Patient-reported outcomes: Assessment and current perspectives of the guidelines of the Food and Drug Administration and the reflection paper of the European Medicines Agency |
| |
| Authors: | Andrew Bottomley Dave Jones Lily Claassens |
| |
| Institution: | 1. EORTC Quality of Life Department, EORTC Headquarters, Avenue E. Mounierlaan, 83/11, Brussels 1200, Belgium;2. AstraZeneca, Alderly Park, Macclesfield, England, United Kingdom;1. Departamento de Química Física i Analítica, ESTCE, Universitat Jaume I, 12071 Castelló, Spain;2. Biopatologia Molecular, Hospital Provincial, Castelló, Spain;1. Department of Urology, New York University Langone Medical Center, New York, NY;2. Division of Urology, Department of Surgery, Faculty of Medicine, University of Ottawa, ON, Canada;1. Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, USA;2. Medicines and Healthcare Products Regulatory Agency, London, UK;3. Therapeutic Products Directorate, Health Products and Food Branch, Health Canada, Ottawa, ON, Canada;1. ICON Commercialisation and Outcomes, Gaithersburg, MD, USA;2. Evidera – Evidence, Value & Access by PPD, Bethesda, MD, USA;3. Department of Health Services, University of Washington, Seattle, WA, USA;4. Pharmaceutical Health Services Research, School of Pharmacy, University of Maryland, Baltimore, MD, USA;5. Director, RareAccess Ltd., London, UK;6. School of Pharmacy, University of Maryland, Royal Oak, MD, USA;1. Outcomes Research, United BioSource Corporation, Bethesda, MD, USA;2. Department of Health Services, University of Washington, Seattle, WA, USA;3. Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA;4. Division of Anti-Infective Products, FDA, Silver Spring, MD, USA;5. IMIM-Hospital del Mar Medical Research Institute, and Catalan Agency for Health Information, Assessment and Quality, Barcelona, Spain;6. Pfizer Global Pharmaceuticals, Pfizer, Inc., San Diego, CA, USA;7. Department of Medical Psychology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany;1. Janssen Research and Development, Titusville, NJ, USA;2. Leidos Biomedical Research in support of the Division of Clinical Research, National Institutes of Health, Bethesda, MD, USA;3. Plymouth University Peninsula Schools of Medicine and Dentistry, Devon, UK;4. Seattle Quality of Life Group, Department of Health Services, University of Washington, Seattle, WA, USA;5. Modus Outcomes, Newton, MA, USA;6. European Medicines Agency, London, UK;7. International Society for Pharmacoeconomics and Outcomes Research, Lawrenceville, NJ, USA;8. Modus Outcomes, Stotfold, UK;9. LORA Group, LLC, Royal Oak, MD, USA;10. Department of Health Services Research, University of Maryland School of Pharmacy, Baltimore, MD, USA |
| |
| Abstract: | AimsPatient-reported outcomes (PROs) have recently gained greater credibility with regulatory bodies aiming to standardise their use and interpretation in RCTs, thereby supporting medicinal product submissions. For this reason, the United States (US) Food and Drug Administration (FDA) and the European Medicines Agency (EMEA) have released guidelines. This review paper provides an overview of the current perspectives and views on these guidelines.MethodTo evaluate the FDA and EMEA PRO guidelines, 47 expert responses to the FDA guidance were qualitatively reviewed. Two reviewers independently extracted data from these letters and checked these responses to warrant consistency and agreement in the evaluation process. A PubMed literature review was systematically examined to obtain supporting evidence or related articles for both the guidance documents.ResultsGenerally, there is agreement between regulatory authorities and the research community on the contents of the FDA and EMEA PRO draft guidance. However, disagreements exist on significant philosophical topics (e.g. the FDA focuses more on conceptual models and symptoms than the EMEA) and design topics (e.g. the FDA is more restrictive on issues of recall bias, blinding of oncology trials and degrees of psychometric validation than researchers and the EMEA). This could influence the approval of PRO claims.ConclusionPRO guidance from the EMEA and FDA has been valuable, and has raised the profile and active debate of PROs in oncology. However, our review of the current opinion shows that there are controversial aspects of the guidance. Consequently, greater latitude should be given to how the guidance is interpreted and applied. |
| |
| Keywords: | |
| 本文献已被 ScienceDirect 等数据库收录！ |
|
