Real-world evidence: new opportunities for osteoporosis research. Recommendations from a Working Group from the European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO).
[en] [en] UNLABELLED: This narrative review summarises the recommendations of a Working Group of the European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO) for the conduct and reporting of real-world evidence studies with a focus on osteoporosis research.
PURPOSE: Vast amounts of data are routinely generated at every healthcare contact and activity, and there is increasing recognition that these real-world data can be analysed to generate scientific evidence. Real-world evidence (RWE) is increasingly used to delineate the natural history of disease, assess real-life drug effectiveness, understand adverse events and in health economic analysis. The aim of this work was to understand the benefits and limitations of this type of data and outline approaches to ensure that transparent and high-quality evidence is generated.
METHODS: A ESCEO Working Group was convened in December 2022 to discuss the applicability of RWE to osteoporosis research and approaches to best practice.
RESULTS: This narrative review summarises the agreed recommendations for the conduct and reporting of RWE studies with a focus on osteoporosis research.
CONCLUSIONS: It is imperative that research using real-world data is conducted to the highest standards with close attention to limitations and biases of these data, and with transparency at all stages of study design, data acquisition and curation, analysis and reporting to increase the trustworthiness of RWE study findings.
Disciplines :
Public health, health care sciences & services
Author, co-author :
Moon, Rebecca J ; MRC Lifecourse Epidemiology Centre, University of Southampton, Southampton, SO16 6YD, UK ; Paediatric Endocrinology, University Hospital Southampton NHS Foundation Trust, Southampton, UK
Reginster, Jean-Yves ; Université de Liège - ULiège > Département des sciences de la santé publique ; WHO Collaborating Center for Epidemiology of Musculoskeletal Health and Ageing, Liège, Belgium
Al-Daghri, Nasser ; Université de Liège - ULiège > Département des sciences de la santé publique > Santé publique, Epidémiologie et Economie de la santé ; Biochemistry Department, College of Science, King Saud University, Riyadh, Saudi Arabia
Thiyagarajan, Jotheeswaran A; Ageing and Health Unit, World Health Organisation, Geneva, Switzerland
Beaudart, Charlotte ; Université de Liège - ULiège > Département des Sciences de l'activité physique et de la réadaptation ; WHO Collaborating Center for Epidemiology of Musculoskeletal Health and Ageing, Liège, Belgium
Bruyère, Olivier ; Université de Liège - ULiège > Département des sciences de la santé publique > Santé publique, Epidémiologie et Economie de la santé ; WHO Collaborating Center for Epidemiology of Musculoskeletal Health and Ageing, Liège, Belgium
Burlet, Nansa ; Université de Liège - ULiège > Département des sciences de la santé publique > Santé publique, Epidémiologie et Economie de la santé
Chandran, Manju; Osteoporosis and Bone Metabolism Unit, Department of Endocrinology, Singapore General Hospital, Singapore, Singapore
da Silva, Mario Coelho; Laboratory of Clinical and Therapeutical Pharmacology, Lisbon, Portugal
Conaghan, Philip G ; Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds, UK ; NIHR Leeds Musculoskeletal Biomedical Research Unit, Leeds, UK
Dere, Willard H ; Department of Internal Medicine, Utah Center for Clinical and Translational Science, University of Utah School of Medicine, Salt Lake City, UT, USA
Diez-Perez, Adolfo; Department of Internal Medicine, Hospital del Mar-IMIM, Autonomous University of Barcelona and CIBERFES, Instituto Carlos III, Barcelona, Spain
Hadji, Peyman ; Frankfurt Centre for Bone Health, Frankfurt, Germany ; Philipps University of Marburg, Hesse, Germany
Halbout, Philippe; International Osteoporosis Foundation, Nyon, Switzerland
Hiligsmann, Mickaël ; Université de Liège - ULiège > Département des sciences de la santé publique > Santé publique, Epidémiologie et Economie de la santé ; Department of Health Services Research, CAPHRI Care and Public Health Research Institute, Maastricht University, Maastricht, the Netherlands
Kanis, John A ; Mary McKillop Institute for Health Research, Australian Catholic University, Melbourne, Australia ; Centre for Metabolic Bone Diseases, University of Sheffield, Sheffield, UK
McCloskey, Eugene V ; MRC Versus Arthritis Centre for Integrated Research in Musculoskeletal Ageing, University of Sheffield, Sheffield, UK ; Mellanby Centre for Musculoskeletal Research, Department of Oncology & Metabolism, University of Sheffield, Sheffield, UK
Prieto-Alhambra, Daniel; Pharmaco- and Device Epidemiology, Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences (NDORMS), University of Oxford, Oxford, UK
Radermecker, Régis ; Université de Liège - ULiège > Département des sciences cliniques > Pharmacologie clinique
Rizzoli, René ; Université de Liège - ULiège > Département des sciences de la santé publique > Santé publique, Epidémiologie et Economie de la santé ; Geneva University Hospitals and Faculty of Medicine, Geneva, Switzerland
Al-Saleh, Yousef ; College of Medicine, King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia ; King Abdullah International Medical Research Center, Riyadh, Saudi Arabia ; Department of Medicine, King Abdulaziz Medical City, Riyadh, Ministry of National Guard-Health Affairs, Riyadh, Saudi Arabia
Silverman, Stuart L; Cedars Sinai Medical Center, Los Angeles, USA
Simon, Lee S ; SDG LLC, Cambridge, MA, USA
Thomasius, Friederike; Frankfurt Centre for Bone Health, Frankfurt, Germany
van Staa, Tjeerd ; Centre for Health Informatics, University of Manchester, Manchester, UK
Laslop, Andrea; Scientific Office, Austrian Medicines and Medical Devices Agency, Vienna, Austria
Cooper, Cyrus ; Université de Liège - ULiège > Département des sciences de la santé publique > Santé publique, Epidémiologie et Economie de la santé ; MRC Lifecourse Epidemiology Centre, University of Southampton, Southampton, SO16 6YD, UK ; NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK ; National Institute for Health Research (NIHR) Musculoskeletal Biomedical Research Centre, University of Oxford, Oxford, UK
Harvey, Nicholas C ; MRC Lifecourse Epidemiology Centre, University of Southampton, Southampton, SO16 6YD, UK. nch@mrc.soton.ac.uk ; NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK. nch@mrc.soton.ac.uk
Real-world evidence: new opportunities for osteoporosis research. Recommendations from a Working Group from the European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO).
