Novartis, a large international pharmaceutical company, applied for the a Class 1 reimbursement of the new drug, RydaptTM, to treat patients with acute myeloid leukaemia who are FLT3 mutation-positive.
Novartis asked Hict for support in the pharmaco-economic section of this reimbursement submission.
This support included:
Hict’s approach consisted of six phases: intake, data collection, CEA support, CEA report, BIA support, and BIA report phase.
Phase 1 – Intake phase
In the intake phase, the context, strategy and key parameters of the submission were discussed and analysed. An in-depth analysis of the provided CEA model, data inputs and sources of this model was performed. Based on the model requirements and the knowledge of BIA model requirements a complementary high-level desk research was performed. A gap analysis matching the available data with the required data was performed. This gap analysis aided in the identification of the optimal data collection approach, and possible adaptations to the proposed approach, which was defined in cooperation with Novartis.
Phase 2 – Data collection
Based on the gap-analysis and data collection approach discussed and validated during the intake phase, the data collection protocol was executed during the data collection phase. In this phase relevant Belgian inputs for the CEA and BIA model were collected. Novartis chose the option yielding the more reliable and credible data, i.e. expert opinion in combination with desk research (instead of only desk research). This phase thus consisted of two major subsections:
Phase 3 – CEA model support
The data collected during the intake and data collection phase was transformed to a set of CEA model inputs according to Belgian health economic guidelines. To ensure the model was correctly populated and results were correctly interpreted, a teleconference call with the model developers was set up. The model was populated and results were generated. The type of results to be generated (e.g. decisions on base case and scenario analyses) were decided on in close collaboration with Novartis.
Model adaptations to the CEA model were proposed to the model developers, to allow the model to correspond maximally to the Belgian clinical practice and context. These adaptations were validated by the model developers, but performed by Hict.
Phase 4 – CEA report support
This phase was designed to generate a CEA report (based on the technical report from Novartis HQ). The report met Belgian reimbursement submission guidelines and clearly described the model, its methodology, the inputs (general and Belgian specific inputs), and the results. This CEA report was enclosed in the reimbursement submission as addenda. A summary of the CEA report was also written in Dutch to be enclosed in the reimbursement submission.
Phase 5 – BIA model
The data collected during the intake and data collection phase was transformed to a set of BIA model inputs according to the Belgian health economic guidelines. The model inputs and types of results to be generated (e.g. decisions on base case and scenario analyses) were decided on in close collaboration with Novartis BE.
Due to the very country specific nature of the existing BIA model, the preference was given to the development of a new BIA model, specifically for Belgium. The added benefit of creating the model from scratch was that the model would be more in line with RIZIV/INAMI expectations (eg. budget lines, OWSA), that extra options could be included and that the model could be made more user-friendly for relevant options (such as: limit population to patients above a certain age, add a second indication with different posology, vary the pricing strategy, etc.).
The robustness of the model results was validated by including a one-way sensitivity analysis (OWSA). The OWSA investigates the main sources of uncertainty by evaluating the effect of changing the value of a single parameter, whilst holding other parameters constant, on the overall model result.
Phase 6 – BIA model report
The BIA report development phase was designed to create a report that met Belgian reimbursement submission guidelines and clearly described the model, its methodology, the inputs, and the results. A summary of the BIA report was also written in Dutch to be enclosed in the reimbursement submission.