Challenges of Clinical Trial Design, Conduct & Endpoints Workstream
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The Challenges of Clinical Trial Design, Conduct & Endpoints Workstream aims to provide solutions to complex questions and hot topics in the field:
- What’s the future of clinical trial design?
- How do we transform clinical endpoints into validated regulatory endpoints?
- How can we maximise outcomes for patients participating in these trials?
The Workstream is currently working on synthesising the group’s experience and knowledge of clinical trial designs into a guidance document. We hope this will inspire the next generation of scientists to facilitate the development of novel drugs and treatments for PH patients. If you’re interested in joining this Workstream please get in touch.
Publications
- Clinical Trial Design in phase 2 and 3 trials for pulmonary hypertension, Sylvia Nikkho, Peter Fernandes, R. James White, Chunqin (CQ) Deng, Harrison W. Farber, Paul A Corris, July 20, 2020.
- Role of biomarkers in evaluation, treatment and clinical studies of PAH , Anna Hemnes, Alexander M.K. Rothman, Andrew J. Swift, Lawrence S. Zisman, November 18, 2020.
- Novel composite clinical endpoints and risk scores used in clinical trials in PAH, Olivier Sitbon, Sylvia Nikkho, Raymond Benza, Chunqin (CQ) Deng, Harrison W. Farber, Mardi Gomberg-Maitland, Paul Hassoun, Christian Meier, Joanna Pepke-Zaba, Krishna Prasad, Werner Seeger, Paul A Corris, November 18, 2020.
- Repurposing of medications for pulmonary arterial hypertension, Mark Toshner, Edda Spiekerkoetter, Harm Bogaard, Georg Hansmann, Sylvia Nikkho, Kurt W. Prins, November 18, 2020. Presentation at PVRI Annual Congress, IDDI Pre-meeting.
- The evolving landscape of pulmonary arterial hypertension clinical trials, Jason Weatherald, Athénaïs Boucly, Anthony Peters, David Montani, Krishna Prasad, Mitchell A Psotka et al. November 26, 2022.
- Evolution and optimization of clinical trial endpoints and design in pulmonary arterial hypertension, Marco Caccamo, Frank E. Harrell, Anna R. Hemnes, 7 August 2023.
- Win Statistics in Pulmonary Arterial Hypertension Clinical Trials, Jason Weatherald, Jude Moutchia, Nadine Al-Naamani, Robyn L. McClelland, Corey E. Ventetuolo, Harold I. Palevsky, Michael O. Harhay, Steven M. Kawut, 1 December 2023.
- Clinical trial design, end-points, and emerging therapies in pulmonary arterial hypertension, Jason Weatherald, Thomas R. Fleming, Martin R. Wilkins, Thomas M. Cascino, Mitchell A. Psotka, Roham Zamanian, Werner Seeger, Nazzareno Galiè, Mardi Gomberg-Maitland, 29 August 2024.
Leaders
CQ Deng, United Therapeutics Corporation
Harm Jan Bogaard, UMC Amsterdam
Supported by
Ben Lavon, Fluidda
Sylvia Nikkho, Bayer AG
Jason Weatherald, Alberta
Want to be part of something special?
All PVRI members are welcome to join an IDDI Workstream or a Task Force.
Our Regional Task Forces don't yet have global coverage. If you're interested in starting one, please contact us.
Interested in accessing global data on PH patient experience?
Almost 4,000 patients and carers across 85 countries completed Phase 1 of our PH Global Patient Survey (PHGPS). Questions across all PH groups included diagnostic tests & timelines, genetic testing, treatments, financial burdens, hospital visits, research participation, quality of life, telemedicine, patient-reported outcome measures, and self-monitoring with digital technology. The findings have the potential to improve patient care, guide future research and help us address unmet needs.