Facilitating bias in cost-effectiveness analysis: CHEERS 2022 and the creation of assumption-driven imaginary value claims in health technology assessment

One of the signal failures in health technology assessment is the absence of consideration given, not only to the standards of normal science, but to those of fundamental measurement. A recent evidence report by the Institute for Clinical and Economic Review (ICER) is emblematic of this failure. Based on a simple linear regression model that translates aggregate scores from the ordinal Menopause-specific Quality of Life Questionnaire (MENQOL) to the ordinal EuroQol EQ-5D-5L, ICER has applied these scores to an assumption driven model simulation to produce preferences, QALYs and incremental cost-per-QALY claims for fezolinetant for moderate to severe symptoms associated with menopause. Unfortunately, the attempt to crosswalk multidimensional or multiattribute ordinal scores is mathematically impossible. The ‘created’ EQ-5D-5L preferences are, as a result, of no interest. The overall result is that the ICER modelled claims for cost-effectiveness fail the required standards for normal science and fundamental measurement. fundamental are impossible. This is unfortunate, although it might be possible to assess certain domains of the MENQOL for their approximation to an interval score with the application of the Rasch Rating Scale Model, this will not support quality of life claims. A preferred approach would be to consider an alternative latent trait for quality of life in menopause, applying Rasch Measurement Theory (RMT), to develop a polytomous instrument that has the required measurement properties. The purpose of this commentary is to point out, as a number of previous commentaries have done, that this framework for creating assumption driven simulated modelled claims has no role in decisions for product assessment, access to formulary and pricing. This commentary expands upon these previous commentaries in placing RMT is the context of a needed paradigm shift to support the evolution of objective knowledge. This is critical if we are to understand, from the individual’s perspective, not only an accurate assessment of the burden of menopause but to see this as part of an on-going research program that has to rely on fundamental measurement.

Section: Standards For Value Claimscontrasting

confidence: 71%

Post-Menopausal Quality of Life Claims: Overlooking the Requirements of Normal Science and Fundamental Measurement in ICER’S Cost-Effectiveness Assessment of Fezolinetant for Moderate to Severe Vasomotor Symptoms

Langley

2023

“…A key element in the creation of value claims is to focus on evidence gaps; covering clinical, quality of life and resource utilization; if data are not available at product launch to enable rapid assessment and reporting of value claims, then outcomes contracting must look to data assembly to support claims over the longer term. Falling back on ICER modelled imaginary approximate information claims is no substitute; after all, there are any number of competing modelled claims that could be created, none of which could have a preferred non-evaluable claim for future realism over any other as they are all assumption driven subject to possible bias 1 17 . From the manufacturer’s perspective ICER and its claim for invented evidence is a major barrier; this must be faced down in the interests not only of the manufacturer but the patient and caregiver in rare disease.…”

Section: Discussionmentioning

confidence: 99%

Formulary Submissions: Value Claims, Protocols and Outcomes Based Contracting in Rare Disease

Langley

2022

Outcomes based payments contracting is in its infancy. The increased attention being given to rare disease place a premium on the ability to engage with payers to ensure that there is an analytical framework relevant to value claims contracting. Rare disease is not, of course, alone; many other chronic disease states may be suitable candidates and have been over the past 10 years or more. Rare disease, however stands apart: (i) the evidence base at product launch is limited; (ii) the therapy costs are often considered prohibitive; and (iii) the target patient population is small. At the same time, those seeking to implement an evidence-based engagement with health systems to support innovative rare disease interventions face a substantive technology assessment barrier. The focus in health technology assessment on assumption driven modeled cost-effectiveness simulations that support imaginary recommendations for cost-effective pricing and access is, however, an avoidable barrier. In the US, this barrier is the business model of the Institute for Clinical and Economic Review (ICER) and one endorsed by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Rare disease can be better served with other tools at our disposal with a proposed new start analytical framework in health technology assessment. The purpose of this brief note is to make the case that this proposed new start focused on single attribute value claims that meet the standards of normal science and fundamental evidence can not only dispense with the ICER imaginary modeling but, with a new start formulary submission package, integrate value claims with assessment protocols to set the stage for effective outcome-based contracting as the default standard for future payer negotiations.

“…This, again, points to the unique nature of HTA: a win-win for all with no hope of a challenge except by changing assumptions with a claim that a new set of assumptions are more ‘realistic’ for an unknown future than an alternative collection. Bias is inevitable 18 .…”

Section: The Statement Of Interest Ismentioning

confidence: 99%

Rejecting False Claims from Markov Simulations in Alzheimer's Disease

Langley

2023

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