Using a disease-specific Patient-Reported Outcome measure as central to a patient-centric approach for clinical trials and routine practice in rare diseases

Patient-Reported Outcome (PRO) measures are increasingly recognized as the ultimate arbitrator of values and concerns that are uniquely meaningful to the patient, and as such are utilized to assess both health-related quality of life and benefit of therapeutic interventions in the context of the various stages of clinical trial development and evaluation, as well as in post-marketing long-term assessment. In routine clinical practice, PROs can serve as the basis for physician-patient dialogue and encourage patient empowerment in furthering patient-centric care. For patients with rare disorders, disease-specific PROs are especially recommended to highlight disease-specific features and concerns in order to more adequately support personalized medical attention.