It is widely known that approximately 300 million people around the world are living with rare conditions. Approximately one third of rare conditions are acknowledged to include neurological components and symptoms due to the condition affecting the number and/or function of nerves. So, even if they are not categorised as a neurological rare condition, patients’ may still cause a wide range of nervous-system derived experiences, including (but not limited to) pain, headaches, seizure-risk, mobility issues, and/or reduced mental capacity.

Rare disease experts with experience in clinical trial design and delivery use their knowledge to build with these conditions in mind. Every child’s experience of their condition is likely to be different, and the breadth of symptoms that rare disease trial participants may experience needs to be acknowledged from the start. 

What’s Inside?

In this white paper we will be exploring some of the modifications and approaches needed to ensure that patient's who experience neuronal effects of their 
condition have positive experiences in research.

Key topics explored include:

• Trial practicalities
• Consent vs assent
• Legal representation and the context to decisions made in and out of the trial setting 
• Remote vs in-clinic visits - the benefits to patients and considerations for sponsors
• Delivering a trial that lasts