Ipad toolkit

Practical tools for sponsors, participants, and champions of patient-centric trial development.

Stay up to date on PRA’s Center for Rare Diseases, our achievements, and new initiatives

Rare Disease Newsletters

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.

Download the Complete Patient-Centric Trial Development Toolkit

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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.

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