Publication date :
August 2023
Journal title :
Osteoporosis International
ISSN :
0937-941X
eISSN :
1433-2965
Publisher :
Springer Science and Business Media Deutschland GmbH, England
RJM has received travel bursaries from Kyowa Kirin. OB has received consulting or lecture fees from Amgen, Aptissen, Biophytis, IBSA, Mylan, Novartis, Orifarm, Sanofi, UCB and Viatris. MC has received honoraria for speaking and chairing engagements from AMGEN, DKSH and Kyowa Kirin. PGC has received speakers bureaus or consultancies from AbbVie, AstraZeneca, Bristol Myers Squibb, Eli Lilly, Galapagos, GlaxoSmithKline, Grunenthal, Merck, Novartis, Pfizer, Stryker and UCB, outside the submitted work. WHD is on the board of Directors for BioMarin, Seres, Mersana and Metagenomi, Chief Advisor to CEO at Angitia and is a former employee and shareholder at Eli Lilly and Amgen. MH has research grants paid to his institution from Amgen, Radius Health, and consulting fees from UCB. EVM reports research support and/or personal fees from Amgen, Eli Lilly, Fresenius Kabi, ObsEva and UCB, outside the submitted work. SO has received consultancy fees from Merck and Pfizer not related to the current work or therapeutic field. RR has speaker bureau or advisory board for Abiogen, Naturex, Nestlé and ObsEva. YAS reports lecture honoraria from Amgen, Eli Lilly and SAJA. SLS has acted as a consultant for Radius and Amgen, received funding from Radius for clinical trial and received travel support from Radius and grant support from Amgen. LSS discloses associations with Astrazeneca, Pfizer, Rigel, Eupraxia, Biosplice, EMDSerono, Horizon, Direct, Lilly, Kaniska, Protalix, Chemomab, TLC, SpineThera, Kyoto, PPD, Galvani, Urica, Transcode, Boehringer Ingelheim, Bristol Myers Squibb, Priovant, Roivant, Ampio, Aura, Aurinia, GSK, Xalud, Neumentum, Neema, Amzell, Applied Bio, Aptinyx, Bexson, Bone Med, Bone Therapeutics, Cancer Prevention, Cerebral Therapeutics, Chemocentryx, Diffusion Bio, Elorac, Enalare, Foundry Therapeutics, Galapagos, Histogen, Gilead, Idera, Intravital, Ingel, Kiel Labs, Mesoblast, Mpathix, Minerva, Regenosine, Samus, Sana, StageBio, Theraly, Unity and Viridian. FT reports personal fees from Amgen, Eli Lilly, Fresenius, Stada and UCB outside the submitted work. CC reports personal fees from ABBH, Amgen, Eli Lilly, GSK, Medtronic, Merck, Novartis, Pfizer, Roche, Servier and Takeda, outside the submitted work. NCH reports personal fees, consultancy, lecture fees and honoraria from Alliance for Better Bone Health, AMGEN, MSD, Eli Lilly, Servier, Shire, Consilient Healthcare and Internis Pharma, outside the submitted work. JYR, NMA, JAT, CB, OB, NB, MCD, WHD, ADP, PH, PH, JAK, DPA, RPR, SLS, TVS and AL report no conflicts of interest related to this work.The ESCEO Working Group was funded by the ESCEO. The ESCEO receives unrestricted educational grants to support its educational and scientific activities from non-governmental organisations, not-for-profit organisations, non-commercial or corporate partners. The choice of topics, participants, content and agenda of the Working Groups as well as the writing, editing, submission and reviewing of the manuscript are the sole responsibility of the ESCEO, without any influence from third parties. PGC is supported in part by the UK National Institute for Health and Care Research (NIHR) Leeds Biomedical Research Centre. The views expressed are those of the authors and not necessarily those of the NIHR, the Department of Health and Social Care or the World Health Organisation.
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