April 2020


Criteria and Process for Initiating and Developing an ISPOR Good Practices Task Force Report

Malone, D., Ramsy, S., Patrick, D., Johnson, F.R., Mullins, C.D., Roberts, M., Wilke, R., and Marshal, D.
Value in Health. 2020;23(4):409-415.

ABSTRACT
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR)’s “Good Practices Task Force” reports are highly cited, multistakeholder perspective expert guidance reports that reflect international standards for health economics and outcomes research (HEOR) and their use in healthcare decision making. In this report, we discuss the criteria, development, and evaluation/consensus review and approval process for initiating a task force. The rationale for a task force must include a justification, including why this good practice guidance is important and its potential impact on the scientific community. The criteria include: (1) necessity (why is this task force required?); (2) a methodology-oriented focus (focus on research methods, approaches, analysis, interpretation, and dissemination); (3) relevance (to ISPOR’s mission and its members); (4) durability over time; (5) broad applicability; and 6) an evidence-based approach. In addition, the proposal must be a priority specifically for ISPOR.

 

Is There a Future for Value-Based Contracting?

Cohen, J.
Value in Health. 2020;23(4):416-417.

ABSTRACT
Value in healthcare is measured in terms of the patient out-comes achieved per dollars spent. As such, when payers and policymakers measure the output of healthcare systems, it is not the volume of services delivered that matters, but rather the outcomes. In light of this, there has been an uptick in interest in value- or outcomes-based contracts. These contracts are supposed to reflect pay-for-performance arrangements, which reimburse for the value a technology or health service adds when it achieves a certain level of improvement in a patient's condition.

Value-based payment mechanisms have been implemented across healthcare sectors. Such mechanisms include alternative payment models for physician reimbursement, hospital value-based purchasing, and value-based arrangements (VBAs) between payers and pharmaceutical manufacturers.1–4VBAs aim to promote greater patient access to effective, but often costly, biopharmaceutical therapies by linking reimbursement, coverage of,or payment for a product to clinical or utilization outcomes.

In the case of biopharmaceuticals, drug makers share with payers in the risk of their drugs’ success or failure (hence the expression“risk-sharing arrangements”).

 

Managed Entry Agreements: Policy Analysis From the European Perspective

Dabbous, M., Chachoua, L., Caban, A., and Toumi, M.
Value in Health. 2020;23(4):425-433.

BACKGROUND
Mounting pressures on the healthcare system, such as budget constraints and new, costly health technologies reaching the market, have pushed payers and manufacturers to engage in managed entry agreements (MEAs) to address uncertainty and facilitate market access.

OBJECTIVE
This study was conducted to illustrate the current landscape of MEAs in Europe and to analyze the main hurdles they face in implementation, providing a policy perspective.

METHODS
We conducted a health policy analysis based on a literature review and described the emergence, classification, current use, and implementation obstacles of MEAs in Europe.

RESULTS
Throughout Europe, uncertainty and high prices of health technologies have pushed stakeholders towards MEAs. Two main types of MEAs were applied heavily, finance-based agreements (FBAs) and performance-based agreements, including individual performance-based agreements and coverage with evidence development (CED). Service-based agreements have not been as heavily considered so far, yet are increasingly used. Many European countries are turning to CEDs to address uncertainty and facilitate market access while negotiating the pricing and reimbursement rates of products. Despite the interest in CEDs, European countries have moved toward FBAs due to the complexities and burdens associated with PBAs.

CONCLUSIONS
Ultimately, in Europe, with the exception of Italy, where MEAs have proven to be inefficient, MEAs are predominantly FBAs dedicated to addressing cost containment from payers’ perspective and external reference pricing from the manufacturers’ perspective. It has been speculated that MEAs will disappear in the medium-term as they are counterproductive for extending patient access and emergence of innovation. To inform value-based decision making and allow early access to innovative medicines, CEDs should be revisited.

Using the Choice Sequence in Time Trade-Off as Discrete Choices: Do the Two Stories Match?

Augestad, L.A., Rand, K., Luo, N., and Barra, M.
Value in Health. 2020;23(4):487-494.

OBJECTIVE
The EQ-5D-5L valuation protocol recommends combining time trade-off (TTO) and discrete choice experiments (DCEs). DCEs that include a duration attribute (DCE ) allow modeling on the quality-adjusted life-year scale. Because the choice sequence in a TTO can be construed as a series of DCE , we used data from a single TTO study to investigate the extent to which DCE values match TTO values when based on identical preferences.

METHODS
In a TTO design in which a fixed set of choices were administered without termination at preference indifference, 202 individuals each valued 10 EQ-5D health states. From identified indifference points, we estimated three sets of TTO values: (i) plotting means and (ii) applying censored regressions at −1 and 1. Using all strict preferences, we (iii) estimated DCE values with a logit model and a bootstrap procedure.

