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
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
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
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
Considering advances in trial designs and master protocols
Innovating through technology, digital health tools, AI and machine learning
Expanding collaborations and partnerships
Addressing ethical and diversity challenges
Discussing long-term vision in rare disease therapy development
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