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ORIGINAL RESEARCH article
Front. Pharmacol.
Sec. Drugs Outcomes Research and Policies
Volume 16 - 2025 | doi: 10.3389/fphar.2025.1536401
From clinical trials to informing clinical decision-making: A review of patientreported outcomes in nononcology medicines approved by the European Medicines Agency
Provisionally accepted
Sarah Sauchelli 1*
Courtney Levy 1 Ari Gnanasakthy 2 Vaidehi Dave 2 Lynda Doward 1 Kristina A Fitzgerald 3 Robyn Carson 3- 1 RTI Health Solutions, Manchester, United Kingdom
- 2 RTI Health Solutions, Durham, North Carolina, United States
- 3 Patient-Centered Outcomes Research, AbbVie Inc, New Jersey, United States
Introduction: Information about a medicine published in the Summary of Product Characteristics (SmPC) and the product’s package leaflet by the European Medicines Agency (EMA) is key to communicate its value to prescribers and patients. The aim of this study was to examine the inclusion of statements related to patient-reported outcomes (PROs) in these documents to communicate patients’ perspectives and experiences of new nononcology medicines.Methods: Nononcology therapeutic indications recommended for approval by the EMA between 2018-2022 were identified. The Public Assessment Report(s) (PAR), SmPC, and package leaflet published for each indication were examined. Information about the indication and characteristics relating to how the PROs were assessed in confirmatory studies was extracted. Results: Most nononcology therapeutic indications (n = 98/140, 70%) contained PRO trial data but less than 50% (n = 64/140, 46%) had PRO-related statements in the SmPC and/or package leaflet. Most statements described treatment benefit (n = 60/64, 94%). Statements were most likely to be included in the SmPC and/or package leaflet if supported by at least 1 randomized controlled trial (n = 52/71, 73%), the endpoint assessed patient-reported symptoms or symptom burden (n = 56/71, 79%), and/or the PRO(s) were assessed as a primary endpoint (n = 24/24, 100%). Discussion: Although trial data pertaining to PROs are reviewed when evaluating nononcology drugs, shortfalls persist in the inclusion of PROs when describing treatment benefit in critical documents used to inform treatment decision-making.
Keywords: Patient-Reported Outcome (PRO), European Medicines Agency (EMA), Summary of Product Characteristics (SmPC), Health-related Quality of Life (HR QoL) outcomes, Package leaflet, clinical trials
Received: 28 Nov 2024; Accepted: 07 Mar 2025.
Copyright: © 2025 Sauchelli, Levy, Gnanasakthy, Dave, Doward, Fitzgerald and Carson. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Sarah Sauchelli, RTI Health Solutions, Manchester, United Kingdom
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.