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BMJ Paediatrics Open Is Committed to Open Peer Review. As Part of This Commitment We Make the Peer Review History of Every Article We Publish Publicly Available bmjpo: first published as 10.1136/bmjpo-2020-000880 on 30 December 2020. Downloaded from BMJ Paediatrics Open is committed to open peer review. As part of this commitment we make the peer review history of every article we publish publicly available. When an article is published we post the peer reviewers’ comments and the authors’ responses online. We also post the versions of the paper that were used during peer review. These are the versions that the peer review comments apply to. The versions of the paper that follow are the versions that were submitted during the peer review process. They are not the versions of record or the final published versions. They should not be cited or distributed as the published version of this manuscript. BMJ Paediatrics Open is an open access journal and the full, final, typeset and author-corrected version of record of the manuscript is available on our site with no access controls, subscription charges or pay- per-view fees (http://bmjpaedsopen.bmj.com). If you have any questions on BMJ Paediatrics Open’s open peer review process please email [email protected] http://bmjpaedsopen.bmj.com/ on September 25, 2021 by guest. Protected copyright. BMJ Paediatrics Open bmjpo: first published as 10.1136/bmjpo-2020-000880 on 30 December 2020. Downloaded from Confidential: For Review Only Does the EU’s Paediatric Regulation work for new medicines for children in Nordic countries? A retrospective database review. Journal: BMJ Paediatrics Open Manuscript ID bmjpo-2020-000880 Article Type: Original research Date Submitted by the 16-Sep-2020 Author: Complete List of Authors: Lepola , Pirkko ; Helsinki University Central Hospital, Children and Adolescents Wang, Siri; Norwegian Medicines Agency Tötterman, Ann Marie; Finnish Medicines Agency Fimea Gullberg, Ninna; Swedish Medicinal products Agency Moll Harboe, Kirstine ; Danish Medicines Agency Kimland, Elin; Swedish Medicinal Products Agency Keywords: Therapeutics, Pharmacology http://bmjpaedsopen.bmj.com/ on September 25, 2021 by guest. Protected copyright. https://mc.manuscriptcentral.com/bmjpo Page 1 of 16 BMJ Paediatrics Open 1 2 3 bmjpo: first published as 10.1136/bmjpo-2020-000880 on 30 December 2020. Downloaded from 4 5 6 7 8 9 I, the Submitting Author has the right to grant and does grant on behalf of all authors of the Work (as defined 10 in the below author licence), an exclusive licence and/or a non-exclusive licence for contributions from authors 11 who are: i) UK Crown employees; ii) where BMJ has agreed a CC-BY licence shall apply, and/or iii) in accordance 12 with the termsConfidential: applicable for US Federal Government For officers Review or employees actingOnly as part of their official 13 duties; on a worldwide, perpetual, irrevocable, royalty-free basis to BMJ Publishing Group Ltd (“BMJ”) its 14 licensees and where the relevant Journal is co-owned by BMJ to the co-owners of the Journal, to publish the 15 Work in this journal and any other BMJ products and to exploit all rights, as set out in our licence. 16 17 The Submitting Author accepts and understands that any supply made under these terms is made by BMJ to 18 the Submitting Author unless you are acting as an employee on behalf of your employer or a postgraduate 19 student of an affiliated institution which is paying any applicable article publishing charge (“APC”) for Open 20 Access articles. Where the Submitting Author wishes to make the Work available on an Open Access basis (and 21 intends to pay the relevant APC), the terms of reuse of such Open Access shall be governed by a Creative 22 Commons licence – details of these licences and which Creative Commons licence will apply to this Work are set 23 out in our licence referred to above. 24 25 Other than as permitted in any relevant BMJ Author’s Self Archiving Policies, I confirm this Work has not been 26 accepted for publication elsewhere, is not being considered for publication elsewhere and does not duplicate 27 material already published. I confirm all authors consent to publication of this Work and authorise the granting 28 of this licence. 29 30 31 32 33 34 35 http://bmjpaedsopen.bmj.com/ 36 37 38 39 40 41 42 43 44 45 on September 25, 2021 by guest. Protected copyright. 46 47 48 49 50 51 52 53 54 55 56 57 58 59 60 https://mc.manuscriptcentral.com/bmjpo BMJ Paediatrics Open Page 2 of 16 version_16092020_corr. Page 1 / 13 1 2 3 Does the EU’s Paediatric Regulation work for new medicines for children in Nordic bmjpo: first published as 10.1136/bmjpo-2020-000880 on 30 December 2020. Downloaded from 4 5 countries? A retrospective database review. 