Validity and Reliability of Value Assessment Frameworks for New Cancer Drugs
Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Com...
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| Vydané v: | Value in health Ročník 20; číslo 2; s. 200 - 205 |
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| Hlavní autori: | , , , , , , , , , , , , |
| Médium: | Journal Article |
| Jazyk: | English |
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United States
Elsevier Inc
01.02.2017
Elsevier Science Ltd |
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| ISSN: | 1098-3015, 1524-4733, 1524-4733 |
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| Abstract | Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN).
To understand the extent to which these four tools can facilitate value-based treatment decisions in oncology.
In this pilot study, eight panelists conducted value assessments of five advanced lung cancer drugs using the ASCO, ESMO, and ICER frameworks. The panelists received instructions and published clinical data required to complete the assessments. Published NCCN framework scores were abstracted. The Kendall's W coefficient was used to measure convergent validity among the four frameworks. Intraclass correlation coefficients were used to measure inter-rater reliability among the ASCO, ESMO, and ICER frameworks. Sensitivity analyses were conducted.
Drugs were ranked similarly by the four frameworks, with Kendall's W of 0.703 (P = 0.006) across all the four frameworks. Pairwise, Kendall's W was the highest for ESMO-ICER (W = 0.974; P = 0.007) and ASCO-NCCN (W = 0.944; P = 0.022) and the lowest for ICER-NCCN (W = 0.647; P = 0.315) and ESMO-NCCN (W = 0.611; P = 0.360). Intraclass correlation coefficients (confidence interval [CI]) for the ASCO, ESMO, and ICER frameworks were 0.786 (95% CI 0.517–0.970), 0.804 (95% CI 0.545–0.973), and 0.281 (95% CI 0.055–0.799), respectively. When scores were rescaled to 0 to 100, the ICER framework provided the narrowest band of scores.
The ASCO, ESMO, ICER, and NCCN frameworks demonstrated convergent validity, despite differences in conceptual approaches used. The ASCO inter-rater reliability was high, although potentially at the cost of user burden. The ICER inter-rater reliability was poor, possibly because of its failure to distinguish differential value among the sample of drugs tested. Refinements of all frameworks should continue on the basis of further testing and stakeholder feedback. |
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| AbstractList | Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN).BACKGROUNDSeveral organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN).To understand the extent to which these four tools can facilitate value-based treatment decisions in oncology.OBJECTIVESTo understand the extent to which these four tools can facilitate value-based treatment decisions in oncology.In this pilot study, eight panelists conducted value assessments of five advanced lung cancer drugs using the ASCO, ESMO, and ICER frameworks. The panelists received instructions and published clinical data required to complete the assessments. Published NCCN framework scores were abstracted. The Kendall's W coefficient was used to measure convergent validity among the four frameworks. Intraclass correlation coefficients were used to measure inter-rater reliability among the ASCO, ESMO, and ICER frameworks. Sensitivity analyses were conducted.METHODSIn this pilot study, eight panelists conducted value assessments of five advanced lung cancer drugs using the ASCO, ESMO, and ICER frameworks. The panelists received instructions and published clinical data required to complete the assessments. Published NCCN framework scores were abstracted. The Kendall's W coefficient was used to measure convergent validity among the four frameworks. Intraclass correlation coefficients were used to measure inter-rater reliability among the ASCO, ESMO, and ICER frameworks. Sensitivity analyses were conducted.Drugs were ranked similarly by the four frameworks, with Kendall's W of 0.703 (P = 0.006) across all the four frameworks. Pairwise, Kendall's W was the highest for ESMO-ICER (W = 0.974; P = 0.007) and ASCO-NCCN (W = 0.944; P = 0.022) and the lowest for ICER-NCCN (W = 0.647; P = 0.315) and ESMO-NCCN (W = 0.611; P = 0.360). Intraclass correlation coefficients (confidence interval [CI]) for the ASCO, ESMO, and ICER frameworks were 0.786 (95% CI 0.517-0.970), 0.804 (95% CI 0.545-0.973), and 0.281 (95% CI 0.055-0.799), respectively. When scores were rescaled to 0 to 100, the ICER framework provided the narrowest band of scores.RESULTSDrugs were ranked similarly by the four frameworks, with Kendall's W of 0.703 (P = 0.006) across all the four frameworks. Pairwise, Kendall's W was the highest for ESMO-ICER (W = 0.974; P = 0.007) and ASCO-NCCN (W = 0.944; P = 0.022) and