Suicide risk and REDCap: facilitating clinical trial inclusion.

Progressive familial intrahepatic cholestasis (PFIC) is a group of rare genetic disorders related to defects in bile transport that can lead to liver failure during childhood. PFIC diseases exhibit considerable intra‐ and inter‐subtype variability in both severity and prognosis. Treatment is challenging, and with no definitive medical therapies available efforts often start with symptom management but progress to surgical interventions, including biliary diversion and liver transplant., , , As with many rare diseases, there are limited guidelines for the management of PFIC disorders. Existing documents are based mainly on expert opinion, underscoring the lack of robust evidence to navigate the clinical course. As a result, patients and families are often left fully dependent upon their individual providers to make complex care decisions regarding treatments and interventions. Critically, how patient values, priorities, and goals are incorporated into these decisions is entirely unknown, and efforts to identify patient‐driven objectives within current treatment and research programs have been lacking. The PFIC Network (PN) is a patient advocacy organization committed to improving the lives of PFIC patients and families while striving for a cure for PFIC and related diseases. Founded in 2018 by caregivers as a novel organization providing resources and support, PN has gained international experience in collaboratively engaging patients and stakeholders to produce disease education materials, create a community for support and advocacy, organize hybrid Family & Scientific Conferences, host the PFIC Network Patient Registry (PNPR, ), and drive research.