Strategic Clinical Trial Design Unlocks Innovative Funding Opportunity

A small biotech sponsor developing a new treatment for carcinoma approached Cytel to leverage Solara^® for assistance with their trial design. Working with a constrained R&D budget, the challenge was to gather the evidence in as cost effective a manner as possible and use a stage-gate approach for the funding.

The sponsor needed to balance innovative design with simplified operational approaches given their resource constraints. The design selection process needed to balance a wide choice of design options, given the complicated nature of the indication, and keep the operational characteristics of the clinical trial simple, so patients would enroll whilst maintaining sufficient stage gates to ensure limited budgets were not wasted if the treatment did not live up to expectations.

The sponsor’s conventional design of 412 patients enrolled across 2.5 years formulated a baseline design that was too expensive for the sponsor to implement and did not offer an opportunity to stage-gate the funding. A clinical trial with an interim look, that provided clearer insight into the likelihood of success of the trial, was required. This was a strategy which could not only lower the overall cost of the study but would likely increase their chances to secure funding from investors.

The goal then was to identify the design that had the greatest probability of success, and use an interim analysis to help the sponsor when communicating to prospective investors as well as the medical community. It was critical to demonstrate a clinically relevant difference between the new compound and the current drug, or standard of care.

To explore elements of the design challenge we will consider:

• Staged Investment
• Innovations
• Operations