A wave of new personalized treatments for cancer have been introduced in recent years, mainly based on RCTs with novel biomarkers/mutations to guide specific targeted therapies. This development often represents a challenge for regulatory and HTA bodies, and prompts a demand for new methods of evidence generation.
The main reason for this is that the trials include fewer patients, have shorter follow-up, and use intermediary endpoints, rather than overall survival. To guide and support oncology drug development there is an increasing need for real world evidence in order to contextualize the results from oncology RCTs.
Furthermore, due to ethical and practical reasons, there is a growing tendency for trials to be run without a randomized control arm, so called single-arm trials, or SATs. Altogether, there is an increasing need for external control arms (ECA) that can support drug development and regulatory decision making.
In our proof of concept research project we will emulate the trial populations in ongoing and completed oncology RCTs, and run analyses in order to see if we can reproduce the outcomes in the standard-of-care (placebo) and experimental treatment arms. Furthermore, we will explore methodological aspects of external control arms, such as emulating intermediary outcomes. In addition, the project will construct synthetic datasets that can be used for feasibility assessments, and data exploration.
We are currently focusing on lung-, breast- and ovarian cancer, but aim to expand our research to other cancer types later.
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MSc. Pharm. Ph.D., Research Director