Background Rare diseases may be life-threatening or chronically debilitating conditions.
Patient care needs are often complex and challenging to coordinate and deliver effectively …

Background Rare diseases can lead to a significant reduction in quality of life for patients
and their families. Ensuring the patients voice is central to clinical decision making is key to …

Background Patient-reported outcome measures (PROMs) are used in health technology
assessment (HTA) to measure patient experiences with disease and treatment, allowing a …

Objectives: A patient reported outcome (PRO) is “any report of the status of a patient's health
condition that comes directly from the patient without interpretation of the patient's response …

Objectives To explore the responsiveness of patient-reported outcomes (PROs) in
interventional studies involving patients with rare lysosomal storage diseases (LSDs). Study …

Background There has been a growing emphasis on health-related quality of life (HRQoL)
as an important outcome in rare disease drug development, although its assessment may be …

Abstract Background Primary Sclerosing Cholangitis (PSC) is a rare chronic, cholestatic liver
condition in which patients can experience a range of debilitating symptoms. Patient …

Our ability to evaluate outcomes which genuinely reflect patients' unmet needs, hopes and
concerns is of pivotal importance. However, much current clinical research and practice falls …

We hope that a wide range of practice types will find this User's Guide helpful. The field of
assessing PROs in clinical practice is continuing to develop and evolve, and the web …

Background Patient-reported outcome measures (PROMs) provide a measure of self-
perceived health status or health-related quality of life. They have been used to support …