Comparing clotting factors attributes across different methods of preference elicitation in haemophilia patients.

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.

Stable isotope data (oxygen-18 and deuterium) from surveys of lakes, wetlands, rivers, and input waters across the South Athabasca Oil Sands region, Alberta, 2007-2009.

Oxygen-18 and deuterium analyses of water samples are provided from a regional survey of lakes, wetlands, soil waters, groundwaters, and snowpack samples collected in the Southern Athabasca Oil Sands (SAOS) region, Alberta, Canada, mainly during 2007-2009. Lake, wetland, and river sampling were conducted by helicopter during late summer, capturing conditions close to peak evaporative enrichment. Shallow soil water from the unsaturated zone was also collected in late summer, whereas deeper groundwaters from Quaternary aquifers, Quaternary channels, and uppermost Cretaceous strata, were collected primarily as part of winter drilling programs by industrial partners.

Advancing the science of patient input throughout the regulatory decision-making process.

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.