Breaking Down Barriers in Support of Patients with Rare Diseases

A limiting challenge remains the complexity of rare disease development and a rapidly evolving landscape. An increasing number of diseases are targeted by large research and development pipelines with increasing complexity to complete studies in a timely manner, as evidenced by the almost 31,000 studies started in the last 10 years across the 675 indications. In addition, there is a growth of indications for which research has never been completed. Success in this space requires collaboration across many stakeholders including families and patients, sites, investigators, labs, advocacy groups, regulators and the various service providers that support clinical trial delivery. Rapid engagement and work with these stakeholders is important in the search for answers.