Archives: Agenda

Fireside Chat: Navigating FDA policy & politics in rare disease drug development under the Trump Administration

Rare disease innovation has always required both scientific courage and regulatory flexibility. Today, under the Trump Administration’s FDA, sponsors are seeing new opportunities for accelerated approvals, digital biomarkers, and innovative endpoints—but also greater scrutiny around confirmatory trials and uncertainty over key incentives like the pediatric voucher program. Meanwhile, political turbulence at FDA and CDC leadership levels underscores just how intertwined policy and science have become.

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