RESULTS
Estimated DCE and TTO values agreed well at the severe end of the quality-adjusted life-year scale, but with decreasing severity, DCE values were higher than TTO-values, with the difference peaking at 0.37 for the mildest health state. Left-censoring TTO values at −1 worsen the agreement for the worst health states and did not affect health states. Right censoring at 1 improved the agreement for mild states.

CONCLUSIONS
TTO and the DCE values estimated from the same preference data diverged, with increasing difference for milder health states. Although the values converged when applying censored regression at +1, we question the validity of this adjustment.


May 2020


Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the ISPOR Personalized Precision Medicine Special Interest Group

Faulkner, E., et al.
Value in Health. 2020;23(5):529-539.

ABSTRACT
Precision medicine is a dynamic area embracing a diverse and increasing type of approaches that allow the targeting of new medicines, screening programs or preventive healthcare strategies, which include the use of biologic markers or complex tests driven by algorithms also potentially taking account of patient preferences. The International Society for Pharmacoeconomics and Outcome Research expanded its current work around precision medicine to (1) describe the evolving paradigm of precision medicine with examples of current and evolving applications, (2) describe key stakeholders perspectives on the value of precision medicine in their respective domains, and (3) define the core factors that should be considered in a value assessment framework for precision medicine. With the ultimate goal of improving health of well-defined patient groups, precision medicine will affect all stakeholders in the healthcare system at multiple levels spanning the individual perspective to the societal perspective. For an efficient, timely and practical precision medicine value assessment framework, it will be important to address these multiple perspectives through building consensus among the stakeholders for robust procedures and measures of value aspects, including performance of precision mechanism; aligned reimbursement processes of precision mechanism and subsequent treatment; transparent expectations for evidence requirements and study designs adequately matched to the intended use of the precision mechanism and to the smaller target patient populations; recognizing the potential range of value-generation such as ruling-in and ruling-out decisions.

 

Competing Views on the English EQ-5D-5L Valuation Set

Norman, R., Olsen, J.A.Value in Health. 2020;23(5):574-575.

ABSTRACT

This issue of Value in Health presents 2 papers with alternative views concerning the UK valuation study of the 5-level EuroQol 5-dimensional questionnaire (EQ-5D-5L). Recent guidance from the National Institute for Health and Care Excellence has recommended that the current value set be replaced and that the 3-level EuroQol 5-dimensional questionnaire is the preferred instrument until this happens. If studies have collected the EQ-5D-5L, then a mapping should be used, in particular that of Van Hout et al.

The EQ-5D-5L valuation study was published in Health Economics in 2018 and has been widely cited in health economic evaluation, related studies conducted in other countries, and methodological work in the health state valuation field. A report conducted by a team at the Policy Research Unit in Economic Methods of Evaluation in Health and Social Care Interventions (EEPRU) raised a series of important issues with the conduct and interpretation of the original valuation study. The paper by Hernández-Alava et al in this issue distills the findings from the EEPRU research report. Then, the EQ-5D-5L valuation study team respond to the concerns raised by the EEPRU team. Many of the issues raised in the EEPRU report have been discussed extensively in recent years. We hope that publishing a condensed form of both the concerns raised in the EEPRU report and the response from the EQ-5D-5L valuation study team will elucidate and promote further engagement with issues of major relevance in this particular case. We also believe that public deliberation informed by the scientific literature provides a means of informing future work in the area using not only the EQ-5D-5L but also other instruments developing a valuation algorithm.

How Are Incremental Cost-Effectiveness, Contextual Considerations, and Other Benefits Viewed in Health Technology Assessment Recommendations in the United States?

Trenaman, L., Pearson, S.D., and Hoch, J.S.
Value in Health. 2020;23(5):576-584.

OBJECTIVES
To review assessments from the Institute for Clinical and Economic Review (ICER) and describe how cost-effectiveness, other benefits or disadvantages, and contextual considerations affect Council members’ assessments of value.

METHODS
Assessments published by the ICER between December 2014 and April 2019 were reviewed. Data on the assessment, intervention, results from cost-effectiveness analyses, and Council members’ votes were extracted. Voting data were examined using bar charts and radar plots. Spearman’s correlations between the number of votes for other benefits and contextual considerations were estimated. Two case studies (tisagenlecleucel and voretigene neparvovec) explored the relationship between different aspects of value and the vote.

