Introduction
Developing paediatric medicines in Europe requires careful planning, regulatory expertise, and early engagement with the European Medicines Agency (EMA). Medicine developers must ensure that medicines intended for children are researched ethically, developed appropriately, and supported by robust scientific evidence. A key component of this process is the Paediatric Investigation Plan (PIP), which outlines the strategy for paediatric development and is typically a mandatory requirement before a Marketing Authorisation Application (MAA) can be submitted. In this article, we explore the purpose of the EU Paediatric Regulation, the role of the Paediatric Committee (PDCO), the requirements for PIPs, waivers and deferrals, and the timelines involved in the PIP evaluation procedure.
The proposed revision to the EU pharmaceutical legislation is expected to introduce significant changes to paediatric development in medicines, including:
- Dissolving the Paediatric Committee (PDCO)
- Creating an initial PIP (iPIP) submission option
- Offering free scientific advice on the design of paediatric studies
In this blog, we explore the current EU paediatric medicine development framework, examine the proposed legislative changes, and discuss the potential impact on medicine developers who must include paediatric studies in their medicine development programmes and get a medicine approved in Europe.
The revised pharmaceutical legislation
In April 2023, the European Union (EU) undertook a comprehensive evaluation of the pharmaceutical legislation. It adopted a proposal for a new pharmaceutical Directive (2023/0132)1 and a new Regulation (2023/0131)2, which revise and replace the existing general pharmaceutical legislation (Directive 2001/83/EC3 and Regulation (EC) No 726/20044). This evaluation also included a proposal to repeal the existing Orphan Medicinal Products Regulation (EC) No 141/20005, and the Paediatric Regulation (EC) No 1901/20066 and to identify strengths and areas for improvement in a new regulation.
On the 11th of December 2025, the EMA announced that the European Parliament and the Council of the European Union reached a political agreement on the reform of the EU pharmaceutical legislation. It is now undergoing a formal review by the European Parliament and the Council.
The proposed new pharmaceutical legislation has far-reaching effects on several regulatory deliverables across all stages of pre-approval medicine development, and for this article, we will discuss the effects on orphan-designated medicinal products.
We hosted a webinar outlining the key EU pre-approval regulatory deliverables required to support successful marketing authorisation and discussed how the upcoming EU pharmaceutical legislation will impact regulatory planning and medicine development.
Watch it here: ‘Future-proofing your EU regulatory deliverables and the impact of the revised pharmaceutical legislation on medicine development’.
Developing paediatric medicines in Europe
The paediatric regulation (EC) No 1901/20066 was created to ensure that medicines for children are of high quality and ethically researched. This is to ensure that children aren’t subjected to unnecessary clinical trials. Currently, the Paediatric Committee (PDCO) is responsible for medicines relating to children.
In the EU, an agreed PIP, or a paediatric waiver, is a prerequisite and a legal requirement for an MAA submission. A PIP details the overall strategy, non-clinical/clinical studies, age-appropriate formulations and proposed timelines for all paediatric age groups (0–18 years).6
A deferral of a PIP may be granted to delay the paediatric development of a medicine in some instances, for example, if there isn’t enough data to support its safety or efficacy in adults. A waiver may be granted when medicine development in children is not needed or is not appropriate, such as for diseases that only affect the adult population; if your medicinal product is ineffective or unsafe for children; or if there is a lack of expected therapeutic benefit in children.6
PIP evaluation procedure
The PIP evaluation procedure follows a rigid timeline, and the EMA allows one submission intake per month.
- PIP submission: This involves a validation period followed by 60 days to the adoption of the Request for Supplementary Information (RSI).7
- Clock-stop: Typically, the applicant is allowed 90 days to respond to the RSI.7
- Response to RSI: PDCO will take 60 days to evaluate before adopting an opinion.7
- Pre-MAA compliance check: Takes approximately 60 days to ensure the applicant has complied with the agreed PIP conditions before submitting an MAA.7
In total, the entire PIP procedure takes approximately 12 months to complete. Therefore, you should plan your paediatric development far in advance and carefully consider the following aspects:
- The paediatric condition and indication
- The use of extrapolation from adults to children
- The design of your paediatric trials
- The development of age-appropriate formulations
- Possible deferrals beyond the MAA submission
- The possibility of full or partial waivers
How will the new pharmaceutical legislation change paediatric medicine development?
