Proposed EU incentives and regulatory obligations for paediatric medicines announced

The European Commission announced its proposed package of measures and incentives for encouraging the development of medicines specifically for children at the EFPIA (the European Federation of Pharmaceutical Industries and Associations) annual meeting in May 2003. The proposals have not been formally published but the Commission proposes a two-step approach, with two separate Regulations governing medicinal products covered by a present or supplementary protection certificate (SPC) and those not covered. An extended impact assessment will be conducted to look at the various possible policy options and the proposals may become more firm early in 2004.

For patented medicines, an additional six months of exclusivity is proposed for new paediatric indications to be added to the existing SPC but this would not be retrospective if the SPC had already expired.

For off-patent medicines, a full marketing authorisation would be needed covering the paediatric indication and may obtain ten years of data protection, instead of the indication being treated as a line extension.

Patented medicines

It is proposed that all marketing authorisation applications for paediatric medicines will have to produce a Paediatric Evaluation Plan (PEP). This would be necessary for new paediatric indications, new dosage forms and new routes of administration of authorised medicinal products unless the requirements for such PEP is waived or deferred. It should contain detailed timings and measures, all relevant paediatric sub-populations and the paediatric formulations.

It would be for the EMEA (the European Agency for the Evaluation of Medicinal Products) to consider the significant benefit to the paediatric population considering the products already authorised for paediatric use. It may request modifications and will give a positive or negative opinion, with the possibility of appeal. The EMEA's opinion would be forwarded to the CPMP (the Committee for Proprietary Medicinal Products) and to the national competent authorities so that the agreed PEP serves as the basis for evaluation of the marketing authorisation application.

Other elements of the draft Regulation are the establishment of a paediatric clinical trials network, an inventory of paediatric therapeutic needs, paediatric protocol assistance and a database on paediatric clinical trials. There may also be the possibility of fee waivers and reductions and the requirement to make a commitment to market the medicine for paediatric uses.

Off-patent medicines

The second draft Regulation proposes a "paediatric marketing authorisation", either for a new formulation of an authorised medicinal product or for a product containing a previously authorised active substance, developed for a new paediatric use. The proposal is that a full marketing authorisation covering the paediatric indication would be needed and the medicine would in effect be a new product with possibly ten years of data protection.

This second draft Regulation calls for the establishment of a Paediatric Study Fund to be administered by EMEA from Community and/or national funds. The priorities for study would be identified from the inventory set up by the Regulation for patented medicines. There may well be the possibility of fee reductions and waivers and a requirement for a commitment to market the medicine.

Better medicines for children

These informal proposals largely reflect the comments received on the Commission's 2002 Consultation Paper "Better Medicines for Children". The paper had received support for the aim of improving the availability of medicines specifically for children though there was some concern that incentives would result in medicines being developed for financial gain rather than for the benefit of children. It is interesting to note that the informal proposal is for an additional six months of exclusivity for new paediatric indications of patented products whereas all parties who expressed views in the consultation recommended 12 months. The Commission's idea of adopting an approach similar to that in that US, which routinely requires studies in paediatric populations as part of the marketing authorisation application requirements seems to have been dropped. However, industry will have a better idea of what to expect after the impact assessment and the publication of the formal proposals early in 2004.

For further information, please contact Zelda Pickup on +44 (0) 207 367 2043, email [email protected]; or Natalie Wood on +44 (0) 207 367 2523, email [email protected].