While we have seen exponential growth in the effort to develop drugs to treat rare diseases in the 30 years since the Orphan Drug Act was introduced, unique challenges remain. Awareness of rare diseases is often low among stakeholders; trial protocols and regulatory pathways may be unclear, especially for investigational rare disease drugs that are first-in-class; and data on disease incidence, onset, progression, and burden are often limited.
Given the small patient populations, strategies to identify and track patients can play a central role in the success of the development and commercialization of rare disease therapeutics. In this article for Clinical Leader, Bhavesh Patel and Connor Bitter review strategies currently used by life sciences companies to support patient identification, recruitment, and tracking and assess how new technologies such as cloud-based platforms could be leveraged to improve tracking and to better support clinical development and market access efforts.
To read the article, click here.