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

scholarly journals Resolving Lingering Problems or Continued Support for Pseudoscience? The ICER Value Assessment Update

2017 ◽  
Vol 8 (4) ◽  
pp. 7
Author(s):  
Paul C. Langley
Keyword(s):  
Evaluation Framework ◽  
Value Assessment ◽  
Assessment Framework ◽  
Health Care Environment ◽  
Care Environment ◽  
Scientific Methods ◽  
The Us ◽  
Evidence Analysis ◽  
The Uk ◽  
Therapy Intervention

The Institute for Clinical and Economic Review (ICER) released its updated value assessment framework in mid-2017. This included refinements to its conceptual structure and modifications to methods of collecting and assessing evidence. Consequent to this release, a number of authors have commented on the updated value framework, addressing the question of whether the latest framework represents a major revision or merely attempts to resolve lingering problems. The purpose of this commentary is twofold: (i) to revisit what are considered to be fundamental flaws in the ICER value assessment framework and (ii) to question whether or not post-release critiques of the value framework address the fundamental weaknesses in the ICER approach: the absence of credible, evaluable and replicable claims for the benefits and harms of a therapy intervention. The commentary argues that while ICER sees the purpose of its value assessment framework as forming ‘the backbone of rigorous, transparent evidence reports’ in placing ‘scientific methods of evidence analysis at the heart of a clearer and more transparent process’ it falls far short of these ideals. Rather, in attempting to replicate in the US health care environment the evaluation framework mandated by the National Institute for Health and Care Excellence (NICE) in the UK, the ICER falls into the trap of generating value claims for product impact that fail to meet the standards of normal science.   Type: Commentary

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 Assessment and Management of Dysphagia in Acute Stroke: An Initial Service Review of International Practice

Geriatrics ◽  
2020 ◽  
Vol 5 (1) ◽  
pp. 4
Author(s):  
Carol A. Fairfield ◽  
David G. Smithard
Keyword(s):  
Acute Phase ◽  
Best Practice ◽  
Evidence Base ◽  
Post Stroke ◽  
Effortful Swallow ◽  
The Us ◽  
Screening Assessment ◽  
The Uk ◽  
Management Of Dysphagia ◽  
The Impact

The international approach to the assessment and management of dysphagia in the acute phase post stroke is little studied. A questionnaire was sent to clinicians in stroke services that explored the current practice in dysphagia screening, assessment, and management within the acute phase post stroke. The findings from four (the UK, the US, Canada, and Australia) of the 22 countries returning data are analysed. Consistent approaches to dysphagia screening and the modification of food and liquid were identified across all four countries. The timing of videofluoroscopy (VFS) assessment was significantly different, with the US utilising this assessment earlier post stroke. Compensatory and Postural techniques were employed significantly more by Canada and the US than the UK and Australia. Only food and fluid modification, tongue exercises, effortful swallow and chin down/tuck were employed by more than fifty percent of all respondents. The techniques used for assessment and management tended to be similar within, but not between, countries. Relationships were found between the use of instrumental assessment and the compensatory management techniques that were employed. The variation in practice that was found, may reflect the lack of an available robust evidence base to develop care pathways and identify the best practice. Further investigation and identification of the impact on dysphagia outcome is needed.

scholarly journals How machine learning is embedded to support clinician decision making: an analysis of FDA-approved medical devices

2021 ◽  
Vol 28 (1) ◽  
pp. e100301
Author(s):  
David Lyell ◽  
Enrico Coiera ◽  
Jessica Chen ◽  
Parina Shah ◽  
Farah Magrabi
Keyword(s):  
Machine Learning ◽  
Decision Making ◽  
Information Processing ◽  
Ejection Fraction ◽  
Medical Devices ◽  
Human Information Processing ◽  
Information Analysis ◽  
Input And Output ◽  
Descriptive Information ◽  
The Us

ObjectiveTo examine how and to what extent medical devices using machine learning (ML) support clinician decision making.MethodsWe searched for medical devices that were (1) approved by the US Food and Drug Administration (FDA) up till February 2020; (2) intended for use by clinicians; (3) in clinical tasks or decisions and (4) used ML. Descriptive information about the clinical task, device task, device input and output, and ML method were extracted. The stage of human information processing automated by ML-based devices and level of autonomy were assessed.ResultsOf 137 candidates, 59 FDA approvals for 49 unique devices were included. Most approvals (n=51) were since 2018. Devices commonly assisted with diagnostic (n=35) and triage (n=10) tasks. Twenty-three devices were assistive, providing decision support but left clinicians to make important decisions including diagnosis. Twelve automated the provision of information (autonomous information), such as quantification of heart ejection fraction, while 14 automatically provided task decisions like triaging the reading of scans according to suspected findings of stroke (autonomous decisions). Stages of human information processing most automated by devices were information analysis, (n=14) providing information as an input into clinician decision making, and decision selection (n=29), where devices provide a decision.ConclusionLeveraging the benefits of ML algorithms to support clinicians while mitigating risks, requires a solid relationship between clinician and ML-based devices. Such relationships must be carefully designed, considering how algorithms are embedded in devices, the tasks supported, information provided and clinicians’ interactions with them.

scholarly journals The Imaginary Worlds of Cure Proportion Modeling: Survivorship and Reference Case Pricing of Transformative Therapies

