CRED Understanding Clinical Development 2024

08/10/2024

Product-specific waivers

• Waivers can be granted in one, several or all paediatric subsets, but only on one (or more) of three legal grounds:

• The condition does not occur in the relevant age subset(s)

• The onus is on the applicant to prove that it doesn’t occur

• The product is likely to be unsafe or ineffective in the relevant age subset(s)

• In practice, this will require data to support the claim

Not often invoked

•

• The product offers no significant benefit over existing treatments in the relevant age subset(s)

• There is more flexibility to be creative here than with the other two legal grounds

Waivers are tied to the PIP condition, not to PIP indications or therapeutic indications

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Waivers based on lack of significant benefit

Possible options include:

•

• The medical needs are already met by existing drugs

• There is no medical need for treatment (even though the condition exists)

• There are too few patients in the age group(s) concerned to informatively investigate benefit-risk in clinical trials

• “No significant benefit because clinical trials are not feasible”

The Organisation for Professionals in Regulatory Affairs

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