Early and Often: Reimagining patient community engagement to improve clinical trials feasibility

The Global Genes Corporate Alliance has developed a new white paper that shows the transformative role of patient advocacy organizations in rare disease drug development. This recording is a panel discussion on that white paper.

The white paper can be downloaded here. 

Speakers:

• Danny Levine, Host, Global Genes Rarecast
• Wendy Erler, Vice President and Global Head of Patient Experience, Patient Advocacy and Patient and Caregiver Insights for Alexion Rare Disease and Co-chair of the Global Genes Corporate Alliance
• Molly White, Vice President of Strategic Initiatives for Dime Therapeutics
• Parisa Sanandaji, Executive Director of Patient Advocacy, Policy, and Engagement for Stoke Therapeutics and incoming Co-chair of the Global Genes Corporate Alliance

Cover page of Early and Often 2025 report

Discussion:

• The Corporate Alliance identified a need to address the lack of meaningful patient engagement in early clinical trial feasibility processes.
• The hypothesis of the white paper was that drug development companies often start early clinical trials without sufficient patient advocacy group engagement.
• Drug developers typically determine feasibility through external counsel and site selection or identifying key principal investigators.
• Patient involvement early in the feasibility process can provide valuable insights and information, leading to time and cost savings, and improved clinical trial success rates.
• The white paper recommends several strategies for improving patient engagement in clinical trial feasibility, including:
  • Establishing a patient advisory board
  • Conducting patient interviews and focus groups
  • Utilizing patient registries and databases
  • Collaborating with patient advocacy organizations
• The panelists discussed their experiences working to drive deeper engagement between their drug development teams and patient communities and the critical role this plays in improving the cost, time, and success rates of rare disease clinical trials.