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

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.

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.    

scholarly journals Supporting Formulary Decisions: The Discovery of New Facts or Constructed Evidence?

2016 ◽  
Vol 7 (2) ◽  
Author(s):  
Paul C Langley
Keyword(s):  
Cost Effectiveness ◽  
Lifetime Cost ◽  
Cost Effective ◽  
Evidence Base ◽  
Normal Science ◽  
Critical Question ◽  
Reference Case ◽  
Life Years ◽  
The One ◽  
The Uk

A critical question, given the growing importance of more targeted therapies to support personalized and precision medicine, is the credibility of the evidence base to support formulary decisions and pricing. On the one hand, for those who subscribe to the reference case model of the National Institute of Health and Care Excellence (NICE) in the UK, the decision rests upon the creation of modeled or simulated imaginary worlds and the application of threshold willingness-to-pay cost-per-QALY thresholds. On the other hand, for those who subscribe to the standards of normal science, the decision rests upon the ability to evaluate competing claims, both clinical and cost-effective, in a timeframe that is meaningful to a formulary committee. If we subscribe to the scientific method where the focus is on the discovery of new facts, untestable claims for clinical benefit and cost-effectiveness, such as created claims for lifetime cost per-quality-adjusted discounted life years (QALYs), are properly relegated to the category of pseudoscience. We have no idea, and will never know, whether the claims are right or even if they are wrong. If formulary decisions are to respect the standards of normal science then there has to be a commitment to claims evaluation. A willingness to accept new products provisionally, subject to an agreed protocol to support the evaluation of clinical and cost-effectiveness claims. This dichotomy between the standards of normal science and pseudoscience is explored in the context of published claims for cost-effectiveness and recommendations for product pricing in the US.   Type: Commentary

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

2019 ◽  
Vol 10 (3) ◽  
pp. 11
Author(s):  
Paul Langley
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
New Products ◽  
Evidence Base ◽  
Cost Utility ◽  
Decision Makers ◽  
Normal Science ◽  
Market Place ◽  
Article Type ◽  
Health Decision

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

scholarly journals National Bariatric Surgery Registries: an International Comparison

2021 ◽  
Author(s):  
Erman O. Akpinar ◽  
Perla J. Marang- van de Mheen ◽  
Simon W. Nienhuijs ◽  
Jan Willem M. Greve ◽  
Ronald S. L. Liem
Keyword(s):  
Standard Of Care ◽  
Population Based ◽  
Substantial Agreement ◽  
Perfect Agreement ◽  
Moderate Agreement ◽  
Real World Evidence ◽  
International Collaborations ◽  
Global Registry ◽  
Registry Report

Abstract Introduction Pooling population-based data from all national bariatric registries may provide international real-world evidence for outcomes that will help establish a universal standard of care, provided that the same variables and definitions are used. Therefore, this study aims to assess the concordance of variables across national registries to identify which outcomes can be used for international collaborations. Methods All 18 countries with a national bariatric registry who contributed to The International Federation for the Surgery of Obesity and Metabolic Disorders (IFSO) Global Registry report 2019 were requested to share their data dictionary by email. The primary outcome was the percentage of perfect agreement for variables by domain: patient, prior bariatric history, screening, operation, complication, and follow-up. Perfect agreement was defined as 100% concordance, meaning that the variable was registered with the same definition across all registries. Secondary outcomes were defined as variables having “substantial agreement” (75–99.9%) and “moderate agreement” (50–74.9%) across registries. Results Eleven registries responded and had a total of 2585 recorded variables that were grouped into 250 variables measuring the same concept. A total of 25 (10%) variables have a perfect agreement across all domains: 3 (18.75%) for the patient domain, 0 (0.0%) for prior bariatric history, 5 (8.2%) for screening, 6 (11.8%) for operation, 5 (8.8%) for complications, and 6 (11.8%) for follow-up. Furthermore, 28 (11.2%) variables have substantial agreement and 59 (23.6%) variables have moderate agreement across registries. Conclusion There is limited uniform agreement in variables across national bariatric registries. Further alignment and uniformity in collected variables are required to enable future international collaborations and comparison. Graphical abstract

“Femininity without Feminism”

2015 ◽  
Vol 43 (3) ◽  
pp. 249-272 ◽  
Author(s):  
Soo-Hyun Mun
Keyword(s):  
Gender Identity ◽  
Social Status ◽  
Gender Politics ◽  
Unintended Consequences ◽  
The Other ◽  
Korean Society ◽  
Feminist Politics ◽  
Identity Based ◽  
The One ◽  
Status Of Women

