The European Medicines Agency’s (EMEA) Paediatric Committee (PDCO) adopted a total of 70 positive opinions and one negative opinion on paediatric investigation plans (PIPs) or waivers from these requirements during its first year of operation.

The PDCO held its first meeting on 4 July 2007 and its latest on 2-4 July 2008. At the anniversary meeting, EMEA executive director Thomas Lönngren said setting up the PDCO was “the most significant achievement of the Agency in 2007”. PDCO chair Dr Daniel Brasseur noted that nearly two-thirds of the decisions issued by the committee in its first year were for as yet unlicensed products, which would “undoubtedly have a dramatic impact on future drug development, both for children and adults”.

Under the European Union’s regulation on medicinal products for paediatric use (No. 1901/2006), companies seeking approval either for a new medicinal product or for a new indication, route of administration or pharmaceutical form of an existing patent-protected drug must submit a PIP detailing their strategy for developing the drug in all subsets of the paediatric population. The trade-off for meeting the requirements of an agreed PIP is a six-month extension to the product’s supplementary protection certificate.

Waivers from these obligations are available where there is evidence that the drug or class of drugs is likely to be ineffective or unsafe in part or all of the paediatric population; the disease or condition targeted by the product occurs only in adult populations (‘class waivers’); or the drug concerned does not present a significant therapeutic benefit over existing treatments for paediatric patients.

In all, the PDCO received 233 validated applications, involving some 420 indications, for PIPS or waivers between August 2007 and July 2008. Of these, 49 were requests for a full waiver covering all conditions and subsets of the paediatric population. On average, every application for a PIP or full waiver includes two indications, which each require a separate scientific evaluation. If an indication covers more than one age group, these must also be assessed separately.

During the year, the PDCO adopted 39 positive opinions on PIPs and 31 on full waivers. The EMEA subsequently adopted decisions on 15 of the PIP opinions and 16 of the waiver opinions.

Not yet authorised

The majority (61%) of the applications for PIPs or waivers concerned medicines that were not yet authorised, while a further 36% were for medicines that were already approved and still under patent – i.e., the applicant was seeking a variation or extension of the original marketing authorisation for a new indication, route of administration or pharmaceutical form.

Applications for off-patent medicines developed specifically for children with an age-appropriate formulation made up 3% of applications to the PDCO.

The therapeutic area most often addressed by applications for PIPs or waivers was oncology, with 19% of the total, closely followed by endocrinology, gynaecology and metabolism (17%), then cardiovascular disorders (13%). Orphan drugs accounted for 17% of applications to the PDCO in its first year, with 73% of these submitted for PIPs and 23% for waivers.

Other activities completed by the Committee during the year included:

- a guidance document on the content and format of data to be collected by EU member states on all existing uses of medicines in the paediatric population;
- a list of class waivers for conditions that do not affect children and for which the requirement to submit a PIP can therefore be waived;
- a proposal, adopted by the EMEA Management Board, for a strategy to implement an EU-wide network of paediatric research;
- a priority list for studies into off-patent medicines in response to a call from the European Commission for funding through the EU’s Seventh Framework Programme;
- a recommendation to the European Commission on a proposal that medicines authorised for use in children should carry a specific symbol (the Commission took up the PDCO’s recommendation that there was no symbol for which the benefits outweighed the risks of medication error).