A review of economic issues for gene‐targeted therapies: Value, affordability, and access

LP Garrison Jr, AW Lo, RS Finkel… - American Journal of …, 2023 - Wiley Online Library
Abstract The National Center for Advancing Translational Sciences' virtual 2021 conference
on gene‐targeted therapies (GTTs) encouraged multidisciplinary dialogue on a wide range …

[HTML][HTML] A review of methodological considerations for economic evaluations of gene therapies and their application in literature

RMT Ten Ham, OH Klungel, HGM Leufkens… - Value in Health, 2020 - Elsevier
Objectives To identify methodological considerations discussed in literature addressing
economic evaluations (EEs) of gene therapies (GTs). Additionally, we assessed if these …

Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies

LP Garrison Jr, B Jiao, O Dabbous - Journal of managed care & specialty …, 2021 - jmcp.org
At an upfront price of 2.125million,theone-timegenetherapyonasemnogeneabeparvov…,arareneuromusculardisorderthatisusuallyfat…
,hasbeencalledthe“mostexpensivedrugever.”Thisflawedcharacterizationraisesimportant …

[HTML][HTML] Value-based pricing for patent-protected medicines over the product life cycle: pricing anomalies in the “Age of Cures” and their implications for dynamic …

LP Garrison Jr, B Jiao, O Dabbous - Value in Health, 2023 - Elsevier
Objectives Conventional cost-effectiveness analysis (CEA) for the value-based pricing of
new medicines largely ignores the implications of limited market exclusivity (ie, patent …

[HTML][HTML] Analysis of patient reported outcomes included in the registrational clinical trials of nivolumab for advanced non-small cell lung cancer

R Zaim, K Redekop, CA Uyl-de Groot - Translational oncology, 2022 - Elsevier
In the era of value-based oncology care, stakeholders are increasingly using patient
reported outcomes (PROs) to guide clinical and regulatory decisions. PROs are also …

Does cost-effectiveness analysis overvalue potential cures? Exploring alternative methods for applying a “shared savings” approach to cost offsets

RH Chapman, VM Kumar, MD Whittington, SD Pearson - Value in Health, 2021 - Elsevier
Objectives To evaluate alternative methods to calculate and/or attribute economic surplus in
the cost-effectiveness analysis of single or short-term therapies. Methods We performed a …

Assessing potential cures: are there distinctive elements of value beyond health gain?

S Hendriks, SD Pearson - Journal of Comparative …, 2021 - becarispublishing.com
Assessing the 'value'of potential cures can be challenging, as some have suggested that
cures may offer distinctive benefits from noncurative treatments. We explore what these …

[引用][C] 4.1 A Review of methodological

RMT ten Ham, OH Klungel, HGM Leufkens… - … , market authorization and …, 2021