2023 - Breaking Down Barriers in Support of Patients with Rare Diseases

There are over 7,000 rare diseases and one in 10 worldwide have a rare disease. Over 675 diseases have some research and development targeting supportive care, treatment or a cure for these patients and with approximately only five percent of indications having a treatment available. There is tremendous opportunity and an even larger patient need.
A limiting challenge remains the complexity of rare disease development and a rapidly evolving landscape. There are an increasing number of diseases targeted by large R&D 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.
This webinar will provide important perspectives from these stakeholders on:
Register to learn about breaking down barriers in support of patients with rare diseases.