Introduction
Despite significant progress fueled by technologies such as gene therapies, there are still no effective therapies for most of the more than 7,000 rare diseases impacting more than 300 million people worldwide. Accurate and timely diagnosis remains problematic. Patient access along with affordability and reimbursement for these novel therapies remain a huge challenge for all stakeholders.
As proud corporate supporters of both NORD and Eurordis, we are committed to working together with key stakeholders to do all we can to accelerate clinical research for rare diseases. To further these efforts, Syneos Health hosted our 2nd Annual Syneos Health Rare Disease Day Symposium on Thursday, February 27, 2020.
The Symposium brought together a diverse group of rare disease experts for thought-provoking presentations and candid discussion about the present and future state of clinical research in rare disease. This year, representatives from the FDA, researchers, patient advocates, industry and the investor community shared perspectives and identified opportunities for the continuing partnership and collaboration needed to drive progress in rare disease research and patient care. This summary report that follows highlights the issues, challenges, resources and solutions covered by our expert presentations and robust discussion.
