Panel Discussion: Technology in Rare Disease Trials: Best Practices for Patient Centricity

Rare Diseases have a significant medical footprint in society, and while most rare diseases might only affect a few hundreds to thousands of people worldwide, they are collectively common, affecting an estimated 25 million to 30 million people in the United States alone.Many of these diseases have a genetic cause, are serious or life-threatening and are hard to diagnose and treat. The U.S. Orphan Drug Act of the U.S. FDA has stimulated industry investment in clinical trials to develop treatments for rare diseases, yet only about 10% of rare diseases have an FDA-approved therapy for their treatment. Rare disease research poses challenges to investigators requiring specific approaches to the design of clinical studies; the discovery, testing, and approval of new treatments, and the training of patients and the medical community to improve access to diagnosis, research and treatments.

In this panel discussion, we sit down with experts in digital health, clinical research and patient engagement to discuss ways in which technology can address the challenges facing clinical development for rare disease.

Watch the panel discussion to learn:

• How does technology address challenges in the design and execution of clinical studies?
• What are the impacts of technology and decentralization on the integration of clinical research within the continuum of rare disease treatment, from diagnosis to treatment?
• How are partnerships with patients, caregivers and patient organizations changing the landscape of rare disease trials and registries?