Masterstudiengang "Drug Regulatory Affairs"
Master-Thesis
The New Paediatric Regulation in the EU - Development, Implications and Comparison with US Experiences in Paediatric Drug Development ***
Martin Watzl (Abschlußjahr: 2007)
Language: English
In the 27 EU Member States, the paediatric population represents more than 100 million people, i.e. about 20 percent of the total population. This is a vulnerable group with developmental, physiological and psychological differences to adults, which makes age and development related research of medicines particularly important.
The absence of paediatric testing and appropriate labelling poses significant risks for children, including adverse reactions and ineffective treatment through underdosing, that could be avoided if such information were provided. Inadequate labelling information may further deny paediatric patients the ability to benefit from therapeutic advances because physicians choose to prescribe existing, off-label or unlicensed products, which might be less effective in the face of insufficient paediatric information about a new medication. The failure to produce drugs in dosage forms that can be used by young children (e.g., liquids or chewable tablets) can also deny them access to important medicinal products.
It may safely be assumed that more than 50 percent or more of medicines used in children in Europe have never been actually studied in this population and are not authorised for such use. Viable mechanisms to enhance paediatric medicinal research in Europe was lacking for a long time. The legislative attempts and efforts made in the US resulted in great benefits for US children. Europe has been slow to follow. Although clearly based upon the US experience, the EU Paediatric Regulation incorporates a similar carrot but a larger stick to ensure companies will undertake paediatric development work for new drugs.
All the key measures in the new EU Paediatric Regulation build on or strengthen the existing framework of Regulations for medicinal products. The core requirements will definitely ensure that medicines are appropriately tested and authorised in the various paediatric populations. The development of medicines for children will be stimulated by a number of incentives and rewards. The additional requirements, facilitating and support measures provide strong support for research by smoothing procedures, providing information and ensuring availability.
The Regulation will definitely cost money: Industry will have to pay for complying with the various requirements, government has to provide adequate infrastructures to make the Regulation work, households, health care professionals and insurers will be faced with slightly higher drug prices, as a result of added paediatric testing. Producers of patented medicines will benefit substantially more than the generic industry. The potential sharing of confidential drug development information between EMEA and FDA hopefully will mean that there will be less duplication of effort for those companies intending to obtain a new product approval in Europe and the US, ultimately resulting in a greater benefit to both paediatric populations.
The overall objective of the EU Paediatric Regulation is to improve the health of the children in Europe. Based on the successful experiences in the US and the comparable approach made in Europe it is concluded that the Paediatric Regulation provides one half of the solution. By changing the economics and legal preconditions, the European Commission hopes to steer consumers, involving health care professionals and households, towards tested and, hence, safer and more effective medicines. If the products are developed and the tested products are indeed prescribed, children will benefit through better treatment, shorter hospitalisation and lower drug consumption and will at the end enjoy a better quality of life. A number of risks and uncertainties related to possible delays in drug development, authorisation and market entry remain. But choice remains the most uncertain factor: the readiness of the industry to focus on the development of paediatric medicines, the response of generic manufacturers to the incentives of a PUMA and the willingness of health care professionals to prescribe tested medicines in disfavour of off-label and unlicensed medicinal products. The final piece regulating prescription practices will have to be provided by policy makers in the health care domain.
Pages: 94