Fundamental Outcome Measurement: Selecting Patient Reported Outcome Instruments and Interpreting the Data they Produce

2022

F1000Res

One of the more unfortunate features of health technology assessment is the tenacity with which leaders in the field and organizations such as the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the Institute for Clinical and Economic Review (ICER) cling to an evaluation framework that fails to meet the standards of normal science. Believers subscribe to a meme that is clearly non-science (metaphysics and pseudoscience) and one that should have been discarded over 30 years ago. Certainly, subscribing to an impossible belief is not unusual; indeed it may make the belief that much stronger. Yet the meme is non-sustainable; it is also pointless as the economic evaluation claims are non-evaluable. There is no acknowledgement of the standards of normal science or the limitations imposed by the axioms of fundamental measurement. The purpose of this commentary is to make the case that the recent release of the Consolidated Health Economic Evaluation Reporting Standards 2022 (CHEERS 22) checklist is misleading; CHEERS 22 fails to address the manifest deficiencies in the approach to economic evaluations endorsed by ISPOR and ICER. Instead, it continues to promote economic evaluations in healthcare that invent evidence and non-empirically evaluable value claims. Given the widespread publicity that has accompanied the release of CHEERS 22, the purpose of this commentary is to detail the deficiencies in CHEERS 22 and propose an alternative framework for economic evaluation in health care to meet the information needs of formulary committees. This means abandoning the standards for economic evaluations that have dominated health technology assessment for 30 years, notably the key role assigned to the mathematically impossible quality adjusted life year (QALY). The proposed new start recommends single attribute evaluable value claims that meet ratio or interval measurement standards and are supported by evaluation protocols.

“…Unfortunately, the same ignorance of fundamental measurement holds for the overwhelming majority of disease specific patient reported outcome (PRO) instruments. 21 , 35 …”

Section: Introductionmentioning

confidence: 99%

Nothing to Cheer About: Endorsing Imaginary Economic Evaluations and Value Claims with CHEERS 22

2022

F1000Res

“…Under certain circumstances, this interval scale may be transformed to a bounded ratio scale, the ideal measure for PRO value claims in therapy response 9 . The failure of the QALY rests on the failure to appreciate that utility or preference scores are ordinal and cannot support multiplication; that requires a ratio scale with a true zero 7 .…”

Section: Normal Science and Fundamental Measurementmentioning

confidence: 99%

“…Standards for PRO claim have been proposed which are consistent with the standards for normal science and Rasch measurement theory (RMT) for ensuring claims have interval properties 7 . In the last resort, as discussed below, this requires recognition of the need for application of Rasch measurement assessments.…”

Section: Introductionmentioning

confidence: 99%

Evidentiary Standards for Patient-Centered Core Impact (PC-CIS) Value Claims

2022

Innov Pharm

Self Cite

Proposals for a patient centered core impact set (PC-CIS) are of little relevance to formulary and health system decisions, let alone patients and providers, unless the elements included in the data set meet the standards of normal science and fundamental measurement. Adhering to these standards will have the effect of focusing on the adequacy of proposed core impact measures, with a filter in place to accept only those that meet the standards not only of the physical sciences but also mainstream economics. and health economics. Fortunately, we are well aware of what the criteria for acceptance and rejection of the core impacts within disease states should be in terms of their required attributes and their relevance for supporting evaluable value claims, notably for patient reported outcomes, Rasch or modern measurement theory. Care must be taken to delineate the core impact elements: separately identifying those that are purely clinical from core patient centric impacts, which is turn should be separated from impacts defined in terms of drug utilization and resource utilization. The purpose of this brief commentary is to set out the required standards for core impact patient-centric value claims and the framework for evaluating those claims. The critical issue for patient-centered core impacts is to recognize the constraints imposed by the standards of fundamental measurement for target patient populations within disease areas; unless these constraints are recognized we will fail. The leads to the role of Rasch or modern measurement theory calibration as the framework for patient centric measures of latent traits or attributes. From these perspectives PC-CIS is premature; until we have agreed standards for measurement for the impact or outcomes for clinical, patient-centric and resource utilization as a core set of disease specific instruments, it seems pointless to push forward to a wider scope when the present evidentiary foundation is so weak.

“…There is, of course, a grey area between exclusively non-PRO clinical endpoints and PRO endpoints used by physicians to judge therapy response, yet all proposed measures must meet the required standards. This is critical because the overwhelming majority of PRO instruments used to assess therapy response may fail to meet the required Rasch measurement standards 10 11 12 13 . Indeed, if there is a proposed core of instruments proposed for assessing response in a rare disease these must all be subject to the same scrutiny against RMT measurement standards (e.g., for polytomous response PRO instruments the concordance with the Rasch Rating Scale Model or Partial Credit Model to support the application of scores as approximations to interval scales for therapy response claims) 6 14 .…”

Section: A New Health Technology Assessment Frameworkmentioning

confidence: 99%

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

2022

Innov Pharm

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.