Abstract

Patient-focused drug development has become a central focus of clinical outcome assessment (COA) research, gaining momentum with its formal launch under the Prescription Drug User Fee Amendments Act V in 2012 and the subsequent initiation of the US Food and Drug Administration (FDA) Patient-Focused Drug Development (PFDD) Guidance series beginning in 2016. As part of this patient centricity in new drug development and global regulatory recognition of the importance of the patient voice, the evolution of qualitative and quantitative research continued with methodological focus expanding beyond large-scale research, pushing COA scientists to consider more narrow populations, including patients living with rare and orphan diseases and syndromes. In fact, the PFDD Guidance series, specifically PFDD Guidance 3 entitled “Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments,” addresses the challenges of applying traditional qualitative and quantitative psychometric methodologies in rare and orphan indications and challenges COA scientists to consider more flexible and mixed approaches, especially for interpreting meaningful change.