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Adaptation to the Nordic — a model in rare disease

Our modelling team adapted a model to the Nordic setting.

What we did

Project background

The client was a world-leading pharmaceutical company working on treatments for rare genetic diseases. They are the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I) and for phenylketonuria (PKU). The client developed a cost-effectiveness model based on a Markov structure for one of their new treatments but encountered that the way of modelling the disease was not completely consistent with what was found in their trial. The model needed to be updated with the new data to create a better representation of the disease and estimate the cost-effectiveness more accurate.

Challenges faced

The whole structure of the model needed to be adjusted to create the appropriate patient flow and distributions in the model. Especially background coding with regard to the sensitivity analyses needed to be rewritten to account for the change in model structure and data inputs. Additionally, the whole model had to be updated with the latest information on pricing.  

Our solution

The model was rebuilt, starting at the inputs and systematically changing the structure and calculations in the model accordingly. Finally, the model was updated and all background coding was cleaned to ensure all analyses could be performed correctly and the code was comprehensible to all individuals working with it. 

Our impact

The success of the project resulted from the updated cost-effectiveness estimates created by the model, and positive results from a quality check on the model and its parameter use. 

Meet the experts

Timon Louwsma, MSc

Timon Louwsma, MSc

Services used

Health economic modeling
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