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 summary, this research project aims to harness Nordic RWD to conduct trial emulation studies and construct ECAs. These emulations will encompass situations when constructing ECAs to SATs, and situations when emulating both the active and the standard-of-care arm in carefully selected phase I-III RCTs, offering valuable context to RCT findings. Additionally, the emulation framework will be employed to conduct hypothetical target trial emulation studies in settings lacking evidence from RCTs and to study the representativeness of RCT populations compared to clinical practise.
We are currently focusing on lung-, breast- and ovarian-, with the aim to expand our research to other cancer types later.
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Christian Jonasson
MSc. Pharm. Ph.D., Research Director
