INNOVATION IN ONCOLOGY: WHY FOCUSING ONLY ON BREAKTHROUGH INNOVATION MAY BE COUNTER-PRODUCTIVE
(Abstract release date: 05/21/15)
EHA Library. Quinn C. 06/12/15; 102819; PB1973
Casey Quinn
Contributions
Contributions
Abstract
Abstract: PB1973
Type: Publication Only
Background
Innovative oncology products are routinely perceived to be those that offer a substantial increase in overall survival (OS), usually above a certain threshold. However, this growing emphasis on significant OS gain (breakthrough innovation), to the exclusion of other clinical and non-clinical gains, undervalues new products and may not capture aspects of treatment that are important to patients. Clinicians, patient groups, and manufacturers argue that progressive innovation in other domains needs to be considered and valued in regulatory and health technology assessment (HTA) decisions.
Aims
The aim of this paper is to bridge the perspectives on progressive innovation and the approaches taken to measure and value innovation among different stakeholders at all levels of drug approval, appraisal and access.
Methods
We conducted a targeted literature search for definitions of innovation in academic and grey literature, covering regulatory, HTA, and industry bodies. Current/proposed OS thresholds were applied to standard of care (SOC) therapies in colorectal cancer (CRC) and non-small cell lung cancer (NSCLC).
Results
The magnitude of OS benefit consistently emerges as key in the definition of innovation from policy (e.g., England’s Cancer Drugs Fund) and clinical (e.g., American Society of Clinical Oncology [ASCO]) perspectives. Regulators and HTA agencies do not provide clear consistent definitions, however. Emphasis is increasingly on ‘clinically meaningful’ change in survival, expressed as minimum thresholds: OS gain >2.5months, HR>0.8; progression-free survival gain >3months, HR>0.5.
Summary
Innovation should not be defined solely on one-time large survival gains but should be evaluated according to the value provided to patients and health systems. Smaller sequential gains in clinical benefit and improvements in quality of life, safety, convenience, and system efficiency should be considered in assessing value and innovation. Products that have smaller outcome gains can be combined to provide an amplification of effectiveness. Similarly, small incremental advances can lead to significant improvemments over time. If the focus moves too far towards breakthrough innovation, these treatment opportunities will be lost. Progressive innovation in these aspects provides opportunities for immediate benefit, including survival until the next therapy is available, and may uncover new clinical pathways with significant cumulative benefit over time. Recognition of this ‘option value’ for future health and research advances is needed.
Type: Publication Only
Background
Innovative oncology products are routinely perceived to be those that offer a substantial increase in overall survival (OS), usually above a certain threshold. However, this growing emphasis on significant OS gain (breakthrough innovation), to the exclusion of other clinical and non-clinical gains, undervalues new products and may not capture aspects of treatment that are important to patients. Clinicians, patient groups, and manufacturers argue that progressive innovation in other domains needs to be considered and valued in regulatory and health technology assessment (HTA) decisions.
Aims
The aim of this paper is to bridge the perspectives on progressive innovation and the approaches taken to measure and value innovation among different stakeholders at all levels of drug approval, appraisal and access.
Methods
We conducted a targeted literature search for definitions of innovation in academic and grey literature, covering regulatory, HTA, and industry bodies. Current/proposed OS thresholds were applied to standard of care (SOC) therapies in colorectal cancer (CRC) and non-small cell lung cancer (NSCLC).
Results
The magnitude of OS benefit consistently emerges as key in the definition of innovation from policy (e.g., England’s Cancer Drugs Fund) and clinical (e.g., American Society of Clinical Oncology [ASCO]) perspectives. Regulators and HTA agencies do not provide clear consistent definitions, however. Emphasis is increasingly on ‘clinically meaningful’ change in survival, expressed as minimum thresholds: OS gain >2.5months, HR>0.8; progression-free survival gain >3months, HR>0.5.
Only one of six CRC products approved since 2000 met the OS threshold published by ASCO of three to five months (Ellis et al. 2014) whereas survival has doubled in that time. None of the products approved for NSCLC since 2005 met the OS threshold, for 3.25 to 4 months, while survival in patients receiving first-line treatment for advanced NSCLC has also doubled.
Summary
Innovation should not be defined solely on one-time large survival gains but should be evaluated according to the value provided to patients and health systems. Smaller sequential gains in clinical benefit and improvements in quality of life, safety, convenience, and system efficiency should be considered in assessing value and innovation. Products that have smaller outcome gains can be combined to provide an amplification of effectiveness. Similarly, small incremental advances can lead to significant improvemments over time. If the focus moves too far towards breakthrough innovation, these treatment opportunities will be lost. Progressive innovation in these aspects provides opportunities for immediate benefit, including survival until the next therapy is available, and may uncover new clinical pathways with significant cumulative benefit over time. Recognition of this ‘option value’ for future health and research advances is needed.
