Previous commentaries in the Formulary Evaluation section of INNOVATIONS in Pharmacy have pointed to the lack of credibility in modeled claims for cost-effectiveness and associated recommendations ...for pricing by the Institute for Clinical and Economic Review (ICER). The principal objection to ICER reports has been that their modeled claims fail the standards of normal science: they are best seen as pseudoscience. The purpose of this latest commentary is twofold: first, to review the latest report by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) on standards and value claims for modeling imaginary cost-per-quality life year (QALY) worlds in health technology assessments and (ii) given ICER’s commitment to ISPOR standards, to consider the credibility of ICER’s QALYs. The concern is that ICER gives the impression that there is a common and agreed utility metric to support QALY constructs and that there is an agreed standard for creating QALYs within the imaginary reference case meme. The purpose of this commentary is to emphasize that there is no ‘gold standard’ QALY, let alone a ‘gold standard’ utility metric. A QALY is what you assume the QALY should be; it is an artificial construct which allows any number of competing QALYs to be generated for the same target population under different imaginary constructs. Similar objections apply to the application of willingness-to-pay thresholds to support ICER’s business case as the sole arbiter, in pole position, of health technology assessments to support pricing recommendations. In the US. A threshold has to be defined for the QALYs and costs captured, by assumption, within the model. Even for the same target patient population a $50,000 cost per QALY threshold will yield different pricing recommendations depending upon the assumptions driving the QALY estimate, such as choice of utility metric, time spent by stage of disease, frequency and severity of adverse events and costs. While this is an obvious point, it appears not to occur to those who, with a limited technical understanding of how the ICER model is constructed (and ICER is not given to extended technical explanations) take at face value the ICER imaginary construct and the QALY value judgements. Of course, it is also beside the point that the ICER reference case imaginary world was never intended to support empirical assessments of the claims made; we have no idea of whether it is right, if it is wrong and, over the lifetime perspective, we will never know and were never intended to know.
Article Type: Commentary
In the wake of the global financial crisis of 2007-09, political economists have typically identified and interrogated speculative logics and credit-debt relations as the markers of financialized ...capitalism. This paper argues that assets, and the contingent processes which turn all manner of things into assets (i.e. 'assetization'), can also be usefully foregrounded to understand the character and movement of financialized capitalism in the contemporary conjuncture, particularly in its Anglo-American heartlands. Centred on assets and assetization, research is refocused on the constitution of political economies of rent and investment, especially as the frontiers of financialized capitalism are extended to further incorporate nature and society. Research into financialized capitalism is also connected more explicitly to wider political debates over intensified inequalities, as the production and distribution of assets is key to wealth disparities and shapes fundamental stratifications across society.
The Great I-QALY Disaster Langley, Paul C
Innovations in pharmacy,
2020, Letnik:
11, Številka:
3
Journal Article
Recenzirano
Odprti dostop
The QALY is an impossible construct; it defies common sense. It fails completely once we consider the axioms of fundamental measurement. Utilities as ordinal scales cannot be used to create QALYS. ...The QALY should never have been introduced to support the value assessment of pharmaceutical products and devices. The result is 30 years of QALY based assessments of pharmaceutical products and devices which are conceptually and technically wrong. They are a charade and will have contributed mistakenly to thousands of formulary decisions. In the search for a common metric to evaluate cost-effectiveness the impossibility of a QALY was overlooked. The result is a disaster, unfolding over decades. Our next steps must be to abandon the QALY paradigm and look ahead to a new value assessment framework.
On 20 February 2020, the Institute for Clinical and Economic Review (ICER) released its draft evidence report to establish the value of innovative therapies in the treatment of cystic fibrosis. ...Following its usual practice, ICER contracted with an outside group to construct a value assessment framework, in this case a microsimulation model, to generate value claims. The primary outcomes for value claims were incremental cost-per-QALY simulations for four target cystic fibrosis populations. The value assessment, in common with the same model applied earlier by ICER in cystic fibrosis, recommended substantial price discounts based on arbitrary threshold cost-per-QALY values. Unfortunately, the entire exercise, as detailed in previous commentaries in INNOVATIONS in Pharmacy is essentially a waste of time. Not only is the reference case model presented by ICER only one of a multiverse of other models, all driven by a selective application of model structure and assumptions, but the fact that the utilities that are applied to hypothetical time spent in different disease stages to modeled QALYs and lifetime cost-per-QALY claims fail to meet fundamental measurement axioms: they are ordinal manifest scores. Applied to target cystic fibrosis target patient groups, the modeled claims are meaningless. From the manufacturer's perspective, in this case Vertex Pharmaceuticals who have developed all the cystic fibrosis therapies 'modeled' by the ICER contractor, their response to ICER claims should be to reject them out of hand; the constructs are imaginary and the outcome claims nonsense.
