Rare diseases affect more than 350 million people worldwide but patients often face limited options for approved therapies. As a result, many patients have joined advocacy groups first and foremost to connect with others struggling with their rare disease, but also to promote research, unite multiple stakeholders and stimulate new possibilities in the therapeutic pipeline. Research and orphan drug development efforts are starting to follow suit by increasingly incorporating patients’ needs and examining potential outcomes.
Addressing clinical challenges in rare disease and orphan drug development
With government-driven financial incentives, advances in genomic technology to identify promising targets for drug development, increasingly organized patient communities, and above average regulatory approval rates, drug developers are motivated to address rare diseases. While these trends are promising for patients with urgent unmet medical needs, orphan drug development still faces many challenges. The very nature of rare disease places pressure on identifying and accessing a sufficient number of patients for clinical trials.