Payers are increasingly concerned with assessing the value of pharmaceutical products to guide reimbursement and pricing decisions, and particularly in countries employing an HTA process, value is often largely based on clinical effectiveness measured as cost-per-QALY. The objective of this study is to assess how the EQ-5D index reacts across a broad range of conditions to clinical changes which are considered significant at the individual disease and patient level. METHODS: A systematic review of published studies assessing the psychometric properties of the EQ-5D, in particular studies reporting statistics on the responsiveness of the EQ-5D index. The Standardised Response Mean (SRM) and Effect Size (ES), which are both measures of signal-to-noise and describe the responsiveness of EQ-5D to clinical change, are compared to benchmark scores from the psychometric literature. We also compare the Visual Analogue Scale (VAS) self-reported health component against the EQ-5D index. RESULTS: In the majority of studies, the responsiveness of EQ-5D to clinical change overall was poor to moderate: In only approx. half of studies was the VAS score within 10% of the EQ-5D index score. Ceiling effects of >15% at baseline where reported in half the studies examined. Only 25% of studies reported ES values within the range considered "moderate" to "good", and 7% of studies reported SRM values in the "moderate" to "good" range. CONCLUSIONS: When considering widely accepted psychometric benchmarks and the relation between self-reported health and EQ-5D index, only a minority of studies (11%) report consistently good performance of the EQ-5D in reflecting clinically important changes. Consequently, a significant proportion of therapeutic value (both from a clinical and patient perspective) is not adequately captured by the existing EQ-5D framework. This has implications for the way new therapies have traditionally been assessed for value, particular as countries start moving towards value based pricing.

Objectives: MG is a debilitating chronic illness characterized by muscle weakness, potentially life-threatening exacerbations and high costs. We sought to further quantify the humanistic and economic burden by reviewing published evidence. Methods: Using PRISMA guidelines, two systematic literature reviews were conducted, one for quality of life (QOL) and one for economic studies, in key biomedical literature databases, Embase, Ovid Medline and Cochrane. Additionally, only studies published between 2009-2019 were identified. Results: A total of 957 abstracts from the QOL search and 521 from the economic search underwent screening. A total of 81 QOL and 41 economic studies were selected. A variety of QOL tools were used, including SF-36, EQ-5D, HADS, MG-ADL, MG-QOL15, QMG and MGC among others. The only study mapping utility values found a range of 0.94-0.20 across MGFA classifications, indicating a major QOL deterioration related to disease severity. Deterioration was further supported in other studies which found increased levels of chronic fatigue, sleep disturbances and anxiety/depression with MG. Refractory patients experienced significantly worse deteriorations across various scales. Employment status and medication adherence were also negatively affected. Of 41 economic studies, 32 were related to cost/healthcare resource use, while 9 described economic models. The average US cost in 2013 was $98,795 per MG hospitalization and the gross hospitalization cost in the HCUP database had risen more than 13-fold since 2003 totalling $546,834,101. Inpatient, outpatient and home costs were found to account for 27%, 23% and 23% of MG healthcare costs. A US claims database found annual costs to be approximately 4 times higher in refractory vs. non-refractory patients ($109,004 vs. $24,196, p,0.001), possibly related to a higher use of IVig/ PLEX, costly therapies according to economic models. Conclusions: Novel treatment strategies are necessitated to help control rising costs and alleviate the humanistic burden associated with MG, especially in refractory patients.

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A211the fully adjusted HR was 2.03; 95% CI (1.13-3.65) among children exposed to SSRIs alone and 4.10; 95% CI (1.35-12.41) among children exposed to combined classes of antidepressant. ConClusions: This study suggests that use of antidepressant during the 2 nd /3 rd trimester of pregnancy may increase the risk of ASD.

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