Vaccine: X (Aug 2024)
Workshop on the design and use of clinical trials with multiple endpoints, with a focus on prevention of RSV
- O. Prunas,
- J. Willemsen,
- J.L. Warren,
- L. Bont,
- J.L. Schwartz,
- J. Atwell,
- E. Begier,
- N. Dean,
- I. Hirsch,
- R. Karron,
- K. Klugman,
- R. Kramer,
- E. Leidman,
- R. Link-Gelles,
- H. Nair,
- CA. Panozzo,
- E. Pelfrene,
- E.A.F. Simões,
- P.G. Smith,
- P. Srikantiah,
- M.E. Sundaram,
- D. Thindwa,
- D.W. Vaughn,
- E. Wilson,
- H.J. Zar,
- V.E. Pitzer,
- D.M. Weinberger
Affiliations
- O. Prunas
- Swiss Tropical and Public Health Institute, Basel, Switzerland; University of Basel, Basel, Switzerland; Corresponding author at: Swiss Tropical and Public Health Institute, Basel, Switzerland.
- J. Willemsen
- Centre for Translational Immunology, University Medical Center Utrecht, Utrecht, the Netherlands; Division of Infectious Diseases, Department of Pediatrics, University Medical Center Utrecht, Utrecht, the Netherlands
- J.L. Warren
- Department of Biostatistics, Yale School of Public Health, Yale University, New Haven, CT USA
- L. Bont
- Division of Infectious Diseases, Department of Pediatrics, University Medical Center Utrecht, Utrecht, the Netherlands
- J.L. Schwartz
- Department of Health Policy and Management, Yale School of Public Health, New Haven, CT, USA
- J. Atwell
- Medical Development, Scientific, and Clinical Affairs, Pfizer Vaccines, Pfizer Inc., Collegeville, PA, USA
- E. Begier
- Medical Development, Scientific, and Clinical Affairs, Pfizer Vaccines, Pfizer Inc., Collegeville, PA, USA
- N. Dean
- Department of Biostatistics and Bioinformatics, Emory University, Atlanta, GA, USA
- I. Hirsch
- Vaccines & Immune Therapies, BioPharmaceuticals R&D, AstraZeneca, Cambridge, UK
- R. Karron
- Department of International Health, International Vaccine Access Center, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
- K. Klugman
- Director of Pneumonia and Pandemic Preparedness Programs, Bill & Melinda Gates Foundation, Seattle, WA, USA
- R. Kramer
- Sanofi Vaccines, Lyon, France
- E. Leidman
- US Centers for Disease Control and Prevention, Atlanta, GA, USA
- R. Link-Gelles
- US Centers for Disease Control and Prevention, Atlanta, GA, USA
- H. Nair
- Centre for Global Health, Usher Institute, University of Edinburgh, Edinburgh, UK
- CA. Panozzo
- Clinical Development, Infectious Diseases, Moderna Therapeutics, Cambridge, MA, USA
- E. Pelfrene
- Office of Vaccines and Therapies for Infectious Diseases, European Medicines Agency, Amsterdam, the Netherlands
- E.A.F. Simões
- Department of Pediatrics, University of Colorado School of Medicine, Aurora, Department of Epidemiology, Colorado School of Public Health and Samshoma Medical Research Inc, Denver, CO, USA
- P.G. Smith
- MRC International Statistics and Epidemiology Group, London School of Hygiene & Tropical Medicine, London WC1E 7HT, UK
- P. Srikantiah
- Bill & Melinda Gates Foundation, Seattle, WA, USA
- M.E. Sundaram
- Marshfield Clinic Research Institute, Center for Clinical Epidemiology & Population Health, Marshfield, WI, USA
- D. Thindwa
- Department of Epidemiology of Microbial Diseases and Public Health Modeling Unit, Yale School of Public Health, Yale University, New Haven, CT USA
- D.W. Vaughn
- Bill & Melinda Gates Foundation, Seattle, WA, USA
- E. Wilson
- Clinical Development, Infectious Diseases, Moderna Therapeutics, Cambridge, MA, USA
- H.J. Zar
- Department of Paediatrics and Child Health, Red Cross War Memorial Children’s Hospital, and SA-Medical Research Council on Child & Adolescent Health University of Cape Town, South Africa
- V.E. Pitzer
- Department of Epidemiology of Microbial Diseases and Public Health Modeling Unit, Yale School of Public Health, Yale University, New Haven, CT USA
- D.M. Weinberger
- Department of Epidemiology of Microbial Diseases and Public Health Modeling Unit, Yale School of Public Health, Yale University, New Haven, CT USA
- Journal volume & issue
-
Vol. 19
p. 100509
Abstract
A meeting held in Lisbon, Portugal, in February 2023 focused on critical aspects of clinical trial design for respiratory syncytial virus (RSV) preventative therapies. The meeting addressed two primary areas: enhancing the efficiency and success of randomized controlled trials (RCTs) for RSV preventative therapies and designing RCTs to better inform post-licensure decision-making. Topics included the selection of primary endpoints, innovative approaches to incorporating multiple endpoints and historical data, and the challenges and benefits of sequential trial designs. The discussion also touched on meta-regression models for obtaining more robust, context-specific estimates of vaccine efficacy. Overall, the meeting underscored the importance of balancing efficiency and robustness in RSV vaccine trial design, while recognizing the need for further discussions involving regulatory and advisory bodies.
