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.
Creating Patient-Centric Trials and Technology for a Better Patient Experience
To commemorate this year’s Patient Experience Week—which celebrates healthcare staff impacting patient experience and provides a focused time for…
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…
Rapid Participation Burden Survey
Quick, flexible, validated patient survey tool for rapid feedback on trial-specific participation burden, helps sponsors refine risk analyses and…