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 …

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 …

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

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

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 …

Patient reported outcomes (PROs) are a measurement based on a report that comes directly
from the patient about the status of their health condition without amendment or …

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 …

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 …

The International Society for Pharmacoeconomics and Outcomes Research Task Force has
presented a detailed report highlighting the challenges associated with capturing clinical …

Objectives Measures for assessing patient-reported outcomes (PROs) that may have initially
been developed for research are increasingly being recommended for use in clinical …