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Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study

PURPOSE: Real-World Data (RWD) studies are increasingly used to support regulatory approvals, reimbursement decisions, and changes in clinical practice for novel cancer drugs. However, few studies have systematically appraised their quality or compared outcomes to pivotal trials. METHODS: All RWD st...

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Autores principales: Boyle, Jemma M., Hegarty, Gemma, Frampton, Christopher, Harvey-Jones, Elizabeth, Dodkins, Joanna, Beyer, Katharina, George, Gincy, Sullivan, Richard, Booth, Christopher, Aggarwal, Ajay
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier Science Ltd 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8442759/
https://www.ncbi.nlm.nih.gov/pubmed/34371443
http://dx.doi.org/10.1016/j.ejca.2021.07.001
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author Boyle, Jemma M.
Hegarty, Gemma
Frampton, Christopher
Harvey-Jones, Elizabeth
Dodkins, Joanna
Beyer, Katharina
George, Gincy
Sullivan, Richard
Booth, Christopher
Aggarwal, Ajay
author_facet Boyle, Jemma M.
Hegarty, Gemma
Frampton, Christopher
Harvey-Jones, Elizabeth
Dodkins, Joanna
Beyer, Katharina
George, Gincy
Sullivan, Richard
Booth, Christopher
Aggarwal, Ajay
author_sort Boyle, Jemma M.
collection PubMed
description PURPOSE: Real-World Data (RWD) studies are increasingly used to support regulatory approvals, reimbursement decisions, and changes in clinical practice for novel cancer drugs. However, few studies have systematically appraised their quality or compared outcomes to pivotal trials. METHODS: All RWD studies (2010–2019) for drugs approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) from 2010 to 2015 for solid organ tumours in the non-curative setting were identified. Quality assessment was undertaken using the Newcastle Ottawa Scale. Survival differences between each RWD study and the pivotal trial were determined using a related sample Wilcoxon signed-rank test. RESULTS: 293 RWD studies for 45 of the 57 drug indications approved by the FDA/EMA were identified. The most common tumour types were prostate cancer (29%, n = 86) and melanoma (15%, n = 43). A quarter of the studies had industry funding. No high-quality studies were identified, and 78% were low quality. Comparative survival analysis between RWD and pivotal trials was possible for 224 studies (37 drug indications). Differences in median survival between the RWD studies and their corresponding trial ranged from −32 months to 21 months (IQR –4·2 months to 1·6 months). Low-quality studies were more likely to report superior survival outcomes (23%) compared to higher quality studies (8%) (p = 0.02). CONCLUSION: RWD study quality for novel cancer drugs is low and of insufficient rigour to inform reimbursement decisions and clinical practice. RWD studies seeking publication should provide a completed quality assessment tool on submission. Greater investment in properly designed RWD studies is required.
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spelling pubmed-84427592021-09-20 Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study Boyle, Jemma M. Hegarty, Gemma Frampton, Christopher Harvey-Jones, Elizabeth Dodkins, Joanna Beyer, Katharina George, Gincy Sullivan, Richard Booth, Christopher Aggarwal, Ajay Eur J Cancer Original Research PURPOSE: Real-World Data (RWD) studies are increasingly used to support regulatory approvals, reimbursement decisions, and changes in clinical practice for novel cancer drugs. However, few studies have systematically appraised their quality or compared outcomes to pivotal trials. METHODS: All RWD studies (2010–2019) for drugs approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) from 2010 to 2015 for solid organ tumours in the non-curative setting were identified. Quality assessment was undertaken using the Newcastle Ottawa Scale. Survival differences between each RWD study and the pivotal trial were determined using a related sample Wilcoxon signed-rank test. RESULTS: 293 RWD studies for 45 of the 57 drug indications approved by the FDA/EMA were identified. The most common tumour types were prostate cancer (29%, n = 86) and melanoma (15%, n = 43). A quarter of the studies had industry funding. No high-quality studies were identified, and 78% were low quality. Comparative survival analysis between RWD and pivotal trials was possible for 224 studies (37 drug indications). Differences in median survival between the RWD studies and their corresponding trial ranged from −32 months to 21 months (IQR –4·2 months to 1·6 months). Low-quality studies were more likely to report superior survival outcomes (23%) compared to higher quality studies (8%) (p = 0.02). CONCLUSION: RWD study quality for novel cancer drugs is low and of insufficient rigour to inform reimbursement decisions and clinical practice. RWD studies seeking publication should provide a completed quality assessment tool on submission. Greater investment in properly designed RWD studies is required. Elsevier Science Ltd 2021-09 /pmc/articles/PMC8442759/ /pubmed/34371443 http://dx.doi.org/10.1016/j.ejca.2021.07.001 Text en © 2021 The Authors https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Original Research
Boyle, Jemma M.
Hegarty, Gemma
Frampton, Christopher
Harvey-Jones, Elizabeth
Dodkins, Joanna
Beyer, Katharina
George, Gincy
Sullivan, Richard
Booth, Christopher
Aggarwal, Ajay
Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study
title Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study
title_full Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study
title_fullStr Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study
title_full_unstemmed Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study
title_short Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study
title_sort real-world outcomes associated with new cancer medicines approved by the food and drug administration and european medicines agency: a retrospective cohort study
topic Original Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8442759/
https://www.ncbi.nlm.nih.gov/pubmed/34371443
http://dx.doi.org/10.1016/j.ejca.2021.07.001
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