Drug Trial Design Depends on Knowledge: Woodcock
CDER director Janet Woodcock says that clinical trial designs, no matter how novel, “will only be as good as the knowledge that underlies them.” In presentation slides prepared for the 12th annual personalized medicine conference, she says that the full clinical development program must be considered, and not just the trial itself.
“No matter how advanced the design,” she says, “you may be in trouble if you have too many variables in play in your very expensive clinical development program.” Variables she cites include disease expression or progression; lack of specificity of diagnosis, prognosis, or predictive biomarkers; unknown or poor performance of clinical outcome assessments; and patient-related outcomes that don’t reflect the patient’s view of disease burden. Advanced design won’t cure these issues, she says, unless exploration of them is built into the trial.
According to Woodcock, targeted, personalized or precision medicine approaches can deliver large treatment effects, making development easier. She reiterates that adequately-performing diagnostic, prognostic, and predictive biomarkers are a key to enrolling the right patient population, and this is not as straightforward as was initially thought. Understanding the performance of clinical outcome assessments is critical to picking the right endpoints, she says, and these items are much more crucial with a smaller treatment effect.