US FDA patient-reported outcome guidance: great expectations and unintended consequences

Abstract

Release of the US FDA patient-reported outcome (PRO) guidance raised expectations within the pharmaceutical industry for the use of PRO measures in support of labeling claims. The FDA developed the guidance with admirable intent, and the recommendations within this document are based on sound scientific principles. However, implementation of the guidance has been somewhat inconsistent within the Study Endpoints and Label Development (SEALD) and across the various FDA-reviewing divisions. Industry and regulatory bodies need to work toward gaining common ground to best support registration of treatments that could extend patients' lives, reduce symptoms, and/or improve health-related quality of life. PROs are valuable tools in communicating these messages, and realistic implementation of the FDA PRO Guidance may truly facilitate this process.

Clinical interpretation of the Uremic Pruritus in Dialysis Patients (UP‐Dial) scale: a novel instrument for the assessment of uremic pruritus

“…Currently, PROs are recognized as valid primary end points in several clinical trials, including chronic kidney disease trials . According to the United States Federal Drug Agency, PRO instruments that reflect the patients’ perspective are crucial to incorporate into new therapeutic interventions trials . Unfortunately, there is no well‐established PRO instruments for UP assessment.…”

One Programme, Four Stakeholders: An Overview of the Utilisation of Patient-Reported Outcomes in Intervention Development to Meet the Needs of Regulators, Payers, Healthcare Professionals and Patients

“…the 2014 FDA Guidance for Industry on Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (Developing Drug Products for Treatment), and the 2014 EMA reflection paper on the use of PRO measures in oncology studies]. Clearly, the regulators recognise PROs as important outcomes for evaluating drugs, biologics, and medical devices, and PRO data have been used as primary and secondary endpoints to support primary biomarkers in both jurisdictions [4][5][6].…”

Using qualitative methods in developing an instrument to identify barriers to self‐care among persons with type 2 diabetes mellitus

“…Patient Reported Outcomes (PROs) (Fehnel et al . ). Various questionnaires have been published to evaluate the self‐care needs of DM II patients, but they present weaknesses such as an inadequate procedure of psychometric validation (to ensure internal/external consistency, the validity of criteria/constructs/content, sensitivity to change, etc.)…”