Regulators in the European Union (EU) have taken several initiatives in the past four years to increase the efficiency of paediatric regulatory processes and boost the development of medicines for children. These achievements are highlighted in the
Some of the key improvements brought by the paediatric action plan include:
- Strengthened focus on unmet medical needs: over the last four years, EMA and relevant stakeholders systematically got together to better identify and raise awareness on the areas where medicines for children are particularly needed, with a goal to shift the research agenda to these areas. For example, multi-stakeholder strategy fora to discuss and agree the needs of children with cancer, and of children with inflammatory bowel disease have taken place. The learnings from these meetings involving clinicians, paediatric patients and their representatives, academia, regulators, HTAs bodies, and developers are being taken into account by EMA when discussing paediatric investigation plans (PIPs) for new medicines.
- Adapting regulatory processes to better support innovation: to facilitate the establishment of PIPs, regulatory processes have been adapted and processes overall simplified. A major outcome of this work is the launch of a pilot phase for a ‘stepwise PIP’ agreement: under this framework, it will be possible in certain cases to agree on a partial development programme, conditional on the development of a full PIP once evidence becomes available over time. This will allow agreeing on PIPs for innovative medicines where crucial information needed to define certain parts of the plan is not yet available, while planning the conditions and milestones for companies to return to EMA’s paediatric committee (PDCO) and discuss the uncertainties once more data are available. More information on this newly developed framework, launched today, is provided in the
guidance for a stepwise PIP pilot
- Increased alignment of data requirements between decision-makers: to facilitate the compatibility of paediatric requirements between regulators, EMA has strengthened its collaboration with international partners, and notably within its paediatric cluster with the US Food and Drug Administration (FDA) and other international regulators. Work was also carried out through the European network of paediatric research at EMA (Enpr-EMA) to align international requirements for paediatric clinical trials authorisation and standards.
In 2017, the EC published a 10-year report on the implementation of the Paediatric Regulation. This report showed an overall success of the Regulation with an increase in authorised medicines for children, but also identified some challenges, noting in particular that certain therapeutic areas (e.g. oncology, neonatology) still lacked sufficient developments for children.
On the basis of this report, EMA together with the EC held a multi-stakeholder workshop in 2018 to identify ways to improve the implementation of the Paediatric Regulation. As a result of this workshop, the paediatric action plan was developed to provide some immediate solutions to these challenges under the current regulatory framework. This work will contribute to the application of the Paediatric Regulation, while the EC is currently finalising a proposal to revise the EU’s pharmaceutical legislation, which will include a revision of the legislative framework applicable for medicines for children. The adoption of the proposal is expected next month.
More information on the Paediatric Regulation
The Paediatric Regulation came into force in the EU in 2007 to encourage manufacturers to research and develop medicines for children’s specific therapeutic needs by using a system of rewards and obliging developers to specifically plan the development of their medicine for children (e.g. by integrating it into the development for adults) and submit a corresponding PIP. A PIP is a development plan aimed at ensuring that the necessary data are obtained through studies in children, to support the authorisation of a medicine for paediatric patients. All applications for marketing authorisation for new medicines have to include the results of studies as described in an agreed PIP, unless the medicine is exempt because of a deferral or waiver.
Source: European Medicines Agency, Actions to support the development of medicines for children, Source link, 2023-02-09 16:19:14