In addition to monopoly protection in the form of a patent and a supplementary protection certificate (SPC), medicinal products may also be protected by a paediatric extension, which extends the lifetime of an SPC by a further six months.
The governing legislation is the Paediatric Regulation (Regulation (EC) 1901/2006). The aim of the legislation, among other things, is to:
"facilitate the development and accessibility of medicinal products for use in the paediatric population, to ensure that medicinal products used to treat the paediatric population are subject to ethical research of high quality and are appropriately authorised for use in the paediatric population, and to improve the information available on the use of medicinal products in the various paediatric populations. These objectives should be achieved without subjecting the paediatric population to unnecessary clinical trials and without delaying the authorisation of medicinal products for other age populations."
The Paediatric Regulation therefore provides that an application for a new marketing authorisation for a medicinal product must include the results of all studies performed and details of all information collected in compliance with an agreed paediatric investigation plan (PIP).
In the case of medicines that are already authorised and protected either by a patent or an SPC, such results must be included in any application for the authorisation of new indications, including paediatric indications.
A PIP is defined in the Paediatric Regulation as:
“a research and development programme aimed at ensuring that the necessary data are generated determining the conditions in which a medicinal product may be authorised to treat the paediatric population”
Furthermore, the PIP "shall specify the timing and the measures proposed to assess the quality, safety and efficacy of the medicinal product in all subsets of the paediatric population that may be concerned. In addition it shall describe any measures to adapt the formulation of the medicinal product so as to make its use more acceptable, easier, safer or more effective for different subsets of the paediatric population."
The procedural steps are as follows:
A PIP should be drawn up and submitted to the EMA with a request for agreement.
The Paediatric Committee of the EMA then adopts an opinion as to whether or not the proposed studies will ensure the generation of the necessary data. Subject to any request for modifications from the applicant, the EMA will adopt a decision, annexing the opinion of the Paediatric Committee.
Assuming that the opinion was a positive one, the paediatric studies should be carried out so that the required results can be included in the application for a marketing authorisation or for a new indication.
A negative opinion may be adopted if the medicine is likely to be ineffective or unsafe in the paediatric population, if the disease to be treated only occurs in the adult population or if the medicine does not represent a significant therapeutic benefit over the existing treatments for paediatric patients. In such cases, production of the relevant results may be waived.
It is also possible for the measures set out in the PIP to be deferred, for instance, if it is appropriate for studies to be carried out only in adults first.
When the application for marketing authorisation is made, the competent authority responsible for granting a marketing authorisation must verify whether the application complies with the relevant requirements, including compliance with the agreed PIP. The Paediatric Committee may be asked to give its opinion as to compliance with the PIP.
Assuming it does, the marketing authorisation may be granted and the relevant results are included in the summary of product characteristics (SmPC) for the product, along with a statement indicating compliance with the agreed PIP. The applicant then receives the six month extension to the SPC.
It should be noted that the extension is granted on the basis that the application for marketing authorisation contains the necessary results. It is not necessary for the product actually to be indicated for use in the paediatric population (for example, if the results show that that would not be appropriate).
If the requirement for compliance with a PIP has been waived or deferred, no results will be included in the SmPC and there will be no six month extension.
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