Janneke van der Kamp
CEO, Norgine
We must address the challenges of expanding the reach of medicines for rare childhood diseases in Europe. Too many children are unable to access new medicines. Here’s how we can fix it.
Rare diseases, including childhood cancers, are a major cause of death for children.R are diseases affect up to 36 million people in the EU;1 three-quarters are known to affect children,2 and about 3 in 10 children affected by a rare disease will die before their fifth birthday.3
Only 5% of rare diseases have effective treatments, leaving most without options.4 A fifth of children with cancer will not survive5 and up to 90% of survivors live with the burden of late effects.6
Turning new science into actual medicines
Scientific innovation is constantly advancing the standard of medicines for rare childhood diseases, but securing equitable access is challenging and complex. Many of the best new medicines are developed by small biotechnology companies that focus on cutting-edge science but lack specific expertise and resources to get their medicines widely approved and distributed.7 Many of these companies are US-based, and there is a significant delay in paediatric medicines reaching patients in the rest of the world.
At Norgine, we know that local expertise is vital, especially in Europe. As a mid-sized pharma company, we are able to partner with small biotechs to bring much-needed medicines to Europe, a process that needs an innovative mindset. It’s important to recognise that innovation does not end in the laboratory. It involves navigating the complex and divergent healthcare systems in Europe to find ways to bring medicines to patients.
Many promising treatments for
children face significant delays or
may never reach patients at all.
Innovation across the whole healthcare process
It is tempting to think that a life-saving children’s medicine will naturally make its way to patients, but this is not the reality. Many obstacles occur long after scientific innovation.
Medicines for children present unique challenges. There are, rightly, special ethical considerations for children in clinical trials; achieving the best risk-benefit balance is extremely important. Trials in rare diseases are especially difficult due to small patient numbers, drawn from diverse groups over wide geographies.8
To solve all of these challenges means working with multiple patient organisations and scientific centres of excellence in line with various nuanced medical guidelines.
Paediatric approvals face complexity
Navigating Europe’s paediatric drug approval processes can be particularly complicated. The new EU Health Technology Assessment (HTA) process and national HTA processes are complex and require different clinical data and study designs, depending on the healthcare system and country.
National health insurance structures vary; clinical data is interpreted based on specific country healthcare system requirements, and there are differing regulations and reimbursement criteria.
To add to the complexity, paediatric medicines must go through additional regulatory steps, requiring pre-approval of detailed plans studying the effects in children of different ages9 — which is time-consuming but vital for safety. Each step must be supported by state-of-the-art manufacturing and local distribution to guarantee consistent quality and supply.
Children’s health can’t afford delays
None of this can be taken for granted. Consequently, many promising treatments for children face significant delays or may never reach patients at all in parts of Europe and beyond. Understanding the path, however complex, is the first step towards finding solutions. Norgine’s agility and our deep regional expertise make us uniquely placed to navigate these processes. We’re proud to do so because the children we serve don’t have time to wait.
This article was developed and funded by Norgine to support dialogue around children’s healthcare in Europe. Find out more at norgine.com
[1] European Commission. 2025. Rare diseases.
[2] Murdoch children’s research institute. Rare genetic disorders.
[3] National Stem Cell Foundation. Rare Childhood Diseases.
[4] US Government Accountability Office. Rare Disease Drugs: FDA Has Steps Underway to Strengthen Coordination of Activities Supporting Drug Development.
[5] Lam CG, et al. Science and health for all children with cancer. Science. 2019;363(6432):1182–86.
[6] National Cancer Institute. Late Effects of Treatment for Childhood Cancer(PDQ®)–Health Professional Version: General Information About Late Effects of Treatment for Childhood Cancer.
[7] Vital Transformation. 2020. The US Ecosystem for Medicines. How new drug innovations get to patients.
[8] Nony P, et al. A methodological framework for drug development in rare diseases. Orphanet J Rare Dis. 2014;9:164.
[9] Zisowsky J, et al. Drug Development for Pediatric Populations: Regulatory Aspects. Pharmaceutics. 2010;2(4):364–88.
