Paediatric oncology drug development – Accelerating the momentum

September was designated Childhood Cancer Awareness Month to bring awareness to childhood cancers, which remain the leading cause of death by disease for children under the age of 14.

It provides an important focus on the children, their families and caregivers affected by childhood cancers and to emphasize the importance of supporting research and the researchers working to fight these devastating conditions. Does this mean children’s health is our focus only once a year? Not at all for sure, EFPIA companies are, more than ever, committed to overcome the obstacles to deliver treatment options. This is our priority as children are our future and deserve all our attention. This is even more important in the midst of the pandemic.

Throughout the course of my career, starting as a pulmonologist at the hospital and then spending time in various roles in regulatory authorities and industry, I have seen the impact diseases can have on children and their families. These last few years I have had the opportunity to see among my industry colleagues a shift in the mindset to considering paediatric strategy early in drug development as an opportunity to address medical needs in children, and no longer as just another task in the process. Similarly, science has evolved, allowing the use of quantitative and extrapolation approaches, and of complex innovative clinical trial designs to optimise drug development.

EFPIA’s We Won’t Rest Campaign has shown that illness never sleeps and neither do we; tireless medical innovation has allowed us to improve the survival rates of patients with diseases like cancer. This aligns with our ultimate goal to better serve both society and the paediatric population with viable and useful therapeutics.

While we believe the Paediatric Regulation has had a significant impact on the delivery of new drugs for children, we expect more new drugs to be delivered in the near future to treat paediatric oncology diseases which are often rare diseases by essence. Despite the implementation of the Paediatric Regulation more than 10 years ago, there are still some challenges sponsors are regularly facing. To this effect, we have proposed some solutions to optimise regulatory requirements and processes within the current regulatory framework, as well as resources.  All these proposals which would benefit PIP delivery and timely patient access to new medicines are detailed further in a publication to be released shortly: the introduction of an integrated scientific discussion throughout the product life cycle for paediatric purpose; paediatric investigation plan (PIP) procedure optimisation; PIP compliance check simplification and alignment of paediatric clinical study reporting requirements with those of adult trials.  

Awareness certainly plays a role to motivate all involved stakeholders and helps them collaborate and participate in education and research initiatives. Multi-stakeholders’ collaborations as well as public private partnerships have shown their value. Some IMI projects such as Conect4Children, EU-PEARL or ITCC-P4 will certainly deliver interesting outputs to help optimise paediatric drug development.

Recently, EPFIA companies have supported the Fostering Age Inclusive Research (FAIR) Initiative. Research into teenage and young adults’ cancer has made slower progress than for any other age group.  One reason is that many clinical trials exclude patients under 18 without medical justification. To confront this problem, the FAIR Working Group was established in 2017 by the ACCELERATE Forum. Made up of experts from Academia, Industry and Parents/Patients advocacy, the group aims to raise awareness and promote change. This is an initiative also widely supported by regulators and advocated in several regulatory guidelines such as the ICH E11(R1) paediatric guideline.

To conclude I cannot resist to quote Nathalie Moll, EFPIA General Director: ‘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’.

Solange Corriol-Rohou

A pulmonologist and immune-allergist by training, she joined AstraZeneca R&D in 2004 and is currently Sr....
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