Archives: Agenda

• Running the trial: innovative approaches and overcoming challenges
• Treating with oncolytic virus intra-tumoral therapy
• Reaching clinical success: findings and future plans

• Comparing outcomes of a DCT model versus non DCT model
• Sharing benefits and lessons learned from experiences within rare disease DCTs

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.

• Understanding regulatory pathways and incentives
• Identifying clinical development challenges and solutions
• Considering pricing, reimbursement and patient advocacy engagement

• Assessing experience in trials with small patient populations an recruitment challenges
• Evaluating ability to adapt and innovate for unique needs of rare disease trials
• Considering regulatory knowledge and patient access

• 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

• 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