Programme Highlights
The Regulatory Framework
- Regulations on medicinal products for paediatric use: the EU and US legislation compared
- The Paediatric Investigation Plan in the EU: the scientific and regulatory viewpoints
- Directives on GCP and clinical trials: specific issues concerning ethics of clinical research in children
- Rare diseases, orphan legislation and how to develop an orphan drug: EU and US perspectives
- Pre-clinical strategies to obtain regulatory approval for a first in children study
- The need for pharmaceutical forms of medicinal products adapted to children
Clinical Trials: Methodology and Networking
- Paediatric clinical trials:
|
| |
- Generalities |
| |
- Managing safety of paediatric clinical trials |
| |
- Maturational changes in organs critical for the disposition and efficacy of drugs |
- Clinical trials in small populations:
|
| |
Level of evidence |
| |
Choice of endpoints and control groups |
| |
Methodological and statistical considerations |
- The European register of clinical trials on medicines for children (DEC-net)
|
- Networks in Paediatric Rheumatology
|
Translational Medicine and Therapeutics for Paediatric Rheumatology
- Biomarkers and surrogate endpoints:
|
| |
- The academic, regulatory, and statistical perspectives |
| |
- Consideration of biomarkers in juvenile idiopathic arthritis |
| |
- Manipulating B-cells in rheumatic diseases |
| |
- Translational medicine in autoinflammatory diseases |
| |
- Proteomics in rheumatology |
- Clinical trials in small populations:
|
| |
Level of evidence |
| |
Choice of endpoints and control groups |
| |
Methodological and statistical considerations |
- Anticipating change in drug development: the emerging era of translational medicine and therapeutics - the perspectives of industry, academia and regulatory agencies
|
- Funding schemes for translational medicine
|