Archives: Agenda

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

• 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

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.

• 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

• Sharing insights on how to use AI to bring drugs to rare disease patients
• Running through step by step processes including pre IND, FDA work, organising and conducting trial
• Discussing challenges, successes and real-world experience

• 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

• 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

• 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