EMA said stepwise PIP pilot is feasible in expediting pediatric drug development

The European Medicines Agency (EMA) has announced that its pilot program for the stepwise pediatric investigation plan (sPIP) has successfully supported timely pediatric development while maintaining scientific rigor. This program will be incorporated into the existing pediatric regulatory landscape.

These were the results of a report released by EMA called “Review of the stepwise paediatric investigation plan (sPIP) pilot: Outcomes and future perspectives.”

“The pilot demonstrated feasibility and value supporting timely paediatric development while ensuring scientific rigour and flexibility for future integration into routine regulatory practice. Future efforts will focus on refining milestone management, embedding the framework into standard regulatory practice, and ensuring long-term monitoring of paediatric development programmes implemented under sPIPs, in order to safeguard sustained regulatory oversight and public health outcomes,” EMA said.

The pilot targets rare or complex pediatric development programs “with major scientific uncertainty.”

The sPIP pilot was launched in February 2023 to increase the efficiency of pediatric drug development and boost the development of treatments for children by allowing sponsors to submit a partial development program until other data elements can be collected. (RELATED: EMA launches ‘stepwise PIP’ pilot, Regulatory Focus 6 February 2023)

EMA received 27 requests to participate in the pilot. Of these, 15 stepwise PIPs were submitted for assessment. The agency rendered an opinion on eight sPIPs.

Four opinions were for neurologic and metabolic genetic disorders, four were in the immunology area, two were in the hepatology space, and one was in the oncology area. Half of the products were classified as new chemical entities, while 37.5% were categorized as biologicals and 12.5% were considered advanced therapy medicinal products (ATMPs).

The report indicated that the eight sPIPs with an adopted opinion were examined in greater detail. The analysis focused on the types of studies included and whether certain key elements were more frequently left undefined at the initial submission stage.

In the initial studies of sPIPs, several key elements were often left undefined. Specifically, sample size was not specified in 12 out of 21 studies, while dosage was not defined in 10 out of 21 studies. Additionally, other undefined elements included endpoints (eight studies), study duration (five studies), statistical analysis methods (six studies), and control measures (two studies).

In conclusion, EMA said that “the sPIP framework accommodates the iterative nature of paediatric medicines development, in which early evidence progressively informs subsequent study design.”

This flexibility can help prevent unnecessary delays in starting studies by facilitating early interactions between sponsors and regulators. This is particularly important for medicines targeting life-threatening or rare pediatric conditions.

The European Medicines Agency (EMA) has agreed to conclude the pilot phase and integrate the sPIP framework into the current regulatory landscape on a voluntary basis. Additionally, EMA will develop and publish updated guidance documents on its website. Finally, EMA has emphasized that the sPIP is a complementary mechanism designed for exceptional cases and should not be viewed as a general alternative to standard Pediatric Investigation Plans (PIPs).

Under EU pharmaceutical legislation, medicine developers are legally required to submit a pediatric investigation plan (PIP) to the European Medicines Agency (EMA) early in the product development process. This plan must be submitted before completing pharmacokinetic or pharmacodynamic studies in adults, or before starting studies in children for medicines intended solely for pediatric use.

sPIP

EMA said stepwise PIP pilot is feasible in expediting pediatric drug development

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