When selecting clinical trial designs, how many design options should a sponsor explore? Would a sponsor feel more confident having an optimized design out of ten potential ones, or simulating several million designs to discover that there are three or four better options? Cytel’s experience suggests that this depends very much on the specific sponsor and the specific conditions of the clinical trial.
This only begs the question, what is a reasonable number of clinical trial designs to explore before feeling confident in a decision? Here are five considerations for sponsors when making this complex decision.
How Quickly Can You Build an Expansive Design Space?
A design space is one that encompasses all of the clinical trial designs available to a sponsor. Depending on a sponsor’s access to statisticians and the tools with which they are equipped, a statistician might be able to build a dozen designs in a few weeks or a few hundred thousand in a matter of minutes. Sponsors should determine as early as possible, how many designs are available to them.
How Many Stakeholders Should Be A Part of the Conversation?
The selection of a clinical trial design can form the backbone of the rest of the clinical trial journey. For some sponsors this means that in addition to CMOs and members of the R&D team, the input of downstream actors like commercial teams might be useful for clinical trial strategy. The challenge here is to ensure that the statistical benefits of each clinical trial design under consideration can be communicated to all. Some software like Cytel’s Solara TM provide visualization tools that enables statisticians to answer questions about design options, in real time.
Do You Have An Objective Way to Rank Designs?
Suppose a Sponsor was given a hundred designs, but was not clear on how to prioritize them. One design might offer slightly higher power than another, but require a timeline extension of a couple of months. Some sponsors may have no trouble with this, while others might see competitors looming on the horizon. Notably, an experienced statistician can not only create designs, but can help a leadership team come up with a strategy based on quantitative decision frameworks . This will ensure that even if a sponsor has hundreds of thousands of designs from which to choose, the selection process will not be ad hoc or overwhelming.
Are You Leaving Anything on the Table?
Oftentimes a sponsor will know that a particular design is a strong candidate given sponsor goals. What we often find at Cytel though, is that sponsors have no way of telling if there are any designs that might be even better. Sometimes there are designs that increase study power without actually requiring much more by way of upfront cost, study timelines or patient samples. The only reason these clinical trial designs aren’t chosen is simply because no one found them before the rush to begin a clinical trial. Realistically, though, even sponsors with firm resource limitations will benefit from knowing these superior designs exist, as it positions them to consider new strategies or even attract more funding. A sponsor should feel confident that none of these designs have been overlooked in the urgency to begin a clinical trial.
What Are the Stakes of Not Exploring Further?
All sponsors want a clinical trial design that will reveal the true value of their product. Some sponsors though, have even more at stake. There might be a competitor on the horizon who will be in a position to disrupt the market if a new medicine is not made available swiftly. More generally, a sponsor might worry that a clinical trial design that fails to reveal the value of a product will also fail to get new medicines to patients who need them most. A number of solid scientific designs will not capture these other stakes, particularly when statisticians are not a part of the strategic discussions.
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About the Author of Blog:
Dr. Esha Senchaudhuri is a research and communications specialist, committed to helping scholars and scientists translate their research findings to public and private sector executives. At Cytel Esha leads content strategy and content production across the company's five business units. She received a doctorate from the London School of Economics in philosophy, and is a former early-career policy fellow of the American Academy of Arts and Sciences. She has taught medical ethics at the Harvard School of Public Health (TH Chan School), and sits on the Steering Committee of the Eastern Division of the Society for Women in Philosophy which is responsible for awarding the Distinguished Woman Philosopher Award.