The Pharmaceutical Journal
Vol 268 No 7189 p349-354
16 March 2002

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EC document seeks to improve safety and range of medicines for children

How medicines for children can be improved is being tackled in a new European Commission consultation document.

The EC says that Europe is dangerously short of medicines that have been developed or adapted for children.

"It is estimated that between 50 and 90 per cent of medicinal products, depending on therapeutic area, used in children have never been specifically evaluated for use in children," it states. This situation leads to use of unauthorised products and use of medicines off-label which can put children's health at risk.

Pharmaceutical companies have traditionally been reluctant to invest in developing or adapting medicines specifically for children because the market is small and the risks associated with paediatric treatments tend to be higher, the document says. However, this lack of specific paediatric medicines can result in problems such as inaccurate dosing because adult medicines have to be adjusted for children and difficulties for children themselves in taking dosage forms intended for adults.

The EC document follows a change in legislation in the US in 1997 in which an additional six-month period of exclusivity to protect a patent was introduced for companies that performed clinical studies in paediatric populations. This has resulted in more than 400 studies starting and 21 labelling changes being made in three years.

Measures similar to those already taken in the US are urgently needed, the document states. Erkki Liikanen, European Commissioner responsible for enterprise and information, said: "To get the best and safest treatments for children across Europe, society must strike the right balance between incentives and regulatory obligations. We need to move forward on this important matter to ensure that both existing and new medicines are adapted to paediatric needs in the most resource-efficient manner."

The EC proposes a number of approaches to tackle the current lack of suitably adapted medicinal products. These include encouraging:

• More paediatric studies of new drugs
• More paediatric studies on existing authorised drugs where there is perceived therapeutic need
• More development of adapted formulations
• More transparency of information on products used in children

Specifically, it proposes that an expert group within the European Medicines Evaluation Agency is created to look at the field of paediatric medicinal products. In addition, a central database would be set up to collect existing data on use of medicinal products in children, including off-label use.

The EC document accepts that market forces are inadequate to stimulate the necessary studies and that incentives are required. It proposes to introduce an additional period of market exclusivity that would apply at the end of an existing patent as a reward for submitting validated clinical studies in children. New applications for market authorisation could routinely require studies in paediatric populations. For products that are already in use, it suggests introducing a new type of market authorisation that would give a period of data protection. Alternatively, independent public funding could be used to fund research.

Sharon Conroy, chair-man of the Neonatal and Paediatric Pharmacists Group, told The Journal that the report was greatly welcomed. "The necessity to use unlicensed and off-label drugs in children is an everyday problem for pharmacists. This is a long-awaited document that provides a welcome starting point to tackle the problems." She supported the suggestion that an approach similar to that taken in the US is followed and the creation of a European network. "The collection of existing data on off-label use has been aired in the group for some time," she added. The group will be submitting an official response to the document to the EC.

Richard Ley of the Association of the British Pharmaceutical Industry said that, in broad terms, the ABPI welcomed much of what was proposed in the document. "However, we do take exception to the fact that the paper lays blame with the pharmaceutical industry that more work has not been done in the area." He pointed out that it is difficult to conduct clinical trials in children.

He added that the ABPI believes that in order to make Europe competitive, the six-month exclusivity extension, as seen in the US, would be too short and should be made one year.

Although Mrs Conroy acknowledged that it is more difficult to conduct trials in children than adults she said that this should not be used as justification for not carrying them out. "Is it ethical to continue to treat children with medicines that have not been properly trialled?" she asked. "We don't know when a drug is used off-licence if it will be metabolised differently in a child so they need to be tested in controlled situations by experienced paediatric investigators which will minimise the risk to children."