Local Real-World Evidence to generate an External Control Arm

The Challenge

An HTA application for an oncology drug was initially submitted to the Danish Medicines Council (DMC) for evaluation as standard treatment. However, the submission faced challenges as the evidence was based on a single-arm trial supported by a MAIC. This led to a negative decision by the DMC.
We were brought in to support the resubmission. Firstly, we provided strategic guidance on what could be improved and addressed to increase the likelihood of a successful outcome in the resubmission.

Solution

To improve the relevance of the matching-adjusted-indirect comparison (MAIC) and to demonstrate the unmet need in Denmark, we conducted an RWE study, leveraging individual patient-level data from the national health registries to model the survival outcomes for current standard of care, depending on the specific treatment line. We then conducted a MAIC to construct a relevant and robust external control arm. This approach strengthened the evidence base, reduced uncertainty, and aligned clinical data with the local healthcare context. Additionally, the cost-effectiveness improved drastically compared to the original submission, illustrating the much higher unmet need of Danish patients compared to the global evidence package.

Impact

In close collaboration with our client, NHTA managed to build a stronger case for the oncology product that resulted in a successful resubmission.