On September 29 2004, the European Commission issued a proposal for a regulation on medicinal products (pharmaceuticals) for paediatric use. The area of medicines for paediatric population - those between 0 and 18 years of age - is viewed as a serious legislative vacuum in the EU. While any medicine must be authorized for use in adults - the authorization depending on the outcome of extensive testing including pre-clinical tests and clinical trials to ensure that it is safe, of high quality and effective - there are no legislative requirements concerning medicines for children.

To stimulate research and testing by pharmaceutical firms on paediatric use, the Proposal is shaped as a combination of obligations and incentives. An applicant may be granted a six month extension of the duration of its patent, if the application for marketing authorization is for a product covered by a patent or by a supplementary protection certificate and is accompanied by the results of all the measures taken under an agreed completed paediatric investigation plan. There also is the prospect of 10 years regulatory exclusivity for off-patent products with a paediatric use marketing authorization.

The Proposal has a broad scope and it would affect:

• medicinal products not yet authorized for marketing;
• authorized medicinal products covered by a patent or a supplementary protection certificate extending patent protection; and
• authorized medicinal products no longer covered by a patent or a supplementary protection certificate.

The main features of the draft Proposals are:

• an advisory expert committee, the Paediatric Board (PB) consisting of ethicists and representatives of the member states, associations of professional and patient representative organizations, and the EMEA Committee for Human Medicinal Products (CHMP) which would give opinions on paediatric investigation plans and on requests for waivers or variations from data requirements;
• the assessment of the results of the tests performed under the legislation by the EMEA CHMP for centrally authorized products, or the national competent authorities for products under the decentralized marketing authorization procedure, which may be after input from the PB;
• a requirement that an application for a marketing authorization for a new medicinal product as well as any application for authorization of a new indication, new pharmaceutical form, or new route of administration must include the results of studies in children according to an agreed paediatric investigation plan and describe how the long-term follow-up of efficacy and possible adverse reactions will be ascertained;
• studies on children may be conducted only if they offer potential benefit to children;
• waivers or deferrals from paediatric data requirements are available in some cases;
• funding for research into paediatric use of off-patent medicines (Medicines Investigation for the Children of Europe - MICE);
• an inventory by the PB to help identify unmet needs of the paediatric population, the extent to which medicines corresponding to these needs are available, and priorities for research as needed to assess paediatric investigation plans;
• member state surveys, and reporting to the EMEA, of all existing uses of medicinal products by health care professionals for paediatric indications and provide these data to the EMEA; and
• creation of a European database with details on all trials started as part of a paediatric investigation plan and on results of all studies conducted in accordance with such plans.

The European Council and the European Parliament are considering the Commission's proposal. Final adoption is not expected before the end of 2006.