Rare diseases are an emerging global public health priority.
November 18, 2020
This webinar will discuss how the COVID-19 pandemic has impacted clinical studies for rare diseases and how patient-centric trial design with regulatory consideration can be utilized to mitigate and manage potential delays and support patient recruitment in rare and orphan clinical trials.
COVID-19 is a serious, life-threatening and fast-spreading viral infection that has had an unexpected global impact. As the world is learning to adapt to a “new normal,” so have clinical trials.
Rare diseases are an emerging global public health priority. There are more than 7,000 identified and classified rare diseases, 71.9% of which are genetic and 69.9% which are exclusively pediatric onset. Following the European definition of the incidence of a “rare disease” (no more than 5 in 10,000 individuals), it is estimated that there are over 440 million individuals globally who are affected by a rare disease at any point in time, excluding rare cancers, infectious diseases and poisonings. Rare diseases are numerous, heterogeneous in nature and geographically disparate. Few of them are preventable or curable, most are chronic, degenerative and many lead to early death. The inherent challenges are derived from their low prevalence, poor scientific knowledge and the scarcity of expertise, as well as their chronic, degenerative and life-threatening nature.
Patient groups, physicians and regulators are eager to support the delivery of rare and orphan clinical trials, allowing flexibility while maintaining the standards necessary to deliver high-quality data. Orphan drugs companies are looking for support and guidance on how to navigate this new challenging clinical landscape and CROs are working to deliver solutions that meet the needs of patients and drug developers.
Join experts from Simbec-Orion, Dr. Carlos Camozzi, Chair, Rare and Orphan Advisory Board; and Dr. Chirag Patel, Director of Regulatory Affairs, in a live webinar on Tuesday, December 8, 2020 at 9am EST (2pm GMT/UK).
For more information, or to register for this event, visit Meeting the Needs of Rare Disease Patients in Clinical Trials During the COVID-19 Pandemic.
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