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The Paradigm Shift From Patient to Health Consumer: 20 Years of Value Assessment in Health.
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Virginia Commonwealth University, Richmond, VA, United States.
Health care is undergoing a "revolution," where patients are becoming consumers and armed with apps, consumer review scores, and, in some countries, high out-of-pocket costs. Although economic analyses and health technology assessment (HTA) have come a long way in their evaluation of the clinical, economic, ethical, legal, and societal perspectives that may be impacted by new technologies and procedures, these approaches do not reflect underlying patient preferences that may be important in the assessment of "value" in the current value-based health care transition. The major challenges that come with the transformation to a value-based health care system lead to questions such as "How are economic analyses, often the basis for policy and reimbursement decisions, going to switch from a societal to an individual perspective?" and "How do we then assess (economic) value, considering individual preference heterogeneity, as well as varying heuristics and decision rules?" These challenges, related to including the individual perspective in cost-effectiveness analysis (CEA), have been widely debated.
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Oral ganaxolone (ZTALMY), a synthetic analogue of the endogenous neuroactive steroid allopregnanolone, acts as a positive allosteric modulator of synaptic and extra-synaptic γ-aminobutyric acid (GABA) type A receptor function in the CNS. In the EU and the UK, it is approved for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged 2-17 years. In a multinational phase III study (Marigold), 17 weeks' therapy with adjunctive ganaxolone, administered orally three times daily with food, significantly reduced 28-day major motor seizure frequency from baseline versus placebo in patients aged 2-19 years with CDD-associated refractory epilepsy.
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Sustainable systems are designed to promote lasting viability and resilience while reducing negative effects on the environment, society, and economy. Like many others, the drug delivery field is facing the challenges of the global environmental crisis. Despite its rapid growth and significant funding, there has been a noticeable slowdown in the rate of advancement, impacting the economy, society, and environment.
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The applications of single-cell and spatial technologies in recent times have revolutionized the present understanding of cellular states and the cellular heterogeneity inherent in complex biological systems. These advancements offer unprecedented resolution in the examination of the functional genomics of individual cells and their spatial context within tissues. In this review, we have comprehensively discussed the historical development and recent progress in the field of single-cell and spatial genomics.
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