Current medical research and opinion
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Meta Analysis Comparative Study
Matching-adjusted indirect treatment comparison of siponimod and other disease modifying treatments in secondary progressive multiple sclerosis.
Background: Siponimod, interferon beta-1a (IFNβ-1a), IFNβ-1b and natalizumab have been evaluated as treatments for secondary progressive multiple sclerosis (SPMS) in separate randomized controlled trials (RCTs), but not head-to-head. These trials included heterogeneous patient populations, which limits the use of standard network meta-analysis (NMA) for indirect treatment comparison (ITC) of relative efficacy. Matching-adjusted indirect comparison (MAIC) aims to correct these cross-trial differences. ⋯ For annualized relapse rate (ARR), with the exception of natalizumab, siponimod was numerically but not statistically superior to all comparators. Conclusions: EXPAND provides evidence of the efficacy of siponimod compared with placebo, and these MAICs complement this by demonstrating improved efficacy of siponimod relative to DMTs. Siponimod offers a significant therapeutic advance that may slow disease progression compared to other DMTs in an EXPAND-like population with secondary progressive disease.
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Meta Analysis Comparative Study
The importance of considering differences in study and patient characteristics before undertaking indirect treatment comparisons: a case study of siponimod for secondary progressive multiple sclerosis.
Background: Indirect treatment comparisons (ITCs) provide valuable evidence on comparative efficacy where head-to-head clinical trials do not exist; however, differences in patient populations may introduce bias. Therefore, it is essential to assess between-trial heterogeneity to determine the suitability of synthesizing ITC results. We provide an illustrative case study in multiple sclerosis (MS) where we assess the feasibility of conducting ITCs between siponimod and interferon beta-1b (IFN β-1b) and between siponimod and ocrelizumab. ⋯ ITCs were not feasible between siponimod and ocrelizumab because study designs and patient populations were too dissimilar to conduct a reliable ITC. Conclusions: This study highlights the importance of conducting a detailed feasibility assessment before undertaking ITCs to illuminate when excessive between-trial heterogeneity would cause biased results. MAIC was performed for siponimod and IFN β-1b in the absence of a head-to-head trial and was considered a more valid approach than a traditional ITC to examine comparative effectiveness.
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Review
Health utility estimation in children and adolescents: a review of health technology assessments.
Objective: Health utility estimates for children and adolescents are critical for cost-utility analyses informing health technology assessment (HTA) authorities' decisions governing access to pediatric treatments. However, in a recent review, only 29% of published pediatric cost-utility models used a utility measure validated for children. We examined utility estimates used in pediatric HTAs. ⋯ When estimating pediatric utility weights, alternative measures should be reviewed for suitability given the model population and health condition. Pediatric and adult utility estimates should be applied appropriately as patients age over time, and caregiver and/or family member utility should be included, where relevant. Gaps exist in utility measures for children aged <4 years and caregivers.
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Observational Study
Chemotherapy treatments, costs of care, and survival for elderly patients diagnosed with cervical cancer: an observational study.
Objective: To describe chemotherapy treatments, associated health care use and costs, and survival for women diagnosed with cervical cancer in the United States. Methods: This was a retrospective cohort study of patients aged ≥65 years, identified in linked Surveillance, Epidemiology, and End Results (SEER) and Medicare databases. Women with a new primary diagnosis of cervical cancer between January 2007 and December 2013 were followed until December 2014. ⋯ Conclusion: Elderly patients with advanced cervical cancer had a poor prognosis, with a median survival of 14 months or less, and had no standard of care for 2L therapy. Systemic chemotherapies pose a substantial economic burden in the range of $7000 to $9000 per patient per month. These results highlight the high unmet medical need among elderly patients with cervical cancer.
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Objective: The aim of this study was to provide recommendations for improving the design of subsequent studies through analysis of the registered coronavirus disease 2019 (COVID-19) clinical trials. Methods: A retrospective analysis of 189 trial retrievals achieved on 20 February 2020. Results: A total of 189 trials are included in the study. ⋯ Study design characteristics: 71% of all studies were randomized, 5% of all studies were blinded, 18% of all studies were multicenter and 76% of all studies were single center. Conclusion: Although many COVID-19 studies include randomization in their design, the lack of additional double-blind and placebo-controlled elements in their designs result in a less robust evaluation of intervention safety and efficacy. Furthermore, similar or repeated research and small sample studies that have less promise in gains of new information have possibly led to a shortage of recruitable patients and become a barrier to the completion of large multicenter clinical trial studies.