A Critical Examination for the Pricing of Eculizumab and Efgartigimod in Myasthenia Gravis

Abstract

The recently released Institute for Clinical an Economic Review’s (ICER’s) final evidence report for eculizumab and efgartigimod in myasthenia gravis is one more example of a commitment to the invention of evidence to support pricing and formulary decisions. This is the ICER business model where the practitioners cling to a belief system that rejects the standards of normal science and, more spectacularly, a passionate belief in the mystical ratio properties of ordinal scales that denies the axioms of fundamental measurement. Without these beliefs ICER’s business model would collapse. This is pseudoscience: a collection of beliefs or practices mistakenly regarded as being based on scientific method. Unfortunately there are many who share ICER’s beliefs, including academic research centers and health decision makers who should know better; in this case the University of Illinois at Chicago, College of Pharmacy, Center for Pharmacoepidemiology and Pharmacoeconomic Research who, under contract to ICER developed the imaginary assumption driven simulation model to support invented claims for pricing and reimbursement for eculizumab and efgartigimod. Whether this shared belief is by accident, ignorance or design is an open question; the fact is that it is widely held to the detriment of patients where insurers and health system fall upon ICER pronouncements as support for pricing and denial of care; in particular the absurd Health Benefit Price Benchmark (HBPB). This HBPB amounts to a neo-eugenic application of a bankrupt methodology. The purpose of this commentary is to focus on two aspects of the ICER-Illinois invented evidence modeling: the first aspect, the downside of assumption driven simulation modeling, the potential creation of a multitude of competing models, the mathematically impossible quality adjusted life year (QALY) and the misguided attempt to deny the axioms of fundamental measurement in mapping ordinal EQ-5D-5L preferences from the ordinal Quantitative Myasthenic Gravis (QMG) score. The second aspect of this commentary is to propose rejecting the analytical dead end of imaginary simulation modeling by proposing standards that should be set for the creation and evaluation of value claims in health technology assessment, in particular need fulfillment quality of life (QoL), that meet the demarcation test to distinguish science from non-science (or pure bunk). The result is that the ICER pricing claims for eculizumab and efgartigimod in myasthenia gravis are nonsense and should be rejected out of hand. Not only should the respective manufacturers ignore them, but this message needs to be transmitted to health system decision makers before care is denied and patients harmed in myasthenia gravis.