Remote endpoints for clinical trials in cystic fibrosis: Report from the U.S. CF foundation remote endpoints task force.

The COVID-19 pandemic necessitated a rapid shift in clinical research to perform virtual visits and remote endpoint assessments, providing a key opportunity to optimize the use of remote endpoints for clinical trials in cystic fibrosis. The use of remote endpoints could allow more diverse participation in clinical trials while minimizing participant burden but must be robustly evaluated to ensure adequate performance and feasibility. In response, the Cystic Fibrosis Foundation convened the Remote Endpoint Task Force (Supplemental Table 1), a multidisciplinary group of CF researchers with remote endpoint expertise and community members tasked to better understand the current and future use of remote endpoints for clinical research. Here, we describe the current use of remote endpoints in CF clinical research, address key unanswered questions regarding their use and feasibility, and discuss the next steps to determine clinical trial readiness.
Competing Interests: Declaration of competing interest Related to this work, MEJ reports employment by the Cystic Fibrosis Foundation (CFF). Unrelated to this work the authors report the following: DB reports consulting fees and stocks from Anagram Therapeutics Scientific Advisory Group and is a member of the Board of Directors for Anagram Therapeutics Scientific Advisory Group. CE reports honoraria for teaching presentations from Vertex and Chiesi. CHG reports grant funding from NIH NIDDK and NCRR, CFF, FDA Orphan Prod. Div. He received consulting fees from Enterprise Therapeutics and honoraria from Gilead and Vertex. He has received travel support from Vertex Pharmaceuticals and Enterprise Therapeutics. He participates on the DSMB for Novartis. He is a deputy editor for Annals of the ATS. He has stock in Air Therapeutics. GH reports grant funding from NIH NHLBI and CFF and support from CFCanada for travel. JEH reports grant funding from CFF and NIH, travel support from CF Canada, consulting fees from Vertex Pharmaceuticals and participation on an advisory board for Vertex Pharmaceuticals. DP reports grant funding from the CFF and consulting fees from Renexion Pharma. MSP reports grant funding from Dexcom Inc, CFF and Vertex Pharmaceuticals. She also reports consulting fees from Anagram Therapeutics and honoraria from Vertex Pharmaceuticals and TD Cowan. She is a member of the CFF DSMB and an associate editor for Endocrine Practice: Journal of the American Association of Clinical Endocrinologists. KAR reports grant funding from the CFF. MS reports grant funding from the CFF and Anagram Therapeutics. She has received consulting fees from Nestle International and Alcresta Therapeutics. She has received travel support from the CFF and serves on the CFF DSMB and reports a leadership role in the CFF. JS reports being a member of the CFF DSMB and advisory boards. ST reports honoraria from Vertex Pharmaceuticals. ETZ reports grant funding from CFF, NIH NHLBI, Vertex Pharmaceuticals. She has received consulting fees from CFF and Vertex Phamaceuticals and honoraria from the CFF and Asosciation for Diagnostic and Laboratory Medicine. She has received travel support from Vertex Pharmaceuticals, CFF and European Cystic Fibrosis Society. She has participated on advisory boards for CFF, CFF Therapeutics Development Network and Vertex Pharmaceuticals. MB, NL, KP and MR have no conflicts to report.
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