Purpose Measuring quality of life in acute asthmatics is challenging, especially when asthma attacks can occur sporadically. Several questionnaires can be used to measure quality of life in this patient group; however, psychometric testing is limited on questionnaires that can be used to estimate Quality Adjusted Life years. The objective of this study is to assess the construct validity (convergent and discriminative validity) and responsiveness of the EuroQol-5-Dimensions 5-Level (EQ-5D-5L), Asthma Quality of Life Utility Index-5 Dimensions (AQL-5D) and Time Trade-Off (TTO) in acute asthma patients. Methods Data from a prospective cohort study were used to test the validity and responsiveness of the EQ-5D-5L, AQL-5D and TTO in asthma patients who were recruited from UK accident & emergency departments or hospital wards. The spearman's rank correlation coefficient, the Kruskal-Wallis test statistic and the standardized response mean were used to test for convergent validity, discriminative validity and responsiveness, respectively. Results One hundred and twenty-one participants were included in the available case analysis. The EQ-5D-5L and AQL-5D showed moderate to strong correlations for convergent validity at baseline, week 4 and week 8. The AQL-5D and TTO showed moderate correlations at week 4 and week 8. No statistical significance was observed for discriminative validity at baseline. Both the EQ-5D-5L and the AQL-5D also showed that they were sensitive to change for the recovery responses. Conclusions The EQ-5D-5L and AQL-5D showed stronger construct validity and responsiveness compared to the TTO. Therefore, both the EQ-5D-5L and AQL-5D should be considered for use in future economic evaluations.

Background Drug costs are increasing in Europe, and there is a heightened need to reduce pressure on healthcare systems. In 2017, oncology, autoimmune disease, and diabetes featured as the three highest therapy areas for drug spend in the EU-28. However, the absolute 1-year drug spend growth for diabetes did not feature within the ten fastest growing therapy areas. Objective This study explores the association between drug spend and disease burden in oncology, autoimmune disease, and diabetes in the EU-28. Methods Oncology, autoimmune disease and diabetes therapeutic areas were investigated using four methodologies. Historical and forecasted drug spend was analysed using the IQVIA MIDAS ® drug sales database. Clinical and economic burden was estimated from targeted literature reviews. Trend analyses compared changes in drug spend with clinical burden using the Global Burden of Disease tool as the epidemiological reference. Cost per quality-adjusted life-years (QALYs) from UK health technology assessments were compared to interpret the health economic value. Results Oncology had the highest historical drug spend and growth compared with autoimmune disease and diabetes. Total drug spend and growth in oncology is forecasted to exceed diabetes by twofold. Increasing oncology drug spend historically did not correspond with reductions in mortality and morbidity. Diabetes had the lowest drug spend and greatest QALY/€1000 spent benefit. Conclusion This study indicates that drug spend may not correlate to clinical burden across diseases. Future research could stimulate debate on whether more equitable drug funding may improve disease management.

Background Evidence of quality of life implications of asthma attacks are limited, particularly when measured on a utility scale, which enables calculating Quality-Adjusted Life-Years (QALYs) and comparisons with other health conditions and services. Therefore, this study sought to estimate the utility loss associated with an asthma-related crisis event (accident and emergency (A&E) attendance or hospital admission). Methods Participants were recruited in a cohort study from A&E and hospital admissions at three UK hospitals. They completed the EuroQol-5 Dimensions 5-Level (EQ-5D-5 L), Asthma Quality of Life Questionnaire (AQLQ), Time trade-off (TTO), and peak flow and symptom diary over 8 weeks, where three different methods (EQ-5D-5 L, AQLQ, and TTO), were used to estimate utilities. The mean difference between two time points were estimated using the Wilcoxon signed rank test. Results From baseline to week 8, mean increases (95% CI) were estimated to be 0.086 (0.019–0.153), 0.154 (0.112–0.196) and 0.132 (0.063–0.201) for EQ-5D-5 L, AQL-5D (preference-based measure derived from AQLQ), and TTO respectively over 8 weeks ( p < 0.01). Conclusion Asthma crisis events are estimated to be associated with a mean utility loss of between 0.086 and 0.132. The utility decrement can be used to assign values to asthma-related crisis events, which can enhance economic evaluations. Trial registration NCT02771678 . Registered 13 May 2016. Electronic supplementary material The online version of this article (10.1186/s12955-019-1138-5) contains supplementary material, which is available to authorized users.

IntroductionHealth Technology Assessment (HTA) agencies have recognized the importance of real-world evidence (RWE) to inform access decision-making and different HTA agencies establish distinct requirements for their local jurisdictions. The objective of this study is to understand the differences of RWE included in HTA reports and HTA agencies’ perception of RWE.MethodsHTA reports from agencies in France, Germany, Spain, Italy, United Kingdom (UK), Canada, Australia and South Korea from January 2011 to November 2021, including original submissions, resubmissions, extensions of original indications and renewals were analyzed.ResultsAcross the eight countries, RWE has been used in nineteen percent of all HTA reports (N=2,960/15,561), with an exponential increase observed between 2019 and 2021. RWE on clinical effectiveness was mostly used in HTA submissions in the UK (twenty-two percent), with twenty-six percent perceived with full acceptance. In contrast, RWE on safety and epidemiology was reported widely in HTA reports in France and Germany (83% and 87%), respectively. Ninety-three percent of RWE received full acceptance in France, followed by forty-four percent in Germany. A mixed picture of the types of RWE included in HTA reports was observed in the other countries, with high variance of acceptance (between 5 to 37%).ConclusionsFrance, Germany, and the UK are the top three countries with a large proportion of HTA reports where RWE was mentioned. The type of RWE used is related to a large extent to the local evidence requirements. For example, RWE around epidemiology was included widely in Germany due to the needs of providing local data for budget impact analyses required by the Federal Joint Committee (G-BA); RWE on tolerability as reported in periodic safety update reports (PSURs) needs to be included in French HTA submissions. RWE on clinical effectiveness has been evaluated the most by the UK HTA bodies.