EMA Pilots Refinable Pediatric Drug Studies Program
The European Medicines Agency (EMA) has launched a new pilot program that gives some study leeway to drugmakers working on pediatric investigation plans (PIP) for innovative children’s medicines.
Under the “stepwise PIP” (sPIP) concept, sponsors can file an initial PIP that may still have some evidentiary gaps, with the expectation that these gaps can be filled in as more evidence emerges, according to new EMA guidance.
The idea emerged from a 2018 workshop co-hosted by the EMA and the European Commission. Multiple stakeholders brainstormed ways the agency could improve pediatric drug development. One such idea was an initially incomplete, but refinable, PIP, according to a concurrently published EMA action plan.
According to the guidance, an sPIP might be appropriate for studies of a first drug for a pediatric disease; a drug whose mechanism of action isn’t yet fully understood or that could affect growth and development; or products intended to address significant unmet pediatric needs in pediatric indications where adult data are lacking.
Sponsors that want to file an sPIP need to justify their request, the guidance said. The plan might be appropriate when critical baseline information isn’t yet defined, including the study design, the target population and even the main study objectives.
Nevertheless, an sPIP has to contain at least a minimum data set that includes the target condition, a preliminary outline of the planned studies based on current available evidence and a proposed PIP completion date. The plan should encompass the current scientific knowledge and the state of drug development in the target field.
The EMA will accept eight sPIPs in the first quarter of this year. These test cases will form the basis of further refinements in the plan.
Read the full stepwise PIP guidance here: https://bit.ly/3ROegeS.
Read the EMA’s action plan for pediatric medicines here: https://bit.ly/3Xc8OU3.