More than a fifth of the people in the European Union are age 16 or under, yet at least half of the medicines in use there have not been tested on children. As a result, the region suffers from a lack of information about the safety and efficacy of pediatric medicines and of off-label pediatric use that medical experts say could cause adverse effects and even death.
Although the problem is of long-standing concern for EU member states, the European Parliament, medical experts, and patient groups, there was no push for a solution until December 2000, when EU health ministers asked the European Commission to address it. A task force made up of members of the commission's public health and enterprise departments proposed that pharma companies do the following:
The European Commission says pharma companies have been reluctant to develop pediatric medicines or to amend adult therapies to meet childrens' needs, because the markets are small and the difficulties of calculating pediatric dosage levels of adult products in clinical trials can lead to serious errors.
The International Conference on Harmonization's guideline on clinical trials for children, adopted by the regulatory agencies of the United States, the European Union, and Japan, and its good clinical practice directive of April 2001 provide for the protection of children in clinical trials. But there are no legal provisions or incentives in Europe for companies to develop medicines for children, even though there is a clear therapeutic need.
To achieve its objectives, the commission is encouraging pediatric studies for marketed products and has proposed to provide an additional period of market exclusivity to companies that submit validated pediatric clinical studies. That incentive would also apply to products still in development.
Another proposal is for a fund to support pediatric research. The European Commission quotes US National Institutes of Health estimates that safety and efficacy studies may cost between $1 and $7.5 million. In Europe, based on an average of 5 million euros per trial, 20 studies could be performed annually at a total cost of about 100 million euros.
The commission also proposes an approach similar to that used in the United States-to require studies in pediatric populations as part of the marketing authorization application process, unless there is no pediatric need for the product. It also wants to set up an EU group of scientific experts, which can match the United States by providing pharma with regulatory oversight while they are developing new medicines, in a way similar to the Union's new orphan medicine regime.
The European Commission welcomes all contributions to the consultation process.