Patient recruitment for clinical trials can be a very challenging aspect of trial design, especially for rare diseases with small and fragmented patient populations. This should therefore be considered early on, and patient sources identified as soon as possible so that collaborations can be set up with those clinical sites with prospective patients. Patient groups are often able to offer an invaluable source of potential recruits for clinical trials through their extensive patient networks.

For the MCDS-Therapy Project, the first patients will be recruited in the UK by Newcastle-upon-Tyne Hospitals NHS Foundation Trust and the Evalina London Children’s Hospital. After a year, recruitment will be expanded to the international sites in the partnership (MCDS therapy partners). For MCDS, as with many rare paediatric diseases, recruitment can be challenging as patients are often misdiagnosed. In addition, there is only a small age window in which patients can be recruited as they must not have reached skeletal maturity. This highlights the importance of international collaboration in drug repurposing projects.

Data collection and storage

Under the new General Data Protection Regulation (GDPR) laws in the EU, health data is considered ‘sensitive data’, which may only be processed with explicit consent or based on medical care need. Patients’ data should be stored in a secure online system and shared only in the contexts that are permitted in the clinical study. For further information on GDPR, click here.