This article is part of our series covering the proposed reforms to the EU pharmaceutical legislation. To view the entire series, click here.

On 4 June 2025, the Council published its stance on the “pharma package”, introducing notable changes that will influence how paediatric medicines are developed across the EU. In this article and following our July 2023 analysis of the European Commission’s proposed reforms to the regulation of paediatric medicinal products (“PMPs”) and our October 2024 review of the EU Parliament's adjustments to these proposals, we examine the Council's key amendments and their potential impact on the pharmaceutical industry.

Key changes at a glance

The Council's position introduces several important changes to the existing framework. Companies will have to submit Paediatric Investigation Plans (PIPs) earlier in the development process—before safety and efficacy studies begin rather than after pharmacokinetic studies are completed. This shift in timing may speed up regulatory review but would also increase early-stage compliance requirements. The Council proposes toextend the derogation from the requirement for full PIPs, clarify the scope of PIP waivers and introduce various procedural adjustments. In detail:

Extension of exemptions from full Paediatric Investigation Plan requirements – Article 74 (2)

The Council has amended the rules for PIPs, specifically Article 74(2) of the proposed new Regulation. This provision creates an exception that allows companies to submit only an “initial” PIP in certain circumstances. The Council’s amendments expand the scope of the exemption to include products for diagnosing and preventing new paediatric conditions (treatments being covered already), and also products for diagnosing, preventing or treating existing conditions using new mechanisms of action.

In practice, this could provide relief for companies in early development phases. This exemption allows companies developing innovative treatments to take a step-by-step approach, with additional requirements to be met later in the development process.

PIP waivers – Article 75 (1)

The amendments to Article 75(1) propose to change the scope of PIP waivers, to apply as follows:

• Adult-only diseases or conditions: Where the disease or condition occurs only in adults, the usual possibility of a waiver would not apply when the product has a mechanism of action that, based on existing scientific data, is relevant for a different disease or condition in children in the same therapeutic area.
• Limited therapeutic benefit: Where the medicinal product is unlikely to provide significant therapeutic benefit over existing methods of diagnosis, prevention, or treatment for paediatric patients, the possibility of a waiver would not apply when the product has a mechanism of action that, based on existing scientific data, is relevant for a different disease or condition in children in the same therapeutic area.

The approach in these proposed amendments seems to directly align with the framework established in the Nycomed decision (Case T-52/09, 14 December 2011): In Nycomed, the General Court rejected the applicant's argument that a PIP waiver should be granted simply because the company restricted its indication to coronary artery disease, which occurs only in adults. The Court held that the assessment must consider what the medicinal product is objectively capable of detecting, not merely the narrow indication proposed by the applicant. Specifically, the Court found that because the ultrasound imaging agent could detect myocardial perfusion defects that may be associated with various diseases occurring in both adult and paediatric populations, a waiver could not be granted even though the specific indication (coronary artery disease) was adult-only.

Article 75(1)(b) codifies this judicial approach by creating a "reverse exception" that prevents companies from obtaining exemptions for adult-only diseases when "the product has a mechanism of action that, based on existing scientific data, is relevant for a different disease or condition in children in the same therapeutic area." This provision effectively implements the Nycomed Court's reasoning that exemptions cannot be justified solely on the grounds that one underlying condition does not occur in children, if the medicinal product has broader applications in the paediatric population.

Both the Nycomed decision and Article 75 require an objective, science-based assessment of the medicinal product's actual capabilities and potential applications in the paediatric population, ensuring that the regulatory framework prioritises comprehensive paediatric medicine development over narrow commercial strategies.

Adjustments to Procedures and Timelines 

The amendments also suggest adjustments to procedural timelines when it comes to the Prolongation of Deferrals (Article 82) and the Procedures in Article 87:

With regard to Article 82(2), the European Medicines Agency (EMA) may now set a deadline for the submission of further documentation, in which case the time limit of 60 days shall be suspended. 

Article 87, concerning the procedure for adopting a decision in relation to a PIP, waiver or deferral, has also been amended to provide a default requirement for the EMA to consult with the Committee for Medicinal Products for Human Use before reaching scientific conclusions, but also requiring scientific principles to determine the situations when consultation is not required to be established. There is also provision for the applicant to request a re-examination of the scientific conclusions within 10 days, and the Agency has 20 days to either change or confirm its conclusions. If no review is requested, the conclusions automatically become final after 10 days. This creates better procedural protections while keeping decision-making efficient.

Annex II (25) modifies the timing requirements for Paediatric Investigation Plan (PIP) submissions. Under the amended provision:

New Requirement:

• PIPs must be submitted to the EMA, before the initiation of safety and efficacy clinical studies
• Submission must be accompanied by an application for approval or exemption

Previous Requirement:

• PIPs were required to be submitted no later than upon completion of pharmacokinetic studies in adults

This change moves PIP submission from after pharmacokinetic studies to before clinical trials begin, potentially speeding up regulatory processes but requiring earlier commitment to paediatric development strategies.

Next steps: trilogue negotiations

The pharma package proposals will now proceed to trilogue negotiations between the Council, European Parliament, and Commission to achieve consensus on the final form of the new Regulation. These negotiations will be crucial in determining the final balance between encouraging paediatric development and ensuring timely patient access to medicines. The outcome could significantly influence how pharmaceutical companies approach paediatric medicine development across the EU, potentially reshaping investment strategies and development timelines for products intended for children.

What should pharmaceutical companies do next?

• Monitor trilogue negotiations closely as they will determine the final regulatory framework
• Review current paediatric development strategies in the context of earlier PIP submission requirements
• Assess whether products may qualify under the new “initial” PIP derogations or the clarified waiver provisions
• Consider the impact of the parallel proposed changes to regulatory data and market protection periods and how these could impact on paediatric product investment decisions

If you need advice on navigating the evolving paediatric medicine regulatory landscape, our pharmaceutical regulatory team is well placed to assist you. Please reach out to the authors for guidance.