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
Country & site selection
Patient identification & recruitment
Timelines & startup complexity
Consultants & collaboration
Conferences & knowledge sharing
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
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