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:
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- Generalities |
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- Managing safety of paediatric clinical trials |
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- Maturational changes in organs critical for the disposition and efficacy of drugs |
- Clinical trials in small populations:
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Level of evidence |
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Choice of endpoints and control groups |
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Methodological and statistical considerations |
- The European register of clinical trials on medicines for children (DEC-net)
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- Networks in Paediatric Rheumatology
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Translational Medicine and Therapeutics for Paediatric Rheumatology
- Biomarkers and surrogate endpoints:
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- The academic, regulatory, and statistical perspectives |
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- Consideration of biomarkers in juvenile idiopathic arthritis |
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- Manipulating B-cells in rheumatic diseases |
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- Translational medicine in autoinflammatory diseases |
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- Proteomics in rheumatology |
- Clinical trials in small populations:
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Level of evidence |
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Choice of endpoints and control groups |
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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
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- Funding schemes for translational medicine
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