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Archives de pédiatrie
Volume 21, n° 10
pages 1129-1138 (octobre 2014)
Doi : 10.1016/j.arcped.2014.07.011
Received : 12 December 2013 ;  accepted : 23 July 2014
ICH E11 : développement d’un médicament pédiatrique : comparaison entre les États-Unis, l’Union Européenne et le Japon
Pediatric drug development: ICH harmonized tripartite guideline E11 within the United States of America, the European Union, and Japan
 

M. Pflieger a, D. Bertram b,
a CS 90632, délégation à la recherche clinique et à l’innovation, hôpital Pierre-Zobda-Quitman, centre hospitalier universitaire de Martinique, 97261 Fort-de-France, Martinique 
b Direction de la recherche clinique et de l’innovation, siège administratif, hospices civils de Lyon, BP 2251, 3, quai des Célestins, 69229 Lyon cedex 02, France 

Auteur correspondant.
Résumé

Pour parer au manque de médicaments pédiatriques, l’International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) a publié en 2000, la ligne directrice ICH E11 intitulée « Clinical Investigation of Medicinal Products in the Pediatric Population ». Son objectif est de promouvoir la recherche clinique en pédiatrie dans les régions ICH : les États-Unis, l’Union Européenne (UE) et le Japon. Cet article compare le cadre législatif, les obligations des industries pharmaceutiques et les mesures incitatives mises en place suite à la publication d’ICH E11. Les États-Unis et l’UE ont élaboré des réglementations spécifiques contrairement au Japon qui a seulement mis en place des mesures incitatives. Aux États-Unis et en Europe, les industries pharmaceutiques doivent, pour tout nouveau médicament, mettre en place un plan d’investigation pédiatrique (PIP) approuvé, dont les résultats sont soumis avec le dossier de demande d’autorisation de mise sur le marché (AMM). En Europe, le PIP doit être soumis pour validation après les études de phase I tandis qu’aux États-Unis, c’est au cours des études de phase II ou III. Un soutien direct des autorités compétentes via les comités d’experts est possible lors de l’élaboration du PIP. Enfin, un système de mesures incitatives, consistant principalement en une prolongation de la période d’exclusivité de mise sur le marché, a été mis en place. La traduction réglementaire de la ligne directrice ICH E11 au sein des régions ICH est maintenant disponible et encourage l’étude et le développement de médicaments pédiatriques, mais il est encore difficile de mesurer quel est son impact.

The full text of this article is available in PDF format.
Summary

To address the lack of appropriate pediatric drugs available on the global market, in 2000 the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) issued the ICH E11 guideline regarding the Clinical Investigation of Medicinal Products in the Pediatric Population. This guideline considerably changes the environment of drug development for children. It has been written specifically to harmonize, promote, and facilitate high-quality and ethical clinical research for children within the ICH regions, i.e., the United States of America (USA), the European Union (EU), and Japan. This article details the various regulations applicable in each ICH region following the publication of the guideline. The framework of rewards, incentives, and obligations for pharmaceutical companies established for the development of pediatric drugs are compared. It appears that the USA and the EU have both developed specific regulations for pediatric drug development while Japan has not. However, in Japan, pharmaceutical companies (PCs) are encouraged to develop pediatric drugs voluntarily, and they may be granted additional months of market exclusivity or the postponement of the drug re-examination deadline. In both the USA and the EU, regulations aimed to increase the number of clinical studies conducted in children, in order to ensure that the necessary data are generated, determining the conditions in which a drug may be authorized to treat the pediatric population. PCs are encouraged to develop pediatric assessment, including pediatric clinical trials, which is described in a pediatric plan submitted to the relevant authorities. A system of rewards for PCs submitting an application for marketing authorization containing pediatric use information has been put in place to cover the additional investment for testing drugs in children. Subject to conditions, these rewards consist in a 6-month extension of the patent or supplementary protection. Regarding the approval for new medicinal products in these two regions, regulations require PCs to include, when it is relevant, a pediatric assessment in their drug research and development plan, which must be approved. Although these regions have implemented the ICH guideline, the regulation differs with respect to the timing of studies in children relative to adults and approval of a pediatric drug development plan. Except for special cases, the pediatric investigation plan in the EU is required to be prepared and submitted to the competent authorities upon availability of adult pharmacokinetic studies (after phase I), which means at an early phase of a new drug development plan. In the USA, the pediatric plan is requested later during the phase II or III trials. In practice, it has become difficult for pharmaceutical industries to develop a practicable clinical program for pediatrics including timelines for studies in children that satisfy both EU and USA authorities. Nevertheless, at an early stage of the development strategy, direct support and advice from competent authorities can be obtained. For the ICH regions, pediatric committees are well-established albeit less structured in Japan. Their roles are to review and assess pediatric plans, to issue recommendations, to advise pharmaceutical companies on the content and format of pediatric data to be methodically collected and analyzed, and to avoid exposing children to unnecessary or redundant clinical trials. This regulatory framework encourages the study and the development of pediatric drugs, but it is still quite difficult to actually measure the impact of the ICH E11 on increasing the number of drugs for pediatric use.

The full text of this article is available in PDF format.

 Publication partielle ou communication préliminaire : Pflieger M. Le développement d’un médicament pédiatrique : la ligne directrice ICH E11 et sa mise en application aux Etats-Unis, dans l’Unions Européenne et au Japon [thèse]. Lyon : université Claude Bernard Lyon 1 ; 2012.



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