Over the last decade we have seen exciting new treatments or even cures for diseases impacting on children such as HIV, Hepatitis C, rheumatoid arthritis or certain cancers. These breakthroughs have been enabled by rapidly advancing science and the European Paediatric Regulation which created a framework to support the development of new medicines for children. Since its inception in 2007, there have been 260 new medicines and indications for treating children. New formulations have been developed to enable children to take their medicines more easily and research continues in this innovative area.
The paediatric research landscape continues to evolve, supported by this important piece of legislation. The number of paediatric development programmes is increasing, there is a growing paediatric research infrastructure and overall more clinical trials to support regulatory submissions. In fact the proportion of paediatric clinical trials since the legislation came into force has increased by 50% between 2007 and 2016 (from 8.25% to 12.4% of all trials conducted in the EU).
We mustn’t stop here. More can be done to increase the number of medicines available for children and we are currently working with other stakeholders to address paediatric needs.
Global collaboration is key. Given the limited number of paediatric patients that can be included in studies, developing medicines for children must be a global exercise. As an industry we stand ready to increase the level of partnership and collaboration with the wider paediatric research community and across other life sciences sectors through programmes like the Innovative Medicines Initiative (IMI).
Improving development speed and efficiency within the current frameworkWe are working on a number of pragmatic steps and measures that can be taken to optimise implementation of the Regulation in the short term that will ultimately improve the efficiency and speed of paediatric development:
* An inventory of disease-based unmet paediatric needs, based on the existing requirements of Article 43 of the Paediatric Regulation, to provide a common basis for strategic decision making on paediatric medicine development. The inventory should indicate clearly for each need if there is research on-going and what type of research, ensuring transparency for all stakeholders of areas where research is most needed and to avoid that the paediatric population is subjected to unnecessary or unfeasible trials. Multiple stakeholders (industry, regulators, epidemiologists, patient groups, paediatric networks) should be involved in this assessment. EFPIA in collaboration with EFGCP held a multi-stakeholder workshop in Amsterdam last December to discuss methodologies for establishing paediatric unmet medical needs in several therapeutic areas. The constructive discussions will be followed up with further conversations in 2020.
* Improving the efficiency of paediatric investigation plans (PIPs) through better integrated scientific and regulatory dialogue, leading to a model where the PIP develops with the evolution of scientific knowledge. The improved PIP process will lead to agreement of development plans that fit better within the global drug development process. It is expected to improve the scientific credibility of the PIP, remove the need for long deferrals for study starting dates and reduce the need for multiple modifications, offering greater certainty to all that the agreed PIPs can be effectively completed. EFPIA is developing its proposal for optimizing the regulatory interactions to improve PIPs and PIP procedures through an integrated paediatric development dialogue within the scope of the continuous regulatory dialogue discussions during product development, and an optimised PIP procedure with opportunities for dialogue and simplified procedures to ensure best use of available resources.
* Improving the international collaboration and references framing the discussion for potential paediatric development plans to ensure a clear and predictable outcome, particularly in the field of oncology. The US efforts led by FDA on molecular targets for oncology can be leveraged to facilitate global convergence.