Developing and marketing products for rare diseases brings unique challenges to biopharmaceutical companies. Disease awareness is low, research costs are high, and there is often no clear regulatory pathway given that products are often first-to-market. Moreover, the relatively small patient populations heighten the importance of patient-finding efforts to ensure that rare disease products reach the patients they are intended to treat. These patient-finding initiatives have three main objectives:
- Fill existing knowledge gaps in the patient journey – an understanding of a patient’s story from symptoms to diagnosis to treatment;
- Understand the drivers and barriers to diagnosis and/or treatment; and
- Help rare disease patients receive the correct diagnosis and subsequent treatment.
Successful patient identification initiatives can lead to more patients receiving potentially lifesaving or life-changing treatment, thereby achieving a product’s full commercial potential. In this Insights, Inderpreet Kambo, Bhavesh Patel, and Connor Bitter discuss these challenges and a new tool developed by CRA, RDNavigator, that provides a tailored approach for rare disease companies to accomplish their patient tracking goals.