Kristensen FB, Husereau D, Huić M, Drummond M, Berger ML, Bond K, Augustovski F, Booth A, Bridges JFP, Grimshaw J, IJzerman MJ, Jonsson E, Ollendorf DA, Rüther A, Siebert U, Sharma J, Wailoo A.
Value in Health. 2019;22(1):13-20.
The systematic use of evidence to inform healthcare decisions, particularly health technology assessment (HTA), has gained increased recognition. HTA has become a standard policy tool for informing decision makers who must manage the entry and use of pharmaceuticals, medical devices, and other technologies (including complex interventions) within health systems, for example, through reimbursement and pricing. Despite increasing attention to HTA activities, there has been no attempt to comprehensively synthesize good practices or emerging good practices to support population-based decision-making in recent years. After the identification of some good practices through the release of the ISPOR Guidelines Index in 2013, the ISPOR HTA Council identified a need to more thoroughly review existing guidance. The purpose of this effort was to create a basis for capacity building, education, and improved consistency in approaches to HTA-informed decision-making. Our findings suggest that although many good practices have been developed in areas of assessment and some other key aspects of defining HTA processes, there are also many areas where good practices are lacking. This includes good practices in defining the organizational aspects of HTA, the use of deliberative processes, and measuring the impact of HTA. The extent to which these good practices are used and applied by HTA bodies is beyond the scope of this report, but may be of interest to future researchers.
Stolk E, Ludwig K, Rand K, van Hout B, Ramos-Goñi JM.
Value in Health. 2019;22(1):23-30.
A standardized 5-level EuroQol 5-dimensional questionnaire (EQ-5D-5L) valuation protocol was first used in national studies in the period 2012 to 2013. A set of problems encountered in this initial wave of valuation studies led to the subsequent refinement of the valuation protocol. To clarify lessons learned and how the protocol was updated when moving from version 1.0 to the current version 2.1 and 2.0, this article will (1) present the challenges faced in EQ-5D-5L valuation since 2012 and how these were resolved and (2) describe in depth a set of new challenges that have become central in currently ongoing research on how EQ-5D-5L health states should be valued and modeled.
Yang F, Devlin N, Luo N.
Value in Health. 2019;22(1):45-49.
To explore how the use of EQ-5D-5L value set and crosswalk from EQ-5D-5L to EQ-5D-3L (and use of 3L value set) would affect cost-effectiveness analysis results for England and six other countries (Canada, the Netherlands, China, Japan, South Korea, and Singapore).
Individual-level utilities derived from primary 5L data using both value set (5L) and crosswalk (c5L) approaches were applied to three Markov models assessing the cost-effectiveness of hemodialysis (HD) and peritoneal dialysis (PD) for end-stage renal disease (ESRD) patients to estimate incremental quality-adjusted life years (QALYs). The mathematic functions between incremental QALY and utility were derived.
5L- and c5L-based incremental QALYs were similar in the model for non-diabetic patients (range: 1.910–2.149, 1.922–2.121). 5L tends to generate more incremental QALYs than c5L in the model for diabetic patients (range: 1.454–1.633, 1.365–1.568) but fewer incremental QALYs in the model for all ESRD patients (range: 0.290–0.480, 0.315–0.493).
The value set and crosswalk approaches may not be used interchangeably in economic evaluation when EQ-5D-5L data are used to estimate utilities. Results of cost-effectiveness analysis using Markov models may be affected by both absolute utilities and their differences.
Johnson FR, Yang JC, Reed SD.
Value in Health. 2019;22(2):157-160.
To develop a tool for testing internal validity of discrete choice experiment (DCE) data, deploy the program, and collect summary test results from a sample of active health researchers to demonstrate the practical utility of the tool in a wide range of health applications.
A previously developed Gauss program had been in use for testing internal validity. The program was translated to MATLAB and adapted, compiled, and deployed. Sixty-seven authors who had coauthored one or more published DCE studies between 2013 and 2016 were contacted by email; provided access to the tool, instructions, and an example data file; and invited to submit test summaries for tabulation.
