Mobility endpoints in marketing authorisation of drugs: what gets the European medicines agency moving?

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• dc.contributor.author Jaeger, Simon U.
• dc.contributor.author Wohlrab, Martin
• dc.contributor.author Schoene, Daniel
• dc.contributor.author Tremmel, Roman
• dc.contributor.author Chambers, Michael
• dc.contributor.author Leocani, Letizia
• dc.contributor.author Corriol-Rohou, Solange
• dc.contributor.author Klenk, Jochen
• dc.contributor.author Sharrack, Basil
• dc.contributor.author García Aymerich, Judith
• dc.contributor.author Rochester, Lynn
• dc.contributor.author Maetzler, Walter
• dc.contributor.author Puhan, Milo A.
• dc.contributor.author Schwab, Matthias
• dc.contributor.author Becker, Clemens
• dc.date.accessioned 2022-04-26T05:57:12Z
• dc.date.available 2022-04-26T05:57:12Z
• dc.date.issued 2022
• dc.description.abstract Background: Mobility is defined as the ability to independently move around the environment and is a key contributor to quality of life, especially in older age. The aim of this study was to evaluate the use of mobility as a decisive outcome for the marketing authorisation of drugs by the European Medicines Agency (EMA). Methods: Fifteen therapeutic areas which commonly lead to relevant mobility impairments and alter the quantity and/or the quality of walking were selected: two systemic neurological diseases, four conditions primarily affecting exercise capacity, seven musculoskeletal diseases and two conditions representing sensory impairments. European Public Assessment Reports (EPARs) published by the EMA until September 2020 were examined for mobility endpoints included in their 'main studies'. Clinical study registries and primary scientific publications for these studies were also reviewed. Results: Four hundred and eighty-four EPARs yielded 186 relevant documents with 402 'main studies'. The EPARs reported 153 primary and 584 secondary endpoints which considered mobility; 70 different assessment tools (38 patient-reported outcomes, 13 clinician-reported outcomes, 8 performance outcomes and 13 composite endpoints) were used. Only 15.7% of those tools distinctly informed on patients' mobility status. Out of 402, 105 (26.1%) of the 'main studies' did not have any mobility assessment. Furthermore, none of these studies included a digital mobility outcome. Conclusions: For conditions with a high impact on mobility, mobility assessment was given little consideration in the marketing authorisation of drugs by the EMA. Where mobility impairment was considered to be a relevant outcome, questionnaires or composite scores susceptible to reporting biases were predominantly used.
• dc.description.sponsorship S.J., M.W., and R.T. are partly supported by the Robert Bosch Stiftung Stuttgart. M.W. reports grants from HORIZON2020 IMI No. 820820, during the conduct of the study. D.S. reports grants from HORIZON2020 IMI No. 820820, during the conduct of the study. M.C. reports personal fees from Takeda Pharmaceuticals, during the conduct of the study; personal fees from Takeda Pharmaceuticals, outside the submitted work. J.K. reports grants from HORIZON2020 IMI No. 820820, during the conduct of the study. J.G.A. reports grants from HORIZON2020 IMI No. 820820, and from AstraZeneca, Chiesi, Esteve, outside the submitted work. W.M. receives or received funding from the European Union, the German Federal Ministry of Education of Research, Michael J. Fox Foundation, Robert Bosch Foundation, Neuroalliance, Lundbeck and Janssen. He received speaker honoraria from Abbvie, Bayer, GlaxoSmithKline, Licher MT, Rölke Pharma and UCB, was invited to Advisory Boards of Abbvie, Biogen, Lundbeck and Market Access & Pricing Strategy GmbH, and is an advisory board member of the Critical Path for Parkinson’s Consortium. He serves as the co-chair of the MDS Technology Task Force. M.P. reports grants from HORIZON2020 IMI No. 820820, outside the submitted work. M.S. is supported by the Robert Bosch Stiftung Stuttgart and reports grants from HORIZON2020 IMI 2 Mobilise D, during the conduct of the study, and grants and non-financial support from Green Cross WellBeing Co. Ltd., Gilead Sciences Inc., Robert Bosch GmbH, and CORAT Therapeutics GmbH, as well as other from Agena Bioscience GmbH, outside the submitted work. C.B. disclosed consultation from E. Lilly and speaker fees from Amgen, Nutricia and Pfizer reports grants from HORIZON2020 IMI No. 820820, during the conduct of the study.
• dc.format.mimetype application/pdf
• dc.identifier.citation Jaeger SU, Wohlrab M, Schoene D, Tremmel R, Chambers M, Leocani L, Corriol-Rohou S, Klenk J, Sharrack B, Garcia-Aymerich J, Rochester L, Maetzler W, Puhan M, Schwab M, Becker C. Mobility endpoints in marketing authorisation of drugs: what gets the European medicines agency moving?. Age Ageing. 2022 Jan 6;51(1):afab242. DOI: 10.1093/ageing/afab242
• dc.identifier.doi http://dx.doi.org/10.1093/ageing/afab242
• dc.identifier.issn 0002-0729
• dc.identifier.uri http://hdl.handle.net/10230/52889
• dc.language.iso eng
• dc.publisher Oxford University Press
• dc.relation.ispartof Age Ageing. 2022 Jan 6;51(1):afab242
• dc.relation.projectID info:eu-repo/grantAgreement/EC/H2020/820820
• dc.rights © The Author(s) 2021. Published by Oxford University Press on behalf of the British Geriatrics Society. This is an Open Access article distributed under the terms of the Creative Commons Attribution NonCommercial-NoDerivs licence (https://creativecommons.org/licenses/by-nc-nd/4.0/), which permits non-commercial reproduction and distribution of the work, in any medium, provided the original work is not altered or transformed in any way, and that the work is properly cited. For commercial re-use, please contact journals.permissions@oup.com
• dc.rights.accessRights info:eu-repo/semantics/openAccess
• dc.rights.uri https://creativecommons.org/licenses/by-nc-nd/4.0/
• dc.subject.keyword European Public Assessment Reports
• dc.subject.keyword Clinical outcome assessment
• dc.subject.keyword Digital outcomes
• dc.subject.keyword Mobility
• dc.subject.keyword Older people
• dc.subject.keyword Real-world mobility
• dc.title Mobility endpoints in marketing authorisation of drugs: what gets the European medicines agency moving?
• dc.type info:eu-repo/semantics/article
• dc.type.version info:eu-repo/semantics/publishedVersion