The Paediatric Investigation Plan (PIP), introduced by Regulation (EC) No 1901/2006, is a research and development programme aimed at ensuring that the necessary data are generated determining the conditions in which a medicinal product may be authorised to treat the paediatric population.
In principle, the PIP shall be submitted early in the product development, no later than upon completion of the human pharmacokinetic studies in adults.
However, there are exceptional cases where there is a lack of crucial information needed to define the PIP in this early phase of the development, such as in the case of:
- a first medicine for a disease (e.g. very rare disease only recently identified in children),
- a medicine whose mechanism of action has not been fully characterized yet and which may have implications for ontogeny related changes,
- a medicine with multiple development options to address significant unmet paediatric need in several paediatric indications where adult data are lacking.
Therefore, EMA is proposing to test through a pilot the concept of the ‘stepwise PIP’ (sPIP) consisting of only a partial development program, conditional on the development of a full PIP once the crucial information has become available. It would rely on predefined steps agreed with the EMA’s Paediatric Committee (PDCO), such as applicants being able to hold discussions with the PDCO once they obtain more data.
Regulatory Pharma Net (RPN) has successfully supported companies in the early dialogue procedures with EMA, including the PIP, and is available to support companies in the preliminary interaction with the Paediatric Medicines (PME) Office to explore the possibility to join to the sPIP pilot program.