EMEA expects 'hundreds of applications' for trials in children

The European Medicines Agency (EMEA) is preparing for 'hundreds of applications' for paediatric investigation plans when its new Paediatric Committee starts work in six months' time.

The Paediatric Regulation, which came into force on 26 January, aims to improve the health of Europe's children by:

• stimulating research and development of medicines for use in children;
• ensuring that medicines used to treat children are appropriately tested and authorised;
• improving the availability of information on the use of medicines in children.

For drugs already on the market companies will also have the option of voluntarily submitting a paediatric investigation plan (PIP) for approval and then conducting trials.

The paediatric committee will assess a company's PIP and then either approve or modify it. The committee will also have the option to grant a waiver if, for example, the drug will not be used in children, although the burden is now on companies to provide compelling evidence that paediatric research does not need to be carried out. The committee can also grant a deferral if the experts believe it is better or safer that paediatric data is collected at a later date.

The findings of studies in children will be analysed by the CHMP in what will become a routine part of the assessment of filings.

The Paediatric Committee will also work with the EU Member States - building on work already performed by the EMEA's Paediatric Working Party (PEG) - to establish an inventory of the therapeutic needs of children, so that focus can be placed on the research, development and authorisation of medicines in areas where there are unmet medical needs. Committee experts will also be advising the EMEA on its development of a European network for clinical trials in children, to be based on existing networks.

'Companies cannot yet apply to the Paediatric Committee, but we are expecting to get several hundred applications in the beginning (when the committee goes live) in June,' said EMEA executive director Thomas Lonngren.

However, he said the EMEA would take neither anticipated filing dates nor when a drug was set to lose patent protection into account when dealing with the expected initial glut of submissions. The legislation demanded that PIPs were processed within 60 days of submission and the EMEA would set out the meet the target.

The initial implementation of the paediatric legislation also needed a light touch, he added and suggested that the use of deferrals may be higher in the early days of the process. Lonngren said: 'We don't want to delay the authorisation of drugs for use in adults.'

At present, children with a wide range of conditions are given lower doses of medication designed for adults, requiring doctors to estimate an appropriate dose. According to the Association of the British Pharmaceutical Industry (ABPI), an estimated 90% of children in neonatal intensive care units are given unlicensed medicines; 45% of medicines used on general children's wards and up to 20% of drugs prescribed to children by GPs are similarly intended for adult use.

This can mean an increased risk of dangerous side-effects or ineffective treatment because of under-dosage, says the ABPI. The new rules seek to prevent children being subjected to unnecessary clinical trials, but acknowledge that ethical concerns are offset by the current risks in giving off-label medicines.

'This new legislation represents a huge opportunity for European research into children's medicines and the UK is probably the most prepared country in the EU to take this forward,' said ABPI medical director Dr Richard Tiner. England, Scotland and Wales all have children's research networks of leading paediatricians in NHS paediatric hospitals sharing knowledge with scientists in industrial and academic institutions, he added.

'Clinical trials obviously raise questions of ethics, plus each stage of a child's development from infant to teenager requires different formulations. As a result patient numbers per drug may be small - especially for rare conditions - in the past this made it difficult for drug companies to recoup the massive costs of research,' Dr Tiner pointed out.

In return for taking on the time and cost of such difficult research, companies will receive a six-month extension to the patent life of new medicines in Europe. For older medicines that are already off-patent, a 10-year data exclusivity incentive is provided for the company that undertakes the research.