Stimulating the development of new medicines for children

Nathalie Moll

Nathalie Moll joined the European Federation of Pharmaceutical Industries and Associations (EFPIA) as Director...
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As a mother, I know that very little impacts our lives like one or more of our children being ill. Every test result, every minor change in symptoms is a high or a low on an emotional rollercoaster. Every decision gets questioned and replayed, every data point gets analysed and then analysed again and again and again.

As well as coping with your child’s emotional and physical needs, you develop a very rapid awareness of the treatment options available, their pros and cons, the latest developments in the relevant therapy area and their impact on your child’s prognosis.
The research and development of new medicines for children is so fundamentally important to us all - for every child, for every family affected by childhood illness, for our clinicians, healthcare systems and for society as a whole. It is why our industry is fully committed to integrating paediatric research in its medicine development programs.  
 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 legislation (2007) which created a framework to support the development of new medicines for children.  Since its inception, 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.
And 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).
Fifteen years ago, we were in a very different place when talking about availability of licensed paediatric medicines in Europe and the rest of the world. Europe’s Paediatric Regulation has played a key role in transforming that landscape and stimulating the development of medicines for children. The Regulation is part of the European Commission’s analysis of pharmaceutical incentives and as an industry we are proposing a number of positive, pragmatic, non-legislative measures that can be taken to optimise the implementation of the Regulation.  These proposals listed below are designed to improve the efficiency and speed of development of paediatric research with a view to further enhance the already positive impact of the Regulation. These are:

- Creating 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.

- Improving the efficiency of Paediatric Investigation Plans (PIPs) through better integrated scientific and regulatory dialogue, leading to a PIP model that 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.

- 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, resulting in the faster development of new medicines for children living with cancer
As long as there are children living with unmet medical needs, as an industry we won’t rest in our commitment to working with our partners in paediatric research to continue to discover, develop and deliver new medicines for them.