Abstract
As described in the previous chapter, it is essential to introduce novel therapeutic agents into treatments of children with cancer to further improve outcomes and quality of life and reduce long-term toxicity. Multistakeholder collaboration, improved understanding of pediatric cancers, early access to appropriate novel anticancer agents for early pediatric investigation, preclinical models, innovative clinical trial designs, international collaboration and alignment between the scientific, regulatory, and payers' requirements from the inception of a development plan are required to accelerate pediatric drug development. This chapter provides an overview of the needs, challenges and potential (practical) solutions to conduct translational and early-phase clinical studies to benefit children with cancer and may also benefit drug development in other pediatric areas.
| Original language | English |
|---|---|
| Title of host publication | Essentials of Translational Pediatric Drug Development |
| Subtitle of host publication | From Past Needs to Future Opportunities |
| Publisher | Academic Press |
| Chapter | 21 |
| Pages | 529-559 |
| Number of pages | 31 |
| ISBN (Electronic) | 978-0-323-88459-4 |
| DOIs | |
| Publication status | Published - 1 Jan 2024 |
| Externally published | Yes |
Keywords
- Cancer treatment
- Chemotherapy
- Early-phase clinical studies
- Immunotherapy
- Molecularly targeted therapy
- Pediatric oncology
- Pediatric translational drug research