIONS: Following China's Health Care System Reform and implementation of UHC, availability and use of medicines has improved in urban areas. However, the affordability of medicines is still a concern.

Systematic Review and Quality Appraisal of Cost-Effectiveness Analyses of Pharmacologic Maintenance Treatment for Chronic Obstructive Pulmonary Disease: Methodological Considerations and Recommendations.

BACKGROUND: Worldwide, chronic obstructive pulmonary disease (COPD) is a highly prevalent chronic lung disease with considerable clinical and socioeconomic impact. Pharmacologic maintenance drugs (such as bronchodilators and inhaled corticosteroids) play an important role in the treatment of COPD. The cost effectiveness of these treatments has been frequently assessed, but studies to date have largely neglected the impact of treatment sequence and the exact stage of disease in which the drugs are used in real life. OBJECTIVE: We aimed to systematically review recently published articles that reported the cost effectiveness of COPD maintenance treatments, with a focus on key findings, quality and methodological issues. METHODS: We performed a systematic literature search in Embase, PubMed, the UK NHS Economic Evaluation Database (NHS-EED) and EURONHEED (European Network of Health Economics Evaluation Databases) and included all relevant articles published between 2011 and 2015 in either Dutch, English or German. Main study characteristics, methods and outcomes were extracted and critically assessed. The Quality of Health Economic Studies (QHES) instrument was used as basis for quality assessment, but additional items were also addressed. RESULTS: The search identified 18 recent pharmacoeconomic analyses of COPD maintenance treatments. Papers reported the cost effectiveness of long-acting muscarinic antagonist (LAMA) monotherapy (n = 6), phosphodiesterase (PDE)-4 inhibitors (n = 4), long-acting beta agonist/inhaled corticosteroid (LABA/ICS) combinations (n = 4), LABA monotherapy (n = 2) and LABA/LAMA combinations (n = 2). All but two studies were funded by the manufacturer, and all studies indicated favourable cost effectiveness; however, the number of quality-adjusted life-years (QALYs) gained was small. Less than half of the studies reported a COPD-specific outcome in addition to a generic outcome (mostly QALYs). Exacerbation and mortality rates were found to be the main drivers of cost effectiveness. According to the QHES, the quality of the studies was generally sufficient, but additional assessment revealed that most studies poorly represented the cost effectiveness of real-life medication use. CONCLUSIONS: The majority of studies showed that pharmacologic COPD maintenance treatment is cost effective, but most studies poorly reflected real-life drug use. Consistent and COPD-specific methodology is recommended.