Navigating The New EU Pharmaceutical Legislation: Key Implications For Pediatric Drug Development

A conversation between UCB Senior Development Strategy Lead Pediatrics Martine Dehlinger-Kremer, Ph.D., and Clinical Leader Executive Editor Abby Proch

A major overhaul of the EU’s pediatric and orphan drug framework is reshaping how therapies are developed, evaluated, and delivered to pediatric and rare disease populations. The new legislation emphasizes earlier planning, scientific flexibility, and equitable access across EU member states. For some biopharmaceutical companies, this update reinforces what they’ve already been doing; for others, it will serve as a catalyst. And for patients, it has the potential to generate more relevant evidence and support more timely access to appropriate treatments. 

In this Q&A, UCB’s Senior Development Strategy Lead Pediatrics Martine Dehlinger-Kremer, Ph.D., unpacks what these changes mean in practice — from earlier pediatric investigational plans to adaptive development strategies — and how companies can take a more integrated, patient-focused approach.

Clinical Leader: You recently attended the Nordic Conference on Pediatric and Orphan Medicines where the theme was the new EU framework on pediatric and orphan drug research. How will this legislation change pediatric drug development?

Martine Dehlinger-Kremer, Ph.D.: This is the most significant pharmaceutical legislation update that we have seen in the last 20 years. Historically, the development was very much driven by regulatory milestones, but what we are seeing now is a much stronger focus on unmet medical needs, meaningful patient impact, and equitable access across the EU. That means biopharmaceutical companies will increasingly be expected not only to generate robust evidence, but also to ensure that the medicines are available in a timely and equitable manner.

Companies will now need to submit a pediatric investigational plan (PIP) earlier in the development process - before safety and efficacy studies start in adults rather than after pharmacokinetic studies are completed. This allows pediatric strategy to be discussed and integrated much earlier in the drug development process. One important evolution of the PIP is the introduction of the Initial PIP, which provides a more flexible and adaptive framework. This is essential for building and refining development plans as new data comes along. Since drug development often evolves as new scientific evidence emerges, having more flexibility within the framework is incredibly important.

We are also seeing a stronger focus on mechanism of action (MOA) and disease biology. That's a notable step because, historically, some therapies were excluded from pediatric development and were based on adult indication alone.

Another change is the growing link between innovation and access. Companies will increasingly need to consider manufacturing, supply, launch planning, and reimbursement much earlier in development rather than waiting until after approval. This element was introduced in response to persistent inequalities in access to medicines across the EU. The reform aims to improve access to medicines for children, increasing the likelihood that they can receive the same treatments regardless of where they live.

We also have to note a change in the EMA committee structure. At the moment, we have committees for pediatrics, orphan drugs, advanced therapies, and more - quite a large number of committees. To be more efficient, only the committees for Medicinal Products for Human Use (CHMP) and Pharmacovigilance Risk Assessment (PRAC) will remain. The pediatric committee and others will go into a more flexible model, such as working groups, working parties, advisory groups, and a pool of experts that can be mobilized on demand by the CHMP and PRAC. By doing this, we will increase efficiency and preserve scientific expertise.

Why does the PIP conversation need to happen even earlier?

In the past, many companies were not thinking early enough about implementing the pediatric strategy and did not always have an initial PIP in place at the right stage. It was very difficult to prepare a full pediatric plan at a very early stage, especially in case of novel pediatric indication or novel mechanism of action, because as more clinical data emerges, strategy often needs to evolve. This frequently resulted in multiple PIP modifications, which created complexity and slowed progress. To avoid this, companies were trying to submit the PIP later.

With the introduction of the Initial PIP in the new legislation, companies developing drugs with a novel pediatric indication or novel mechanism of action, will no longer be required to commit a fully detailed pediatric development plan at an early stage. Instead, they are able to outline a broader strategy initially and refine it as science develops.

This creates earlier and more meaningful scientific dialogue without sacrificing rigor. It also better reflects how modern drug development actually works, especially as we move toward personalized and mechanism of action-driven therapies. 