2019 ◽  
Vol 10 (3) ◽  
pp. 17
Author(s):  
Paul Langley
Keyword(s):  
Incremental Cost ◽  
Business Model ◽  
Fair Value ◽  
Normal Science ◽  
Value Assessment ◽  
Assessment Framework ◽  
Short Term ◽  
Reference Case ◽  
Case Methodology ◽  
Article Type

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

scholarly journals Comparative study of updated Clinical Trial Regulations in India with respect to Australia, Europe, Japan and US

2021 ◽  
Vol 9 (1) ◽  
pp. 48-61
Author(s):  
Devender Kumar ◽  
Shivali Rahi ◽  
Arpana Rana
Keyword(s):  
Clinical Trial ◽  
Medical Devices ◽  
Pharmaceutical Products ◽  
European Medicines Agency ◽  
Pharmaceutical Companies ◽  
Drug Candidates ◽  
Geographical Regions ◽  
The Us ◽  
The World ◽  
Scientific Standards

The national regulatory authorities, such as the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), Central Drugs Standard Control Organization and the Australian Therapeutic Goods Administration (TGA), approve every drug that is prescribed for patients around the world. The process of approval undertaken by pharmaceutical companies for drug candidates consists of a series of phases to ensure the product is safe and effective at treating the disease. In this paper, we will compare the clinical trial regulations of India with respect to Australia, Europe, Japan & USA in order to determine the safety and efficacy of pharmaceutical products like drugs, biologics and medical devices in different geographical regions and to confirm the clinical trials studies follow strict scientific standards.

Machine-Learnt Bias? Algorithmic Decision Making and Access to Criminal Justice

2020 ◽  
Vol 20 (2) ◽  
pp. 99-100
Author(s):  
Malwina Anna Wojcik
Keyword(s):  
Risk Assessment ◽  
Decision Making ◽  
Criminal Justice ◽  
Law Enforcement ◽  
Criminal Justice System ◽  
Justice System ◽  
Crime Mapping ◽  
The Us ◽  
Court Proceedings ◽  
The Uk

The pressure on the criminal justice system in England and Wales is mounting. Recent figures reveal that despite a rise in recorded crime, the number of defendants in court proceedings has been the lowest in 50 years. This indicates a crisis of access to criminal justice. Predictive policing and risk assessment programmes based on algorithmic decision making (ADM) offer a prospect of increasing efficiency of law enforcement, eliminating delays and cutting the costs. These technologies are already used in the UK for crime-mapping and facilitating decisions regarding prosecution of arrested individuals. In the US their deployment is much wider, covering also sentencing and parole applications.

scholarly journals Value Assessment in Cystic Fibrosis: ICER’s Rejection of the Axioms of Fundamental Measurement

2020 ◽  
Vol 11 (2) ◽  
pp. 8
Author(s):  
Paul Langley
Keyword(s):  
Cystic Fibrosis ◽  
Stakeholder Involvement ◽  
Normal Science ◽  
Value Assessment ◽  
Assessment Framework ◽  
Multiattribute Utility ◽  
Public Comment ◽  
A Value ◽  
Article Type ◽  
Utility Scores

One of the features of the ICER stakeholder involvement in the development of ICER evidence reports is the ability for public comment. Unfortunately, and this may just a miscommunication, the replies from ICER to public comments frequently miss the point or fail to provide backup for their claims. The purpose of this commentary is to review ICER’s responses to public comments by the author on the just released final evidence report on cystic fibrosis. The message is quite simple: the ICER value assessment framework lacks credibility. It fails to meet the standards of normal science. This is seen in ICERs apparent ignorance or rejection of the axioms of fundamental measurement which point quite clearly to the mathematical impossibility of creating QALYs from generic multiattribute utility scores. The ICER report also fails standards by creating a model from prior assumptions; there is no logical basis for constructing a value assessment claim. Either ICER should withdraw its value claims or admit the dubious basis on which the model is built, as a duty to its readership.   Article Type: Commentary    

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 Rush to Judgement: Imaginary Worlds and Cost-Outcomes Claims for PCSK9 Inhibitors

2017 ◽  
Vol 8 (2) ◽  
Author(s):  
Paul C Langley
Keyword(s):  
Willingness To Pay ◽  
Decision Makers ◽  
Normal Science ◽  
Pcsk9 Inhibitors ◽  
Ongoing Debate ◽  
Drug Prices ◽  
Price Adjustments ◽  
The Us ◽  
The Uk ◽  
Cost Outcomes

Pricing by the manufacturers of the two PCSK9 inhibitors recently approved by the FDA, evolocumab and alirocumab, has led to an ongoing debate over the value of these interventions in clinical practice. In the US, the Institute for Clinical and Economic Review (ICER) has been in the forefront of those who have argued that, in the context of notional incremental willingness-to-pay thresholds, manufacturers should reduce drug prices substantially. This conclusion has been echoed in a number of other technology assessments, notably in assessments by the National Institute of Health and Care Excellence (NICE) in the UK. At the same time, other evaluations have reported favorably, arguing that at current US prices, the two products meet willingness-to-pay benchmarks. The purpose of this commentary is not to argue for or against current PCSK9 pricing policies but to point out that the case made for possible price adjustments rest upon technology assessments that fail to meet the standards of normal science. Modeled claims that are properly classified as pseudoscience. The claims made are non-evaluable. Formulary committees, rather than accepting these claims at face value, should step back and work with manufacturers to develop claims that are targeted, robust, evaluable and replicable in a timeframe where feedback on PCSK9 outcomes are meaningful to health system decision makers.   Type: Commentary

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