Twenty-one researchers from 10 countries contributed test results from a total of 55 DCE data sets. Fifty-one studies included at least two out of a possible six tests. Attribute dominance was the most common test, and stability had the highest failure incidence. Only three summaries included a transitivity test, and no failures were detected.
It was possible to evaluate multiple internal validity checks for most data sets even when the experimental design did not explicitly include tests. Nevertheless, internal validity is rarely reported. Free availability of the tool for testing data quality could improve both reporting and more careful design of DCE studies to help validate and interpret stated preference data.
Clément V, Raimond V.
Value in Health. 2019;22(2):220-224.
This paper constitutes the first attempt to draw lessons from the recent uptake of health economic evaluation of innovative drugs in the French regulatory framework.
Taking the example of new direct-acting antivirals against hepatitis C virus, the paper asks whether and how the cost-effectiveness (CE) opinions issued by the French National Health Authority improve the information available to support the pricing decisions.
The analysis compares the assessment of these drugs based on three different sources: CE opinions, clinical opinions, and the published cost-utility analyses (CUA) available in the literature and identified through a systematic review.
The results show that CE opinions bring to the fore three issues prone to impact the incremental cost utility ratio and those were not available to the decision maker through clinical opinions or published CUA: the stage of treatment initiation, the modeling of the disease progression, and the uncertainty around the efficacy rates.
France has introduced the criterion of the cost per QALY gained in the pricing and regulation of innovative pharmaceuticals since 2013. Our analysis shows that the use of CUA does enhance the information available to the decision makers on the value of the treatments.
Atkinson TM, Schwartz CE, Goldstein L, Garcia I, Storfer DF, Li Y, Zhang J, Bochner BH, Rapkin BD.
Value in Health. 2019;22(2):225-230.
Patient response burden is often raised as a human subject concern in consideration of the length or complexity of patient-reported outcome (PRO) instruments used in oncology.
To quantify patient response burden and identify its predictive factors.
Data were collected presurgically during a prospective trial that used a comprehensive symptom and health-related quality-of-life (HRQOL) PRO assessment. A subset of patients also completed HRQOL interviews. Response burden was captured using an internally developed six-item instrument. Demographic and clinical characteristics as well as HRQOL scores were examined as potential predictors using hierarchical regression. Response burden was used to predict participant dropout at the first follow-up interval.
A total of 275 patients (mean age 67.5 years; 23.6% female) completed surveys (n = 126) or surveys in addition to interviews (n = 149). Patients experienced low response burden (mean 12.19 ± 11.65). Repetitive questions were identified by 60 patients (21.8%), whereas 31.6% indicated that additional information should be gathered; 35 patients (12.7%) identified repetitive questions and expressed a desire for additional items. Low self-reported cognitive function was a significant predictor of higher response burden (β = −0.20; t(270) = −3.38; P = 0.01; model-adjusted R = 0.04). Response burden was not a significant predictor of study dropout.
Despite completing a large battery of PRO measures and interviews, patients reported minimal response burden, with nearly one-third expressing that more questions should have been asked. Patients with lower cognitive function are more likely to report higher response burden when completing PRO measures. Further examination of patient characteristics related to response burden may reveal useful pathways for tailoring patient-centered interventions.
Huo J, Aloia TA, Xu Y, Chung TH, Sheu T, Tina Shih YC.
Value in Health. 2019;22(3):284-292.
For patients with hepatocellular carcinoma (HCC) not eligible for surgical resection, radiofrequency ablation (RFA) is a promising technique that reduces the risk of disease progression.
To evaluate whether the trend of image guidance for RFA is moving toward the more expensive computed tomography (CT) technology and to determine the clinical benefits of CT guidance over the ultrasound (US) guidance.
A cohort of 463 patients was identified from the Surveillance, Epidemiology, and End Results and Medicare–linked database. The temporal trends in use of image guidance were assessed using the Cochrane–Armitage test. The associations between modality of image guidance and survival, complications, and costs were assessed using the Cox regression model, the logistic regression model, and the generalized linear model, respectively.
