Module232025

12/05/2025

• Are study endpoints and measures appropriate and validated in paeds • Is the disease the same in children as in adults • Will new endpoints need to be developed • Measurements of subjective symptoms, such as pain, require • different assessment instruments for different ages of patient Protocol: Endpoints

The Organisation for Professionals in Regulatory Affairs

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Protocol: Additional Safety monitoring

• Also need to assess the effects on: • Growth and development, e.g. steroids • IQ and educational impact, e.g. anticonvulsants • Sexual maturation and fertility • beware of how it will have to be established • Long term follow up will therefore usually be required

The Organisation for Professionals in Regulatory Affairs

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