Module232025

07/05/2025

Control Group

Placebo

Active comparator

• Can we ethically conduct placebo controlled trials in countries in which: • No other drugs have paediatric approvals, or • Other drugs are approved but not accessible? • Would children in such countries be “used as a means to an end”? • Are we withholding treatment of proven benefit from children?

Blinding issue?

•

• COVID vaccine – the Oxford model

What about children with a different condition? Can we administer an investigational drug to children with a different condition, in which the drug is expected to be ineffective?

The Organisation for Professionals in Regulatory Affairs

37

Study Rationale

• EMA and FDA “encouraging” pediatric development for all new drugs • EMA will not grant MAA unless PIP is filed and – in some cases – activated • The fact that the EMA or FDA has agreed a specific study design with a sponsor does not per se make the study ethical • Trials being required for conditions which are well-treated in children but not adults, e.g., ALL – delaying adult approvals

Study Rationale

38

19