The Regulation aims to ensure that medicines for use in children are of high quality, ethically researched and authorised appropriately and improving the availability of information on the use of medicines for children. It aims to achieve this without subjecting children to unnecessary trials or delaying the authorisation of medicines for use in adults.
The Regulation dramatically changed the regulatory environment for paediatric medicines in Europe. Its main impact was the establishment of the Paediatric Committee (PDCO), which is responsible for coordinating the Agency's work on medicines for children. The Committee's main role is to determine the studies that companies must carry out on children as part of paediatric investigation plans (PIPs). The PDCO replaced the Agency's previous Paediatric Working Group.
The European Commission has conducted a comprehensive review of the strengths and weaknesses of the EU regulatory framework governing paediatric medicines.
The evaluation found that the Paediatric Regulation has fostered the development and availability of medicines for children. It has redirected private and public investment towards previously neglected areas through incentives, obligations and rewards.
However, it also found that it has not sufficiently managed to support development in areas where the need for medicines is greatest, and has increased costs for healthcare systems. Even so, the legislation brought benefits for children that appear to outweigh the costs imposed on both industry and society.
The European Commission evaluated the Paediatric Regulation together with the Orphan Regulation, with similar findings for both.
Both regulations set out to address the lack of medicines available for the patient groups concerned, and they often address the same therapeutic areas, since many childhood diseases are rare.
The outcome of this review is intended to guide future legislative changes and shape the EU’s pharmaceutical strategy.
The Paediatric Regulation is comprised of:
In October 2017, the European Commission published a ten-year report on implementation of the Paediatric Regulation.
The report shows an increase in medicines for children in many therapeutic areas in the last ten years, most notably in rheumatology and infectious diseases. However, it also shows that little progress has been made in diseases that only affect children or where the disease shows biological differences between adults and children, particularly rare diseases.
As a next step, the Commission and EMA and its PDCO have developed an action plan to improve the implementation of the Regulation.
The ten-year report takes into account an EMA/PDCO report on experience with implementing the Regulation and a study on its impact. The Commission also held a public consultation on the report to get stakeholder feedback in 2016.
In June 2013, the European Commission published a report on the first five years of the Regulation. This concludes that paediatric development has become a more integral part of the overall development of medicinal products in the EU, with the Regulation working as a major catalyst to improve the situation for young patients:
The report includes an interim analysis of the experience based on a range of sources, including the results of a public consultation on the experience gained with the first five years of the Regulation, building on the Agency's five-year report to the European Commission.
It also identifies some areas for improvement, such as the low uptake of paediatric-use marketing authorisations (PUMAs) by companies. More information can also be found in Successes of the Paediatric Regulation after five years, published by the Agency in 2013.