6 7 Correspondence: 1 8 Pirkko Lepola , 9 1University of Helsinki and Helsinki University Hospital, Department of Children and Adolescents 10 Address: Stenbäckinkatu 11, 00029 Helsinki, Finland 11 Tel.: +358-50-5142646 12 Confidential: For Review Only e-mail: [email protected] 13 14 15 Co-authors: 16 Siri Wang2, Ann Marie Tötterman3, Ninna Gullberg4, Kirstine Moll Harboe5 and Elin E. Kimland4 17 2Norwegian Medicines Agency (NoMA), N-0213 Oslo, Norway; [email protected] 18 3Finnish Medicines Agency (FIMEA), FI-00034 FIMEA, Finland; [email protected] 19 4Swedish Medicinal products Agency (MPA), 751 03 Uppsala, Sweden; [email protected] 20 21 (N.G.); [email protected] (E.K.) 22 5Danish Medicines Agency, 2300 Copenhagen S, Denmark; [email protected] 23 24 Keywords: 25 child; age-appropriate formulation; marketing authorisation; paediatric medicines; paediatric 26 27 regulation; medicines availability; access to medicines 28 29 What is known about the subject? 30 The aim of the Paediatric Regulation (EU 1901/2006) is to improve the health of children by 31 facilitating the development and availability of new medicines. 32 33 34 In 2017, the European Medicines Agency (EMA) published 10-year report, and concluded, 35 that a significant number of new medicines for children have been authorised. http://bmjpaedsopen.bmj.com/ 36 37 Marketing authorisation is no guarantee that a medicine is available for patients, and the 38 access to medicines varies across countries. 39 40 41 What this study adds? 42 In 2019, 22 to 36% of the new medicinal products authorised for children (between 2007 43 and 2016) were not marketed across the four Nordic countries. 44 45 on September 25, 2021 by guest. Protected copyright. 46 Of the new paediatric formulations, 23 to 49% were not marketed, and a significant 47 proportion had never been marketed across the four Nordic countries. 48 49 Despite the intentions of the EU Paediatric Regulation, medicines targeted at children are 50 not all marketed, risking limitations in access and availability. 51 52 53 54 55 56 57 58 59 60 https://mc.manuscriptcentral.com/bmjpo Page 3 of 16 BMJ Paediatrics Open version_16092020_corr. Page 2 / 13 1 2 3 ABSTRACT bmjpo: first published as 10.1136/bmjpo-2020-000880 on 30 December 2020. Downloaded from 4 5 Objective 6 7 The aim of this study was to assess the marketing status of the new paediatric medicinal products 8 authorised for children between 2007 and 2016 reflecting the product availability in four Nordic 9 countries. 10 11 Design 12 Confidential: For Review Only 13 This is a retrospective analysis of the national Medicine Agency’s databases in Denmark, Finland, 14 Norway and Sweden. 15 16 Setting 17 18 New medicinal products with paediatric indications and new formulations were reviewed according 19 to the list of products in the Annex of European Medicines Agency´s (EMA) EU Paediatric Regulation 20 21 10-year report. The products were classified according to national marketing status; whether a 22 product was still authorised and whether the product was marketed at the time of the study period 23 between January and March 2019. 24 25 Main outcome measures 26 27 The main outcomes are the proportions of the new products which still had a valid marketing 28 authorisation and proportions of products that were being marketed, both in terms of the sums of all 29 countries and separately for each country. 30 31 Results 32 33 Across the four countries, 22 to 36% of the new medicinal products initially authorised for children 34 (between 2007 and 2016) were not marketed. Of the new formulations relevant to children, 23 to 49% 35 were not marketed, and a significant proportion of these products had never been marketed. http://bmjpaedsopen.bmj.com/ 36 37 Conclusions 38 39 This study reflects the reality of the implementation of the Paediatric Regulation. The results show 40 that several new medicinal products and new formulations targeted at children are not marketed. This 41 directly affects the availability of these medicines. These findings indicate the need to further 42 43 investigate how to facilitate the availability of new medicines for children across Europe. 44 45 on September 25, 2021 by guest. Protected copyright. 46 INTRODUCTION 47 48 The benefit of new therapies has not reached children to the same extent as the adult population 49 throughout the history of drug development. Children still lack age-appropriate medicines, dosing and 50 51 labelling information for many medicinal products. Increased knowledge and revised attitudes have 52 prompted practical actions to improve the situation in the form of new legislation. In Europe, the 53 Paediatric Regulation (EU 1901/2006 and 1902/2006) was implemented on 26th of January 2007 [1]. 54 55 The aim of the Paediatric Regulation is to improve the health of children in Europe by facilitating the 56 development and availability of medicines for children. To achieve this, the regulation includes a 57 system of obligations, rewards, and incentives for the pharmaceutical industry.
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