Paediatric Investigation Plans: Planning for Children from the Start

For most new medicines in the EU, studying the product in children is not optional. Under the Paediatric Regulation, a paediatric investigation plan (PIP) must be agreed early in development — and sponsors who leave paediatric planning until late routinely find it delays their adult marketing authorisation.

The Paediatric Regulation

Regulation (EC) No 1901/2006 reshaped how paediatric medicines are developed in Europe. Its central mechanism is the PIP: a development plan describing the studies a company will conduct to generate data on a medicine’s use in the paediatric population. The Regulation also created the Paediatric Committee (PDCO) at the EMA, which agrees, modifies and oversees PIPs.

When a PIP is required

A PIP is generally required for any application for a new marketing authorisation, and for certain variations — new indications, pharmaceutical forms or routes of administration — of products still covered by a patent or supplementary protection certificate. Crucially, the results of the agreed PIP studies, or an agreed waiver or deferral, must be in place before the application can be validated. Two mechanisms provide flexibility:

• Waivers, where paediatric study is not appropriate — for example, a disease that does not occur in children.
• Deferrals, which allow paediatric studies to be completed after the adult authorisation, so the adult launch is not held up.

Rewards and obligations

The Regulation balances obligation with incentive. Companies that complete an agreed PIP may qualify for:

• a six-month extension of the supplementary protection certificate; or
• for orphan medicines, two additional years of market exclusivity.
• A dedicated authorisation type, the Paediatric Use Marketing Authorisation (PUMA), exists for off-patent products developed specifically for children.

These rewards depend on full compliance with the agreed plan, not merely on completing some paediatric work.

Common pitfalls

• Starting PIP discussions too late, so paediatric requirements dictate — and delay — the adult filing timeline.
• Treating the agreed PIP as fixed; plans often need modification as data emerge, and modifications must themselves be agreed with the PDCO.
• Underestimating the operational challenge of paediatric formulation development and recruitment.
• Missing the link between PIP completion and the reward, and forfeiting the SPC extension.

How PQRA helps

PQRA supports sponsors across the paediatric lifecycle — assessing whether a PIP, waiver or deferral applies, preparing and negotiating the plan with the PDCO, managing modifications as development progresses, and aligning paediatric timelines with the adult programme so the marketing authorisation is not delayed. We also advise on the rewards available and how to secure them.

Get in touch to plan your paediatric strategy early.