Small molecule orphan drugs: Status quo, challenges, and perspectives
The development of orphan drugs in the past year has continued to trend upwards in the US, the European Union, and Japan. This is due to the financial incentives offered through regulation, with orphan designation appearing to be successful at generating opportunities and positive value for drug sponsors and patients. Despite that, many rare diseases remain without effective treatments despite the rising number of approved orphan drug products. In addition, the decision to invest in the orphan drug space is more complex than simply responding to regulatory incentives.
In this webinar, experts will discuss the key drivers that motivate drug sponsors in developing orphan drugs. In addition, experts will explore the challenges of orphan drug development and manufacturing and will discuss how the industry is leveraging strategic partnerships to help mitigate these challenges to create a viable orphan drug business model.
Key learning objectives:
- Understand the drivers of growth in orphan drug investment and development
- Learn how to navigate the complex development and manufacturing space of orphan drugs
- Discuss practical considerations for supply chain when bringing an orphan drug to the clinic and market
