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

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

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 …

Purpose New patient reported outcome (PRO) measures are regularly developed to assess
various aspects of the patients' perspective on their disease and treatment. For these …

Objectives: Rare diseases are often heterogeneous in their progression and response to
treatment, with only a small population for study. This provides challenges for evidence …

The science of measuring patient-reported outcomes (PROs) has advanced substantially in
recent decades, allowing evaluation of how patients feel and function in clinical research …

Abstract Background Rare diseases (RDs) affect a small number of people within a
population. About 5000 to 8000 distinct RDs have been identified, with an estimated 6% to …

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 Patient Reported Outcome Measures (PROMs) are being increasingly
introduced in clinical registries, providing a personal perspective on the expectations and …