Cancer prevention trials can take years to generate meaningful clinical outcomes, delaying critical development decisions. In this case study, Marc Buyse, shows how IDDI helped enable earlier, data-driven decisions in a Lynch syndrome trial by linking early measurable endpoints to long-term outcomes and applying interim analyses throughout the study.
Looking to improve success rates in oncology trials? See how adaptive Bayesian design can increase phase 3 success.
Working in complex or rare diseases? Explore how endpoint strategy can support regulatory approval.
Key Challenges in Cancer Prevention Trials
- Long timelines to observe endpoints such as cancer incidence or survival
- Lack of widely accepted standards for early endpoints
- Difficulty balancing speed vs. statistical confidence
- Regulatory expectations for clinically meaningful outcomes
The IDDI Approach
Drawing on deep expertise and biostatistical excellence, IDDI:
- Identified early measurable endpoints linked to long-term outcomes
- Designed a group sequential trial with interim analyses
- Enabled data-driven decisions earlier in development
- Ensured alignment with regulatory expectations
Sponsor Benefit
- Reduce time to key decisions in oncology trials
- Improve probability of success without increasing sample size
- Avoid costly late-stage failures
- Maintain credibility with regulators (FDA, EMA)
Planning a cancer prevention or oncology trial?
Talk to our statistical experts about designing endpoints that accelerate decision-making while preserving rigor.
Read the full video transcript
A situation where IDDI did make a difference was our work with a company that was designing a preventive cancer trial for patients with Lynch syndrome. Lynch syndrome is a genetic condition that raises the risk of many types of cancer, in particular colon cancer. Doing trials in cancer prevention is challenging for many reasons, in particular because you need a lot of patients, and also there are less accepted standards than for treatment trials. The challenge in the case of Lynch syndrome was to identify endpoints that would be observed early enough to conclude to the efficacy (or lack of thereof) of the preventive treatment early enough, and yet with sufficient confidence that the treatment would have an effect on the late outcomes of interest, specifically clinical evidence of colon cancer or death from cancer, both of which occur much later than early endpoints such as immune response or the development of adenomas. The trial design that was ultimately adopted was a group sequential design monitored by an independent committee that would recommend a course of action to the company at every interim pre-specified analysis.
