Introduction: Emerging, systematic approaches for capturing patient input, such as preference elicitation, can provide valuable information for the benefit-risk assessment of medical products for treating bleeding disorders, such as haemophilia.
Aim: This study aims to identify existing and develop new methods to capture, rank and summarize preference scores for clotting factor therapies.
Methods: Haemophilia patient preference data were compiled from studies identified through literature review and publicly available US FDA patient-focused drug development meeting documents.
The US Food and Drug Administration (FDA) understands the value of patient input in the regulatory decision-making process and has worked to enhance meaningful engagement. In recent years, there has been an increased scientific demand for more systematic and quantitative approaches to incorporate patient input throughout the medical product lifecycle, including to inform regulatory benefit-risk assessments. The use of patient preference information (PPI), elicited using established scientific methods, is a promising strategy for accomplishing this.
View Article and Find Full Text PDFPurpose: To identify factors associated with early treatment discontinuation of three agents commonly prescribed for women with low bone density.
Methods: A telephone survey was conducted in 2000 to 2001 in a random sample of women aged 45 years or older who had bone density T-scores -1.0 or lower and who had initiated treatment with hormone replacement therapy, raloxifene, oral endronate.