Focussing only on breakthrough OS gains Regulatory and HTA agencies should balance clinical and economic gains and societal and patient preferences when evaluating innovation in a new therapy.
Abstract: PB1973
Type: Publication Only
Background
Innovative oncology products are routinely perceived to be those that offer a substantial increase in overall survival (OS), usually above a certain threshold. However, this growing emphasis on significant OS gain (breakthrough innovation), to the exclusion of other clinical and non-clinical gains, undervalues new products and may not capture aspects of treatment that are important to patients. Clinicians, patient groups, and manufacturers argue that progressive innovation in other domains needs to be considered and valued in regulatory and health technology assessment (HTA) decisions.
Aims
The aim of this paper is to bridge the perspectives on progressive innovation and the approaches taken to measure and value innovation among different stakeholders at all levels of drug approval, appraisal and access.
Methods
We conducted a targeted literature search for definitions of innovation in academic and grey literature, covering regulatory, HTA, and industry bodies. Current/proposed OS thresholds were applied to standard of care (SOC) therapies in colorectal cancer (CRC) and non-small cell lung cancer (NSCLC).
Results
The magnitude of OS benefit consistently emerges as key in the definition of innovation from policy (e.g., England’s Cancer Drugs Fund) and clinical (e.g., American Society of Clinical Oncology [ASCO]) perspectives. Regulators and HTA agencies do not provide clear consistent definitions, however. Emphasis is increasingly on ‘clinically meaningful’ change in survival, expressed as minimum thresholds: OS gain >2.5months, HR>0.8; progression-free survival gain >3months, HR>0.5.
Summary
Innovation should not be defined solely on one-time large survival gains but should be evaluated according to the value provided to patients and health systems. Smaller sequential gains in clinical benefit and improvements in quality of life, safety, convenience, and system efficiency should be considered in assessing value and innovation. Products that have smaller outcome gains can be combined to provide an amplification of effectiveness. Similarly, small incremental advances can lead to significant improvemments over time. If the focus moves too far towards breakthrough innovation, these treatment opportunities will be lost. Progressive innovation in these aspects provides opportunities for immediate benefit, including survival until the next therapy is available, and may uncover new clinical pathways with significant cumulative benefit over time. Recognition of this ‘option value’ for future health and research advances is needed.
Type: Publication Only
Background
Innovative oncology products are routinely perceived to be those that offer a substantial increase in overall survival (OS), usually above a certain threshold. However, this growing emphasis on significant OS gain (breakthrough innovation), to the exclusion of other clinical and non-clinical gains, undervalues new products and may not capture aspects of treatment that are important to patients. Clinicians, patient groups, and manufacturers argue that progressive innovation in other domains needs to be considered and valued in regulatory and health technology assessment (HTA) decisions.
Aims
The aim of this paper is to bridge the perspectives on progressive innovation and the approaches taken to measure and value innovation among different stakeholders at all levels of drug approval, appraisal and access.
Methods
We conducted a targeted literature search for definitions of innovation in academic and grey literature, covering regulatory, HTA, and industry bodies. Current/proposed OS thresholds were applied to standard of care (SOC) therapies in colorectal cancer (CRC) and non-small cell lung cancer (NSCLC).
Results
The magnitude of OS benefit consistently emerges as key in the definition of innovation from policy (e.g., England’s Cancer Drugs Fund) and clinical (e.g., American Society of Clinical Oncology [ASCO]) perspectives. Regulators and HTA agencies do not provide clear consistent definitions, however. Emphasis is increasingly on ‘clinically meaningful’ change in survival, expressed as minimum thresholds: OS gain >2.5months, HR>0.8; progression-free survival gain >3months, HR>0.5.
Only one of six CRC products approved since 2000 met the OS threshold published by ASCO of three to five months (Ellis et al. 2014) whereas survival has doubled in that time. None of the products approved for NSCLC since 2005 met the OS threshold, for 3.25 to 4 months, while survival in patients receiving first-line treatment for advanced NSCLC has also doubled.
Summary
Innovation should not be defined solely on one-time large survival gains but should be evaluated according to the value provided to patients and health systems. Smaller sequential gains in clinical benefit and improvements in quality of life, safety, convenience, and system efficiency should be considered in assessing value and innovation. Products that have smaller outcome gains can be combined to provide an amplification of effectiveness. Similarly, small incremental advances can lead to significant improvemments over time. If the focus moves too far towards breakthrough innovation, these treatment opportunities will be lost. Progressive innovation in these aspects provides opportunities for immediate benefit, including survival until the next therapy is available, and may uncover new clinical pathways with significant cumulative benefit over time. Recognition of this ‘option value’ for future health and research advances is needed.
Focussing only on breakthrough OS gains Regulatory and HTA agencies should balance clinical and economic gains and societal and patient preferences when evaluating innovation in a new therapy.
{{ help_message }}
{{filter}}