the lowest for ICER-NCCN (W = 0.647; P = 0.315) and ESMO-NCCN (W = 0.611; P = 0.360). Intraclass correlation coefficients (confidence interval [CI]) for the ASCO, ESMO, and ICER frameworks were 0.786 (95% CI 0.517-0.970), 0.804 (95% CI 0.545-0.973), and 0.281 (95% CI 0.055-0.799), respectively. When scores were rescaled to 0 to 100, the ICER framework provided the narrowest band of scores.The ASCO, ESMO, ICER, and NCCN frameworks demonstrated convergent validity, despite differences in conceptual approaches used. The ASCO inter-rater reliability was high, although potentially at the cost of user burden. The ICER inter-rater reliability was poor, possibly because of its failure to distinguish differential value among the sample of drugs tested. Refinements of all frameworks should continue on the basis of further testing and stakeholder feedback.CONCLUSIONSThe ASCO, ESMO, ICER, and NCCN frameworks demonstrated convergent validity, despite differences in conceptual approaches used. The ASCO inter-rater reliability was high, although potentially at the cost of user burden. The ICER inter-rater reliability was poor, possibly because of its failure to distinguish differential value among the sample of drugs tested. Refinements of all frameworks should continue on the basis of further testing and stakeholder feedback. Abstract Background Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN). Objectives To understand the extent to which these four tools can facilitate value-based treatment decisions in oncology. Methods In this pilot study, eight panelists conducted value assessments of five advanced lung cancer drugs using the ASCO, ESMO, and ICER frameworks. The panelists received instructions and published clinical data required to complete the assessments. Published NCCN framework scores were abstracted. The Kendall's W coefficient was used to measure convergent validity among the four frameworks. Intraclass correlation coefficients were used to measure inter-rater reliability among the ASCO, ESMO, and ICER frameworks. Sensitivity analyses were conducted. Results Drugs were ranked similarly by the four frameworks, with Kendall's W of 0.703 ( P = 0.006) across all the four frameworks. Pairwise, Kendall's W was the highest for ESMO-ICER ( W = 0.974; P = 0.007) and ASCO-NCCN ( W = 0.944; P = 0.022) and the lowest for ICER-NCCN ( W = 0.647; P = 0.315) and ESMO-NCCN ( W = 0.611; P = 0.360). Intraclass correlation coefficients (confidence interval [CI]) for the ASCO, ESMO, and ICER frameworks were 0.786 (95% CI 0.517–0.970), 0.804 (95% CI 0.545–0.973), and 0.281 (95% CI 0.055–0.799), respectively. When scores were rescaled to 0 to 100, the ICER framework provided the narrowest band of scores. Conclusions The ASCO, ESMO, ICER, and NCCN frameworks demonstrated convergent validity, despite differences in conceptual approaches used. The ASCO inter-rater reliability was high, although potentially at the cost of user burden. The ICER inter-rater reliability was poor, possibly because of its failure to distinguish differential value among the sample of drugs tested. Refinements of all frameworks should continue on the basis of further testing and stakeholder feedback. Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN). To understand the extent to which these four tools can facilitate value-based treatment decisions in oncology. In this pilot study, eight panelists conducted value assessments of five advanced lung cancer drugs using the ASCO, ESMO, and ICER frameworks. The panelists received instructions and published clinical data required to complete the assessments. Published NCCN framework scores were abstracted. The Kendall's W coefficient was used to measure convergent validity among the four frameworks. Intraclass correlation coefficients were used to measure inter-rater reliability among the ASCO, ESMO, and ICER frameworks. Sensitivity analyses were conducted. Drugs were ranked similarly by the four frameworks, with Kendall's W of 0.703 (P = 0.006) across all the four frameworks. Pairwise, Kendall's W was the highest for ESMO-ICER (W = 0.974; P = 0.007) and ASCO-NCCN (W = 0.944; P = 0.022) and the lowest for ICER-NCCN (W = 0.647; P = 0.315) and ESMO-NCCN (W = 0.611; P = 0.360). Intraclass correlation coefficients (confidence interval [CI]) for the ASCO, ESMO, and ICER frameworks were 0.786 (95% CI 0.517–0.970), 0.804 (95% CI 0.545–0.973), and 0.281 (95% CI 0.055–0.799), respectively. When scores were rescaled to 0 to 100, the ICER framework provided the narrowest band of scores. The ASCO, ESMO, ICER, and NCCN frameworks demonstrated convergent validity, despite differences in conceptual approaches used. The ASCO inter-rater reliability was high, although potentially at the cost of user burden. The ICER inter-rater reliability was poor, possibly because of its failure to distinguish differential value among the sample of drugs