A public health emergency creates a challenging environment for the study of experimental vaccines, necessitating the need for novel, flexible and responsive trial designs. In a paper published in the journal Science Translational Medicine, a group of researchers led by University of Florida faculty describes key considerations for the design and analysis of such trials.
“The most significant challenge during a public health emergency is that it is hard to design a study that places the vaccine in populations at high risk for the disease,” said lead author Dr. Natalie Dean, an assistant professor in the department of biostatistics at the University of Florida College of Public Health and Health Professions and the UF College of Medicine. “In order to tell if the vaccine works, populations need to be naturally exposed, but for outbreaks, disease incidence is very unpredictable. Outbreaks may end quickly, before there is time to implement a clinical trial. We need new trial design strategies to address these challenges.”
The new recommendations come from a group of statisticians, clinical trialists, infectious disease modelers and researchers, which include the paper’s senior author, Dr. Ira Longini, a UF professor of biostatistics. They were convened by the World Health Organization’s Research and Development Blueprint and charged with establishing standard procedures to rapidly evaluate experimental vaccines during public health emergencies while maintaining the highest scientific and ethical standards.
In their paper, the team outlines major study design elements and challenges specific to the Blueprint priority diseases and to the context of public health emergencies. They also illustrate some of the trade-offs and methodological options.Friday Letter Submission, Publish on July 12