More than 90% of the 7,000 rare diseases known to science don’t have a single FDA approved treatment. We believe there’s no time to waste in bringing meaningful treatments to lives affected by rare disease.
We also believe in empowering every stakeholder to make rare disease clinical research as efficient, effective and successful as possible.
We know from experience, that patient-centric rare disease clinical trials that reduce barriers to participation create efficiencies.
This toolkit was developed to support sponsors, patient communities, and other stakeholders to operationalize true patient-centricity in clinical development.
What to ask for when you’re interested in a clinical trial: A guide for rare disease patients and caregivers
Considering a clinical trial? This handy guide will help you ask about the support you’ll receive to make your participation easier and make informed…
Patient-Centric Protocol Risk Assessment Tool
Expert-validated tool that enables sponsors to identify potential trial efficiency risks driven by participation burden in any draft protocol
Patient-Centric Trial Development Toolkit
Learn more about our practical tools for sponsors, participants and champions of patient-centric trial development and how you can start using them…