CRED Understanding Clinical Development 2024

08/10/2024

Stepwise PIP (sPIP)

• Recent innovation – currently a pilot scheme

Concept is:

•

• Recognition that there may be insufficient evidence to facilitate full PIP preparation

• Agree some elements of the PIP as usual

• Develop the remainder in conjunction with PDCO over time

A potential outbreak of common sense…?

…but why did it take 16 years to realise that you can’t plan a full paediatric development without any adult efficacy data?

The Organisation for Professionals in Regulatory Affairs

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PIP modifications

• You can request a PIP modification at any time

• No limit on the number of modifications to a PIP

• 60-day review procedure with no clock-stop

• Modifications required for good scientific reasons are not a problem…

• …but modifications based on company preference without good scientific back-up will very likely fail

• Modifications for technical reasons are often needed

• e.g. Study design is as planned but initiation (FPFV) was a month later than stated in the PIP Decision

The Organisation for Professionals in Regulatory Affairs

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