Confronting the Challenges of Rare Disease: Finding Solutions Across the Entire Life Cycle

Confronting the Challenges of Rare Disease:

Finding Solutions Across the Entire Product Life Cycle

Six experts, working in patient recruitment, clinical development, drug safety,
product launch, and commercialization review unique hurdles faced in the rare disease market in this article.

In small patient populations where medical experience is limited and payer policy is vague, big solutions are required to overcome challenges.  In this article, our clinical trial experts, epidemiologists, and patient access professionals cover the topics important to working with small patient populations, including:

  • Reaching rare disease patients through advocacy groups and social media
  • Meeting real-world evidence requirements
  • Incorporating natural history studies
  • Managing expanded access programs
  • Designing and running high-touch patient support services