Previous commentaries in the Formulary Evaluation section of INNOVATIONS in Pharmacy have pointed to the lack of credibility in modeled claims for cost-effectiveness and associated recommendations ...for pricing and access by the Institute for Clinical and Economic Review (ICER). The principal objection to ICER reports has been that their modeled claims fail the standards of normal science: they are best seen as pseudoscience. The purpose of this latest commentary is to provide a critique of the recently released ICER 2020 Value Assessment Framework (VAF). Although ICER has taken upon itself the pole position in health technology assessments and recommendations for product pricing in the US health care system, the incremental, lifetime cost-per-QALY modeling methodology should not be taken seriously. The creation of imaginary modeled worlds, built entirely from assumption, fails the demarcation test between science and pseudoscience. The ICER evidence reports are best seen as the health technology assessment equivalent of 'intelligent design' in counterpoint to 'natural selection'. It is surprising, therefore, that health care decision makers should take ICER's recommendations seriously as providing 'approximate information' for formulary decision making. What is not appreciated is that the claims made by ICER lack credibility, are impossible to evaluate and lack the ability to be replicated across treatment settings. Indeed, the models presented under the guise of a 'state of the art' value assessment were never intended to support evaluable claims. We have no idea and will never know if they are right or if they are wrong. ICER's position becomes even more untenable once the models presented are assessed in detail. Without in any way supporting the ICER methodology, it is worth noting that all too often ICER's claims for incremental QALYs in specific models are based upon what appears to be, from the limited evidence presented, a casual and ad hoc assemblage of utility scores from diverse constructs. This is a critical weakness given the role attributed by ICER to the modeled cost-per-QALY claims as central to ICERs imaginary value assessment. ICER also overlooks the fact that the utility scores it captures from the literature to populate its imaginary reference case world lack objectivity. They are ordinal rather than interval measures. To apply these manifest scores to time spent in a disease stage and then aggregate these over different disease stages is nonsensical. The critical issue is one of instrument development. The case made here is for the application of Rasch Measurement Theory (RMT) to construct a unidimensional instrument with interval properties, in this case from the needs fulfillment construct of quality of life (QoL). Unless an instrument meets RMT standards in its development, the logic of Rasch modeling to achieve fundamental measurement standards means that other scales are, by definition, ordinal. It is absurd to 'assume' they are interval. RMT is designed to create instruments to evaluate change and test hypotheses. In the absence of instruments that have RMT properties, the cost-per-QALY reference case modelling meme collapses. It is an analytical dead end. If we are to support a meaningful scientific program to discover new facts to support health care delivery and improve the lives of patients, caregivers and their families, then ICER should be put to one side.
On 9 December2019, the Institute for Clinical and Economic Review (ICER) released its final evidence report to establish the value of oral semaglutide (Novo Nordisk) for Type 2 diabetes (T2DM). A key ...element in this report was the development of a lifetime cost effectiveness microsimulation model based on a small sample of NHANES diabetes respondents. The model contrasted oral semaglutide added to current antihyperglycemic treatment for T2DM. The purpose of the model was to estimate outcomes that included life years (LYs) gained, an estimate of equal value life years gained (evLYGs), QALYs gained, clinical events, cost per major adverse cardiovascular events (MACE) avoided and total costs for each intervention over a lifetime time horizon. Previous commentaries in INNOVATTIONS in Pharmacy have provided detailed critiques of the ICER modeling framework. While this model differs from previous ICER models, the result is still a framework that constructs a so-called evidence base that fails the demarcation test. It is best described as pseudoscience. The model creates, by assumption, an imaginary world. The claims made for oral semaglutide by ICER should not be taken seriously by health care decision makers. The purpose of this commentary is to point to the limitations of the model with particular reference to the utility metrics employed, the resulting claims for quality adjusted life years (QALYs) and consequent recommendations for price discounting and affordability.
One of the features of the ICER stakeholder involvement in the development of ICER evidence reports is the ability for public comment. Unfortunately, and this may just a miscommunication, the replies ...from ICER to public comments frequently miss the point or fail to provide backup for their claims. The purpose of this commentary is to review ICER's responses to public comments by the author on the just released final evidence report on cystic fibrosis. The message is quite simple: the ICER value assessment framework lacks credibility. It fails to meet the standards of normal science. This is seen in ICERs apparent ignorance or rejection of the axioms of fundamental measurement which point quite clearly to the mathematical impossibility of creating QALYs from generic multiattribute utility scores. The ICER report also fails standards by creating a model from prior assumptions; there is no logical basis for constructing a value assessment claim. Either ICER should withdraw its value claims or admit the dubious basis on which the model is built, as a duty to its readership.
A number of commentaries have been published over the past 4 years by the present author on the manifest flaws in the reference case value assessment framework of the Institute for Economic and ...Clinical Review. The recent release of the evidence report on sickle cell disease continues ICER's commitment to what has been described as the creation of imaginary worlds to support value assessment. The purpose of the present commentary is to continue the critiques that have been presented for earlier evidence reports. This is important because of the apparent willingness to take ICER's recommendations at face value rather than a critical review of the value assessment framework. The case presented here points to a number of weaknesses in the ICER framework: (i) the fabrication of imaginary constructs with a lifetime cost-per-incremental QALY framework; (ii) the consequent failure to meet the standards of normal science; (iii) the illogical reliance of assumptions drawn from the literature to create future scenarios; (iv) the rejection of hypothesis testing in favor of 'approximate information' and (v) a belief that in the construction of QALYS that the EQ-5D-3L utility scale has ratio properties. This last point is demonstrably false which means that the ICER value assessment framework collapses. It is impossible mathematically, a failure to meet the axioms of fundamental measurement, for an ordinal utility scale to be combined with time spent in a disease state. The result is that the pricing and access recommendations for Crizanlizumab, Voxelotor and L-glutamine in sickle cell disease (SCD) are complete nonsense and should be rejected.
