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2021. FINDINGS FROM THE PATIENT-FOCUSED DRUG DEVELOPMENT FORUM ON PRIMARY SCLEROSING CHOLANGITIS

Plain-Language Summary

The Our Voices survey was developed by the PSC Partners Registry team to identify gaps in understanding PSC symptoms, gaps in communication between patients and physicians, and areas of future research to support successful drug development for PSC. The survey captured patient experience related to impact of symptoms with over 50% of participants reporting they could not fulfill their work or school duties when PSC symptoms were at their worst. The survey showed a high willingness to participate in research, prioritizing research focused on improving liver function over symptom management.  Participation in this research led to the development of the Patient-Focused Drug Development Forum, supported by the U.S. Food and Drug Administration.

AASLD The Liver Meeting Poster #630



Conclusions:


Findings from the Our Voices survey informed the development of the externally-led PFDD Forum for PSC and highlight the need for a more robust, validated Patient Reported Outcome Measure (PROM) for PSC. Overall, our results demonstrate the willingness of the PSC patient community to share experiences and guide the development of PROMs, which requires further perspective and expertise from the PSC research community.




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