Thinking of the Individual in Rare Disease Clinical Trials

Rare diseases present unique challenges in drug development due to their low prevalence and the diverse nature of each individual condition. Traditional drug development approaches rely on large-scale research to create medicines that cater to the "average patient," leaving those with rare diseases underserved.

However, there is hope on the horizon with the emergence of personalized/precision medicine. This innovative approach to treatment has gained popularity, particularly in the field of cancer, and has revolutionized drug development. By tailoring therapies to the specific genetic makeup and characteristics of each patient, personalized medicine offers a promising solution for improving outcomes in rare diseases.

What’s Inside?

• In this white paper, we delve into the changes that personalized/precision medicine brings to the table, exploring its potential impacts and limitations on developing therapies for rare diseases.
• We hear from a patient advocate with a rare condition as well as health care professionals - what are their opinions on the advancements of precision/personalised medicine?
• An Indepth analysis of the impact of personalized/precision medicine in rare disease
• An exploration of the realities of designing for real-world: finding commonality amongst individuals
•  Valuable insights and considerations for the future, ensuring that this transformative approach can be effectively applied to children with rare diseases.​