Practical tools for sponsors, participants, and champions of patient-centric trial development.
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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.
Explore the toolkit
Patient Involvement Value Dossier
Increasing evidence supports that patient involvement in development of clinical trials provides value to sponsors by de-risking trial efficiency
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
Rapid Participation Burden Survey
Quick, flexible, validated patient survey tool for rapid feedback on trial-specific participation burden, helps sponsors refine risk analyses and…