Improving the Paediatric Framework: Aligning Ambition with Reality in the Revision of the General Pharmaceutical Legislation

As part of the broader revision of the EU’s General Pharmaceutical Legislation (GPL), the integration and update of the Paediatric Regulation is a timely and necessary development. The revised framework provides an opportunity to build on the strengths of the existing Regulation, while addressing long-standing challenges that have slowed or discouraged the development of medicines for children.

EUCOPE supports the Commission’s ambition to create a more supportive environment for paediatric innovation. However, several elements of the current proposal, in particular the five-year deferral cap[1] and the lack of significant improvements on Paediatric Investigation Plan (PIP) procedural timelines, risk undermining that objective.

Below, we outline key considerations and recommendations to ensure the revised framework is both scientifically sound and operationally feasible and efficient.

Deferral Durations: The Risks of a Rigid Five-Year Cap

As part of its proposal to revise the GPL, the European Commission introduced a five-year time limit for completing deferred measures under a PIP. Deferrals are granted with a view to “ensuring that research is conducted only when safe and ethical and that the requirement for study data in the paediatric population does not block or delay the authorisation of medicinal products for other populations”[2]. Albeit not explained in the legislative text, a time limit on deferrals appears to aim at reducing delays in paediatric development and ensuring that studies are conducted in a timely manner.

While these aims are valid and widely shared, the proposed five-year cap is not justified and fails to reflect the scientific, operational, and ethical complexities that characterise paediatric clinical research. In areas such as oncology, rare diseases, and advanced therapies, paediatric studies often require long timelines to adapt formulations, enrol small and heterogeneous patient populations, comply with age-specific ethical standards, and ensure adequate observation follow-ups.

The current system, under which the European Medicines Agency (EMA) assesses and approves deferral timelines based on product- or development-specific justifications, already provides a flexible and science-based mechanism. Importantly, the system allows for adaptation in response to unforeseen delays, emerging scientific data, or new regulatory requirements. Therefore, we believe that the system does not necessarily require legislative changes. The European Medicines Agency and European Commission’s DG SANTE (Directorate-General for Health and Food Safety) action plan on paediatrics[3] acknowledged that completing a paediatric development plan depends on a variety of factors, and a limited number of measures were put in place to simplify and expedite paediatric trials (design and conduct). However, it remains unclear how these incremental changes would justify an expectation that in the future deferrals should in all cases be for five years maximum, or – as possibly implied by proposed rules – will only require one adaptation during the PIP life cycle.

Imposing a fixed five-year cap could force companies to start paediatric studies before they have the necessary adult data, making it harder to design effective and feasible studies. Rather than driving earlier availability of paediatric data, the rigidity of the proposed cap could dissuade investment in paediatric development, particularly in areas where studies are already highly complex. Furthermore, what would happen should developers fail to complete deferred measures in time despite their best efforts?

In conclusion, placing an arbitrary cap on the duration of a deferral, irrespective of the specifics in question, is tantamount to imposing a legal “minimum speed limit” on paediatric drug development, which is unprecedented, and potentially damaging both for developers and, more importantly, for patients.

EUCOPE therefore recommends that the five-year limit is removed, and that the existing, science-based assessment of deferral timelines is maintained (we acknowledge the Parliament’s edits to recital 112 in this respect). This way, we can ensure sufficient time for PIPs, while balancing the need to develop more paediatric medicines as expeditiously as realistic, with the imperative to safeguard the safety and quality of both clinical trials and the medicines themselves.

Procedural Timelines: Efficiency Requires More Than Defined Deadlines

Lengthy PIP procedures remain a significant barrier to timely paediatric development. Due to the complexity of the development issues at stake, the amount of background information to be reviewed, and the number of measures to be discussed, it is common for a PIP procedure to last 6 – 9 months altogether, and at times (much) longer. Developers experience PDCO requests for important modifications to the proposed plan until the last stage of the PIP procedure, with limited time available for impact assessment and company internal alignment. All the while, in other regions other than EU there are no paediatric requirements, or there are more efficient processes to discuss and agree paediatric development plans. For instance, in the US, Orphan Medicinal Products (OMPs) are exempt from the requirement to submit a Paediatric Study Plan (PSP). For companies operating in the rare disease space and developing drugs for both the EU and the US, this exemption represents a significant advantage.

While the revised legislation includes some adjustments to PIP procedural timelines, these may not translate into shorter procedures in practice. Under the proposed revised framework:

• After positive validation of the application (timelines unchanged), standard PIP procedures would take 90 days from start to decision (currently: 60 days from start to opinion), however, the active review time can be extended for a maximum of 90 days (currently: maximum of 60 days) for handling the agency’s requests for modification.
• “Stepwise” (initial) PIPs foresee shorter initial timelines (70 days from start to opinion) but remain subject to similar extension rules (extension of up to 70 days in case of request for modification following assessment of the proposed plan).

The new proposed EMA structure aims to streamline the final stages of the process, including PIP decision-making. However, it’s worth noting that the proposed regulation sets a 180-day timetable (or 140 days for stepwise PIPs), compared to the current 120-day timeline. While the finer details, such as clock-stop rules and the role of any paediatric working party or expert group, will be outlined in future EMA guidance, there’s a possibility that PIP approvals could take longer under the new system than they do now.

In practice, developers still face long and uncertain timelines before initiating paediatric studies. Without a rethinking of the proposed legislative timelines and/or meaningful change to the operational handling of procedures, including validation, request-for-modification management, the revised timelines may offer limited benefit, or even delay things further. We recommend that proposed timelines be reconsidered with a view to expedite the involved regulatory processes.

Conclusion: A Framework That Works in Practice

The revision of the Paediatric Regulation offers a unique opportunity to deliver a more efficient and innovation-friendly environment for paediatric R&D in the EU. EUCOPE supports the EU Commission’s intention to streamline procedures and improve regulatory predictability.

However, this ambition must be matched with operational realism. The proposed five-year deferral cap risks introducing rigid constraints that do not reflect the complexity of paediatric development. And procedural efficiencies will depend not only on timelines set in legislation, but also on how procedures are applied and managed in practice.

Th EU’s competitiveness in paediatric research will depend on a framework that is flexible, proportionate, and aligned with scientific and operational realities. EUCOPE remains committed to supporting a regulatory environment that encourages investment in paediatric innovation, while ensuring timely development and access to high-quality medicines for children.

---

[1] Article 81(3) of the Commission’s proposal for the revised Regulation

[2] Recital 112 of the Commission’s proposal for the revised Regulation

[3] Action 3 – Ensuring timely completion of paediatric investigation plans

Read more EUCOPE Lab Talks here