This article contributes to the debate between the merits of the ‘politics of presence’ versus the ‘politics of ideas’ by examining the case of the first female Korean president, Park Geun-Hye. On the one hand, Park did not represent ‘the ideas’ of feminist politics. While her gender identity was widely propagated and accepted, it did not transform into deliberate identity-based politics. On the other hand, she contributed to the elevation of women’s social status through various unintended consequences, although Park’s ‘femininity without feminism’ inevitably led to the negligence of gender politics in her government. Indeed, Park’s existence, rather than her intention, stimulated the debate on the role and status of women in Korean society and enabled the rise of a number of first females in various sectors. In sum, the ‘politics of presence’ was triggered even without overt political measures.

scholarly journals Human Creativity and Consciousness: Unintended Consequences of the Brain’s Extraordinary Energy Efficiency?

2020 ◽  
Author(s):  
Tim Palmer
Keyword(s):  
Energy Efficiency ◽  
Human Brain ◽  
Quantum Physics ◽  
Unintended Consequences ◽  
Large Network ◽  
Active Neuron ◽  
Available Energy ◽  
Human Creativity ◽  
The One ◽  
The Brain

It is proposed that both human creativity and human consciousness are (unintended) consequences of the human brain’s extraordinary energy efficiency. The topics of creativity and consciousness are treated separately, though have a common sub-structure. It is argued that creativity arises from a synergy between two cognitive modes of the human brain (which broadly coincide with Kahneman’s Systems 1 and 2). In the first, available energy is spread across a relatively large network of neurons. As such, the amount of energy per active neuron is so small that the operation of such neurons is susceptible to thermal (ultimately quantum decoherent) noise. In the second, available energy is focussed on a small enough subset of neurons to guarantee a deterministic operation. An illustration of how this synergy can lead to creativity with implications for computing in silicon are discussed. Starting with a discussion of the concept of free will, the notion of consciousness is defined in terms of an awareness of what are perceived to be nearby counterfactual worlds in state space. It is argued that such awareness arises from an interplay between our memories on the one hand, and quantum physical mechanisms (where, unlike in classical physics, nearby counterfactual worlds play an indispensable dynamical role) in the ion channels of neural networks. As with the brain’s susceptibility to noise, it is argued that in situations where quantum physics plays a role in the brain, it does so for reasons of energy efficiency. As an illustration of this definition of consciousness, a novel proposal is outlined as to why quantum entanglement appears so counter-intuitive.

scholarly journals Post QALY: Scientific Approaches to Real World Formulary Decision Making

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

Prevention and education evidence review: Gambling-related harm

2021 ◽  
Author(s):  
Margo Hilbrecht ◽  
Sally M. Gainsbury ◽  
Nassim Tabri ◽  
Michael J. A. Wohl ◽  
Silas Xuereb ◽  
...  
Keyword(s):  
At Risk ◽  
Research Evidence ◽  
Grey Literature ◽  
Evidence Base ◽  
Unintended Consequences ◽  
Evidence Based ◽  
Negative Consequences ◽  
Age Cohorts ◽  
Gambling Harm ◽  
Harm Prevention

This report supports an evidence-based approach to the prevention and education objective of the National Strategy to Reduce Harm from Gambling. Applying a public health policy lens, it considers three levels of measures: universal (for the benefit of the whole population), selective (for the benefit of at-risk groups), and indicated (for the benefit of at-risk individuals). Six measures are reviewed by drawing upon a range of evidence in the academic and grey literature. The universal level measures are “Regulatory restriction on how gambling is provided” and “Population-based safer gambling/responsible gambling efforts.” Selective measures focus on age cohorts in a chapter entitled, “Targeted safer gambling campaigns for children, youth, and older adults.” The indicated measures are “Brief internet delivered interventions for gambling,” “Systems and tools that produced actual (‘hard’) barriers and limit access to funds,” and “Self-exclusion.” Since the quantity and quality of the evidence base varied by measure, appropriate review methods were selected to assess publications using a systematic, scoping, or narrative approach. Some measures offered consistent findings regarding the effectiveness of interventions and initiatives, while others were less clear. Unintended consequences were noted since it is important to be aware of unanticipated, negative consequences resulting from prevention and education activities. After reviewing the evidence, authors identified knowledge gaps that require further research, and provided guidance for how the findings could be used to enhance the prevention and education objective. The research evidence is supplemented by consultations with third sector charity representatives who design and implement gambling harm prevention and education programmes. Their insights and experiences enhance, support, or challenge the academic evidence base, and are shared in a separate chapter. Overall, research evidence is limited for many of the measures. Quality assessments suggest that improvements are needed to support policy decisions more fully. Still, opportunities exist to advance evidence-based policy for an effective gambling harm prevention and education plan.

