Implementing alternative methodology like RWD or AI to obtain and model data when clearly defined clinical trial roadmaps and regulatory pathways seldom exist
Discussing the necessity and ethics of placebos for orphan drug trials: Can we do something different?
How can you run studies with limited populations, does the clinical trial model work in this area of research?
Turning to patients to help reduce waste, shorten development times and ensure the right outcomes are selected and measured effectively
Shedding light on the ACT-EU Multistakeholder partnership advisory board: an overview from a patient and policy perspective
A focus on the prospective approach to improve trial readiness: long-term preparation for a successful trial
Growing the 4 components of trial readiness: Community, Site, Science, Regulator
Engaging with the rare disease community and advocacy groups to grow involvement of well-informed patients
Leveraging investigator expertise for pragmatic trial designs and patient identification
Building the evidence, from natural history to endpoint development, to support informed decisions by health authorities
Key patient recruitment considerations: Diversity Action Plans, Paediatrics et al: enrolling specific populations in rare disease studies
Trends, opportunities, and challenges from AI
Finding the right balance between protecting data and enabling innovation
Preparing medical device applications amidst geopolitical changes and managing evolving cyber and privacy laws
Establishing long term foundations under cost control
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