EMA launches ‘stepwise PIP’ pilot

The European Medicines Agency (EMA) on Monday launched a pilot program intended to allow greater flexibility for sponsors of innovative medicines in developing pediatric investigation plans (PIPs). Under the Stepwise PIP framework, EMA and sponsors may agree to a partial pediatric development program before agreeing to a full PIP later, as additional information about a drug becomes available.
 
The concept of a stepwise PIP stems from a 2018 multi-stakeholder workshop hosted by EMA and the European Commission (EC) on ways to improve the landscape for pediatric drugs in the EU. During a focus group involving industry and academia, participants called for a more “evolutionary” approach to agreeing to PIPs for medicines where knowledge at the time of PIP submission is limited. The announcement of the pilot also coincides with the publication of a report on the steps EMA has already taken or plans to take to improve pediatric drug development in the region.
 
According to EMA, the sPIP framework will enable drugmakers to move forward with development with a “minimum set of data … as a basis which should consider the state-of-the-art scientific knowledge and current drug development experience in the relevant field,” then return to the agency’s pediatric committee (PDCO) to update the plan as new information becomes available.
 
“This will allow, in selected cases, to leave some elements of a PIP open to further refinement after first agreement, with a commitment to fill the gaps once certain milestones are reached, and necessary scientific evidence becomes available,” EMA said. “Moreover, the key elements to be specified in PIP opinions have been revised in order to focus only on essential elements at an appropriate level of detail.”
 
EMA explains how the sPIP pilot will work, when participation would be justified, as well as practical aspects of sPIP submission in a new guidance.
 
“The sPIP concept will be piloted starting in the first quarter of 2023 with 8 sPIP adopted initial opinions in order to gather experience to shape the process accordingly and to inform decision making on its use in the future,” the EMA said in its guidance. “The scope and the process may be further defined once the experience from the initial pilot is analyzed.”
 
“In cases where the sPIP is envisaged to be applied, a dedicated pre-submission meeting with the assessment team is strongly recommended,” the agency added. “In the presence of relevant existing guidance and precedence (including agreed PIPs in similar therapeutic areas) it is not expected that a stepwise PIP approach would be warranted in most cases.”
 
In its report on efforts to promote pediatric drug development, EMA lists five steps it has taken or plans to take in the wake of the 2018 workshop. The agency notes that it has brought together key stakeholders in recent years to identify and raise awareness of areas where pediatric drugs are acutely needed with the aim of shifting to research to those areas.
 
EMA adds that it has better aligned data requirements between decision-makers; which it says has improved compatibility and collaboration regarding pediatric requirements between European and international regulators.