Patient-Reported Outcomes in Orphan Drug Labels Approved by the US Food and Drug Administration

09/16/2019

Dr. Eleanor Perfetto, NHC Executive Vice President, Strategic Initiatives, co-authored a paper titled, “Patient-Reported Outcomes in Orphan Drug Labels Approved by the US Food and Drug Administration,” which was published by ISPOR Value in Health.

“In recent years, there has been increasing recognition of the need to assess treatment benefit from the patient’s perspective. The extent of patient-reported outcome (PRO) data included in labeling for rare disease treatment is largely unknown. The objective of this study was to review trends over time for PRO-based labeling granted by the US Food and Drug Administration (FDA) for orphan drugs.”