Archives: Agenda

Building transparency into rare disease gene therapy trials: what patients and caregivers need to know

  • Understanding why transparency is critical to patient trust and retention in gene therapy trials
  • Recognizing operational challenges unique to gene therapy that impact patients and caregivers
  • Identifying strategies to embed patient and caregiver voices into protocol and schedule design
  • Sharing practical take away communication practices that can strengthen partnerships between trial teams, advocates, and families

Medically underserved populations in rare disease research

  • Discussing population inclusion and access to care and clinical trials regardless of diversity and socioeconomics, with a focus on improving patient care
  • Mental health and well-being: the emotional toll rare diseases take on patients and caregivers
  • Insights on the differences between represented and underrepresented populations on their barriers to care
  • Identifying realistic ways to improve opportunities for all patients

Optimizing rare disease clinical trial designs

  • Reviewing challenges in designing a rare disease trial
  • Discussing approaches to optimize a rare disease clinical trial design
  • Considering incorporating a competitive mindset in designing a rare disease clinical trial