Key Takeaways
- Industry is “eager” to use stepwise pediatric investigation plans (sPIPs) following an EU initiative piloting this approach to pediatric medicines development.
- sPIP are designed to alleviate the administrative burden for industry and regulators and ensure pediatric developments are not delayed.
- However, the European Medicines Agency said the future of sPIPs in Europe will depend on pharmaceutical legislation negotiations, which are ongoing.
- The EMA plans to publish a report later this year based on eight sPIP opinions granted under the pilot, and it will work with industry on a joint journal publication.
Pharmaceutical companies could benefit from a new approach to developing pediatric investigation plans (PIPs) under which they can agree on a partial development plan with the European Medicines Agency when some important information is unavailable.
The EMA said earlier this month that companies generally had a positive experience with its stepwise PIP pilot (sPIP), which is designed to alleviate the administrative burden for industry and regulators and ensure pediatric developments are not delayed.
It said that industry was “eager to integrate sPIP into business practices.”
However, a spokesperson for the agency subsequently told the Pink Sheet that while the stepwise PIP approach is included in the legislative proposals for the revision of the EU pharmaceutical legislation, it “will depend on the outcome of the legislative process whether this will be formalized in the new legislation.”
The European Commission adopted a legislative package including a new regulation and a new directive in April 2023, to overhaul and replace the EU pharmaceutical legislation, which is two decades old. Negotiations on the reform are still ongoing.
Under the sPIP pilot, which was first launched in 2023, companies and the regulator can agree on a partial development program if some critical PIP elements cannot be identified, such as study design, populations and main objectives. The pilot was part of an EU push to make pediatric regulatory processes more efficient and stimulate the development of medicines for use in children.
The sPIP process can be used by companies in cases where it is difficult to identify these elements, for instance if the medicine is innovative or intended for a rare disease. The EMA said that of the 15 sPIP submissions made so far, most submissions focused on rare pediatric genetic disorders, followed by oncology.
Industry feedback on pre-submission meetings with the EMA as part of the pilot “was generally positive, highlighting approachability, discussion, and flexibility,” according to an EMA report on the 13th industry stakeholder platform on research and development support.
“The current implementation was appreciated as it did not require separate procedural requirements or timetables, making the sPIP another pathway to a full PIP. Clear guidance on which PIPs are applicable to the sPIP approach was suggested, with potential criteria expansion beyond the pilot’s scope,” the EMA stated.
Industry can expect an EMA report on the pilot, which will focus on opinions issued for eight sPIPs using the new approach, in “either Q2 or Q3” of this year, the EMA spokesperson said.
The agency also plans to build on the experience from both industry and regulators to prepare a joint article for publication in a journal, although the details of this have yet to be released.
The agency will also establish a monitoring mechanism for follow-up on agreed sPIP applications in terms of life-cycle management, including the establishment of performance indicators based on the initial proposals.
Updates On The Pilot
Of the 15 sPIPs that have been submitted using the pilot so far, eight have received an opinion from the EMA to date. The other seven remain under review, but will be evaluated eventually.
“The stepwise PIPs included in the pilot will be followed over the whole development period until the PIP is completed to assess the long-term impact of this approach,” the EMA spokesperson added.
Companies can continue submitting requests following the stepwise PIP approach, “as long as applications adhere to the criteria as published,” the spokesperson explained, despite the fact that the pilot itself has now ended.
“The stepwise PIP remains a plan to be agreed early on during the development stage of the medicinal product for adult patients, and its outcome is a fully developed PIP at the end of the process, same as for a conventional PIP,” the spokesperson stated.
Guidance on sPIPs was released in early 2023 for companies. In January 2024, the EMA stated that its pediatrics committee (PDCO) would focus on further developing sPIPs by reviewing the pilot project.
PIPs are development plans that the EU started requiring of drug developers many years ago to help ensure that the necessary data are obtained through studies in children to support the authorization of medicine for children. All applications for the marketing authorization of new medicines must include the results of studies as described in an agreed PIP, unless the medicine is exempt because of a deferral or waiver.
