European paediatric medicines regulation

The European Paediatric Medicines Regulation is well into its fourth year. Following early concerns that the challenges of compliance outweighed the benefits of the incentives offered, many new procedures and processes have been circulated to aid compliance.

The regulation, which came into force in January 2007, requires drug developers to submit a Paediatric Investigation Plan (PIP) to the Paediatric Committee (PDCO).

The PIP was introduced after estimations that 50% to 90% of medicines used to treat children have never been specifically studied in or approved for the paediatric population. Prescribing errors, lack of paediatric formulations or confusing medication labelling has resulted in injuries and fatalities to babies and children, a few of which were fatal.

The regulation is designed to improve the health of children in Europe by ensuring paediatric medicines are subject to high-quality research and clinical development. All medicines must be ethically researched and authorised appropriately. The resulting products are duly authorised across the EU and, in turn, there is more information on the use of medicines developed specifically for children and more transparency in paediatric clinical trials. The legislation guards against subjecting children to unnecessary trials and delaying the authorisation of medicines for use in adults.

The PIP is a plan for adequately defining the efficacy and safety of certain medicinal products in all proposed and approved indications across all paediatric age groups - namely preterm neonates, term neonates, infants and toddlers, children (aged 2-11 years) and adolescents (up to 18 years).

This development plan can be modified at a later stage, with increasing knowledge and if the applicant encounters such difficulties with its implementation as to render the plan unworkable or no longer appropriate.

The PDCO may grant a waiver from submitting a PIP for certain indications, paediatric age groups and/or a deferral of the start or completion of certain studies or measures in the PIP. The PDCO has granted waivers for classes of drugs that are used to treat conditions only occurring in adults, however in all other cases, waivers must be applied for on a product-specific basis.

Incentives and opportunity

The Paediatric Regulation dramatically changes the regulatory environment for paediatric medicines in Europe. Upon its introduction, it provoked mixed opinion.

While the R&D-based industry believes that over time, it will allow Europe to reclaim a central role in innovative drug development, it has been far less popular with the generics community.

However, ways of benefiting from the new processes have gradually garnered approval.

Alongside the obligatory requirements, the new regulations provide incentives to develop paediatric forms and doses of medicines currently only approved for adults, the main one being a six-month extension to patent protection for innovator companies that test their products on children.

There is also a separate paediatric marketing authorisation for off-patent medicines that are developed for children and a special fund to encourage research into paediatric forms of such products. Furthermore, the regulation sets up a paediatric committee at the European Medicines Agency that will agree paediatric investigation plans with companies and determine research priorities.

It has therefore been shown possible to profit from the incentives on offer as a result of the new legislation, and as the field of paediatric trials develops, possible to identify key market opportunities for on and off-patent drugs.

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