Clinical Development Challenge - Use Of RWA To Understand Natural History

A biotechnology company, focused on precision medicine received an FDA request to provide a natural history of the rare, oncology indication for which they were developing a new therapy. With limited information available about the disease, and a small patient population, the client needed to leverage real-world data to generate evidence that could satisfy the regulatory request. However, a small organization without an in-house real-world evidence team, they did not have the adequate resources, connection, or the expertise available to conduct this natural history study. With extensive experience in this domain, Cytel proposed to function as the virtual real-world evidence (RWE) department for the client. A two-step process was established to deliver more information that would inform regulatory decision-making and help the client get its therapy approved more quickly and cost-effectively.