We are pleased to invite you to the IDDI & MVA complimentary meeting to explore the challenges of the transition from early (phase 1, expansion cohorts and single-arm phase 2) trials to later phases of clinical development.
The transition from early (phase 1, expansion cohorts and single-arm phase 2) trials to later phases of clinical development poses several challenges. While many of these challenges are of strategic and financial natures, some usual methodological pitfalls can be addressed in a somewhat systematic fashion.
In this presentation,
- We will explore these pitfalls and attempt to outline the key aspects that need to be taken into account to mitigate risks and optimize the chances of successful drug development in oncology, even though the concepts to be discussed are applicable to any other indication where precision medicine is of interest.
- The most salient issues to be discussed include:
- “Regression to the mean” (a key reason why phase 3 results often do not reproduce those from phase 2),
- The overreliance on historical data, misconceptions about the impact on sample size from randomizing early on,
- The assessment of more promising subgroups for development of precision therapy in biomarker-defined subsets, and some key aspects related to accelerated approval by the US Food and Drug Administration and conditional marketing authorization by the European Medicines Agency.
