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Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective

Immuno-oncologics (IOs) differ from chemotherapies as they prime the patient’s immune system to attack the tumor, rather than directly destroying cancer cells. The IO mechanism of action leads to durable responses and prolonged survival in some patients. However, providing robust evidence of the lon...

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Autores principales: Quinn, Casey, Garrison, Louis P, Pownell, Anja K, Atkins, Michael B, de Pouvourville, Gérard, Harrington, Kevin, Ascierto, Paolo Antonio, McEwan, Phil, Wagner, Samuel, Borrill, John, Wu, Elise
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7359062/
https://www.ncbi.nlm.nih.gov/pubmed/32661115
http://dx.doi.org/10.1136/jitc-2020-000648
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author Quinn, Casey
Garrison, Louis P
Pownell, Anja K
Atkins, Michael B
de Pouvourville, Gérard
Harrington, Kevin
Ascierto, Paolo Antonio
McEwan, Phil
Wagner, Samuel
Borrill, John
Wu, Elise
author_facet Quinn, Casey
Garrison, Louis P
Pownell, Anja K
Atkins, Michael B
de Pouvourville, Gérard
Harrington, Kevin
Ascierto, Paolo Antonio
McEwan, Phil
Wagner, Samuel
Borrill, John
Wu, Elise
author_sort Quinn, Casey
collection PubMed
description Immuno-oncologics (IOs) differ from chemotherapies as they prime the patient’s immune system to attack the tumor, rather than directly destroying cancer cells. The IO mechanism of action leads to durable responses and prolonged survival in some patients. However, providing robust evidence of the long-term benefits of IOs at health technology assessment (HTA) submission presents several challenges for manufacturers. The aim of this article was to identify, analyze, categorize, and further explore the key challenges that regulators, HTA agencies, and payers commonly encounter when assessing the long-term benefits of IO therapies. Insights were obtained from an international, multi-stakeholder steering committee (SC) and expert panels comprising of payers, economists, and clinicians. The selected individuals were tasked with developing a summary of challenges specific to IOs in demonstrating their long-term benefits at HTA submission. The SC and expert panels agreed that standard methods used to assess the long-term benefit of anticancer drugs may have limitations for IO therapies. Three key areas of challenges were identified: (1) lack of a disease model that fully captures the mechanism of action and subsequent patient responses; (2) estimation of longer-term outcomes, including a lack of agreement on ideal methods of survival analyses and extrapolation of survival curves; and (3) data limitations at the time of HTA submission, for which surrogate survival end points and real-world evidence could prove useful. A summary of the key challenges facing manufacturers when submitting evidence at HTA submission was developed, along with further recommendations for manufacturers in what evidence to produce. Despite almost a decade of use, there remain significant challenges around how best to demonstrate the long-term benefit of checkpoint inhibitor-based IOs to HTA agencies, clinicians, and payers. Manufacturers can potentially meet or mitigate these challenges with a focus on strengthening survival analysis methodology. Approaches to doing this include identifying reliable biomarkers, intermediate and surrogate end points, and the use of real-world data to inform and validate long-term survival projections. Wider education across all stakeholders—manufacturers, payers, and clinicians—in considering the long-term survival benefit with IOs is also important.
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spelling pubmed-73590622020-07-16 Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective Quinn, Casey Garrison, Louis P Pownell, Anja K Atkins, Michael B de Pouvourville, Gérard Harrington, Kevin Ascierto, Paolo Antonio McEwan, Phil Wagner, Samuel Borrill, John Wu, Elise J Immunother Cancer Position Article and Guidelines Immuno-oncologics (IOs) differ from chemotherapies as they prime the patient’s immune system to attack the tumor, rather than directly destroying cancer cells. The IO mechanism of action leads to durable responses and prolonged survival in some patients. However, providing robust evidence of the long-term benefits of IOs at health technology assessment (HTA) submission presents several challenges for manufacturers. The aim of this article was to identify, analyze, categorize, and further explore the key challenges that regulators, HTA agencies, and payers commonly encounter when assessing the long-term benefits of IO therapies. Insights were obtained from an international, multi-stakeholder steering committee (SC) and expert panels comprising of payers, economists, and clinicians. The selected individuals were tasked with developing a summary of challenges specific to IOs in demonstrating their long-term benefits at HTA submission. The SC and expert panels agreed that standard methods used to assess the long-term benefit of anticancer drugs may have limitations for IO therapies. Three key areas of challenges were identified: (1) lack of a disease model that fully captures the mechanism of action and subsequent patient responses; (2) estimation of longer-term outcomes, including a lack of agreement on ideal methods of survival analyses and extrapolation of survival curves; and (3) data limitations at the time of HTA submission, for which surrogate survival end points and real-world evidence could prove useful. A summary of the key challenges facing manufacturers when submitting evidence at HTA submission was developed, along with further recommendations for manufacturers in what evidence to produce. Despite almost a decade of use, there remain significant challenges around how best to demonstrate the long-term benefit of checkpoint inhibitor-based IOs to HTA agencies, clinicians, and payers. Manufacturers can potentially meet or mitigate these challenges with a focus on strengthening survival analysis methodology. Approaches to doing this include identifying reliable biomarkers, intermediate and surrogate end points, and the use of real-world data to inform and validate long-term survival projections. Wider education across all stakeholders—manufacturers, payers, and clinicians—in considering the long-term survival benefit with IOs is also important. BMJ Publishing Group 2020-07-12 /pmc/articles/PMC7359062/ /pubmed/32661115 http://dx.doi.org/10.1136/jitc-2020-000648 Text en © Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. http://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See http://creativecommons.org/licenses/by-nc/4.0/.
spellingShingle Position Article and Guidelines
Quinn, Casey
Garrison, Louis P
Pownell, Anja K
Atkins, Michael B
de Pouvourville, Gérard
Harrington, Kevin
Ascierto, Paolo Antonio
McEwan, Phil
Wagner, Samuel
Borrill, John
Wu, Elise
Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective
title Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective
title_full Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective
title_fullStr Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective
title_full_unstemmed Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective
title_short Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective
title_sort current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective
topic Position Article and Guidelines
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7359062/
https://www.ncbi.nlm.nih.gov/pubmed/32661115
http://dx.doi.org/10.1136/jitc-2020-000648
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