RESULTS
Thirty-one ICER assessments were reviewed, which included 51 value votes and 17 votes on other benefits and contextual considerations. On average, interventions with lower cost-effectiveness ratios received a higher proportion of high and intermediate value votes; however, there was heterogeneity across assessments. Of other benefits or disadvantages, having a novel mechanism of action received the most votes (n = 138), and reducing health disparities received the fewest (n = 24). Of contextual considerations, treating a condition that has a severe impact on length and quality of life received the most votes (n = 164). There was a strong positive correlation between votes for reduced caregiver/family burden and improving return to work/productivity (ρ = 0.88, P .05). Two case studies highlighted that factors beyond cost-effectiveness can lead to lower (tisagenlecleucel) or higher (voretigene neparvovec) assessments of value.

CONCLUSION
Council members’ judgments about the value of interventions are influenced by other benefits or disadvantages and contextual considerations but anchored by cost-effectiveness.

Informing Patient-Centered Care Through Stakeholder Engagement and Highly Stratified Quantitative Benefit–Harm Assessments

Aschmann, H.E., et al.
Value in Health. 2020;23(5):616-624.

OBJECTIVES
In a previous project aimed at informing patient-centered care for people with multiple chronic conditions, we performed highly stratified quantitative benefit–harm assessments for 2 top priority questions. In this current work, our goal was to describe the process and approaches we developed and to qualitatively glean important elements from it that address patient-centered care.

METHODS
We engaged patients, caregivers, clinicians, and guideline developers as stakeholder representatives throughout the process of the quantitative benefit–harm assessment and investigated whether the benefit–harm balance differed based on patient preferences and characteristics (stratification). We refined strategies to select the most applicable, valid, and precise evidence.

RESULTS
Two processes were important when assessing the balance of benefits and harms of interventions: (1) engaging stakeholders and (2) stratification by patient preferences and characteristics. Engaging patients and caregivers through focus groups, preference surveys, and as co-investigators provided value in prioritizing research questions, identifying relevant clinical outcomes, and clarifying the relative importance of these outcomes. Our strategies to select evidence for stratified benefit–harm assessments considered consistency across outcomes and subgroups. By quantitatively estimating the range in the benefit–harm balance resulting from true variation in preferences, we clarified whether the benefit–harm balance is preference sensitive.

CONCLUSION
Our approaches for engaging patients and caregivers at all phases of the stratified quantitative benefit–harm assessments were feasible and revealed how sensitive the benefit–harm balance is to patient characteristics and individual preferences. Accordingly, this sensitivity can suggest to guideline developers when to tailor recommendations for specific patient subgroups or when to explicitly leave decision making to individual patients and their providers.


June 2020


Defining Patient Engagement in Research: Results of a Systematic Review and Analysis: Report of the ISPOR Patient-Centered Special Interest Group

Harrington, R., et al.
Value in Health. 2020;23(6):677-688.

OBJECTIVES
Lack of clarity on the definition of “patient engagement” has been highlighted as a barrier to fully implementing patient engagement in research. This study identified themes within existing definitions related to patient engagement and proposes a consensus definition of “patient engagement in research.”

METHODS
A systematic review was conducted to identify definitions of patient engagement and related terms in published literature (2006-2018). Definitions were extracted and qualitatively analyzed to identify themes and characteristics. A multistakeholder approach, including academia, industry, and patient representation, was taken at all stages. A proposed definition is offered based on a synthesis of the findings.

RESULTS
Of 1821 abstracts identified and screened for eligibility, 317 were selected for full-text review. Of these, 169 articles met inclusion criteria, from which 244 distinct definitions were extracted for analysis. The most frequently defined terms were: “patient-centered” (30.5%), “patient engagement” (15.5%), and “patient participation” (13.4%). The majority of definitions were specific to the healthcare delivery setting (70.5%); 11.9% were specific to research. Among the definitions of “patient engagement,” the most common themes were “active process,” “patient involvement,” and “patient as participant.” In the research setting, the top themes were “patient as partner,” “patient involvement,” and “active process”; these did not appear in the top 3 themes of nonresearch definitions.

CONCLUSION
Distinct themes are associated with the term “patient engagement” and with engagement in the “research” setting. Based on an analysis of existing literature and review by patient, industry, and academic stakeholders, we propose a scalable consensus definition of “patient engagement in research.”

 

Research and Policy Impact of Trials Published by the UK National Institute of Health Research (2006-2015)

Carroll, C., Tattersal, A. 
Value in Health. 2020;23(6):727-733.

OBJECTIVES
Health technology assessment aims to inform and support healthcare decision making, and trials are part of that process. The purpose of this study was to measure the impact of a sample of trials in a meaningful but robust fashion.

METHODS
All randomized controlled trials funded and published by the UK National Institute of Health Research in the Health Technology Assessment journal series and other peer-reviewed journals were identified for 2006 to 2015. Citation analysis was performed for all trials, and quantitative content analysis was undertaken on a purposive sample to determine whether impact could be categorized as “instrumental” (ie, having a clear influence on key research and policy publications).

