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 …
The Evolution of Open-Label Extensions and Rare Disease Studies
When running early development studies, sponsors must consider whether or not to provide open-label, long-term treatment at the end of their randomized, placebo-controlled trial. In the past, sponsors have been hesitant to offer open-label extensions. But with our industry’s increasing focus on patient-centric care, sponsors are now weighing their options to incorporate more …