Surrogate end points in the design of immunotherapy trials: emerging lessons from type 1 diabetes.
Approximately 5% of people in developed countries suffer from 1 of approximately 80 classified autoimmune diseases. The sheer scale of the clinical problem captures the interests of health policy makers, academics, funding bodies and pharmaceutical companies in equal measure. In recent decades, immunologists have gained a good understanding of disease pathogenesis, which has led to the development of various potential therapies. The next challenge is to establish which therapies have superior efficacy, sustainability and safety. Therapeutic trials that depend on clinical end points are long lasting and expensive. In this Opinion article we offer a perspective on the future of clinical trial design in which the process is significantly shortened by making greater use of laboratory measures to determine therapeutic outcome.
Roep
Bart O
BO
Leiden University Medical Center, National Diabetes Expert Center, Leiden, The Netherlands. boroep@lumc.nl
Peakman
Mark
M
D000818:Animals; D015415:Biomarkers / Q000097:blood*; D002986:Clinical Trials as Topic; D003922:Diabetes Mellitus, Type 1 / Q000097:blood / Q000628:therapy*; D006801:Humans; D007167:Immunotherapy; D012107:Research Design
D015415:Biomarkers