Health economic analysis of Rydapt for treatment of AML patients in Belgium

  • Client: Novartis
  • Period: October 2017 – January 2018
  • Contact: Ann Tanghe
  • Domain: HEMAR
  • Keywords: Reimbursement, Leukaemia, Expert opinion

Project description

Novartis, a large international pharmaceutical company applied for the Class 1 reimbursement of the new drug, Rydapt, 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:
  • 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 the 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
    • Adaptations of an existing CEA full report to the Belgian context
  • BIA support
    • Development of a BIA model tailored to the Belgian context
    • Population of the BIA model with the collected data, and generation of results
    • Development of a BIA report
    • Development of a Dutch summary BIA report

Approach

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:

  1. Desk research and unit cost collection
    • Type of information collected included: product costs, unit costs (bone marrow biopsy, hospitalisation, blood tests, blood transfusions, consultations), life tables, patient characteristic, nurse at home costs, FLT3 testing, SCT cost, terminal care costs, and epidemiological data.
  2. Expert opinion
    • Sources such as MZG-MKG national data, literature, RIZIV/INAMI national database, and so on were consulted.
    • Development of 2 surveys: one on epidemiology and clinical practice of AML in Belgium and one on the treatment of AEs
    • Processing of all data collected via the survey
    • Individual interviews with 4 experts to discuss the results of the surveys
    • Development of 2 reports
      • Expert opinion report on epidemiology and clinical practice
      • AE cost report

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.

Results

  • Gap-analysis between required data and available data.
  • Kick-off meeting in which strategy of the reimbursement submission (CEA and BIA) was determined in cooperation with Novartis.
  • Data collection of all key information to populate the CEA and BIA model (including specification of the sources).
  • Survey on the current Belgian clinical practice and epidemiology.
  • Survey on the current Belgian resource use necessary to treat specific AEs.
  • A transparent and detailed AE cost calculation model in Excel, consisting of the resource use obtained from the experts, the derived resource use consensus, the unit costs and the total cost calculation.
  • Expert opinion report on the clinical practice: a report describing the applied methodology, questions asked, results obtained, and conclusions made.
  • AE cost report: a report structurally presenting how the AE costs were determined (including the applied methodology) and discussing the resource use, unit costs and total cost for each AE.
  • Validated and populated CEA model, adapted for Belgium.
  • Clear and validated CEA report, conform KCE guidelines and RIZIV/INAMI expectations, describing the model, methodology, inputs and results.
  • Summary CEA report (NL) describing the data inputs and model results.
  • Validated and populated BIA model for Belgium, with results, including a one-way sensitivity analysis.
  • Clear and validated BIA report, conform KCE guidelines and RIZIV/INAMI expectations, describing the model, methodology, inputs and results.
  • Summary BIA report (NL) describing the model, inputs and results.

Your contact person

Ann Tanghe
Senior Manager
Ann Tanghe
By submitting this form, you accept the Mollom privacy policy.
MeetDistrict | Ottergemsesteenweg-Zuid 808 B/354 | 9000 Gent | Belgium | Phone: +32 (0)92 77 77 84 | BTW BE 0866 039 556 | RPR/RPM Brugge | e-mail: info@hict.com