Paediatric medicines: Overview

The European Medicines Agency has a number of important tasks and responsibilities relating to the development of paediatric medicines. These responsibilities, granted through the European Union (EU) Paediatric Regulation, enable the Agency to stimulate research into the uses of medicines in children and to lead to their authorisation in all age groups.

Before the Paediatric Regulation came into effect in 2007, many medicines authorised in Europe were not studied adequately or authorised in children. This caused difficulties for prescribers and pharmacists treating children, as well as for their patients and carers.

The Regulation introduced sweeping changes into the regulatory environment for paediatric medicines, designed to better protect the health of children in the EU. The main change was the creation and operation of the Paediatric Committee to provide objective scientific opinions on paediatric investigation plans (PIPs), development plans for medicines for use in children.

A report is available on the improvements brought by EMA and the European Commission's action plan to boost the availability of medicines for children and increase the efficiency of paediatric regulatory processes.

This closing report highlights EU regulators' main initiatives and achievements in this field between 2018 and 2022. They include:

• Strengthening focus on unmet medical needs, such as for children with cancer and inflammatory bowel disease
• Adapting regulatory processes to better support innovation, such as launching a pilot to test a  stepwise PIP  agreement enabling the authorisation of medicines for children
• Increasing alignment of data requirements between decision-makers, for instance EMA collaborating more closely with the US Food and Drug Administration (FDA)

EMA and the European Commission first published the action plan in 2018. It aimed to address the challenges identified by the European Commission’s ten-year report on the implementation of the Paediatric Regulation.

It also took account of ideas on How to better apply the Paediatric Regulation to boost the development of medicines for children collected during a 2018 multi-stakeholder workshop.

The European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children.

Enpr-EMA's main objective is to facilitate studies in order to increase availability of medicines authorised for use in the paediatric population. It provides access to European and international specialty and multi-specialty networks who can help developing a PIP and recruiting patients for clinical trials.

The Agency works closely with its international partners on medicines for children:

• it holds regular meetings with the United States Food and Drug Administration (FDA) within the paediatric cluster to exchange information on applications and topics related to development and to help support global development plans for paediatric medicines. For more information, see Partners & networks: United States: Cluster activities.
• it participated in the launch of the World Health Organization (WHO) initiative Make Medicines Child Size.
• it is a member of the Paediatric Medicines Regulators' Network (PmRN). This is a network of national medicines regulatory authorities set up by the WHO in 2010, which promotes the quality and availability of medicines for children and contributes to capacity-building.

The Agency acknowledges that it may be helpful to consult children and young people as part of the Committee's work. It has therefore established principles for the involvement of young patients, consumers, and their carers, including identifying in which situations it may be helpful to seek their input and defining what is expected from them when they are consulted.

This follows a public consultation on the concept paper on the involvement of children and young people at the Paediatric Committee .

Guidance is available for medicine developers on how they should present the use of extrapolation of efficacy and safety data from adults to children in their regulatory applications. 

This covers the methodology medicine developers use to identify the clinical data required to show that the therapeutic response in children can be similar to that in adults.

The guidance complements EMA’s framework for paediatric extrapolation in medicine development, which is described in the reflection paper available via the link below:

• Extrapolation of efficacy and safety in paediatric medicine development - Scientific guideline