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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...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier Science Ltd
2021
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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. |
format | Online Article Text |
id | pubmed-8442759 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Elsevier Science Ltd |
record_format | MEDLINE/PubMed |
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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