Implementing multi-level network meta-regression for time-to-event outcomes: A case study in relapsed refractory multiple myeloma
Multi-level network meta-regression (ML-NMR) leverages individual patient data (IPD) and aggregate data (AD) from a network of randomized controlled trials (RCTs) to assess the comparative efficacy of multiple treatments, while adjusting for between-study differences. We provide an overview of ML-NMR for time-to-event outcomes and apply it to an illustrative case study, including example R code. (Source: Value in Health)
Source: Value in Health - April 26, 2024 Category: International Medicine & Public Health Authors: Dylan Maciel, Jeroen P. Jansen, Sven L. Klijn, Kevin Towle, Devender Dhanda, Bill Malcolm, Shannon Cope Source Type: research

Treatment effect waning assumptions: A review of NICE Technology Appraisals
Review National Institute of Health and Care Excellence (NICE) technology assessments to gain insights into the implementation of treatment effect waning, whereby the hazard or survival in an assessed technology converges to that of the comparator. This analysis aims to contribute to inform future guidance in this area. (Source: Value in Health)
Source: Value in Health - April 26, 2024 Category: International Medicine & Public Health Authors: Laura A. Trigg, G.J. Melendez-Torres, Ahmed Abdelsabour, Dawn Lee Source Type: research

How do the Institute for Clinical and Economic Review ’s Assessments of Comparative Effectiveness Compare to the German Federal Joint Committee’s Assessments of Added Benefit? A Qualitative Study
We compared the Institute for Clinical and Economic Review ’s (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee’s (G-BA) added benefit ratings, and explored what factors may explain disagreement between the two organizations. (Source: Value in Health)
Source: Value in Health - April 25, 2024 Category: International Medicine & Public Health Authors: Michael J. DiStefano, Steven D. Pearson, David M. Rind, Antal Zemplenyi Source Type: research

Can Generalized Cost-effectiveness Analysis (GCEA) Leverage Meaningful Use of Novel Value Elements in Pharmacoeconomics to Inform Medicare Drug Price Negotiation?
Decision-makers considering using cost-effectiveness analysis (CEA) to inform health-technology assessment (HTA) must contend with documented and controversial shortfalls of CEA, including its assumption of disease severity independence and static pricing. ISPOR has recently introduced novel value elements besides direct healthcare cost and effectiveness for the patient, and these should be captured in CEA . While novel value elements advance our understanding of “what” should be measured (e.g. value of hope, severity of disease, health equity, etc.), there is limited direction on “how” to measure them in conventio...
Source: Value in Health - April 25, 2024 Category: International Medicine & Public Health Authors: William V. Padula, Peter Kolchinsky Tags: Policy Perspectives Source Type: research

EQ-5D-5L population norms for China derived from a national health survey
To develop the EQ-5D-5L (5L) population norms for China; and to assess the relationship between various factors and 5L data. (Source: Value in Health)
Source: Value in Health - April 25, 2024 Category: International Medicine & Public Health Authors: Danlin Li, Zitong Wang, Xinyi Nie, Nan Luo, Yibo Wu, Chenwei Pan, Pei Wang Tags: Preference-Based Assessments Source Type: research

Estimating the Allocation of the Economic Value Generated by Utilization of All-Oral Direct-Acting Antivirals for Hepatitis C in the United States, 2015 –2019
Between 2013 –2019, several all-oral direct-acting antivirals (DAAs) were launched with the potential to cure patients with hepatitis C virus (HCV). They generated economic value in terms of the health gains for patients and cost-savings for the US healthcare system. We estimated the share of this value alloca ted to four manufacturers vs society. (Source: Value in Health)
Source: Value in Health - April 22, 2024 Category: International Medicine & Public Health Authors: Louis P. Garrison, Boshen Jiao, Zizi Elsisi, Alon Yehoshua, Roy Koruth, Bruce Kreter, Jens Grueger Source Type: research

Health Fluctuations in Dementia and its Impact on the Assessment of Health-related Quality of Life using the EQ-5D-5L
To quantify health fluctuations, identify affected health-related quality of life (HRQoL) dimensions, and evaluate if fluctuations affect the HRQoL instruments recall period adherence in people living with dementia (PlwD). (Source: Value in Health)
Source: Value in Health - April 20, 2024 Category: International Medicine & Public Health Authors: Bernhard Michalowsky, Lidia Engel, Maresa Buchholz, Niklas Weber, Thomas Kohlmann, Feng Xie Source Type: research

Subjective well-being population norms and inequalities in Hungary: a large cross-sectional, internet-based survey
This study aims to provide subjective well-being (SWB) population norms in Hungary and explore the contribution of explanatory factors of SWB inequality among the Hungarian adult general population. (Source: Value in Health)
Source: Value in Health - April 17, 2024 Category: International Medicine & Public Health Authors: Thao T.P. Nguyen, Fanni Rencz, Valentin Brodszky Source Type: research

