Proof of Concept / Health Economics & Outcomes Research
Monte Carlo
Budget Impact Model
A probabilistic budget impact model simulating the financial consequences of bezuclastinib entering the systemic mastocytosis market against Ayvakit (avapritinib). This proof of concept demonstrates AMCP-format payer analytics with 10,000 Monte Carlo iterations, tornado sensitivity analysis, and three-scenario comparison — built entirely in-browser with no backend dependencies.
Disclaimer: This is a proof-of-concept demonstration using synthetic parameters and assumptions. All cost inputs, epidemiological estimates, and market share projections are illustrative and do not represent actual pricing, clinical data, or commercial strategies of Blueprint Medicines, Cogent Biosciences, or any other entity. This tool is NOT intended for formulary decision-making and should NOT inform actual payer negotiations.
Running 10,000 Monte Carlo iterations...
Technical Architecture
Monte Carlo Engine
10,000 iterations with seeded PRNG (Mulberry32) for full reproducibility. Each iteration independently samples 16 parameters from their respective distributions, computes the population funnel, and calculates two-world cost differentials.
Distribution Sampling
Normal (Box-Muller), Beta (Marsaglia-Tsang gamma method), and Triangular (inverse CDF) distributions. Beta distributions model bounded proportions while Triangular captures expert-elicited ranges with asymmetric uncertainty.
Sensitivity Analysis
One-way tornado analysis varies each parameter ±20% from base case while holding all others constant. Ranks parameters by absolute swing in net budget impact, identifying the key cost drivers for payer negotiation strategy.
References
- Sullivan SD, et al. Budget impact analysis — principles of good practice: report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force. Value Health. 2014;17(1):5-14.
- AMCP Format for Formulary Submissions, Version 4.1. Academy of Managed Care Pharmacy. 2020.
- Gotlib J, et al. Avapritinib for advanced systemic mastocytosis (PATHFINDER). N Engl J Med. 2021;384(14):1343-1353.
- DeAngelo DJ, et al. Bezuclastinib for advanced systemic mastocytosis (APEX): Phase 2 results. Blood. 2023;142(Suppl 1):Abstract 620.
- Jennings S, et al. The Mastocytosis Society survey on mast cell disorders: prevalence and clinical phenotype. J Allergy Clin Immunol Pract. 2014;2(1):70-76.
- Briggs AH, et al. Model parameter estimation and uncertainty analysis: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force. Med Decis Making. 2012;32(5):722-732.