To advance therapy development for (rare) diseases, it is essential that physicians, pharmacists, and researchers are introduced to this field during their training. RARE-NL supports educational initiatives, which are already emerging in many places across the Netherlands. For example, a minor in Academic Pharma is offered to medical students in Leiden, and a specialisation in Development of Cell, Drug and Gene Therapies will be launched in the Biomedical Sciences master’s programme in Nijmegen.

“Our shared goal is to accelerate the availability of affordable treatments for patients with rare diseases,” says Dr Saco de Visser, Scientific Director of FAST. “Collaboration and knowledge exchange within the FAST hub RARE-NL are crucial in this regard. Education is an integral part of this effort, ensuring that future professionals are equipped with the necessary knowledge on therapy development so they can actively contribute to it or apply new therapies more effectively.”

Anyone interested in developing educational modules for future physicians, pharmacists, or researchers can draw on the RARE-NL network. “Sharing teaching materials and faculty allows us to provide a more comprehensive perspective,” says Dr Jeroen van Smeden, Director of Education at the Centre for Human Drug Research (CHDR). This is also reflected in discussions with Dr Rick Greupink (Radboudumc) and Prof. Dr Teun van Gelder (LUMC) about their educational programmes. “We will certainly invite lecturers from RARE-NL and other organisations, such as the CCMO (Central Committee on Research Involving Human Subjects) and the CBG (Medicines Evaluation Board),” Greupink adds.

Minor programme for medical students

Van Gelder and colleagues developed a 10-week minor programme for medical students in Leiden, which was offered for the second time in autumn 2025: “Before the start of their third year, students can choose from several minors. Our minor, Academic Pharma, immerses medical students in the full trajectory of drug development, from molecule to regulatory approval. It is a complex pathway, with many challenges and potential obstacles. The focus is on developing treatments for rare diseases, such as gene therapies, as these are particularly aligned with academic therapy development.”

The minor also covers antibiotic development and drug repurposing. Guest lecturer Rosan Kreeftmeijer-Vegter (EATRIS) engages students with the serious game Repurpolis, developed in collaboration with FAST, which explores the challenges and trade-offs involved in repurposing. More recently, EATRIS and FAST launched a similar serious game on ATMP development, Genetropolis. In addition, Prof. Dr Agnes Kant provides lectures on adverse event reporting and pharmacovigilance at LAREB, where she serves as director, while Van Smeden offers students insight into early-phase drug development at CHDR. Van Gelder concludes: “It is rewarding to teach highly motivated students who have specifically chosen this topic. Of course, we cannot teach them everything in 10 weeks, but we hope to spark sufficient interest for them to pursue this field later in their careers, for example within one of the many companies at the Leiden Bio Science Park.”

Master’s programme in Nijmegen

In Nijmegen, funding from the Dutch Ministry of Education, Culture and Science (sector plan funding) has been used to accelerate therapy development for rare diseases through the Therapy Accelerator for Rare Diseases. “We are strongly committed to research, but we also want to give this topic a prominent place in education,” says Greupink. Together with colleagues, he is developing a specialisation in Development of Cell, Drug and Gene Therapies within the Master’s in Biomedical Sciences, which will start in the 2026–2027 academic year. “We aim to train professionals who can work both in academia and in the pharmaceutical industry as researchers, or, for example, in regulatory bodies such as the CBG.”

The programme builds on Nijmegen’s expertise in innovative therapies, including antisense oligonucleotides, cell therapies, and gene therapies. Both preclinical and clinical development, up to and including market authorisation, are covered. Experts from other university medical centres within the RARE-NL network will contribute as guest lecturers, alongside specialists in ethics and regulation. A key component of the programme is a six-month internship at a research institute, company, or regulatory authority. “The strength of a master’s programme is that students come from diverse academic backgrounds and receive multidisciplinary training. We aim to educate professionals who can act as connectors in their future roles. After all, therapy development requires professionals who are versatile and able to operate across domains.”

CHDR as an educational ‘hub’

In addition to the initiatives in Nijmegen and Leiden, other university medical centres and universities are also responding to the growing demand for professionals with expertise in therapy development for rare diseases and drug repurposing. Van Gelder notes: “This year, we hosted colleagues from Rotterdam who are developing their own educational programmes and are interested in adopting elements from our minor.” De Visser and Van Smeden also deliver guest lectures at various institutions, including within the Master’s in Personalised Medicine in Amsterdam. They will also contribute to the Nijmegen master’s programme.

“Education is one of the key ways in which we work towards our goals within FAST and RARE-NL,” says De Visser. “I am pleased that we can draw on the expertise of Jeroen van Smeden and his colleagues at CHDR. He is fully dedicated to educational development. CHDR provides its Teaching Resource Centre (TRC) free of charge to students worldwide. It is a beautifully designed online pharmacology resource with a distinctive visual language that is highly suited for teaching drug development. They also run a large clinical pharmacology training programme in collaboration with LUMC. Thanks to their expertise in educational development, CHDR serves as an important hub for education.” Van Smeden adds: “Educational development and capacity building are strategic priorities for our organisation. Moreover, educational collaboration often leads to new forms of cooperation, including in research and drug development.”

Connecting knowledge across the development chain

De Visser concludes: “The bottom line is that professionals at different stages of their training gain relevant knowledge on drug repurposing and therapy development for rare diseases. RARE-NL brings together this knowledge and the associated teaching materials across the entire development chain and can play a connecting role between university medical centres. This also applies to specific expertise on how to ensure patient access to these therapies, including regulatory aspects and appropriate reimbursement.”