EMA review of stepwise paediatric investigation plan pilot

‍The European Medicines Agency (EMA) has published its review of the stepwise paediatric investigation plan (sPIP) pilot launched in 2023. The sPIP is intended to manage exceptional paediatric development programmes in which critical data are unavailable at the time of submission of initial Paediatric Investigation Plan (PIP), thereby constraining evidence-based planning of key study elements. The Report concluded that the stepwise approach demonstrated feasibility and value where it was used in exceptional cases.

‍The eight sPIP opinions adopted by the EMA during the pilot phase covered products intended for the treatment of neurologic and metabolic genetic disorders, immunology, hepatology and oncology. The most commonly undefined key elements in the initial sPIPs were sample size and dosage, with sample size not defined in 12 out of 21 studies and dosage not defined in 10 out of 21 studies. Other undefined elements included endpoints (not defined in eight studies), duration of study (not defined in five studies), statistical analysis (not defined in six studies), and control (not defined in two studies).

The time from PIP submission to agreement was similar to that of conventional PIPs, at around 12 months. This suggests that the clock-stop period remains a significant factor in the overall timeline for PIP approval. Moreover, the modification process remains bound by the 60-day timeline without any clock-stop provision, making it difficult to accommodate the complex revisions anticipated under the sPIP framework.

The sPIP process continues to be available with updated Guidance published on the EMA website