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…
PRA would like to express deepest gratitude and recognition to members of the 2020-2021 Rare Disease Advisory Council for their immense contributions to the development of this Toolkit. PRA is especially grateful for the deep understanding of lived reality of rare disease patients and caregivers, the deep understanding of clinical development, and the willingness to join us on a journey of collaborative development that RDAC members have contributed.
Shortening the Path to Rare Disease Diagnosis with a Patient-Centric Approach
Many rare disease patients struggle for years before they know what their symptoms indicate, and even longer to find a meaningful treatment. Learn…
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…
Download the Patient-Centric Trial Development Toolkit
Download 4 practical tools for patient-centric trial development including: Patient Involvement Value Dossier, Patient-Centric Protocol Risk…