All too often organizations embrace standards for health technology assessment that fail to meet the standards of normal science. A continuing puzzle is why the axioms of fundamental measurement are ...ignored by researchers such as the University of Washington Model Group in constructing lifetime cost-per-QALY claims. The University of Washington Model Group is not alone; it is an accepted article of faith that multiattribute utility scales can be manipulated as if they had ratio scale properties, which they do not. This commitment to pseudoscientific claims, embracing intelligent design rather than natural selection, is endorsed by professional groups such as ISPOR as well as by self-appointed arbiters of value assessment such as ICER. Perhaps the answer is peer pressure rather than ignorance of the axioms of fundamental measurement. More to the point, if you have been an advocate of imaginary simulations a Damascene epiphany creates both psychological and professional challenges. After all, if cost-per-QALY constructs are rejected, then it is difficult to see what options there are for those attempting to model cost-effectiveness claims. If it is just ignorance of the axioms of fundamental measurement then a reasonable question is why these axioms, readily available on any number of internet sites, are ignored in health technology assessment programs. The purpose of this commentary is to review the ICER September 11
2020 evidence report in ulcerative colitis, with particular reference to ICER's responses to questions raised in the public comment period on the measurement properties (or their absence) for utility scales; in this context the EQ-5D instruments. The critique pointed out that the utility scores had ordinal properties. ICER, without proof, disputed this statement asserting that health economists believed (or assumed) they were ratio scales. This is nonsensical. ICER has two options: first, to continue to believe that the EQ-5D instruments had ratio properties or second, to acknowledge that they indeed only had ordinal properties, rejecting their many modeled claims for pricing and access. Not surprisingly, the possibility of a Damascene epiphany was rejected. ICER maintained its assertion that health economists, presumably all of them, believe or possibly just assume for analytical convenience that the EQ-5D-3L and similar measures are in fact on a ratio scale. This introduces a new concept in fundamental measurement: a ratio scale without a true zero but with negative values. ICER is quite prepared to admit that negative I-QALYs are possible and their lifetime cost-per-incremental I-QALY modelling can yield negative I-QALYs.
Previous commentaries in the Formulary Evaluation section of INNOVATIONS in Pharmacy have pointed to the lack of credibility in modeled claims for cost-effectiveness and associated recommendations ...for pricing by the Institute for Clinical and Economic Review (ICER). The principal objection to ICER reports has been that their modeled claims fail the standards of normal science: they are best seen as pseudoscience. The purpose of this latest commentary is to consider the recently released ICER evidence report for Janus Kinase (JAK) Inhibitors. As ICER continues, in the case of JAK Inhibitors, to apply its modeled cost utility framework with consequent recommendations for pricing adjustments, these recommendations also lack credibility. In contrast with previous ICER evidence reports, the present report adopts only a 12-month timeframe, one due, in large part, to ICER being unable to justify assumptions to drive its construction of imaginary worlds beyond 12 months. This commentary emphasizesagain, why the ICER methodology fails to meet the standards of normal science. Claims made by ICER for the competing JAK Inhibitor therapies lack credibility, are impossible to evaluate, let alone replicate across treatment settings. Even so, it is important to examine a number of key elements in the ICER invention of the 12-month JAK Inhibitor imaginary world. While this does not imply any degree of acceptance of the ICER methodology, one element that merits particular attention is thefailure of the ICER modeling to meet logically defensible measurement standards in its application of generic health related quality of life (HRQoL) ordinal metrics to create its QALY claims. The failure to meet the required standards of fundamental measurement means that the cost-per-QALY claims are invalid. This raises the issue of the application of Rasch Measurement Theory (RMT) in instrument development and the potential role of patient centric outcome (PCO) instruments that represent the patient voice in value claims. The case made here is that the ICER approach should be abandoned as an unnecessary distraction. If we are to meet standards for the discovery of new facts in therapy response then our focus must be on proposing credible, evaluable and replicable claims within disease states. Instruments, such as the Rheumatoid Arthritis Quality of Life (RAQoL)questionnaire that build on the common construct that QoL is the extent to which human needs are fulfilled should be the basis for value claims. HRQoL Instruments that are clinically focused and reflect the value calculus of providers and not patients in measuring response by symptoms and activity limitations are irrelevant. This puts to one side the belief that incremental cost-per-QALY models, the construction of imaginary worlds are, in any sense, a 'gold standard'; a meme embraced by the health technology assessment profession. Claims for incremental cost per QALY outcomes and recommendations for pricing and access driven by willingness to pay thresholds are irrelevant to formulary decisions.
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