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Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments

Background: Advances in cancer medicines have resulted in tangible health impacts, but the magnitude of benefits of approved cancer medicines could vary greatly. Health Technology Assessment (HTA) is a multidisciplinary process used to inform resource allocation through a systematic value assessment...

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Autores principales: Trapani, Dario, Tay-Teo, Kiu, Tesch, Megan E., Roitberg, Felipe, Sengar, Manju, Altuna, Sara C., Hassett, Michael J., Genazzani, Armando A., Kesselheim, Aaron S., Curigliano, Giuseppe
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9406873/
https://www.ncbi.nlm.nih.gov/pubmed/36005193
http://dx.doi.org/10.3390/curroncol29080455
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author Trapani, Dario
Tay-Teo, Kiu
Tesch, Megan E.
Roitberg, Felipe
Sengar, Manju
Altuna, Sara C.
Hassett, Michael J.
Genazzani, Armando A.
Kesselheim, Aaron S.
Curigliano, Giuseppe
author_facet Trapani, Dario
Tay-Teo, Kiu
Tesch, Megan E.
Roitberg, Felipe
Sengar, Manju
Altuna, Sara C.
Hassett, Michael J.
Genazzani, Armando A.
Kesselheim, Aaron S.
Curigliano, Giuseppe
author_sort Trapani, Dario
collection PubMed
description Background: Advances in cancer medicines have resulted in tangible health impacts, but the magnitude of benefits of approved cancer medicines could vary greatly. Health Technology Assessment (HTA) is a multidisciplinary process used to inform resource allocation through a systematic value assessment of health technology. This paper reviews the challenges in conducting HTA for cancer medicines arising from oncology trial designs and uncertainties of safety-efficacy data. Methods: Multiple databases (PubMed, Scopus and Google Scholar) and grey literature (public health agencies and governmental reports) were searched to inform this policy narrative review. Results: A lack of robust efficacy-safety data from clinical trials and other relevant sources of evidence has made HTA for cancer medicines challenging. The approval of cancer medicines through expedited pathways has increased in recent years, in which surrogate endpoints or biomarkers for patient selection have been widely used. Using these surrogate endpoints has created uncertainties in translating surrogate measures into patient-centric clinically (survival and quality of life) and economically (cost-effectiveness and budget impact) meaningful outcomes, with potential effects on diverting scarce health resources to low-value or detrimental interventions. Potential solutions include policy harmonization between regulatory and HTA authorities, commitment to generating robust post-marketing efficacy-safety data, managing uncertainties through risk-sharing agreements, and using value frameworks. Conclusion: A lack of robust efficacy-safety data is a central problem for conducting HTA of cancer medicines, potentially resulting in misinformed resource allocation.
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spelling pubmed-94068732022-08-26 Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments Trapani, Dario Tay-Teo, Kiu Tesch, Megan E. Roitberg, Felipe Sengar, Manju Altuna, Sara C. Hassett, Michael J. Genazzani, Armando A. Kesselheim, Aaron S. Curigliano, Giuseppe Curr Oncol Review Background: Advances in cancer medicines have resulted in tangible health impacts, but the magnitude of benefits of approved cancer medicines could vary greatly. Health Technology Assessment (HTA) is a multidisciplinary process used to inform resource allocation through a systematic value assessment of health technology. This paper reviews the challenges in conducting HTA for cancer medicines arising from oncology trial designs and uncertainties of safety-efficacy data. Methods: Multiple databases (PubMed, Scopus and Google Scholar) and grey literature (public health agencies and governmental reports) were searched to inform this policy narrative review. Results: A lack of robust efficacy-safety data from clinical trials and other relevant sources of evidence has made HTA for cancer medicines challenging. The approval of cancer medicines through expedited pathways has increased in recent years, in which surrogate endpoints or biomarkers for patient selection have been widely used. Using these surrogate endpoints has created uncertainties in translating surrogate measures into patient-centric clinically (survival and quality of life) and economically (cost-effectiveness and budget impact) meaningful outcomes, with potential effects on diverting scarce health resources to low-value or detrimental interventions. Potential solutions include policy harmonization between regulatory and HTA authorities, commitment to generating robust post-marketing efficacy-safety data, managing uncertainties through risk-sharing agreements, and using value frameworks. Conclusion: A lack of robust efficacy-safety data is a central problem for conducting HTA of cancer medicines, potentially resulting in misinformed resource allocation. MDPI 2022-08-16 /pmc/articles/PMC9406873/ /pubmed/36005193 http://dx.doi.org/10.3390/curroncol29080455 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Trapani, Dario
Tay-Teo, Kiu
Tesch, Megan E.
Roitberg, Felipe
Sengar, Manju
Altuna, Sara C.
Hassett, Michael J.
Genazzani, Armando A.
Kesselheim, Aaron S.
Curigliano, Giuseppe
Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments
title Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments
title_full Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments
title_fullStr Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments
title_full_unstemmed Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments
title_short Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments
title_sort implications of oncology trial design and uncertainties in efficacy-safety data on health technology assessments
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9406873/
https://www.ncbi.nlm.nih.gov/pubmed/36005193
http://dx.doi.org/10.3390/curroncol29080455
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