Archives: Agenda

Rare disease clinical trials face unique and often underappreciated risks that can disrupt timelines, data integrity, and patient engagement. Jim will discuss both overt and nuanced risk factors, offering proactive strategies to identify, mitigate, and manage challenges across the trial lifecycle for smoother, more efficient execution

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

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

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.

• Finding innovative solutions for patient engagement
• Collaborating with advocacy groups, patient organizations and care givers to extend patient reach
• Utilizing decentralized trials with remote visits and monitoring to reduce patient burden where possible

• Considering ways to boost recruitment through investigator meetings and recruitment workshops
• Communicating effectively and working together with sites to assist in recruitment
• Sharing recruitment challenges and successes from a rare disease trial that started during Covid-19 pandemic

• 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

• Creating vendor selection criteria and processes
• Establishing shared goals and KPIs to foster trust and alignment
• Managing expectations on timelines, budgets and deliverables
• Evaluating vendor capability in finding and supporting rare disease patients
• Monitoring vendor performance, governance and communication plans