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

Utilisation, expenditure, and treatment patterns associated with calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) reimbursed subject to a managed access protocol in Ireland.
This study presents data on utilisation, expenditure and treatment patterns with CGRP mAbs available under a managed access protocol in Ireland, to a cohort of treatment refractory patients (failed three or more previous treatments) with chronic migraine. (Source: Value in Health)
Source: Value in Health - April 12, 2024 Category: International Medicine & Public Health Authors: Amelia Smith, Karen Finnigan, Sarah Clarke, Michael Barry, Claire Gorry Source Type: research

A Systematic Review and Quality Assessment of Cardiovascular Disease-Specific Health Related Quality of Life Instruments Part I: Instrument Development and Content Validity
This study aimed at systematically identifying and assessing the content validity of CVD-specific HRQoL instruments in clinical studies. (Source: Value in Health)
Source: Value in Health - April 10, 2024 Category: International Medicine & Public Health Authors: Xue Li, Rui Li, Meixuan Li, Liang Yao, Harriette Van Spall, Kun Zhao, Yunxiang Chen, Feiyi Xiao, Qiang Fu, Feng Xie Tags: Systematic Literature Review Source Type: research

Testing the psychometric properties of 9 bolt-ons for the EQ-5D-5L in a general population sample
We aim to assess the psychometric performance and added value of nine existing bolt-ons (breathing problems, cognition, hearing, self-confidence, skin irritation, sleep, social relationships, tiredness and vision) for the EQ-5D-5L in a general population sample. (Source: Value in Health)
Source: Value in Health - April 8, 2024 Category: International Medicine & Public Health Authors: Fanni Rencz, Mathieu F. Janssen Source Type: research

The PICOTS-ComTeC Framework for Defining Digital Health Interventions: An ISPOR Special Interest Group Report
Digital health definitions are abundant, but often lack clarity and precision. We aimed to develop a minimum information framework to define patient-facing digital health interventions (DHIs) for outcomes research. (Source: Value in Health)
Source: Value in Health - April 1, 2024 Category: International Medicine & Public Health Authors: Zsombor Zrubka, Annette Champion, Anke-Peggy Holtorf, Rossella Di Bidino, Jagadeswara Rao Earla, Artem T. Boltyenkov, Masami Tabata-Kelly, Carl Asche, Anita Burrell Tags: ISPOR Report Source Type: research

Table of Contents
(Source: Value in Health)
Source: Value in Health - April 1, 2024 Category: International Medicine & Public Health Source Type: research

Editorial Board
(Source: Value in Health)
Source: Value in Health - April 1, 2024 Category: International Medicine & Public Health Source Type: research

Do Quality-Adjusted Life-Years Discriminate Against the Elderly? An Empirical Analysis of Published Cost-Effectiveness Analyses
This study aimed to compare published cost-effectiveness analyses (CEAs) on patients aged ≥65 years and those aged (Source: Value in Health)
Source: Value in Health - March 26, 2024 Category: International Medicine & Public Health Authors: Feng Xie, Ting Zhou, Brittany Humphries, Peter J. Neumann Tags: Economic Evaluation Source Type: research

Methods for Including Adverse Events in Economic Evaluations: Suggestions for Improvement
Inclusion of relevant effectiveness and safety outcomes in economic evaluation of health technologies is required to aid efficient healthcare decisions. Our objective was to identify the key issues related to the inclusion of adverse events (AEs) in economic evaluation and explore perspectives for good practice recommendations to handle these issues. (Source: Value in Health)
Source: Value in Health - March 26, 2024 Category: International Medicine & Public Health Authors: Salah Ghabri, Dalia Dawoud, Michael Drummond Tags: Methodology Source Type: research

Development of a value assessment framework for pediatric health technologies using multi-criteria decision analysis: expanding the value lens for funding decision-making
Health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multi-criteria decision analysis (MCDA) approach. (Source: Value in Health)
Source: Value in Health - March 26, 2024 Category: International Medicine & Public Health Authors: Cindy L. Gauvreau, Leighton Schreyer, Paul Gibson, Alicia Koo, Wendy J. Ungar, Dean Regier, Kelvin Chan, Robin Hayeems, Jennifer Gibson, Antonia Palmer, Stuart Peacock, Avram E. Denburg Source Type: research

Anti-VEGF Drugs Compared with Panretinal Photocoagulation for the Treatment of Proliferative Diabetic Retinopathy: A Cost-Effectiveness Analysis
To evaluate the cost-effectiveness of anti-vascular endothelial growth factor drugs (anti-VEGFs) compared with panretinal photocoagulation (PRP) for treating proliferative diabetic retinopathy (PDR) in the UK. (Source: Value in Health)
Source: Value in Health - March 26, 2024 Category: International Medicine & Public Health Authors: Matthew Walton, Laura Bojke, Mark Simmonds, Ruth Walker, Alexis Llewellyn, Helen Fulbright, Sofia Dias, Lesley A. Stewart, Tom Rush, David H. Steel, John G. Lawrenson, Tunde Peto, Robert Hodgson Source Type: research

Flexible approaches based on multi-state models and microsimulation to perform real-world cost-effectiveness analyses: an application to pcsk9-inhibitors
This study aims to show the application of flexible statistical methods in real-world cost-effectiveness analyses applied in the cardiovascular field, focusing specifically on the use of PCSK9 inhibitors for hyperlipidaemia. (Source: Value in Health)
Source: Value in Health - March 26, 2024 Category: International Medicine & Public Health Authors: Caterina Gregorio, Federico Rea, Francesca Ieva, Arjuna Scagnetto, Carla Indennidate, Chiara Cappelletto, Andrea Di Lenarda, Giulia Barbati Source Type: research

Towards Patient-Centered Drug Approval for Treatment of Rare Diseases
This commentary seeks to improve the design and analysis of trials undertaken to obtain approval of drugs for treatment of rare diseases. (Source: Value in Health)
Source: Value in Health - March 26, 2024 Category: International Medicine & Public Health Authors: Charles F. Manski Source Type: research

Tackling Challenges in Assessing the Economic Value of Tumor-Agnostic Therapies: A Cost-Effectiveness Analysis of Pembrolizumab as a Case Study
Assessing the value of tumor-agnostic therapies (TAD) is challenging given the potential variability in treatment effects, trials with small sample sizes, different standards-of-care (SoC), and lack of comparative data from single-arm basket trials. Our study developed and applied novel methods to assess the value of pembrolizumab compared to SoC to inform coverage decisions. (Source: Value in Health)
Source: Value in Health - March 26, 2024 Category: International Medicine & Public Health Authors: Yilin Chen, Peter Martin, Lurdes Y.T. Inoue, Anirban Basu, Josh J. Carlson Source Type: research

Do QALYs discriminate against the elderly? An empirical analysis of published cost effectiveness analyses
Critics of quality-adjusted life years (QALYs) argue that it discriminates against older individuals. However, little empirical evidence has been produced to inform this debate. To compare published cost effectiveness analyses (CEAs) on patients aged ≥65 years and those aged (Source: Value in Health)
Source: Value in Health - March 26, 2024 Category: International Medicine & Public Health Authors: Feng Xie, Ting Zhou, Brittany Humphries, Peter J. Neumann Source Type: research