scholarly journals 2020. Impact of Local Antimicrobial Stewardship Guidelines through a Cellphone App to Lower Hospital-acquired MDR Infections in a Private Hospital in Mexico City

2019 ◽  
Vol 6 (Supplement_2) ◽  
pp. S678-S678
Author(s):  
Marco Villanueva Reza ◽  
Lorena Lanz-Zubiria ◽  
Israel Rojas de Ita ◽  
Paulo Castañeda-Méndez ◽  
Esperanza Aleman Aguilar ◽  
...  
Keyword(s):  
Antimicrobial Stewardship ◽  
Private Hospital ◽  
Standard Of Care ◽  
Hospital Staff ◽  
Antimicrobial Drugs ◽  
Hospital Acquired Infection ◽  
Healthcare Program ◽  
Local Patterns ◽  
The One ◽  
Hospital Acquired

Abstract Background Hospital-acquired multi-drug-resistant organisms (HA-MDRO) infections are associated with increased cost, length of stay, morbidity, and mortality. The prevalence of HA-MDRO has been increasing worldwide. We look to describe how the implementation of local guidelines as an app available for tablets or cellphones in our hospital in 2014 decrease the prevalence of HA-MDRO. Methods Local guidelines were implemented as an electronic application available and intended for hospital staff only. This app gives local patterns of resistance for bacteria with treatment recommendations alongside suggested antimicrobial drugs for empiric treatment. Descriptive statistics were performed. Results In 2013, before implementing the app, HA-MDRO were reported in 25% of hospital-acquired infection. After the introduction of the app, compliance in 2014 was 56.7% with HA-MDRO reported in 20%, 2015 use was 60.7% and HA-MDRO in 18%, 2016 compliance 68.7% and HA-MDRO in 16%, 2017 compliance in 73.7% and HA-MDRO in 14%, lastly in 2018 prevalence was 15% of HA-MDRO while use of local guidelines was 78.7%. Different type of infections (VAP, HAP, UTI, SSI, BSI) also decreased while following guidelines recommendations. Conclusion These programs are needed as a part of the healthcare program to control the HA-MDRO morbidity and complications. Simple friendly-user electronic applications such as the one implemented in our hospital led to higher compliance and lower hospital-acquired infections. Antimicrobial stewardship programs and local guidelines should be part of the standard of care of all hospitals. Disclosures All authors: No reported disclosures.

scholarly journals Designing and using incentives to support recruitment and retention in clinical trials: a scoping review and a checklist for design

Trials ◽  
2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Beth Parkinson ◽  
Rachel Meacock ◽  
Matt Sutton ◽  
Eleonora Fichera ◽  
Nicola Mills ◽  
...  
Keyword(s):  
Scoping Review ◽  
Pay For Performance ◽  
Evidence Base ◽  
Unintended Consequences ◽  
Current Evidence ◽  
Incentive System ◽  
Design Issues ◽  
Recruitment And Retention ◽  
Design Choice ◽  
Current Evidence Base

Abstract Background Recruitment and retention of participants are both critical for the success of trials, yet both remain significant problems. The use of incentives to target participants and trial staff has been proposed as one solution. The effects of incentives are complex and depend upon how they are designed, but these complexities are often overlooked. In this paper, we used a scoping review to ‘map’ the literature, with two aims: to develop a checklist on the design and use of incentives to support recruitment and retention in trials; and to identify key research topics for the future. Methods The scoping review drew on the existing economic theory of incentives and a structured review of the literature on the use of incentives in three healthcare settings: trials, pay for performance, and health behaviour change. We identified the design issues that need to be considered when introducing an incentive scheme to improve recruitment and retention in trials. We then reviewed both the theoretical and empirical evidence relating to each of these design issues. We synthesised the findings into a checklist to guide the design of interventions using incentives. Results The issues to consider when designing an incentive system were summarised into an eight-question checklist. The checklist covers: the current incentives and barriers operating in the system; who the incentive should be directed towards; what the incentive should be linked to; the form of incentive; the incentive size; the structure of the incentive system; the timing and frequency of incentive payouts; and the potential unintended consequences. We concluded the section on each design aspect by highlighting the gaps in the current evidence base. Conclusions Our findings highlight how complex the design of incentive systems can be, and how crucial each design choice is to overall effectiveness. The most appropriate design choice will differ according to context, and we have aimed to provide context-specific advice. Whilst all design issues warrant further research, evidence is most needed on incentives directed at recruiters, optimal incentive size, and testing of different incentive structures, particularly exploring repeat arrangements with recruiters.

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