Drug development is facing a new challenge in Europe. A well intended legislation in force since 2007 is handled by the European Medicines Agency (EMA) in collaboration with all EU regulatory authorities. Nobody will object to the wish to let children participate as early as possible from pharmaceutical progress. But the reality is different from the visions of most people that welcomed a decade ago an upcoming European pediatric legislation.
A veritable machinery has been established, consisting of the EU paediatric committee (PDCO) with 33 members and 33 alternates, representing each EU member state, the CHMP (which later decides about drug registration), representatives from health care organizations and from patients’ groups. PDCO members and alternates together form a group of 66 people that will decide about each company’s proposals.
The PDCO is supported by a group of 20 EMA pediatric coordinators that take care of procedural issues. Diseases such as melanoma or postmenopausal disorders have now been declared pediatric diseases, and even a drug specifically designed to treat preterm neonates needs the PDCO’s blessing in the form of an approved pediatric investigation plan (PIP), or EMA will not validate a submission.
In contrast to the US, orphan drugs need a PIP, too. At the end of the one year PIP procedure most companies reach an agreement, with considerable costs and efforts. It is an asymetrical dialogue. There will always be a tension between the logic of public health and the logic of market driven processes. The good thing is that drug development without at least thinking about children is no longer possible. With up to 16 pediatric studies for new drugs it is a question of time until companies will avoid therapeutic areas with heavy EMA/PDCO pediatric requests.
Dr. Rose will be participating in the World Pediatric Drugs Congress this coming April. He will be speaking on the topic ‘European pediatric legislation: an evolutionary step or potential roadblock,’ and participating in a panel on ‘Harmonizing pediatric regulation for a global market.’ Register now to hear him and other pediatric drug experts share their insights.