The Imaginary Worlds of ISPOR:  Modeled Cost-Effectiveness Claims Published in Value in Health from January 2016 to December 2016

2020

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 emphasizes again, 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 the failure 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. Article Type: Commentary

Section: The Icer Reference Casementioning

confidence: 99%

More Unnecessary Imaginary Worlds - Part 1: The Institute for Clinical and Economic Review’s Evidence Report on Janus Kinase (JAK) Inhibitors in Rheumatoid Arthritis

2020

“…Over the past 30 years, literally thousands of modeled claims have been presented in the literature, including leading health technology assessment journals. Annual reviews of the status of cost-effectiveness or modeled claims in the three journals, Pharmacoeconomics , Value in Health and the Journal of Medical Economics found that the majority of models presented non-evaluable claims (typically lifetime cost-per-QALY) 20 , 21 , 22 , 23 , 24 . Where models were funded by a manufacturer a high proportion supported, in their modeled cost-per-QALY assessment, the manufacturer’s product.…”

Section: The Icer Reference Casementioning

confidence: 99%

Another Rush to Judgement: The Imaginary Worlds of ICER and Recommendations in Duchenne Muscular Dystrophy

2019

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 report for Duchenne muscular dystrophy (DMD). As ICER has continued in the case of DMD to apply its modeled cost utility framework with consequent recommendations for pricing adjustments, these recommendations also lack credibility. This commentary emphasizes again not only why the ICER methodology fails to meet the standards of normal science but to point to the importance in rare diseases for accelerated approval, while recognizing that evidence generation will continue. While this assessment of the ICER DMD model does not imply any support for this methodology, a key point is the application of quality of life measures which fail to capture the experience of patients with DMD and, importantly, the interests of both patients and caregivers. While ICER would argue that even with a limited evidence base it is important to address issues of pricing and access for new products, their reports are used as justification for coverage and reimbursement by insurers and health system decision makers without recognition of their lack of scientific merit. This rush to judgement by ICER must raise concerns about potentially adverse formulary decisions that result in access restrictions on new products. If ICER is to make a contribution to the entry of new products in the health market place then it should consider an alternative methodology that generates claims that are empirically evaluable in a timeframe relevant to health decision makers. As it stands, ICER’s recommendation should be rejected. This is not a research program that meets accepted scientific standards but one that relies on the willingness of an audience to accept the proposition that evidence is constructed not discovered. Article Type: Commentary

“…Over the past 30 years, literally thousands of modeled claims have been presented in the literature, including leading health technology assessment journals. Annual reviews of the status of cost-effectiveness or modeled claims in the three journals, Pharmacoeconomics , Value in Health and the Journal of Medical Economics found that the majority of models presented non-evaluable claims (typically lifetime cost-per-QALY) 18 , 19 , 20 , 21 , 22 . Where models were funded by a manufacturer a high proportion supported, in their modeled cost-per-QALY assessment, the manufacturer’s product.…”

Section: The Icer Reference Casementioning

confidence: 99%

The Imaginary Worlds of Cure Proportion Modeling: Survivorship and Reference Case Pricing of Transformative Therapies

2019

On August 6, 2019 the Institute for Clinical and Economic Review (ICER) released a set of proposed adaptations to its value assessment framework methods where the intervention under review was considered a ‘single or short-term transformative therapy’ (SST). These adaptations are intended to ‘complement and build upon the upcoming update to the overall ICER assessment framework…’. The purpose of this commentary is to review the proposed cure proportion modeling reference case framework for assessing the value of SSTs together with ICER’s ‘recommendations for fair value-based pricing …’. Following previous commentaries on the ICER value assessment framework, the question raised is whether the proposed cure proportion modeling standards meet those of normal science: is the modeling proposed capable of generating value claims for the intervention that are credible, evaluable and replicable? The proposed standards for transformative therapies do not change the underlying commitment to reference case modeling. At the same time, the cure proportion modeling proposed adaptations have to be seen in the context of the concerns expressed by ICER that their reference case model can be used to justify substantial one-off SST pricing. This follows from the ICER incremental cost per QALY willingness to pay thresholds where the SST QALY gains are sufficient, at even a $50,000 QALY cut-off, to support SST pricing in the millions of dollars. ICER has two options: (i) abandon the imaginary reference case methodology, which is the ICER core business model and would represent an ironic reversal, or (ii) attempt to bolt-on adaptations, possibly incorporating revised survivorship profiles using cure proportion modeling, that supports a modified imaginary reference case ‘rescue’ model for SSTs designed specifically to generate pricing recommendations that may be considered affordable. Article Type: Commentary