Module 6

02/10/2024

Target Product Profile Other considerations: Paediatric population

Paediatric Regulation 1901/2006

• TPP needs to consider the different paediatric population (all age range) wrt to target indication, treatment and duration of completing clinical studies in paediatric population. • Paediatric Investigation Plan (PIP) consisting of clear clinical study designs, timelines, measures with statistical plan will need to be devised and agreed by EMA Paediatric Committee. Submission ideally after adult PK studies are completed. • If development is too early to plan then a PIP deferral is required to be devised in time for the adult MAA filing. • If disease, condition or treatment is not intended for the paediatric population then a wavier (part or full) needs to be applied. • Clinical development must be in accordance with an agreed PIP. Modification of the PIP is allowed within justified scientific reasoning. The development plan should plan for the timelines for anticipated PIP modifications. • All MAAs with proposed paediatric indication requires a completed approved PIP with successful compliance check. • Incentives: 6 months SPC extension. PUMA: 8+2+1 data protection rule. Paediatric orphan drug: extra 2 years market exclusivity.

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

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Target Product Profile Summary

• TPP is useful resource for industry and agencies for first MA and line extensions

Agency

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• TPP can allow constructive dialogue regarding proposed promotional claims

• Efficient use of agency meeting time

• Useful in scientific advice meeting with regulatory agencies

Industry

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• Provides a clear link to ensure teams constantly ensure that the data support the appropriate claims

• Can facilitate production of an annotated label and identify gaps in the development

Useful Reference: FDA Guidance for Industry Target Product Profile — A Strategic Development Process Tool

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

22

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