Paediatric Medicinal Products
Therapies with medicinal products often expose children and adolescents up to 17 years of age (hereinafter referred to as ‘children’) to special risks. Medicinal products have not always been tested in this population, and marketing authorisations for them are likewise lacking. The development of paediatric medicinal products on the basis of existing products which are long time approved for adults is not obligatory. A better incentive policy for voluntary research could help.
Only 150 out of 1,200 clinical studies per year in Germany include children as study subjects. The EU ‘Regulation on paediatric medicinal products’ of 2007 aimed to create more medicinal products which are safe, age-adapted and have been tested in children. It made paediatric studies obligatory for the development of new medicines and has by this achieved an improvement of the situation in the area of new medicines: According to information provided by the ʻEuropean Medicines Agency’ (EMA), and the ʻPaediatric Committee’ (PDCO) which is in charge of the assessment of paediatric studies, the number of marketing authorisations for children solely in the centralised procedure has more than doubled: from 31 in the period from 2004 to 2006 to 68 in the period from 2012 to 2014.
Few research incentives for PUMAs
The situation for medicinal products which are already approved for adults for a longer time is different. For these, paediatric research by the marketing authorization holder is voluntary. An authorization for the paediatric use of a medicinal product which is already approved for adults is called ‘PUMA’ (Paediatric Use Marketing Authorisation). Such an authorization can be granted for all paediatric indications, for all or specified age groups or for the development of pharmaceutical forms which are suitable for children. The compensation for the holder of a PUMA is a ten year data protection. This market exclusivity for the product should at least compensate for the financial research expenditure. However, it seems to exist only in name because it is undermined by national health systems, and not only in Germany. Substitution of medicinal products with the same active substance according to the aut-idem rule basically obstructs market-exclusivity. In addition, in Germany PUMAs must undergo the sophisticated procedure of the early benefit assessment conducted by the Federal Joint Committee (G-BA).
IKAM-initiative argues for improvements in the existing market
As a member of the ‘Initiative Medicines for Children e.V.’ (IKAM), the BAH is committed to medicinal products which are safe and have been tested in children. Consistent implementation of the PUMA concept pursuant to the EU regulation could improve the situation in terms of the existing market. The benefit of a PUMA should in the future – similar to orphan medicinal products which are intended for the treatment of rare diseases – be considered as established, as the medicinal product has already cleared high regulatory hurdles. The G-BA should merely assess the extent of the benefit. Furthermore, intelligent incentives are needed in order to bring the development and marketing of medicinal products which have specifically been developed for children also to economic success.