Though there are thousands of rare diseases affecting more than 300 million people worldwide, only a small percentage of them have treatments. Considering this and the fact that over 50% of rare disease patients are children, the high unmet need for rare disease therapies has exponentially increased the number of drugs in development. Currently, according to Pharma projects, around 30% of all investigational products globally are in rare diseases, proving that rare disease drug development is moving higher up the pharma industry’s agenda.
Nevertheless, due to the myriad of challenges involved in rare disease drug development, it typically takes on average four years longer for rare disease medicines to successfully advance through clinical trials and on to FDA approval relative to non-rare disease medicines.
As a result, sponsors must be more strategic in their approach to rare disease drug development and leverage innovations and strategies to help them overcome these challenges to speed up development to bring much-needed treatments to patients. This webinar will explore these strategic considerations and innovations for accelerating the drug development of rare diseases. Our listeners will also understand the importance of patient- and site-centric solutions in aiding this process as well as gain insight into the Ergomed Rare Disease Innovation Center.
- Rare Disease Overview and Market
- Key Strategic Considerations for Rare & Orphan Drug Development
- Innovations that Accelerate Rare Disease Drug Development
- Patient and Site Centric Solutions
- Ergomed Rare Disease Innovation Center
- Q&A with Zizi Imatorbhebhe, MBA, MS, PMP®, Senior Vice President, Global Strategy & Development, Rare Disease, Ergomed
