Multiple Sclerosis and the Comparative Value Disease Modifying Therapy Report of the Institute for Clinical and Economic Review (ICER)

2018

In August 2018, CVS Health released a position paper detailing policies in place and those being implemented to help reduce the costs of drugs. This paper introduced three new strategies for reducing costs. These are (i) zero out of pocket costs for chronic disease through a preventive drug list; (ii) reducing the launch price through adoption of modeled cost-per-QALY outcomes by the Institute for Clinical and Economic Review (ICER) to guide clients to exclude drugs launched at a price of greater than $100,000 per QALY; and (iii) introducing tools to be used by doctors, pharmacists and consumers to create greater transparency in understanding the real cost of drugs. The purpose of this commentary is to consider the second of these strategies, the application of a willingness to pay threshold as a viable strategy for impacting launch costs. The arguments presented here are that while modeled cost-per-QALY claims are a staple of formulary committee deliberations in many single payer health systems, their adoption by CVS Health fails to take into account not only the imaginary nature of the modeled construct utilized to generate the cost-per-QALY estimate and its shortcomings but the fact that alternative model structures may render invalid the application of willingness-to-pay thresholds. The case is made that CVS Health could adopt a more rigorous process of formulary assessment to support both preliminary assessments of new products and also an ongoing process of formulary review that challenge manufacturers to justify pricing over product patent life. This process should capitalize on the development of formulary evaluation platforms, potentially involving blockchain technology and smart contracting, for therapy interventions in targeted patient populations. Introducing a more rigorous formulary process, in particular the requirement for claims evaluation protocols, will not only assist CVS Health in restraining price increases over the life of the product but, for the first time in the US, put manufacturers on notice that patently unreasonable pricing policies and claims for product performance can be systematically and effectively challenged. Article Type: Commentary

Section: The Icer Modeling Frameworkmentioning

confidence: 99%

CVS Health and the Imaginary Worlds of the Institute for Clinical and Economic Review (ICER)

2018

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

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