tested. Refinements of all frameworks should continue on the basis of further testing and stakeholder feedback. Background: Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN). Objectives: To understand the extent to which these four tools can facilitate value-based treatment decisions in oncology. Methods: In this pilot study, eight panelists conducted value assessments of five advanced lung cancer drugs using the ASCO, ESMO, and ICER frameworks. The panelists received instructions and published clinical data required to complete the assessments. Published NCCN framework scores were abstracted. The Kendall's W coefficient was used to measure convergent validity among the four frameworks. Intraclass correlation coefficients were used to measure inter-rater reliability among the ASCO, ESMO, and ICER frameworks. Sensitivity analyses were conducted. Results: Drugs were ranked similarly by the four frameworks, with Kendall's W of 0.703 (P = 0.006) across all the four frameworks. Pairwise, Kendall's W was the highest for ESMO-ICER (W = 0.974; P = 0.007) and ASCO-NCCN (W = 0.944; P = 0.022) and the lowest for ICER-NCCN (W = 0.647; P = 0.315) and ESMO-NCCN (W = 0.611; P = 0.360). Intraclass correlation coefficients (confidence interval [CI]) for the ASCO, ESMO, and ICER frameworks were 0.786 (95% CI 0.517–0.970), 0.804 (95% CI 0.545–0.973), and 0.281 (95% CI 0.055–0.799), respectively. When scores were rescaled to 0 to 100, the ICER framework provided the narrowest band of scores. Conclusions: The ASCO, ESMO, ICER, and NCCN frameworks demonstrated convergent validity, despite differences in conceptual approaches used. The ASCO inter-rater reliability was high, although potentially at the cost of user burden. The ICER inter-rater reliability was poor, possibly because of its failure to distinguish differential value among the sample of drugs tested. Refinements of all frameworks should continue on the basis of further testing and stakeholder feedback. |
| Author | Broder, Michael S. Cohen, Joshua T. Mukherjea, Arnab Zambrano, Jenelle M. Huynh, Julie Copher, Ronda Mei, Matthew Knoth, Russell Elkin, Elena B. Bentley, Tanya G.K. Neville, Thanh H. Popescu, Ioana Lee, Jackie |
| Author_xml | – sequence: 1 givenname: Tanya G.K. surname: Bentley fullname: Bentley, Tanya G.K. email: tbentley@pharllc.com organization: Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA – sequence: 2 givenname: Joshua T. surname: Cohen fullname: Cohen, Joshua T. organization: Tufts Medical Center, Boston, MA, USA – sequence: 3 givenname: Elena B. surname: Elkin fullname: Elkin, Elena B. organization: Memorial Sloan Kettering Cancer Center, New York, NY, USA – sequence: 4 givenname: Julie surname: Huynh fullname: Huynh, Julie organization: Hematology Oncology of San Fernando Valley, Encino, CA, USA – sequence: 5 givenname: Arnab surname: Mukherjea fullname: Mukherjea, Arnab organization: Health Sciences Program, California State University, East Bay, Hayward, CA, USA – sequence: 6 givenname: Thanh H. surname: Neville fullname: Neville, Thanh H. organization: Department of Medicine, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA – sequence: 7 givenname: Matthew surname: Mei fullname: Mei, Matthew organization: City of Hope National Medical Center, Duarte, CA, USA – sequence: 8 givenname: Ronda surname: Copher fullname: Copher, Ronda organization: Eisai Inc., Woodcliff Lake, NJ, USA – sequence: 9 givenname: Russell surname: Knoth fullname: Knoth, Russell organization: Eisai Inc., Woodcliff Lake, NJ, USA – sequence: 10 givenname: Ioana surname: Popescu fullname: Popescu, Ioana organization: Department of Medicine, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA – sequence: 11 givenname: Jackie surname: Lee fullname: Lee, Jackie organization: Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA – sequence: 12 givenname: Jenelle M. surname: Zambrano fullname: Zambrano, Jenelle M. organization: Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA – sequence: 13 givenname: Michael S. surname: Broder fullname: Broder, Michael S. organization: Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/28237195$$D View this record in MEDLINE/PubMed |
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| Copyright | 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Copyright © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved. Copyright Elsevier Science Ltd. Feb 2017 |
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| Snippet | Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical... Abstract Background Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American... Background: Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society... |
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| Title | Validity and Reliability of Value Assessment Frameworks for New Cancer Drugs |
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