What kind of operational challenges might this new legislation present for biotech companies?

The Initial PIP is very favorable for biotech companies, especially those that have a highly innovative product but may not yet have all the knowledge needed to develop a complete pediatric plan. They will still need the complete PIP, but under this new legislation they can submit it step by step.

This differs from the current regulatory framework under which if they were not able to submit a full PIP at the point required by the pediatric regulation - after pharmacokinetic studies in adults – it could be viewed as being late. That created operational pressures, particularly for smaller teams with limited resources.

How will this update impact UCB?

In general, this new legislation reflects what UCB is already doing. But at the same time, the new framework introduces some additional complexities, particularly around conditional incentives, access obligations, and operational readiness. That means we will need to think more holistically about development, access, evidence, generation, and life cycle planning. This regulation reinforces the need to continue embedding pediatric considerations early in asset strategy and to maintain close engagement with regulators and external stakeholders.

How does this update align with FDA expectations?

There is a difference in the timing in the U.S. and in Europe. The PSP, pediatric study plan, for the U.S. is to be submitted 60 days after the end-of-the -Phase 2 meeting, whereas in the EU, the requirement is currently after pharmacokinetics in adults. It's always been earlier than the U.S. and now it will be even earlier.

There is also an important difference in how pediatric requirements apply to orphan drugs. In the U.S., orphan-designated drugs were historically exempt from PREA and thus PSP requirements. However, in 2017, the U.S. enacted the RACE (Research to Accelerate Cures and Equity) for Children Act and since August 2020, certain oncology products with orphan designation became subject to pediatric requirements. Under this Act, a PSP is required for orphan designated cancer therapies if they are molecularly targeted drugs directed at a target that is substantially relevant to the growth or progression of a pediatric cancer, even if the adult indication itself does not occur in children.

Under current legislation, the EU does not have a “MOA PIP”; waivers are granted based on the adult indication when the disease or condition occurs in adults only. Under the new pharmaceutical legislation, however, waivers will no longer be granted if, based on existing scientific evidence, the product’s mechanism of action targets a molecular pathway relevant to pediatric diseases within the same therapeutic area.

A key difference is that in the U.S. this “MOA pediatric plan” applies only to oncology products. In the EU, under the new pharma legislation, the MOA PIP will apply to all products.

What best practices can you offer for companies trying to align with the pediatric aspect of this new framework?

I recommend companies look at pediatric development at a very early stage, to look at their assets and plan their pediatric strategy right from the beginning. I would also recommend involving all key stakeholders early in the development of the asset to ensure close collaboration with the regulators. This should also include early cross-functional engagement within the company, bringing together market access, and health technology assessment (HTA) expertise, to enable alignment on the pediatric strategy.

Ultimately, this is about patients. Our goal should be to ensure that children are not an afterthought in drug development and that, when a medicine has the potential to benefit pediatric patients, there is a clear and practical path to generate the right evidence and make that medicine available to them in a timely and equitable way.

About The Expert:

Martine Dehlinger-Kremer, Ph.D., brings 30+ years of experience in the research industry, with recognized expertise in global regulatory affairs, medical affairs, maternal–fetal medicine, and pediatric development. Driven by the goal of ensuring safe, effective treatments are accessible to every child, she is currently senior development strategy lead for pediatrics at UCB Biosciences, focusing on advancing innovative approaches to pediatric drug development.

She’s held numerous senior leadership and advisory roles, including President of the European CRO Federation (EUCROF) and chair of its pediatric working group.

Dr. Dehlinger-Kremer serves as a board member of the European Forum for Good Clinical Practice (EFGCP), co-chair of its Children’s Medicines Working Party, an advisory board member of the International Children’s Advisory Network (iCAN), and an Enpr-EMA contributor.

Her contributions have been recognized through inclusion in PharmaVOICE’s 100 Most Inspiring People in Life Sciences, the TOPRA Award for Regulatory Excellence, and an honorary membership of EUCROF.