The past decade has witnessed a remarkable surge in rare disease research leading to an impressive pipeline of promising investigational products. Novel approaches, including genomics, data mining and computational repurposing of drugs will continue to fuel discovery and consequent clinical development of medicines. In this webinar, a panel of three rare disease experts will discuss current trends in orphan drug research, the role of open-label extension studies in rare disease development and the unique recruitment challenges for rare disease trials. The convergence of next generation genomics, advances in clinical trials operations, and the strong commitment of all stakeholders active in the field will bring us closer to the ultimate goal of improved health for people living with rare diseases.

What will you learn from this webinar

  • What are the current trends in the orphan space
  • How to harvest the most information from small population trials
  • What motivates patients to participate in clinical trials
  • What are the imperatives and pitfalls of open-label extension trials

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