Dr. Terry Derks and Prof. Dr. Jos Kosterink are the RARE-NL liaisons from the University Medical Center Groningen (UMCG). Their strength lies in combining care and pharmacy: Derks is a paediatrician specialised in metabolic diseases and coordinator for rare diseases, while Kosterink is a hospital pharmacist and head of Clinical Pharmacy and Pharmacology. With these complementary perspectives, they successfully bridge patient care, research, and drug development — ensuring innovations ultimately reach patients.
UMCG has a long tradition of working with rare diseases. This is no coincidence, according to paediatrician Derks. University medical centres are a “last resort,” particularly for this broad group of conditions. Paediatricians have traditionally been confronted with “difficult scenarios,” which, together with the strong involvement of parents, may have fostered the drive to innovate. “I think we can be an example for others,” Derks notes.
Hospital pharmacist Kosterink observes that both pharmacists and physicians are actively engaged in rare disease research and care. “The collaboration is there, and the medicines are available. When needed, we produce them ourselves or import them.” A striking example of such collaboration is the CAR T-cell research project. Together with universities and biotech companies, UMCG is optimising the CAR T-cell production process for local manufacturing within the hospital. This not only has the potential to lower the costs of these expensive therapies, but Kosterink sees another major advantage: “By producing these living medicines ourselves, we gain deeper insight into their behaviour, have greater control over quality, and can innovate faster.”
Although UMCG has both extensive expertise and suitable patient populations for research, both experts see added value in a national platform like RARE-NL. Kosterink explains: “Ultimately, we want medicines to reach patients. We often stop at proof of principle, and beyond that, individual university hospitals often lack the knowledge and experience to take the next steps. RARE-NL does have that.”
Derks adds: “The platform can help strengthen our centres of expertise and ensure that we continue learning from each other — both within UMCG and across the country.”
