Drug development in pediatric oncology – challenges and opportunities – reflections from European regulators

ABSTRACT

Introduction: Ten years of the Paediatric Regulation in Europe has resulted in progress in drug development for children. Nonetheless, drug development remains a challenge, particularly in the area of pediatric oncology, as cited by the European Commission’s 10-year Report published in 2017.

Areas covered: In order to further improve the timely availability of novel medicines for children with cancer, further collaborative efforts are needed. Whilst the regulatory framework might present challenges, it provides key tools necessary to support pediatric drug development.

Expert Opinion: It is the regulators together with all stakeholders who need to apply these tools by means of concerted, innovative, and inclusive ways of thinking in order to make it a true success. This requires all stakeholders to reflect on their individual responsibilities in this process, not only to create a level of understanding amongst each other but also to push their own boundaries and challenge established ways of thinking for the benefit of the patient, a principle which holds true for pediatric drug development in general. This paper reflects on these challenges and the opportunities for regulators and key stakeholders.

KEYWORDS:

• Pediatric oncology
• Paediatric Regulation
• Rare diseases
• Orphan Drug Regulation
• Pediatric drug development

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Corrigendum

Article highlights

• Ten years of the Paediatric Regulation positively changed the landscape of pediatric drug development, but for pediatric oncology further progress is to be made.

• Further collaborative efforts are needed; the regulatory tools are sufficient but need to be used by means of concerted, innovative, and inclusive ways of thinking.

• Regulators need to use the existing regulatory tools in a pragmatic and proportionate way that allows focusing on detailed pediatric development plans for the most promising products to be investigated without delay, in an environment which provides the resources to complete these studies in a timely fashion.

• Academia needs to make choices and take the responsibility to judge preclinical as well as early clinical data individually, as well as in relation to other existing and novel compounds regarding the appropriateness to proceed to full pediatric development.

• Industry needs to create a pediatric inclusive R&D process, discuss their pipeline products for certain disease groups early with academia, but also industry colleagues, engage early with regulators, and make use of all regulatory incentives.

• Patients should be consulted regularly, helping industry, academia, and regulators understand better what is a promising new product from a patient perspective, what are relevant clinical trial end points, and feasible study designs.

• Existing platforms, such as ACCELERATE, should become global, including not only US colleagues but also from developing countries, in order to establish global perspective on understanding of the unmet needs and to foster a truly international approach to pediatric oncology drug development.

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Acknowledgments

The views of the authors are their own and should not be considered to represent the opinions of their respective organisations or those of the EMA’s committees.

Declaration of interest

The authors have no other relevant affiliationsor financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussedin the manuscript apart from those disclosed.

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Funding

This paper was not funded.