Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Towards the unmet medical need of children – Reflection on future changes of the EU paediatric legislation ***

Dr. Sibylle Eisbach (Abschlußjahr: 2022)

Summary
Language: English
While considerable strides have been made in the field of paediatric medicine, even after almost fifteen years of the Paediatric Regulation being in force, a significant unmet medical need remains in the paediatric population. More medicinal products are available than were two decades ago, but children remain an underserved group in many therapeutic areas.
The thesis at hand analyses the available evaluations and assessments of the Paediatric Regulation along with stakeholder feedback and points out opportunities and challenges of a revision of the Paediatric Regulation.
Not all provisions of the Paediatric Regulation have been successful, while others are successful but require some minor adjustments. The various objectives agreed on after the initial evaluations have already addressed some of the identified challenges, but some aspects of how PIPs are conducted and how medicinal products for small populations will be approved still need to be reconsidered. This is especially true in light of new modalities and new approaches – cell and gene therapy and personalised medicine are only two examples where the revised Paediatric Regulation, and indeed the whole revised pharmaceutical legislation in Europe, needs to be future-proof and ready for continuous innovation. This comes on top of the generally difficult area of conducting clinical trials in the paediatric population. Smart trial design, the use of real-world evidence, and the continued dialogue between sponsors and regulators will be essential in order to develop new approaches to paediatric medicinal product development.
Open dialogue - between sponsors and regulators, between different sponsors, between different regulatory agencies as well as between patient advocates, regulators, and sponsors, and between all those groups and payer representatives - as well as open mindedness will be essential in order to address the unique challenges of paediatric medicinal product development. Nevertheless, it is expected that issues around the development of medicinal products for children will remain and that these need to be addressed on a case-by-case basis and learnings need to be derived from each, which should be shared at multi-stakeholder meetings, especially when it concerns new and innovative approaches.
With this groundwork laid, the revised Paediatric Regulation has the potential to address the unmet medical need of children and become a new milestone for the development of paediatric medicinal products that are safe, efficacious, and of high quality.
Pages: 94