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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doaj-aa6ef09e5c874ebe921c812fb3fc41192021-07-13T15:00:20ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262020-07-018210.1136/jitc-2020-000648Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspectiveCasey Quinn0Louis P Garrison1Anja K Pownell2Gérard de Pouvourville3Samuel Wagner4John Borrill5Elise Wu61 PRMA Consulting, Fleet, UK 2 CHOICE Institute, University of Washington, Seattle, Washington, USA1 PRMA Consulting, Fleet, UK4 ESSEC Business School, Cergy-Pontoise, France8 Bristol-Myers Squibb, New York, New York, USA8 Bristol-Myers Squibb, New York, New York, USA8 Bristol-Myers Squibb, New York, New York, USAImmuno-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.https://jitc.bmj.com/content/8/2/e000648.full |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Casey Quinn Louis P Garrison Anja K Pownell Gérard de Pouvourville Samuel Wagner John Borrill Elise Wu |
spellingShingle |
Casey Quinn Louis P Garrison Anja K Pownell Gérard de Pouvourville Samuel Wagner John Borrill Elise Wu Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective Journal for ImmunoTherapy of Cancer |
author_facet |
Casey Quinn Louis P Garrison Anja K Pownell Gérard de Pouvourville Samuel Wagner John Borrill Elise Wu |
author_sort |
Casey Quinn |
title |
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_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_sort |
current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective |
publisher |
BMJ Publishing Group |
series |
Journal for ImmunoTherapy of Cancer |
issn |
2051-1426 |
publishDate |
2020-07-01 |
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. |
url |
https://jitc.bmj.com/content/8/2/e000648.full |
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