Commemoration of the International Day for Clinical Trials 19 May 2026

How are these two themes connected? What do children, their relatives, and patients with rare diseases think about participating in clinical trials? What collaborations exist for clinical trialsin Norway and the Nordic countries? And how, practically speaking, are trials on rare diseases and children organised?

- Clinical trials on treatments for rare diseases are important for patients and professionals. They provide patients with earlier access to new innovative drugs, and professionals gain practical experience in administering the treatment, its effects, and the need for follow-up. Clinical trials for rare diseases are particularly challenging because there are very few patients who can be included in each individual country, and they can be difficult to locate in electronic records, says Olve Moldestad, head of the Partnership for Rare Diseases. He is also the head of the Newborn Screening Department at the Division of Paediatric and Adolescent Medicine at Oslo University Hospital (OUS).

Per Kristian Knudsen at NorPedMed, a national network for paediatric research, states that clinical trials involving children are important for the same reason: to ensure that children have access to new treatments.

- New treatments are often approved much later for children than for adult patients, and up to 70% of medications for children in hospitals are used off-label. Therefore, more clinical trials for children are necessary to provide them with quicker access to new treatments. At the same time, children are a vulnerable group, and there are strict ethical and regulatory requirements for clinical trials, says Knudsen, who is also a senior physician at the Clinical Research Unit for Children at Oslo University Hospital.

At the meeting, specific examples of trials on CAR-T treatment for leukaemia, trials involving children with rare cancers, trials on achondroplasia (dwarfism), and trials on rare eye diseases will be presented, to name a few.

The meeting is organised by NorTrials and NorCRIN, in collaboration with the Norwegian Childhood Cancer Association, NorPedMed, the Partnership for Rare Diseases, the National Centre for Rare Diagnoses, and The Nordic Society of Paediatric Haematology and Oncology (NOPHO).

- International collaboration, especially in the Nordic region, is important for clinical trials involving children and for rare diseases. Because there are few patients in each country, we should collaborate across borders to achieve a sufficient number of participants in the trials, says Moldestad.

Clinical Trials Day is celebrated worldwide on 20 May each year. On this date in 1747, James Lind initiated what is often regarded as the first randomised clinical trial. The study took place aboard a ship, with 12 sailors divided into 6 groups, and concluded that oranges and lemons were a cure for scurvy.

Register for the event here: Microsoft Virtual Events Powered by Teams