There have been several studies that show that innovative trial design is required and in August 2018, FDA launched a program that would encourage novel clinical trial designs. They have issued guidance on novel trial design such as an expansion cohort which seeks to expand the clinical trial by allowing various cohorts into early phases that expose the patients to different conditions/environment for the drug. Expansion cohort guidance is here.
Interestingly, a randomized placebo controlled clinical trial has always been thought of as the golden test of a interventional drug or device. The benefits are many: decrease of bias, decrease in patient drop out in control groups and for subjective quality of life measures – it allows for unbiased assessment. However, in some cases, such as hematologic malignancy, FDA even supports unblinding of the data and alternates to a placebo-controlled trial as listed in this document.
This general guidance is coming under an umbrella of adaptive clinical trial design which is defined in the FDA document as “a clinical trial design that allows for prospectively planned modifications to one or more aspects of the design based on accumulating data from subjects in the trial”
Two important factors in many of the adaptive clinical trial designs is an interim analysis wherein the data accumulated for a period is analyzed and a decision is made on the future of the trial. Second, multiple arms of the trial can facilitate continuation or stopping of the arms to manage the complexity of the trial and its analysis.
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In many wearable device trials the sponsors have struggled with placebo controls since, after all, what is your placebo for a wearable device worn on the wrist? A watch that does not collect data? Or no watch? And what do you compare it to?
These new clinical trial designs may not only be very useful in complex oncology trials but will also be useful for these wearable device makers.