Treatment needs and expectations for Fabry disease in France: development of a new Patient Needs Questionnaire.

ABSTRACT:

Background

Fabry disease (FD) is a rare, X-linked, inherited lysosomal disease caused by absent or reduced α-galactosidase A activity. Due to the heterogeneity of disease presentation and progression, generic patient-reported outcome (PRO) tools do not provide accurate insight into patients' daily lives and impact of disease specific treatments. Also, the French National Health Authority, (HAS) actively encourages a patient-centric approach to improve the quality of care throughout the patient journey. In response to this initiative, we aimed to develop and validate a specific, self-reported, Patient Needs Questionnaire for people living with Fabry disease to appraise patient needs and expectations towards their treatment (PNQ Fabry). This endeavour was led with the help of French

SUBMITTER: Noel E 

PROVIDER: S-EPMC6894302 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature