scholarly journals To Dream the Impossible Dream: The Commitment by the Institute for Clinical and Economic Review to Rewrite the Axioms of Fundamental Measurement for Hemophilia A and Bladder Cancer Value Claims

2020 ◽  
Vol 11 (4) ◽  
pp. 22
Author(s):  
Paul Langley
Keyword(s):  
Lifetime Cost ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Pharmaceutical Products ◽  
Normal Science ◽  
Value Assessment ◽  
New Paradigm ◽  
Dead End ◽  
Patient Reported ◽  
New Vision

Understandably, after 30 years of ignoring the axioms of fundamental measurement, advocates of creating approximate information through the construction of lifetime cost-per-QALY worlds are somewhat unnerved by the realization that their methodology is incompatible with those axioms. This is made all the more unnerving when it is pointed out that this incompatibility was pointed out over 30 years ago, following the formalization of those axioms almost 80 years ago. Why this was overlooked is a mystery. The result was a commitment to the application of ordinal utility and other patient reported outcome measures to support claims for response to competing therapies; most egregiously, the advocacy of cost-per-QALY lifetime models and willingness to pay thresholds to support recommendations for pricing and access to pharmaceutical products and devices. Although this incompatibility has been pointed out in respect of simulation modeling, to groups such as the Institute for Clinical and Economic Review (ICER) they press on, producing evidence reports and recommendations for emerging products that fail the standards of normal science.  While these are an analytical dead end, ICER has nowhere else to go. This is their business model; to admit otherwise would mean withdrawing their many evidence reports and admit they were wrong. ICER has rejected this; rather it has decided, together with its academic consultants, to challenge the axioms of fundamental measurement, to produce a parallel measurement universe that can sustain QALYs and the imaginary simulation lifetime models. The purpose here is to make clear that ICER is manifestly wrong and that there is no way it can maintain its credibility in pursuing this path. This is achieved by a deconstruction of the arguments put forward by ICER to defend its new vision of the axioms of fundamental measurement, a vision which provides a case study in the distinction between justified belief and opinion.  Fortunately, we have the framework for a new paradigm in value assessment; a paradigm that recognizes the standards of normal science and rejects belief in an alternative reality consistent with fundamental measurement axioms.

scholarly journals Value Assessment, Real World Evidence and Fundamental Measurement: Version 3.0 of the Minnesota Formulary Submission Guidelines

2020 ◽  
Vol 11 (4) ◽  
pp. 12
Author(s):  
Paul C. Langley
Keyword(s):  
Evidence Base ◽  
Pharmaceutical Products ◽  
Normal Science ◽  
Value Assessment ◽  
New Paradigm ◽  
Therapeutic Class ◽  
New Information ◽  
Real World Evidence ◽  
Fatal Flaw ◽  
New Evidence

This latest version of the Minnesota guidelines is intended to reassert the application of the standards of normal science in formulary submissions for new and existing pharmaceutical products and devices. This represents a paradigm shift from the existing value assessment standards which are focused on imaginary or I-QALY modeling of lifetime claims. The proposed new paradigm rejects this as pseudoscience; a failure to recognize the standards of normal science, in particular a failure to recognize the constraints of fundamental measurement. As a result, current health technology assessment is dominated by value assessments that create claims that are neither credible, nor empirically evaluable or replicable. The fatal flaw is the failure to recognize that QALYS are an impossible mathematical construct (hence the term I-QALY). The proposed paradigm recognizes that if there are claims for product value then, regardless of whether the claim is for clinical impact, quality of life or resource utilization, all claims must be empirically evaluable. If not, then they should be rejected. The Minnesota guidelines propose a new evidence based approach to formulary assessment, together with ongoing disease area and therapeutic class reviews. The focus is on claims that are specific to target patient populations that are claims for specific attributes and are consistent with the axioms of fundamental measurement. Manufacturers are asked to support claims assessment through protocols detailing the evidence base for claims assessment, the timelines for those assessments and the process by which claims assessments are reported back to formulary committees. Value assessment leads naturally to value contracting, revisiting provisional prices as new information is discovered and delivered to the formulary committee.    

What about the Customer? The Patient’s Dilemma

2014 ◽  
Author(s):  
Michael Tansey
Keyword(s):  
Clinical History ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Pharmaceutical Products ◽  
Medical Product ◽  
Fda Guidance ◽  
Department Of Health ◽  
Patient Reported ◽  
William Osler

Apparently no one was listening to Sir William. In a study carried out 80 years after Osler’s exhortation, physicians interrupted patients, on average, after 18 seconds (H. Beckman and R. Frankel, 1984). It is common to take for granted the very people who are essential for our continued paid employment and the success of our endeavors, namely our customers. For example, some airline staff refer to passengers as “self-loading freight” or SLF (www. pprune.org), which, though mildly amusing and certainly accurate, is hardly indicative of a warm and friendly service-focused attitude. Attitudes in the pharmaceutical business are no different. Throughout the development and marketing cycles, the emphasis is primarily on satisfying intermediaries such as regulators, opinion leaders, prescribers, and insurers. Important though these intermediaries may be, they are not the customers. Patients are the sole customers. Only patients pay for their drugs (whether directly or through insurance or taxation) and only patients consume pharmaceutical products and experience the benefits and adverse effects. Only patients decide whether or not to continue taking a drug. Put simply, if there were no patients, there would be no pharmaceutical industry. Sir William Osler was talking in the context of clinical history taking and examination, but his comment applies more broadly. Over the past 10 to 15 years, there has been slowly increasing recognition that there are virtually no aspects of development, registration, and commercialization of a drug in which listening to the patient does not improve the outcome. Guidance issued by the FDA (Guidance for Industry, Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims, U.S. Department of Health and Human Services, Food and Drug Administration, December 2009) has gone a long way in helping to redirect and focus the industry. The implications are broad and go beyond the traditional areas of patient input such as quality of life measures and can significantly influence trial design, approval, labeling, and reimbursement.

Patient-Reported Outcome Measures in Adults with Hand Trauma: A Systematic Review

2015 ◽  
Vol 27 (2-4) ◽  
pp. 145-157
Author(s):  
Daniel Alexander Waltho ◽  
Manraj Nirmal Kaur ◽  
Mary Ellen Gedye ◽  
Achilleas Thoma
Keyword(s):  
Systematic Review ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Hand Trauma ◽  
Patient Reported

Optimizing electronic capture of patient-reported outcome measures in oncology clinical trials: lessons learned from a qualitative study

2020 ◽  
Vol 9 (17) ◽  
pp. 1195-1204
Author(s):  
Florence D Mowlem ◽  
Brad Sanderson ◽  
Jill V Platko ◽  
Bill Byrom
Keyword(s):  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Lessons Learned ◽  
Structured Interviews ◽  
Patient Reported ◽  
Oncology Clinical Trials ◽  
Anticancer Treatment ◽  
Fit For Purpose ◽  
The Impact ◽  
Electronic Implementation

Aim: To understand the impact of anticancer treatment on oncology patients’ ability to use electronic solutions for completing patient-reported outcomes (ePRO). Materials & methods: Semi-structured interviews were conducted with seven individuals who had experienced a cancer diagnosis and treatment. Results: Participants reported that the following would impact the ability to interact with an ePRO solution: peripheral neuropathy of the hands (4/7), fatigue and/or concentration and memory issues (6/7), where they are in a treatment cycle (5/7). Approaches to improve usability included: larger, well-spaced buttons to deal with finger numbness, the ability to pause a survey and complete at a later point and presenting the recall period with every question to reduce reliance on memory. Conclusion: Symptoms associated with cancers and anticancer treatments can impact the use of technologies. The recommendations for optimizing the electronic implementation of patient-reported outcome instruments in this population provides the potential to improve data quality in oncology trials and places patient needs at the forefront to ensure ‘fit-for-purpose’ solutions.

Impact of Depression on Patient-Reported Outcome Measures After Lumbar Spine Decompression

Spine ◽  
2018 ◽  
Vol 43 (6) ◽  
pp. 434-439 ◽  
Author(s):  
Robert K. Merrill ◽  
Lukas P. Zebala ◽  
Colleen Peters ◽  
Sheeraz A. Qureshi ◽  
Steven J. McAnany
Keyword(s):  
Lumbar Spine ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported

scholarly journals The effects of competition and bundled payment on patient reported outcome measures after hip replacement surgery

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Fanny Goude ◽  
Sverre A. C. Kittelsen ◽  
Henrik Malchau ◽  
Maziar Mohaddes ◽  
Clas Rehnberg
Keyword(s):  
Hip Replacement ◽  
Health Gain ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Economic Incentives ◽  
Bundled Payment ◽  
Treatment Groups ◽  
Replacement Surgery ◽  
Hip Replacement Surgery ◽  
Patient Reported

Abstract Background Competition-promoting reforms and economic incentives are increasingly being introduced worldwide to improve the performance of healthcare delivery. This study considers such a reform which was initiated in 2009 for elective hip replacement surgery in Stockholm, Sweden. The reform involved patient choice of provider, free establishment of new providers and a bundled payment model. The study aimed to examine its effects on hip replacement surgery quality as captured by patient reported outcome measures (PROMs) of health gain (as indicated by the EQ-5D index and a visual analogue scale (VAS)), pain reduction (VAS) and patient satisfaction (VAS) one and six years after the surgery. Methods Using patient-level data collected from multiple national registers, we applied a quasi-experimental research design. Data were collected for elective primary total hip replacements that were carried out between 2008 and 2012, and contain information on patient demography, the surgery and PROMs at baseline and at one- and six-years follow-up. In total, 36,627 observations were included in the analysis. First, entropy balancing was applied in order to reduce differences in observable characteristics between treatment groups. Second, difference-in-difference analyses were conducted to eliminate unobserved time-invariant differences between treatment groups and to estimate the causal treatment effects. Results The entropy balancing was successful in creating balance in all covariates between treatment groups. No significant effects of the reform were found on any of the included PROMs at one- and six-years follow-up. The sensitivity analyses showed that the results were robust. Conclusions Competition and bundled payment had no effects on the quality of hip replacement surgery as captured by post-surgery PROMs of health gain, pain reduction and patient satisfaction. The study provides important insights to the limited knowledge on the effects of competition and economic incentives on PROMs.

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