OBJECTIVES: Patient-reported outcomes (PRO) have received increasing attention from regulatory agencies regarding intended use of the data for promotional labeling claims. However, some disease areas and/or regulatory bodies necessitate the use of PRO data to substantiate product efficacy for securing approval. Therefore, the research objective was to determine how many of the final product development guidance available from EMEA and FDA for clinical/medical research indicate PRO as a mandatory component of efficacy.
METHODS: Final guidance documents from the EMEA and FDA were reviewed for mention of PRO. EMEA Guidance documents that fell under the following categories were excluded: Clinical Pharmacology and Pharmacokinetics, Blood and Blood Forming Organs, Blood products (including biotech alternatives), and Herbals. Included in FDA Guidance review were those listed under the “Clinical/Medical” heading. The following data were abstracted from each guidance: guidance number, name, issue date, disease area, body system classification, PRO requirement, PRO endpoint hierarchy, and a summary of the PRO language included in the guidance. PRO statements were then characterized within each of the following categories (yes/no): signs/symptoms, function/feeling, HRQOL, or patient global rating.
RESULTS: Of the 134 final guidance documents reviewed (EMEA = 81, FDA = 53), 52 mention PRO (EMEA n = 39; FDA n = 13). Within EMEA, PRO is indicated as primary (n = 5) or secondary (n = 22; of which 4 are secondary and/or exploratory) or both (n = 12).The majority of PRO statements are characterized as sign and symptom measures followed by HRQOL measures. Within FDA, 5 required PRO and 8 suggest use of PRO. The majority of PRO statements are characterized as sign and symptom measures, followed by measures of function/feeling.
CONCLUSIONS: PRO data in many disease areas are viewed by regulatory agencies as supportive evidence of the primary endpoint. PRO data are essential in the support of product submissions to regulatory stakeholders, especially within EMEA.
