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

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

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Post QALY: Scientific Approaches to Real World Formulary Decision Making

INNOVATIONS in pharmacy ◽

10.24926/iip.v11i2.3375 ◽

2020 ◽

Vol 11 (2) ◽

pp. 19

Author(s):

Paul Langley ◽

Stephen McKenna

Keyword(s):

Decision Making ◽

Medical Devices ◽

Evidence Base ◽

Pharmaceutical Products ◽

Normal Science ◽

Value Assessment ◽

Scientific Methods ◽

Article Type ◽

The Us ◽

The Uk

The purpose of this anniversary supplement for Innovations in Pharmacy is to consider and / or propose modern, scientific methods for determining the evidence base for the fair pricing and accessibility of pharmaceutical products and medical devices. At present, such decisions are based on the construction of imaginary value assessment models that fail to meet the standards of normal science. Such a business model has been adopted by the Institute for Clinical and Economic Review (ICER) in the US, NICE in the UK and in a number of other countries. Article Type: Call for Papers

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Economic evaluation of early intervention services

The British Journal of Psychiatry ◽

10.1192/bjp.191.51.s19 ◽

2007 ◽

Vol 191 (S51) ◽

pp. s19-s22 ◽

Cited By ~ 17

Author(s):

Paul McCrone ◽

Martin Knapp

Keyword(s):

Early Intervention ◽

Cost Effectiveness ◽

Evidence Base ◽

Cost Utility ◽

Decision Makers ◽

Economic Evaluations ◽

Utility Analysis ◽

Early Intervention Services ◽

Scarce Resources ◽

Intervention Services

SummaryEarly intervention services have been introduced in a number of countries, but the evidence base to support them is limited. In particular there are very few economic evaluations, which are crucial if decision-makers are to have a better understanding of how scarce resources can be used appropriately. This paper discusses the different approaches used in economic evaluations and shows how these differ in the way in which outcomes are measured. The most useful forms of evaluation are cost-effectiveness and cost-utility analysis. We describe how the results of evaluations can be interpreted using incremental cost-effectiveness ratios and acceptability curves. Finally, the paper summarises some key evidence to date on early intervention services and economic evaluations currently being undertaken

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Yet another Ersatz World: The ICER Final Evidence Report for Additive Cardiovascular Therapies

INNOVATIONS in pharmacy ◽

10.24926/iip.v10i4.2337 ◽

2019 ◽

Vol 10 (4) ◽

pp. 22

Author(s):

Paul Langley

Keyword(s):

Cardiovascular Disease ◽

Cost Effectiveness ◽

Ad Hoc ◽

Analytical Framework ◽

Quality Adjusted Life Year ◽

Decision Makers ◽

Normal Science ◽

Article Type ◽

Value Judgements ◽

Principal Objection

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 Additive Cardiovascular Disease therapies. This report should not be taken seriously in its claims for cost-effectiveness and pricing in cardiovascular disease (CVD). The analytical framework applied by ICER fails to meet the standards of normal science in demarcating science from pseudoscience. Irrespective of the value judgements and recommendations of an ICER report, these lack credibility. They were never intended to be evaluable and replicable across treatment settings. The claims made are constructed, driven by assumption, and should be put to one side by health system decision makers. In this review the focus is on to the ICER modeled estimates of utility scores in CVD, the insistence on utilizing a generic utility algorithm (the EQ-5D-3L) and the consequent quality adjusted life year (QALY) estimates. Two issues are raised that will be the subject of future commentaries: the lack of appreciation of fundamental measurement and (ii) the importance of the patient voice in benefit claims. Given the importance in the ICER methodology of QALYS, the ad hoc nature of the ordinal utilities introduced to the cardiovascular model must raise concerns over the role the ICER evidence report may play in health care decision-making. These concerns extend to the claim by ICER that, on ICER’s own affordability threshold for individual new molecular entities, the anticipated uptake of these therapies may raise questions of overall affordability. Again, we are dealing with an arbitrary construct that may adversely impact patient access. Article Type: Commentary

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How to choose tools and programs that are worth the investment

Phi Delta Kappan ◽

10.1177/00317217211043633 ◽

2021 ◽

Vol 103 (1) ◽

pp. 54-57

Author(s):

Fiona Hollands ◽

Anna Kushner

Keyword(s):

Decision Making ◽

Evidence Base ◽

Cost Utility ◽

Cost Utility Analysis ◽

Decision Makers ◽

Evidence Based ◽

Utility Analysis ◽

Program Strategy ◽

Impossible Task ◽

Education Decision

Making evidence-based decisions in schools seems like an impossible task when hundreds of activities are available, the evidence base fails to address most of them, and budget deadlines are immovable. In addition, different stakeholders hold different views on what it means for a program, strategy, or intervention to “work.” Fiona Hollands and Anna Kushner describe a 10-step decision-making framework developed in collaboration with practicing education decision makers to tackle these kinds of complex decisions. It is based on cost-utility analysis, which addresses two main questions: What resources are required to implement a program, and how useful or satisfying is each program to stakeholders?

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Medicaid Formulary Decisions and the Institute for Clinical and Economic Review: Abandoning Pseudoscience in Imaginary Pharmaceutical Pricing Claims

INNOVATIONS in pharmacy ◽

10.24926/iip.v12i1.3702 ◽

2021 ◽

Vol 12 (1) ◽

pp. 11

Author(s):

Paul Langley

Keyword(s):

Simulation Models ◽

Standard Of Care ◽

Evidence Base ◽

Quality Adjusted Life Year ◽

Unintended Consequences ◽

Normal Science ◽

Pharmaceutical Pricing ◽

Real World Evidence ◽

Health Decision ◽

The One

Medicaid formulary committees and other gatekeepers face a difficult task. On the one hand they can utilize technical expertise in evaluating the real world evidence for clinical, quality of life and resource utilization claims for competing products while on the other hand they may be asked to assess claims built by simulation models for pricing and product access. A common option has been to take modeled claims from third parties such as the Institute for Clinical and Economic Review (ICER) at face value without challenging the model structure, its assumptions and its incremental cost-per-QALY claims set against competing products or the existing standard of care. Unfortunately, from the available evidence, it seems clear that many formulary assessment groups, last but not least those for whom the ICER modeling claims are targeted, have little if any appreciation of the limitations of ICER modeling. There are two substantive issues: (i) a failure to appreciate the limitations imposed by the standards of normal science for credible, empirically evaluable and replicable product claims and (ii) an understanding of limitations imposed by the axioms of fundamental measurement. In the latter case, a failure to recognize that the quality adjusted life year (QALY) is an impossible mathematical construct (hence the I-QALY). To these limitations should be added the potential for constructing competing imaginary claims. Surprisingly, ICER has provided the ideal opportunity to construct competing claims with the launch in late 2020 of the ICER Analytics cloud platform. Formulary committees and other health decision makers should be aware that claims based on the ICER Analytics platform together with competing lifetime modelled claims all fail the standards of normal science. Factoring these into formulary decisions is not only misguided but may have unintended consequences for pricing and access that may disadvantage significantly patients and caregivers. We have spent too much time debating the merits or otherwise of the I-QALY for targeted patient groups with the parties failing to recognize that the focus on simulated cost-per-I-QALY value assessments is a mathematical folly; I-QALY claims are a chimera. The I-QALY, at long last, should be abandoned together with modelled lifetime simulations. Medicaid formulary decision makes should rethink the required evidence base for formulary decisions and negotiations. Care should be taken to revisit previous negotiations where ICER recommendations have been utilized to support pricing and access.

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