RESULTS
The search identified 133 relevant trials. The citation rate per trial was 102.97. Of the 133 trials, 129 (98%) were cited in 1 or more systematic reviews or meta-analyses (mean per trial = 7.18, range = 0-44). Where they were cited, the trials were used in some form of synthesis 63% of the time. Ninety-one of the 133 (68%) trials were found to be cited in 1 or more guidance or policy document (mean per trial = 2.75, range = 0-26) and had an instrumental influence 41% of the time. The publication of these trials’ results in journals other than the Health Technology Assessment journal appears to enhance the discoverability of the trial data. proved to be very useful in identifying unique policy and guidance documents.

CONCLUSIONS
These trials have impressive citation rates, and a sizeable proportion are certainly being used in key publications in a genuinely instrumental manner.

Computing the Expected Value of Sample Information Efficiently: Practical Guidance and Recommendations for Four Model-Based Methods 

Kunst, N., et al.
Value in Health. 2020;23(6):734-742.

ABSTRACT
Value of information (VOI) analyses can help policy makers make informed decisions about whether to conduct and how to design future studies. Historically a computationally expensive method to compute the expected value of sample information (EVSI) restricted the use of VOI to simple decision models and study designs. Recently, 4 EVSI approximation methods have made such analyses more feasible and accessible. Members of the Collaborative Network for Value of Information (ConVOI) compared the inputs, the analyst’s expertise and skills, and the software required for the 4 recently developed EVSI approximation methods. Our report provides practical guidance and recommendations to help inform the choice between the 4 efficient EVSI estimation methods. More specifically, this report provides: (1) a step-by-step guide to the methods’ use, (2) the expertise and skills required to implement the methods, and (3) method recommendations based on the features of decision-analytic problems.

 

Acceptability and Validity of the EQ-5D in Patients Living With Dementia

Michalowsky, B., et al.  
Value in Health. 2020;23(6):760-767.

OBJECTIVES
To assess the acceptability and validity of the 3 levels of the EQ-5D (EQ-5D-3L) compared with the Quality of Life in Alzheimer’s Diseases (QoL-AD) in patients living with dementia.

METHODS
The analysis was based on 560 dyads of persons with dementia and their caregivers of the multicenter observational study of dementia care networks in Germany (DemNet-D). Health-related quality of life was assessed by face-to-face interviews using the EQ-5D-3L (self-rating) and the QoL-AD (self- and proxy-rating). The number of missing values, the score ranges (observed vs possible range) and the floor and ceiling effects were used to assess the acceptability. We used one-way analyses of variance and multivariate linear regression models to evaluate the discriminative ability. The convergent validity was assessed using Spearman's correlation coefficient (r ) and multivariate regression models.

RESULTS
The EQ-5D index had a higher response rate (89% vs 84%) and a comparable floor (>1%) but a higher ceiling effect (18% vs >1%) compared with the QoL-AD. Both measures can significantly differentiate between different stages of general health, instrumental activities of daily living, and depression. The EQ-5D index and the visual analog scale self-rating scores strongly correlated with the QoL-AD self-rating (r = 0.644 and 0.553, respectively) but not with the proxy-rating score (r = 0.314 and r = 0.170, respectively), which was confirmed by multivariate regression analyses.

CONCLUSIONS
The results satisfy acceptability, discriminative ability, and convergent validity for moderately cognitively and functionally impaired patients living with dementia. The EQ-5D-3L performed comparably with the QoL-AD, and could, therefore, be used in economic evaluations in dementia. The differences between self- and proxy-ratings should be evaluated and considered in the interpretation of health-related quality of life scores.

An Investigation of the Stability of Patients’ Treatment Preferences Over the Course of a Clinical Trial

Allanson, P., et al.  
Value in Health. 2020;23(6):760-767.

OBJECTIVES
The usefulness of discrete choice experiments (DCEs) to inform clinical guidelines rests on the assumption that patients facing the same treatment choice at different points in time will express the same preferences. This study provides the first investigation, to our knowledge, to specifically focus on the stability of patients’ treatment preferences over the course of a clinical trial.

METHODS
The same DCE was completed by participants at baseline and final post-treatment assessment in a trial of the efficacy of alternative topical treatments for actinic keratosis as a means for the prevention of skin cancer. The study assesses both the consistency of stated treatment choices and the stability of population-level preference parameter estimates and analyzes how the former is influenced by design aspects of the DCE.

RESULTS
No evidence was found of population-level preference parameter instability over the course of the trial despite only a moderate strength of choice consistency. Choice consistency is negatively related to task difficulty with weak evidence of the existence of ordering effects over the sequence of choice tasks.

CONCLUSIONS
The results provide no evidence that the timing of a DCE within a clinical trial significantly influences population-level treatment preference estimates.

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