The use of CT-guided RFA increased sharply, from 20.7% in 2002 to 75.9% in 2011. Compared with CT-guided RFA, those who received US-guided RFA had comparable risk of periprocedural and delayed postprocedural complications. Stratified analyses by tumor size also showed no statistically significant difference. In adjusted survival analysis, no statistically significant difference was observed in overall and cancer-specific survival. Nevertheless, the cost of CT-guided RFA ($2847) was higher than that of US-guided RFA ($1862).
Despite its rapid adoption over time, CT-guided RFA incurred higher procedural costs than US-guided RFA but did not significantly improve postprocedural complications and survival. Echoing the American Board of Internal Medicine’s Choosing Wisely campaign and the American Society of Clinical Oncology’s Value of Cancer Care initiative, findings from our study call for critical evaluation of whether CT-guided RFA provides high-value care for patients with HCC.
Holleman MS, Uyl-de Groot CA, Goodall S, van der Linden N.
Value in Health. 2019;22(3):322-331.
Risk-sharing arrangements (RSAs) can be used to mitigate uncertainty about the value of a drug by sharing the financial risk between payer and pharmaceutical company. We evaluated the projected impact of alternative RSAs for non–small cell lung cancer (NSCLC) therapies based on real-world data.
Data on treatment patterns of Dutch NSCLC patients from four different hospitals were used to perform “what-if” analyses, evaluating the costs and benefits likely associated with various RSAs. In the scenarios, drug costs or refunds were based on response evaluation criteria in solid tumors (RECIST) response, survival compared to the pivotal trial, treatment duration, or a fixed cost per patient. Analyses were done for erlotinib, gemcitabine/cisplatin, and pemetrexed/platinum for metastatic NSCLC, and gemcitabine/cisplatin, pemetrexed/cisplatin, and vinorelbine/cisplatin for nonmetastatic NSCLC.
Money-back guarantees led to moderate cost reductions to the payer. For conditional treatment continuation schemes, costs and outcomes associated with the different treatments were dispersed. When price was linked to the outcome, the payer's drug costs reduced by 2.5% to 26.7%. Discounted treatment initiation schemes yielded large cost reductions. Utilization caps mainly reduced the costs of erlotinib treatment (by 16%). Given a fixed cost per patient based on projected average use of the drug, risk sharing was unfavorable to the payer because of the lower than projected use. The impact of RSAs on a national scale was dispersed.
For erlotinib and pemetrexed/platinum, large cost reductions were observed with risk sharing. RSAs can mitigate uncertainty around the incremental cost-effectiveness or budget impact of drugs, but only when the type of arrangement matches the setting and type of uncertainty.
Feng Y, Devlin N, Bateman A, Zamora B, Parkin D.
Value in Health. 2019;22(3):355-361.
The distribution of EQ-5D-3L values (health state profiles, weighted by value sets) often shows two distinct groups, arising from both the distribution of profiles and the characteristics of value sets. To date, there is little evidence about the distribution of EQ-5D-5L values.
To explore the distribution of EQ-5D-5L profiles; to compare the distributions of EQ-5D-5L values arising from the English value set (EVS) and a ‘mapped’ value set (MVS); and to develop further the methods used to investigate clustering within EQ-5D data.
We obtained data from Cambridgeshire Community Services NHS Trust containing EQ-5D-5L profiles before treatment for three patient groups: community rehabilitation (N=6919); musculoskeletal physiotherapy (N=19999); and specialist nursing services (N=3366). Values were calculated using the EVS and MVS. Clusters were examined using the k-means method and Calinski–Harabasz pseudo-F index stopping rule.
We found no evidence for clustering of EQ-5D-5L values arising from the classification system and no strong or consistent evidence of clustering arising from the EVS. There was clearer evidence of clustering using the MVS, with two being the optimal number of clusters. The clusters that were found for the EVS were very different from the MVS clusters.
Unlike the EQ-5D-3L, clustering of EQ-5D-5L values does not seem to be driven by clustering of its profile. This suggests the EQ-5D-5L is superior in that it is less likely to generate artefactual clusters – however, clusters may still result from using value sets such as MVS that have the tendency to generate them.