Happy Clinical Trials Day !!
Clinical Trials Day is celebrated today to recognize the day that James Lind of the British Royal Navy decided to study citrus as a therapy to cure scurvy in 1774. We have come a long way since.
Cell and gene therapies pose unique challenges in proper cell collection, processing, post-processing and distribution logistics making it crucial to understand the nuances and be innovative in designing the clinical trials with right patient selection (inclusion/exclusion criteria) to answer the question of safety (Phase1) and efficacy (Phase2) and learn from the pitfalls of past clinical trials in the field.
Especially while studying therapies for rare diseases it is imperative that we are innovative not only test the safety but also have efficacy measures embedded so as to improve much needed access for critical patients.
Ethically most important are long term prospective (Phase 3 and 4) trials as they will help understand the real statistical significance and implications by overcoming biases.