There are new exciting novel pro-hemostatic therapeutics in early phase clinical trials for hemophilia and hemophilia inhibitor patients. Yet, it is difficult to design randomized trials to compare these agents, or compare them with standard treatment, given the small sample size and competing studies for such patients. It is critical to develop novel approaches to compare new agents in rare populations. For example, ...more »
We need new ideas in how to make clinical trials more efficient and cost effective. (Randomized) comparison of different methods for accrual might be of interest; blinded versus unblinded trials; behavioral controls versus placebo controls or usual care controls might be explored.
There is a need to utilize insights gained from T4 translation research and implementation science to inform the design and execution of early-stage translational research and clinical trials.
Alignment of regulatory, healthcare, and research arms of the government is poor. There is a need to improve the design, quality and usefulness of data from regulatory studies to address major clinical questions and also to facilitate scientific inquiry. This is a barrier to evidence based medicine and improved treatments.
Will adaptive trial design improve clinical research for acute lung injury?
Research to "fix" spinal cord injury in humans, has been insanely hyped, rare in reality, and very disappointing in its clinical applicability to human patients. After a parade of rat models, mouse models, cat models, dog models, African green monkey models, pig models, guinea pig models, hamster models, rabbit models, gerbil models, etc. one wonders whether most researchers or funders will ever have any interest in ...more »