PhD defence H.Y. (Chrissy) van Beurden-Tan

Dissertation in short:

The following doctoral thesis “Multiple myeloma evidence-based decision-making using phase 3 randomized controlled trial evidence” is describing how phase 3 RCT data can be utilized to support evidence-based decision making for determining cost-effectiveness of treatment sequences in multiple myeloma. 
Before cost-effectiveness can be calculated, synthesizing all available evidence is required. This thesis shows how to do this for treatments in newly diagnosed and relapsed and/or refractory (R/R) MM patients in two separate Bayesian network meta-analyses (NMA) based on the commonly used clinical outcome (i.e. progression-free survival). Another NMA was conducted in RR MM treatments using a different – less common - clinical outcome (i.e. response) to compare the treatments. This different choice of outcome requires a different NMA method and changes the ranking of the R/R MM treatments compared to the conventional NMA method. The correlation between response and time to next treatment was also investigated using a competing risk analysis based on patient level data from four newly diagnosed MM HOVON studies. Finally, the conventional NMA results from this thesis are integrated into an existing patient simulation model based on real-world evidence from the Dutch PHAROS registry to estimate the cost-effectiveness of treatment sequences of novel MM treatments.
Hence, this research shows it is possible to develop a cost-effectiveness patient simulation model for MM treatment sequences based on phase 3 RCT data. And it highlights that HTA bodies should be aware of different data synthesis methods resulting in slightly different rankings of treatments.