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New Information on Old Medicinal Products: A Cross-Sectional Analysis of Guidance for Paediatric Use for Substances on the European Priority List of Off-Patent Medicinal Products

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Abstract

Background

As part of the European Paediatric Regulation, the European Medicines Agency (former European Medicines Evaluation Agency) and the Paediatric Working Party (precursor for the Paediatric Committee) revised a priority list for studies on off-patent medicinal products in 2007 where a need for studies on paediatric medicinal products was emphasised.

Objectives

We aimed to evaluate the status of guidance for paediatric use in the Summary of Product Characteristics for medicinal products on the priority list as well as the presence and status of Paediatric Investigation Plans for these medicinal products.

Methods

We included active pharmaceutical ingredients on the priority list authorised through the centralised procedure and/or marketed in Denmark. The status of guidance for paediatric use (indication, posology and/or contraindication) was reviewed from the most recent Summary of Product Characteristics uploaded on the European Medicines Agency or the Danish Medicines Agency website as of November 2020. Information on Paediatric Investigation Plans status (Paediatric Committee opinion, completion and waivers granted) was retrieved from the European Medicines Agency website.

Results

A total of 121 active pharmaceutical ingredients were included in this study. Seventy-one percent had guidance for paediatric use in the Summary of Product Characteristics for at least one paediatric subpopulation, more often concerning adolescents (70%) and children (70%) as compared with neonates (41%) and infants (49%). The guidance included a paediatric indication in 46% of the cases, but less often a contraindication (13%). Thirty-three active pharmaceutical ingredients had an agreed Paediatric Investigation Plan, six of these were completed.

Conclusions

Most active pharmaceutical ingredients from the priority list had guidance for paediatric use in the Summary of Product Characteristics. However, there is still an unmet need in relation to guidance for use for the youngest paediatric subpopulation.

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Authors and Affiliations

  1. Copenhagen Centre for Regulatory Science, Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark

    Ann-Katrine Birkelund Mogensen, Helle Christiansen, Marie Louise De Bruin & Christine Erikstrup Hallgreen

  2. Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands

    Marie Louise De Bruin

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Contributions

AKM led the study conception and design, data collection, quality of information assessment of monitoring instructions, data analysis and interpretation of data. CEH, HC and MLDB were involved in the study concept and design, data analysis and interpretation of data. All four authors participated in the manuscript preparation, editing and revision and agreed upon the final version of the paper. All four authors agree to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

Corresponding author

Correspondence to Christine Erikstrup Hallgreen.

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No external funds were used in the preparation of this article.

Conflicts of interest/competing interests

Christine Erikstrup Hallgreen is employed by the University of Copenhagen at the Copenhagen Centre for Regulatory Science (CORS). CORS is a cross-faculty university anchored institution involving various public (Danish Medicines Agency, Copenhagen University) and private stakeholders (Novo Nordisk, Lundbeck, Ferring Pharmaceuticals, LEO Pharma) as well as patient organisations (Rare Diseases Denmark). The centre is purely devoted to the scientific aspects within the regulatory field and with a patient-oriented focus whilst the research is not a company-specific product or directly company related. Helle Christiansen is a PhD student at CORS. Her project is funded by a grant from Lundbeck A/S to the CORS. At the time of the study, Marie Louise De Bruin was an employee at CORS. Currently, she is employed by Utrecht University to conduct research under the umbrella of the Utrecht Centre for Pharmaceutical Policy and Regulation. This centre receives no direct funding or donations from private parties, including the pharma industry. Research funding from public-private partnerships, for example, IMI, The Escher Project (http://escher.lygature.org/), is accepted under the condition that no company-specific study is conducted. The centre has received unrestricted research funding from public sources, for example, World Health Organization, Netherlands Organisation for Health Research and Development, the Dutch National Health Care Institute, European Commission Horizon 2020, the Dutch Medicines Evaluation Board and the Dutch Ministry of Health. Ann-Katrine Birkelund Mogensen was during the study period from August 2020 to January 2021 a master thesis student at the University of Copenhagen at CORS. During this period, AKM worked part-time as a student assistant in the generic pharmaceutical company Sandoz A/S. After graduation in January 2021, and currently, AKM is working at Sandoz A/S. Sandoz A/S was not involved in any aspect of this study.

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Data collected for the purpose of this study are included in this published article as supplementary information.

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Mogensen, AK.B., Christiansen, H., De Bruin, M.L. et al. New Information on Old Medicinal Products: A Cross-Sectional Analysis of Guidance for Paediatric Use for Substances on the European Priority List of Off-Patent Medicinal Products. Pediatr Drugs 24, 679–687 (2022). https://doi.org/10.1007/s40272-022-00530-1

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