“For years, I’ve been devising optimal strategies for increasing the probability of success in clinical trials by using carefully chosen clinical endpoints, often matched with appropriate biomarkers and diagnostics,” says Dr. Khatry. “And I can report that an expedited, optimized study with these factors is essential to ensuring clinical trial efficiency.”

Dr. Khatry says the first step in the design process is clearly understanding how the drug works and what the appropriate clinical endpoint should be. Next the margin of clinically meaningful difference must be determined to demonstrate the superiority, equivalence, or non-inferiority of the new drug versus the standard of care, depending on the type of study design to be used. That margin of difference must be acceptable to clinicians, patients, and regulators such as the U.S. Food and Drug Administration (FDA), he adds.

“Appropriate biomarkers and diagnostics can assist in stratifying patient subsets and in choosing optimal statistical methodology and tools,” says Dr. Khatry. “Identifying the right statistical methodology and tools will ensure robust data analysis for evidence generation, interpretation of study results, and communication of study findings. Other factors we consider are the required sample size, which will affect the trial’s length and cost, and how best to recruit participants.

“Also, to increase success probability, part of the design is to run computer simulations of the studies to ensure that they are adequately powered to succeed with the sample sizes proposed and they are using realistic assumptions related to the chosen clinical endpoints,” explains Dr. Khatry. “The computer simulations provide assurance for trial success. Where relevant, Westat builds in provisions for interim analysis of study data to allow us to increase study sample size if necessary to maintain study power for success.

“When clinical trials are efficiently designed and conducted, they are less costly and promise a higher success rate,” continues Dr. Khatry. “This means the drug can come to market sooner, and insurance companies are more likely to pay for it. It’s a win-win for the patient and all stakeholders.”