The Long Coronavirus Disease (COVID) Symptom and Severity Score: Development, Validation, and Application
The primary focus of this research is the proposition of a methodological framework for the clinical application of the long coronavirus disease (COVID) Symptom and Severity Score (LC-SSS). This tool is not just a self-reported assessment instrument developed and validated but serves as a standardized, quantifiable means to monitor the diverse and persistent symptoms frequently observed in individuals suffering from long COVID. (Source: Value in Health)
Source: Value in Health - April 17, 2024 Category: International Medicine & Public Health Authors: Gengchen Ye, Yanan Zhu, Wenrui Bao, Heping Zhou, Jiandong Lai, Yuchen Zhang, Juanping Xie, Qingbo Ma, Zhaoyao Luo, Shaohui Ma, Yichu Guo, Xuanting Zhang, Ming Zhang, Xuan Niu Source Type: research

Cost-effectiveness of axicabtagene ciloleucel for adult patients with relapsed or refractory follicular lymphoma in the United States
The results of a recent single-arm trial (ZUMA-5) of axicabtagene ciloleucel (axi-cel) for relapsed/refractory (r/r) FL demonstrated high rates of durable response and tolerable toxicity among treated patients. To quantify the value of axi-cel compared to standard of care (SOC) to manage r/r FL patients who have had at least two prior lines of systemic therapy (3L+), a cost-effectiveness model was developed from a US third-party payer perspective. (Source: Value in Health)
Source: Value in Health - April 16, 2024 Category: International Medicine & Public Health Authors: Olalekan O. Oluwole, Markqayne D. Ray, Katherine L. Rosettie, Graeme Ball, Jorge Jacob, S. Pinar Bilir, Anik R. Patel, Caron A. Jacobson Source Type: research

Economic Evaluations of CAR-T Cell Therapies for Hematologic and Solid Malignancies: A Systematic Review
This study aims to systematically review evidence on the cost-effectiveness of chimeric antigen receptor (CAR)-T therapies for patients with cancer. (Source: Value in Health)
Source: Value in Health - April 16, 2024 Category: International Medicine & Public Health Authors: Kednapa Thavorn, Emily Thompson, Srishti Kumar, Aliisa Heiskanen, Anubhav Agarwal, Harold Atkins, Risa Shorr, Terry Hawrysh, Kelvin Kar-Wing Chan, Justin Presseau, Daniel A. Ollendorf, Ian D. Graham, Jeremy M. Grimshaw, Manoj Mathew Lalu, Surapon Nochaiwo Tags: Systematic Literature Review Source Type: research

Validating health economic models with the Probabilistic Analysis Check dashBOARD (PACBOARD)
This study aimed to develop and test a generic dashboard to systematically explore the workings of HE models and validate their model parameters and outcomes. (Source: Value in Health)
Source: Value in Health - April 16, 2024 Category: International Medicine & Public Health Authors: Xavier G.L.V. Pouwels, Karel Kroeze, Naomi van der Linden, Michelle M.A. Kip, Hendrik Koffijberg Tags: Methodology Source Type: research

Decision Makers Should Avoid the Health Years in Total (HYT) Approach: A Response to Dr Basu
(Source: Value in Health)
Source: Value in Health - April 15, 2024 Category: International Medicine & Public Health Authors: Mike Paulden, Chris Sampson, James F. O ’Mahony, Eldon Spackman, Christopher McCabe, Jeff Round, Tristan Snowsill Tags: Letter to the Editor Source Type: research

The Inflation Reduction Act: An Opportunity to Accelerate Confidence in US Real-World Evidence.
The objective of this commentary is to explore the implications of the IRA for US pharmaceutical companies, with a specific focus on the role of real-world evidence (RWE) in the context of Medicare reforms. (Source: Value in Health)
Source: Value in Health - April 15, 2024 Category: International Medicine & Public Health Authors: Paul Arora, Alind Gupta, Emma Mackay, Bart Heeg, Kristian Thorlund Source Type: research

It takes two to tango. Setting out the conditions in which performance-based risk-sharing arrangements work for both parties.
Faster regulatory approval processes often fail to achieve faster patient access. We seek an approach, using performance-based risk-sharing arrangements (PBRSA), to address uncertainty for payers regarding the relative effectiveness and value for money of products launched through accelerated approval schemes.One important reason for risk-sharing is to resolve differences of opinion between innovators and payers about a technology ’s underlying value. To date, there has been no formal attempt to set out the circumstances in which risk-sharing can address these differences. (Source: Value in Health)
Source: Value in Health - April 12, 2024 Category: International Medicine & Public Health Authors: Adrian Towse, Elisabeth Fenwick Source Type: research