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The Paediatric Regulation in the European Union


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Five years ago on January 26, 2007 a new regulation came into force with the objective to improve the health of children in Europe. In the past very often medicines have been given to children without prior investigations in this patient population. The assumption was that children were smaller adults. However, this is not true. Therefore, Regulation EC 2006/1901 on medicinal products for paediatric use has been established to facilitate the development and availability of medicines for children aged 0 to 18 years. The regulation was implemented to ensure that medicines for children are of high quality, developed under ethical criteria and authorized in accordance to the requirements in the EU.

 

In accordance with the regulation a specific scientific committee has been established, the Paediatric Committee. This committee consists of five members of the Committee for Medicinal Products for Human Use, one member and one alternate appointed by each European Union (EU) Member State that is not represented by the members appointed by the CHMP, three members and alternates representing healthcare professionals as well as three members and alternates representing patient associations. The main role of the PDCO is to assess Paediatric Investigation Plans and adopt opinions on them. However, the PDCO is not responsible for the assessment of marketing authorization applications (MAAs) for paediatric indications.

 

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Since July 26, 2008 it is mandatory for MAAs concerning new products to include “Information relating to Paediatrics” as part of Module 1 of the CTD. An MAA is only valid if the dossier contains the following information:

  • Results of paediatric studies conducted as specified in a PIP approved by the PDCO, this includes the check for compliance with the agreed PIP
  • Decision on granting a product-specific/class waiver
  • Decision on granting a deferral

Since January 26, 2009 the above is also mandatory for a medicinal product which is already approved in the EU and which is protected either by a Supplementary Protection Certificate (SPC) or by a patent which qualifies for granting a SPC extension and for which it is planned to introduce the following:

  • New indications, incl. paediatric indication
  • New pharmaceutical forms
  • New routes of administration

According to the Regulation EC 2006/1901 a PIP should include details of the timing and the measures proposed to demonstrate the quality, safety and efficacy of the medicinal product in the paediatric population.

 

This means that the PIP has to include information on tests and studies investigating the quality, the safety and the efficacy of the medicinal product in compliance with criteria which are applicable for marketing authorization. If relevant for the medicinal product and condition(s) concerned the PIP will include information on the appropriateness of the dosage form(s) used in adults or the development of specific formulations for certain age groups, additional nonclinical investigations necessary to amend the nonclinical programme, and clinical studies investigating the safety and efficacy of the product. The PIP will indicate the timing of the specified tests and studies and will cover all subsets of the paediatric population, i.e. from preterm to adolescents. It will compromise all indications the current and proposed ones.

 

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The Regulation EC 2006/1901 also stipulates early considerations on the PIP since Article 16 requires submission of the plan “…not later than upon completion of human pharmaco-kinetic studies in adults…”. This is certainly very challenging for pharmaceutical companies since at this point in time of the development of a medicinal product data are limited and the proof of concept has not been confirmed. However, as more and more information on the product will be available during development the PIP can be adapted to the new data and modified accordingly.

Modifications of the PIP are of importance since the content of the PIP is binding for the applicant and a compliance check will be conducted prior to MAA or variation submissions. This compliance check will be conducted by the PDCO for all submission types whether these are centralized or decentralized or mutual recognition procedures. It needs to be emphasized that the conducted studies will be scrutinized during the compliance check and the study reports will be compared in detail with the agreed PIP.

The necessity of preparing a PIP and conducting a paediatric development programme will very much depend on the condition(s) the medicinal product is intended for and safety concerns. There are three criteria when a PIP will be waived by the PDCO; this concerns products which are:

  • are likely to be ineffective or unsafe in part or all of the paediatric population;
  • are intended for conditions that occur only in adult populations;
  • do not represent a significant therapeutic benefit over existing treatments for paediatric patients.

The EMA has published a list of class waivers summarizing conditions for which no PIP is necessary (see Opinion of the Paediatric Committee on a class waiver on condition). In any case it is recommended to get feedback from the PDCO whether or not the class waiver applies to the particular product and the target indication. For conditions not listed the sponsor can also apply for a product specific waiver if one or more of the criteria listed above are met. The product specific waiver can cover all age groups or just some.

 

There is also the option to ask for a deferral to initiate or complete the paediatric development. The application for deferral will be included in a full PIP together with a sound justification why the deferral is necessary. Reasons for a deferral could be safety issues, e.g. that it is essential to have more data from studies conducted in adults and/or more nonclinical data needs to be obtained before starting the clinical development in the paediatric population. Furthermore, the development of specific paediatric formulation(s) might be difficult and time consuming.

 

No doubt the Paediatric Regulation means additional obligations and burden for the pharmaceutical industry. However, there are also incentives. These incentives include:

  • SPC extension for on-patent drugs (additional 6 months)
  • Extended marketing exclusivity for Orphan drugs (additional 2 years)
  • Paediatric Use Marketing Authorisation (PUMA) for off-patent drugs (8+2 years data protection)

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