Objectives: Multiple methods are available for collecting health preference information. However, information on the design and analysis of novel methods is limited. This article aims to provide the first introduction into the design and analysis of multidimensional thresholding (MDT).
View Article and Find Full Text PDFPRODUCTS with janus kinase (JAK) inhibition have been shown to promote hair regrowth in patients with alopecia areata (AA). To guide drug-approval and treatment decisions, it is important to understand patients' willingness to accept the potential risks of JAK inhibition in exchange for potential benefits. We quantified the treatment preferences of adult (≥18 years) and adolescent patients (12-17 years) with AA in the US and Europe to determine the trade-offs they are willing to make between benefits and risks.
View Article and Find Full Text PDFObjectives: To understand industry practices and challenges when submitting patient experience data (PED) for regulatory decisions by the US Food and Drug Administration (FDA).
Methods: A two-part online survey related to collection, submission, and use of PED by FDA in regulatory decision-making (part 1) and a best-worst exercise for prioritizing potential PED initiatives (part 2) was completed by industry and contract research organization (CRO) members with ≥ 2 years of recent experience with patient-reported outcome (PRO), natural history study (NHS), or patient preference (PP) data; and direct experience with FDA filings including PED.
Results: A total of 50 eligible respondents (84% industry) completed part 1 of the survey, among which 46 completed part 2.
Hepsin, a type II transmembrane serine protease, is commonly overexpressed in prostate and breast cancer. The hepsin protein is stabilized by the Ras-MAPK pathway, and, downstream, this protease regulates the degradation of extracellular matrix components and activates growth factor pathways, such as the hepatocyte growth factor (HGF) and transforming growth factor beta (TGFβ) pathway. However, how exactly active hepsin promotes cell proliferation machinery to sustain tumor growth is not fully understood.
View Article and Find Full Text PDFObjectives: An increasing number of methods are used to elicit health preference information. It is unclear whether different elicitation methods produce similar results and policy advice. Here, we compared the results from a discrete choice experiment (DCE) and multidimensional thresholding (MDT) that were conducted in the same sample.
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