Overcoming Rare Disease Clinical Trials Challenges

Over 90% of rare diseases have no treatment. There are more than 6,000 known rare diseases. Approximately 80% of them have a genetic component—75% affect children—and most rare diseases are chronic conditions, many of which progress over time and are potentially fatal.

Understanding the rare disease framework is key to being able to offer study participants adapted solutions and as such, positively contribute to important and necessary clinical research conducted on rare diseases.

• What makes rare disease clinical trials so difficult?
• How can we contribute to making this a smoother experience for the patients, their caregivers, and the clinical trials industry?

Find the answers in this case study.