Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Medicines for Children, a Regulatory Target!

Dr. Cornelia Schmidt (Abschlußjahr: 2003)

Europe is seriously short of medicines developed for or adapted to children. More than 50% of the medicines used to treat children in Europe today have never been specifically tested for them. This master thesis gives an overview on the current situation, the reasons why children's medicines is underdeveloped, the initiatives started in the past and the solutions and proposals which have been introduced in the US and Europe in order to counteract this situation.

Pharmaceutical companies are often reluctant to invest in child-specific medicines, as the market for them is relatively small and the development program is both, difficult and time consuming. Thus the return of investment is unsatisfactory in many cases. According to applicable law the regulatory authorities neither have the authorisation to commit pharmaceutical companies to conduct clinical trials in children nor do they have the right to grant rewards for these efforts. A change of this situation is crucial for the improvement of the availability of paediatric medicines.

In the US, experience with paediatric regulatory initiatives is available for both, on- and off-patent substances. In the EU, a proposal for ensuring that children will get the medicines they need, was set out in a Commission consultation paper "Better Medicines for Children" in February 2002. Possible incentives include a longer period of intellectual property protection (6 months) to recompense work on innovative medicines, and a new type of marketing authorisation ("kid marketing authorisation") that would provide data protection (5 years) to new paediatric uses of older products. Furthermore the provision of a fund for pre-clinical and clinical research has been suggested.

In order to account for the specific preconditions and goals, different regulatory procedures and incentives have been proposed to obtain paediatric information for patented and off-patent drugs. The adoption of a period of data protection has been selected for the "kid marketing authorisation" rather than market exclusivity as there is no intention to block the market to similar products. It would also allow other companies than the originator to exploit known medicinal products and develop specific formulations. Only the paediatric indication would benefit from data protection.

Products for which a protection of intellectual property exists, can profit from an extension of the patent or supplementary protection certificate. The prolonged period of the property protection defends from generic competition until this additional period has elapsed. Data protection would not be appropriate because patented products do not benefit from such a provision, if data protection runs in parallel to the patent. An extension of data protection at the end of the supplementary protection certificate or patent run also would be counterproductive as paediatric research should be started as early as possible. An adequately prolonged protection period may be an acceptable incentive for pharmaceutical companies which will be obliged to perform paediatric trials as a marketing authorisation requirement in the future, unless the medicinal product is unlikely to be used in children.

The chances to obtain paediatric information for products with intellectual property seem to be more likely promising. The prospect of success to achieve paediatric labelling for medicinal products which are at least equally important to children, i.e. off-patent products, still appears to be a major challenge as also shown by the experience from the USA. The European Commission will present two separate regulations governing patented and off-patent products. It is expected that the proposals will be adopted in 2004.

Pages: 41