Table 2 Examples of practical points to consider in writing a protocol for a multi-centre paediatric study.
Protocol item | Points to consider |
|---|---|
PD endpoints | Ensure that pharmacodynamic (PD) endpoints for efficacy and/or safety have been validated for the paediatric study population and are clinically relevant43 |
Safety | Use the paediatric safety specification and paediatric risk management plan as the rationale for safety data collection and analyses (including Data Safety Monitoring Board reviews)46 |
Investigations (laboratory and vital signs) | Plan for differences in local paediatric laboratory reference values |
Ensure that age group-specific reference values for paediatric laboratory values and vital signs are used, and that results are interpreted by a paediatric specialist (e.g. paediatric electrocardiograms (ECG) should be read by a paediatric cardiologist)47 | |
Consider limitations on biosampling and interventional investigations in children48 | |
Ensure investigations and equipment used are adapted to the age group (e.g. an infant will usually not do a sitting blood pressure, the age appropriate practice is to take the blood pressure when the child is sleeping) | |
Aim for integrating trial procedures and follow-up into routine paediatric care to keep disruption for children and their families to a minimum | |
Diagnostic criteria and standard treatments | Plan for differences in diagnostic criteria, treatments and comorbidities in children across different investigator sites |
