Better medicines for children: Collaboration is the key to transforming pediatric drug development
Author
Isabella Presch, MD, MBA, is a Senior Medical Director at Fortrea. She is a certified pediatric surgeon with more than 10 years of clinical experience across therapeutic areas in academia as well as private and public hospitals. Her pharmaceutical industry experience spans more than 20 years across several indications, including a focus on hematologic rare diseases across Phase I–IV. Dr. Presch has broad experience developing and overseeing global clinical development programs and collaborating with internal and external stakeholders, including the FDA, PMDA, EMA, PEI and reimbursement agencies. She also has supported global and local medical affairs, including the launch of innovative treatments in rare hematologic disease and immunology and has significant experience supporting due diligence activities. Dr. Presch is a strategic, cross-functional global leader with experience supporting rare disease, advanced therapies and pediatric clinical development activities across several therapeutic areas. She was recognized in 2022 with the prestigious Labcorp Chairman’s Award for her outstanding contributions.
The European Forum for Good Clinical Practice (EFGCP) Better Medicines for Children Conference 2026 in Amsterdam brought together leaders from across the pediatric development ecosystem under the theme, “Collaborating to Implement Innovative Approaches in Pediatric Development.” Regulators, industry leaders, patient advocates, clinicians, researchers and policymakers gathered to discuss how innovation and collaboration can help improve health outcomes for children worldwide. 1,2
Several themes emerged throughout the conference, highlighting both the progress being made and the work that remains to accelerate access to better medicines for children.
Children deserve more than adapted adult medicines
Too often, medicines are developed for adults first, with pediatric evidence generated years later. Yet children are not simply “small adults.” Their diseases, physiology, developmental stages and treatment needs require dedicated research and tailored development strategies.
Discussions throughout the conference reinforced that improving access to innovative therapies for children requires early planning, scientific innovation and a willingness to challenge traditional development paradigms. Ethical considerations, age-appropriate formulations, novel endpoints and patient-centered study designs all play an essential role.
The clear message: pediatric development must begin with the child in mind, not as an afterthought.
Success requires an entire ecosystem
One of the strongest messages from the conference was that no single organization can solve the challenges of pediatric drug development alone.
Meaningful progress requires collaboration among:
- Pharmaceutical and biotechnology companies
- Regulatory authorities
- Clinical investigators and healthcare providers
- Patients and caregivers
- Patient advocacy groups
- Research networks
- Health Technology Assessment (HTA) bodies and payers
- Contract research organizations (CROs)
Every stakeholder brings a unique perspective that helps create better pediatric clinical trials, stronger evidence and ultimately faster access to treatment.
The critical role of regulators and HTA
Discussions led by European Medicines Agency (EMA), Food and Drug Administration (FDA) and Pharmaceuticals and Medical Device Agency (PMDA) representatives throughout the meeting emphasized the importance of engaging regulators early to create efficient, scientifically robust development pathways. Whether through innovative trial designs, formulations or Chemistry, Manufacturing and Controls (CMC) regulatory collaboration remains central to accelerating access for children.
Equally important was the focus on market access and HTA. Pediatric innovation only benefits children if therapies are ultimately accessible and reimbursed. Increasingly, sponsors need to consider regulatory and HTA evidence requirements in parallel, ensuring that studies generate data that demonstrate not only safety and efficacy, but also meaningful value for patients, families, healthcare systems and society.
The future of pediatric development depends on aligning regulatory, clinical, patient and access perspectives from the very beginning.
Patient-centered approaches in pediatric drug development
Another clear takeaway from the conference was the growing recognition that children and families are essential collaborators in pediatric research.
Patient organizations and caregivers contribute invaluable insights into what matters most: meaningful outcomes, practical study designs, reduced trial burden and improved access to treatments. Their voices help shape development programs that are not only scientifically sound but also relevant to real-world needs.
The best innovations occur when research is built with patients, not merely for patients.
How Fortrea supports better medicines for children
The themes discussed throughout the conference highlighted the increasing complexity of pediatric drug development. Advancing therapies for children requires deep therapeutic experience, pediatric regulatory understanding, patient-centric thinking and the ability to connect stakeholders across a highly complex ecosystem.
At Fortrea, our teams support sponsors through:
- Pediatric clinical development strategy
- Rare disease and orphan drug development
- Cell and gene therapy programs
- Global regulatory and operational planning
- Patient-focused study design and recruitment strategies
- Long-term follow-up programs
- Evidence generation to support both regulatory and market access needs
Through Fortrea’s Rare Diseases, Advanced Therapies and Pediatrics (RAPT) experience, we work alongside sponsors, investigators, advocacy groups, regulators and healthcare providers to help bring innovative therapies to children faster and more effectively.
The future of pediatric drug development depends on collaboration
One thing became abundantly clear during the conference: the future of pediatric medicine will be built through collaboration.
Scientific breakthroughs are creating unprecedented opportunities for children with rare diseases and previously untreatable conditions. However, transforming innovation into real-world patient benefits requires alignment across the entire healthcare ecosystem.
Industry must innovate. Regulators must enable. Patients must be heard. HTA bodies must help ensure access. Research organizations must execute with excellence.
And together, the industry must never lose sight of the ultimate objective: delivering better medicines to children who cannot afford to wait.
Thank you to the organizers and participants of the EFGCP Better Medicines for Children Conference 2026 for fostering these important conversations and driving meaningful collaboration across our industry.