@article{37ace0ee0dee4bcdafec0d325a3418f4,
title = "Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy",
abstract = "Aim: Delayed diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) represents a missed opportunity for intervention. This study estimates the health benefits of timely diagnosis and treatment with tafamidis. Methods: A disease simulation model was developed to predict health outcomes under scenarios of timely and delayed diagnosis and treatment. Efficacy and quality of life (QoL) profiles were derived from the pivotal tafamidis trial and diagnostic delay durations from the literature. Results: Timely diagnosis and treatment were predicted to extend mean life expectancy by 5.46 and 7.76 years, relative to delayed diagnosis, for wild-type and hereditary ATTR-CM, respectively. Corresponding QALY gains were 4.50 and 6.22. Conclusion: Timely diagnosis and treatment with tafamidis are predicted to significantly improve survival and QoL for ATTR-CM patients.",
keywords = "ATTR-CM, diagnosis, disease simulation model, health outcomes, tafamidis, transthyretin amyloid cardiomyopathy",
author = "Rozenbaum, {Mark H.} and Samuel Large and Rahul Bhambri and Michelle Stewart and Robert Young and Doornewaard, {Alexander Van} and Noel Dasgupta and Ahmad Masri and Jose Nativi-Nicolau",
note = "Funding Information: This work was supported by Pfizer. MH Rozenbaum, S Large, R Bhambri and M Stewart are employees of Pfizer and own stock and/or stock options. R Young and A van Doornewaard are employees of Health Economics and Outcomes Research Ltd and received fees from Pfizer in relation to this study and development of the manuscript. J Nativi-Nicolau{\textquoteright}s institution received funding for clinical trials for Pfizer, Akcea and Eidos and Educational Grants from Pfizer. N Dasgupta has been a consultant for Pfizer, Ionis, Akcea and Alynlam. J Nativi-Nicolau has been a consultant for Pfizer, Eidos, Akcea and Alnylam. A Masri{\textquoteright}s institution received research grants from Pfizer, Akcea and Ultromics. A Masri has been a consultant for Eidos, Ionis and Cytokinetics. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Medical writing support was provided by Jo Whelan of Health Economics and Outcomes Research Ltd, funded by Pfizer. Publisher Copyright: {\textcopyright} 2021 ",
year = "2021",
month = aug,
doi = "10.2217/cer-2021-0071",
language = "English (US)",
volume = "10",
pages = "927--938",
journal = "Journal of Comparative Effectiveness Research",
issn = "2042-6305",
publisher = "Future Medicine Ltd.",
number = "11",
}