EU Council Adopts EC Regulation on Paediatric Medicines

The Council of the European Union (EU) announced today that it has adopted the EC Regulation on medicinal products for paediatric use.

Today's press release states that the new Regulation aims to improve child health, as considered in more detail in earlier background material accompanying the original proposal. The Regulation sets out specific requirements for the authorisation and use of medicines in children, in response to a concern that around 50% of medicines used to treat children are in fact not tested or authorised on children. The improvement to the health of children in Europe is said to be likely to come from better research, development and authorisation of paediatric medicines. The new Regulation creates a new system through various strategies, which are stated as aimed at achieving better availability of paediatric medicines throughout the Community and removing obstacles to trade of medicinal products within the Community.

As well as dealing with the appropriate standards of research, clinical trial, and authorisation, the Regulation also looks to improve information on the use of paediatric medicines. Obligation in this area are to be accompanied by certain incentives created in the Regulation.

Of particular interest is the incentive with respect to medicines still protected by monopoly (patent or supplementary protection certificate (SPC)). The Regulation introduces new obligations to submit (as part of the procedure for market authorisation) results of clinical studies in accordance with a paediatric investigation plan or proof of a waiver where the medicines of no paediatric use. These obligations are accompanied by an incentive in the form of the extension of exclusive rights (an additional 6 months) on the basis that it takes longer to test and authorise paediatric medicines. In the case of SPCs, extensions are available only for products authorised in all Member States and for which marketing authorisation procedures are complete.

In the drafts leading to final text, discussions rejected proposals to exclude extensions for products that have already received the protection of data or market exclusivity for the same paediatric use in the EU. Similarly, the Commission rejected earlier drafts seeking to amend the proposed text to exclude an extension of the SPC for products that have already benefited from patent protection covering the paediatric use. The Commission claimed that such exclusions would compromise the policy objective of encouraging greater research into medicines for children. Nevertheless, in keeping with this rationale, importantly the extensions are available as an incentive only for research entered into after the new Regulation has entered into force.

However, in May this year, the European Court of Justice (ECJ) ruled out SPCs for new formulations of known active ingredients, in the Massachusetts Institute of Technology (C-431/04). This was despite an earlier opinion of the Advocate-General. The Court ruled: "Article 1(b) of the Regulation must be interpreted so as not to include in the concept of 'combination of active ingredients of a medicinal product' a combination of two substances, only one of which has therapeutic effects of its own for a specific indication, the other rendering possible a pharmaceutical form of the medicinal product which is necessary for the therapeutic efficacy of the first substance for that indication." This may limit the incentive in respect of extensions of SPCs for new uses (paediatric) of known products.

Regarding off-patent paediatric medicines, the Regulation introduces a new kind of marketing authorisation, referred to as the Paediatric Use Marketing Authorisation (PUMA). Such medicines must have been appropriately tested for paediatric use. The Regulation also includes provisions on the funding of research into the use of off-patent medicines for paediatric purposes.

Medicinal products for the treatment of serious diseases affecting fewer than 5 in 10 000 people in the Community are often referred to as "orphan drugs," because of their limited interest to the pharmaceutical industry. The Orphan Drug Regulation currently provides for 10 years market exclusivity, in order to provide incentives to bring such treatments to market, which the new Regulation is expected to extend to 12.

The Regulation also creates a new scientific committee, an expert committee within the European Medicines Agency (EMEA). This Committee will be primarily responsible for the assessment and agreement of paediatric investigation plans and waivers (described above).

The new Regulation amends 3 European instruments - Council Regulation (EEC) No 1768/92 creating supplementary protection certificate; Regulation (EC) No 726/2004 for authorisation and supervision of medicinal products for human and veterinary use (establishing the EMEA); and the Clinical Trials Directive 2001/20/EC. The original proposal text and amended text are available. The text of the adopted Regulation is likely to be published in the Official Journal in the next couple of weeks.