The Paediatric Committee (PDCO) of the European Medicines Agency released last year the first ‘5-year Report to the European Commission on the experience acquired on the Paediatric Regulation’.
Since the Paediatric Regulation came into force in the European Union (EU) in 2007, pharmaceutical companies have had a legal obligation to develop all new medicines for children as well as for adults through a paediatric investigation plan (PIP), unless they obtain an exemption (waiver). A similar obligation also applies, in some cases, to medicinal products that are already authorised in the EU. Pharmaceutical companies that develop a medicine for children in accordance with the requirements of the Regulation may obtain specific rewards (for example, a prolongation of the duration of the patent).
All applications for PIPs and waivers have to be submitted for review by the PDCO, the scientific body in charge of discussing the paediatric development of medicines, and to agree with the pharmaceutical companies on the clinical studies, pharmaceutical forms and measures that must be performed in children (from 0 to 18 years). These studies may be deferred, for example if the PDCO considers that experience in adults is needed before paediatric clinical trials are conducted. Additionally, the PDCO may grant a waiver from the obligation to develop a medicine in paediatric populations when the condition being addressed by the medicine does not exist in children, when the medicine is not likely to offer any benefit for children or when it is unsafe to use the product in children. This helps to avoid unnecessary paediatric studies.
“In the near future sick children can expect to be treated like adults with medicinal products having been formulated and investigated according to their specific needs. A constant dialogue between the PDCO and different drug developers (academia, industry and also parent associations) has enabled us to come closer to this goal. Further efforts are now under way to optimise the activities and enlarge the scope of paediatric diseases deserving proper treatment offering better lives to young patients. Children are our future,” explains the Chair of the PDCO, Daniel Brasseur.
What has been achieved in five years
Between the first meeting of the PDCO in July 2007 and January 2013, the PDCO has assessed 1340 new applications for a PIP or product-specific waiver, among other activities, of which 76% related to new medicinal products (not authorised at the time of PIP or waiver application). For 613 of these applications, the PDCO adopted opinions agreeing a PIP (most of which include a deferral of one or more of the studies).
In total, 29 PIPs have been completed in full compliance with the PDCO Opinion. These have led to new paediatric indications for 24 medicines, and to new pharmaceutical forms appropriate for children for 7 medicines. Data from the other five completed PIPs also provided important information, which did not support the use in children but was detailed in the product information for the benefit of health care professionals.
As a consequence of the requirements of the Paediatric Regulation, 10 new medicinal products were authorised with a paediatric indication by the EU Commission, based on the scientific opinion of the Agency between 2008 and 2012. In the same period, 30 medicinal products, already authorised centrally (18) or nationally (12), received a new paediatric indication developed under the Paediatric Regulation.
Finally, the results of pre-existing paediatric studies, submitted to the Agency, have led so far to 65 paediatric-related changes to the product information for 149 active substances analysed by a separate Committee of Member States, hosted by EMA, the Coordination Group for Mutual Recognition & Decentralised Procedures – Human (CMDh).
Projects in 2013 and beyond
In 2013 and beyond, the Agency and the PDCO plan to work in the following areas, among others:
- simplification of applications and opinions for PIPs to adjust the appropriate level of detail of the studies/measures in the PDCO opinions;
- further increase of transparency on the activities of the PDCO: this will include publication of full opinions, including key elements of the studies, and a new document, “Summary of the PDCO Opinion”, written in simple language for all stakeholders;
- expand the role of the (Enpr-EMA) in facilitating and coordinating clinical trials in children;
- increase monitoring activities on the completion of PIPs, including the analysis of the annual reports on deferred studies provided by the pharmaceutical companies. This report provides an update on progress with paediatric studies in accordance with the decision of the Agency agreeing the PIP and granting a deferral;
- plan for the data to be provided to the European Commission for the 10-year report on the Paediatric Regulation (as per Art. 50.3 of the Paediatric Regulation);
- greater involvement of children and their parents in the opinion-making process. In 2012, the Agency launched a public consultation on a concept paper describing its plans to develop a framework of interaction outlining how the views of children and young people can be incorporated as part of the Committee’s work. The PDCO is now reviewing all of the comments received.
July 2007 – January 2013: the PDCO in numbers
- 1340 applications for PIPs or waivers assessed
- 613 opinions agreeing a PIP
- 276 opinions granting a product-specific waiver
- 491 opinions agreeing modifications to an agreed PIP
- 38 negative opinions, 31 of which relate to a request for a PIP or waiver and seven related to a request for modification of an agreed PIP
- 37 opinions of positive final compliance check (attesting the completion of the studies detailed in the PIP at the time of marketing authorisation application)