A US-based oncology biotech sponsor, with multiple subsidiaries, sought ICON’s expertise to create a more cost-trial design for their phase 2 and 3 single oncology molecule trial. The molecule was under investigation for multiple tumour types, backed by scientific support and limited funding from a major pharmaceutical sponsor. Compounded by multiple clinical programs without prior approvals and fast-track designation in one indication, the biotech sponsor faced significant challenges. ICON was engaged to provide statistical design options which would help achieve sponsor objectives while also being accepted by health authority reviewers.

The sponsor faced numerous challenges in this project, including:

Extended development times: Historically, the sponsor’s trials were conducted using limited utility dose formulations that were not suitable for phase 3 development. This led to prolonged timelines, delaying the potential benefits of their treatment reaching patients in need.

Uncertainty from prior data: The data from previous trials introduced a high level of uncertainty, making it challenging to confidently plan the next stages of clinical development.

Budget constraints: the sponsor’s clinical development budget was significantly limited, imposing constraints on their ability to explore various options and approaches for optimising the trial design.

Pressure for publishable trial results: The sponsor’s shareholders and executive board were eager to see publishable trial data results. The demand for positive outcomes added further pressure to trial planning and execution.

By acknowledging and addressing these challenges, ICON aimed to provide alternatives to the existing study design that would align with regulatory requirements, expedite the study timeline, and optimise the use of available resources, whilst considering the variable data and change in dose formulation.

With a focus on the sponsor’s development budget and timeline concerns, ICON presented two adaptive design options.

Phase 2/3 design: This option involved exploring multiple new dose formulation options, with selection for phase 3 dosing based on the findings from phase 2.

Phase 3 design with interim analysis: A smaller initial investment was made possible through sample size re-estimation at the interim stage, based on conditional power. ICON’s consulting team guided the implementation of the interim analysis without publication, with the expectation that passing the interim threshold would sufficiently send a positive signal to the market.

After evaluating both study design alternatives, the sponsor chose the second option, to mimimise their initial investment and secure executive board approval. Their highest priority was to achieve early efficacy success and file for regulatory approval with a smaller up-front cost.