PIPs will no longer be reviewed by PDCO and instead will fall under the purview of the CHMP.1,2
Additionally, alongside the existing PIP requirements, the new regulation proposes that you can submit an initial PIP (iPIP), as provided for in Article 74 of the revised Regulation.2 The iPIP is for new active substances intended to treat a novel paediatric condition or an existing condition via a new mechanism of action. It is only expected to cover measures known at the time of submitting an iPIP; however, you must justify why you are unable to submit a complete PIP. You must also include timings for when you will submit updated and final versions of the PIP.2
Additionally, the EMA will provide free scientific advice “on the design and conduct of the various tests and studies necessary to demonstrate the quality, safety and efficacy of the medicinal product in the paediatric population,” per Article 89 of the revised Regulation.2
PIP waivers
Per Article 75 of the revised regulation,2 paediatric development may be waived if:*
- The medicinal product or class of medicinal products is likely to be ineffective or unsafe in part or all of the paediatric population,
- that the disease or condition for which the specific medicinal product or class is intended occurs only in adult populations, unless except when the product displays a mechanism of action, including where its action is directed at a specific molecular target or biological pathway, that on the basis of existing scientific data, is relevant for a different disease or condition in the same therapeutic area in children than the one for which the specific medicinal product or class of medicinal products is intended for in the adult population,
- that the specific medicinal product is likely to not represent a significant therapeutic benefit over existing methods of diagnosis, prevention or treatments for paediatric patients, except when the product displays a mechanism of action, including where its action is directed at a specific molecular target or biological pathway, that on the basis of existing scientific data, is relevant for a different disease or condition in the same therapeutic area in children than the one for which the specific medicinal product or class of medicinal products is intended for in the adult population.
*Updated text compared to the current paediatric regulation in italics
Currently, applicants can benefit from PIP rewards. For non-orphan authorised medicines, you can obtain a 6-month extension to the supplementary protection certificate. For orphan-designated medicines, if a PIP is agreed and completed before the 10-year orphan exclusivity expires, then the market exclusivity is extended by 2 years. However, the revised pharmaceutical regulation has proposed that this will no longer be the case; instead, the major reward for completing a PIP will be a 6-month extension to the supplementary protection certificate for both orphan and non-orphan medicinal products.2
Summary
The development of paediatric medicines in Europe is governed by the EU Paediatric Regulation, which aims to improve the availability of safe, effective, and appropriately researched medicines for children. A Paediatric Investigation Plan (PIP) is generally required before an MAA submission and must outline the proposed paediatric development strategy, including studies, formulations, and timelines. Depending on the circumstances, companies may apply for a waiver or deferral where paediatric development is unnecessary, inappropriate, or requires postponement. As the PIP evaluation process can take around 12 months, including compliance checks, early planning is essential to avoid delays in regulatory submissions. Understanding PIP requirements and engaging proactively with the EMA and PDCO can help streamline paediatric medicine development and support successful market authorisation in Europe.
The revised pharmaceutical legislation is proposing to make several changes to how paediatric medicines are developed. For pharmaceutical and biotechnology companies, understanding these evolving requirements will be essential for effective regulatory planning and long-term commercial success.
How Somerville Development Partners can help
Our team brings decades of combined experience across both consultancy and industry settings. This dual perspective enables us to balance regulatory rigour with scientific and operational realities.
We don’t just advise, we embed within your team to deliver high-quality regulatory solutions to help pharmaceutical and biotech companies develop safe and efficacious medicines for patients.
Whatever your needs, we are your trusted life sciences partner:
Long-term (12–24 months) projects, such as an MAA, new drug application or biologics license application.
Ad-hoc or complex development services such as regulatory due diligence, paediatric development or orphan designation if you are developing medicines for rare diseases.
Short-term (3–6 months), discrete projects to address a particular need for resources or expertise.
