The COVID-19 pandemic has impacted drug development, and rare disease drug development in particular. A number of sponsors have put some of their rare disease drug development programs on hold as the industry in general grapples with challenges in this new clinical trial environment. Some of the primary challenges include patients' difficulty in getting to sites due to movement restrictions placed by various government bodies, the reluctance of patients to be involved in trials at this time, hospital sites especially diverting resources to care for COVID-19 patients, and interruption of gene and cell therapies, which require extended hospital visits to prepare or receive treatments.
The pandemic and the numerous challenges involved in rare disease drug development have heightened the need to adapt innovations and strategic considerations to accelerate development. Careful consideration and planning in the key areas mentioned in this paper will help to overcome many of the challenges.
