development of a new Patient Needs Questionnaire
In France, two associations actively represent Fabry patients, participate in and promote medical research: Association des Patients de la Maladie de Fabry (APMF, apmf-fabry.org) and Vaincre les Maladies Lysosomales (VML, www.vml-asso.org). In a patient-centric approach, involving these associations in developing a patient needs instrument is highly recommended to provide a patient perspective.
In this context, the authors identified the need to develop a questionnaire to evaluate and measure treatment expectations for patients living with Fabry disease to heighten the clinical picture and allow physicians tomanage the treatment more astutely.