Health economic analyses of a CAR T-cell therapy for the treatment of pALL patients in Belgium

This support includes:

• Collection of the necessary Belgian cost data for the CEA and BIA model
• Development and implementation of a data collection strategy for the required CEA and BIA inputs
• CEA support
  • Critical review of an existing CEA model and (clinical) inputs
  • Population of the CEA model with the collected data, and generation of results
  • Development of a Dutch summary CEA report
• BIA support
  • Critical review of an existing BIA model and (clinical) inputs
  • Population and adaptation of the BIA model with the BE-specific collected data, and generation of results
  • Development of a Dutch summary BIA report

Approach

Hict’s approach consists of six phases:

Phase 1 – Intake phase

In the intake phase, the context, strategy and key parameters of the submission are discussed and analysed. An in-depth analysis of the provided CEA and BIA model, data inputs and sources of these models are performed. Based on the requirements of the models a complementary high-level desk research was performed, resulting in a gap analysis matching the available data with the required data. This gap analysis aids in the identification of the optimal data collection approach, and possible adaptations to the proposed approach.

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 are collected. This phase consists of two major subsections:

Desk research and unit cost collection

• Type of information collected includes: unit costs, life tables, patient characteristic, SCT cost, terminal care costs and epidemiological data.
• Sources such as MZG-MKG national data, literature, RIZIV/INAMI national database, etc. are consulted.

Expert opinion

• Development of 2 surveys: one on epidemiology and clinical practice of pALL in Belgium and one on the treatment of Adverse Events in the target population.
• Processing of all data out of the completed surveys
• Individual interviews with 4 experts to discuss the results of the surveys
• Development of an expert opinion report, detailing the results of the survey and interviews on epidemiology, clinical practice, and resource use needed to treat the adverse events considered.

Phase 3 – CEA model support

The data collected during the intake and data collection phase is transformed to a set of CEA model inputs according to Belgian health economic guidelines. To ensure the model is correctly populated and results are correctly interpreted, close communication with the model developers is facilitated. The model is populated and results are generated. The type of results to be generated (e.g. decisions on base case and scenario analyses) was decided in close collaboration with Novartis.

Model adaptations to the CEA model were performed, to allow the model to correspond maximally to the Belgian clinical practice and context. These adaptations were performed by Hict and validated by the model developers.

Phase 4 – CEA report support

During this phase a Dutch summary report on the CEA was generated, which describes the model, its inputs and results. This summary report is enclosed in the reimbursement submission dossier.

Phase 5 – BIA model

The data collected during the intake and data collection phase is transformed to a set of BIA model inputs according to 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.

The BIA model was populated with Belgian data, and where needed model adaptations are performed. Results were generated and discussed in detail with Novartis.

Phase 6 – BIA model report

The BIA report development phase is designed to create a summary report in Dutch to accompany the reimbursement submission. The report describes the BIA model, its methodology, the inputs, and the results.