Cargando…
Trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: A retrospective cohort study
BACKGROUND: The objective of this review was to investigate trends in clinical trial design, specifically, the primary outcomes used, interpretation of results, and the magnitude of the benefits described in phase III controlled clinical trials in the first‐line treatment of patients with advanced n...
Autores principales: | , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons Australia, Ltd
2019
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6449273/ https://www.ncbi.nlm.nih.gov/pubmed/30868737 http://dx.doi.org/10.1111/1759-7714.13024 |
_version_ | 1783408807712391168 |
---|---|
author | Fernández‐López, Cristina Calleja‐Hernández, Miguel Ángel Balbino, Jaime Espín Cabeza‐Barrera, José Expósito‐Hernández, José |
author_facet | Fernández‐López, Cristina Calleja‐Hernández, Miguel Ángel Balbino, Jaime Espín Cabeza‐Barrera, José Expósito‐Hernández, José |
author_sort | Fernández‐López, Cristina |
collection | PubMed |
description | BACKGROUND: The objective of this review was to investigate trends in clinical trial design, specifically, the primary outcomes used, interpretation of results, and the magnitude of the benefits described in phase III controlled clinical trials in the first‐line treatment of patients with advanced non‐small cell lung cancer (NSCLC). METHODS: Seventy‐six trials published between 2000 and 2012 were selected from a total of 122 identified in a structured search. RESULTS: Overall survival (OS) was evaluated as the primary study endpoint in 50 (65.8%) trials, followed by progression‐free survival (PFS) in 15 (19.7%), and other variables, such as toxicity, quality of life (QoL), and response rate in 11 (14.5%). Ten (66.7%) out of 15 clinical trials using PFS as the primary endpoint were published between 2010 and 2012. Median overall survival (mOS) was 9.90 months (interquartile range: 3.5) with an increase of 0.384 months per year of publication (P < 0.001). A statistically significant improvement in mOS was obtained in only 13 (18.8%) trials. A total of 41 (53.9%) studies concluded that the result was positive. Of these, only 16 (39.1%) showed a statistically significant benefit in OS. QoL was assessed in 46 trials (60.5%) and of these, 10 (21.7%) reported significant improvements. CONCLUSIONS: These findings raise important questions about how clinical benefits are measured in clinical trials in advanced NSCLC. Appropriate clinically relevant outcome variables should be established and validated, and post‐marketing studies should be requested by regulatory authorities to ensure meaningful clinical benefits in OS and QoL. |
format | Online Article Text |
id | pubmed-6449273 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | John Wiley & Sons Australia, Ltd |
record_format | MEDLINE/PubMed |
spelling | pubmed-64492732019-04-15 Trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: A retrospective cohort study Fernández‐López, Cristina Calleja‐Hernández, Miguel Ángel Balbino, Jaime Espín Cabeza‐Barrera, José Expósito‐Hernández, José Thorac Cancer Original Articles BACKGROUND: The objective of this review was to investigate trends in clinical trial design, specifically, the primary outcomes used, interpretation of results, and the magnitude of the benefits described in phase III controlled clinical trials in the first‐line treatment of patients with advanced non‐small cell lung cancer (NSCLC). METHODS: Seventy‐six trials published between 2000 and 2012 were selected from a total of 122 identified in a structured search. RESULTS: Overall survival (OS) was evaluated as the primary study endpoint in 50 (65.8%) trials, followed by progression‐free survival (PFS) in 15 (19.7%), and other variables, such as toxicity, quality of life (QoL), and response rate in 11 (14.5%). Ten (66.7%) out of 15 clinical trials using PFS as the primary endpoint were published between 2010 and 2012. Median overall survival (mOS) was 9.90 months (interquartile range: 3.5) with an increase of 0.384 months per year of publication (P < 0.001). A statistically significant improvement in mOS was obtained in only 13 (18.8%) trials. A total of 41 (53.9%) studies concluded that the result was positive. Of these, only 16 (39.1%) showed a statistically significant benefit in OS. QoL was assessed in 46 trials (60.5%) and of these, 10 (21.7%) reported significant improvements. CONCLUSIONS: These findings raise important questions about how clinical benefits are measured in clinical trials in advanced NSCLC. Appropriate clinically relevant outcome variables should be established and validated, and post‐marketing studies should be requested by regulatory authorities to ensure meaningful clinical benefits in OS and QoL. John Wiley & Sons Australia, Ltd 2019-03-13 2019-04 /pmc/articles/PMC6449273/ /pubmed/30868737 http://dx.doi.org/10.1111/1759-7714.13024 Text en © 2019 The Authors. Thoracic Cancer published by China Lung Oncology Group and John Wiley & Sons Australia, Ltd This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. |
spellingShingle | Original Articles Fernández‐López, Cristina Calleja‐Hernández, Miguel Ángel Balbino, Jaime Espín Cabeza‐Barrera, José Expósito‐Hernández, José Trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: A retrospective cohort study |
title | Trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: A retrospective cohort study |
title_full | Trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: A retrospective cohort study |
title_fullStr | Trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: A retrospective cohort study |
title_full_unstemmed | Trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: A retrospective cohort study |
title_short | Trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: A retrospective cohort study |
title_sort | trends in endpoint selection and result interpretation in advanced non‐small cell lung cancer clinical trials published between 2000 and 2012: a retrospective cohort study |
topic | Original Articles |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6449273/ https://www.ncbi.nlm.nih.gov/pubmed/30868737 http://dx.doi.org/10.1111/1759-7714.13024 |
work_keys_str_mv | AT fernandezlopezcristina trendsinendpointselectionandresultinterpretationinadvancednonsmallcelllungcancerclinicaltrialspublishedbetween2000and2012aretrospectivecohortstudy AT callejahernandezmiguelangel trendsinendpointselectionandresultinterpretationinadvancednonsmallcelllungcancerclinicaltrialspublishedbetween2000and2012aretrospectivecohortstudy AT balbinojaimeespin trendsinendpointselectionandresultinterpretationinadvancednonsmallcelllungcancerclinicaltrialspublishedbetween2000and2012aretrospectivecohortstudy AT cabezabarrerajose trendsinendpointselectionandresultinterpretationinadvancednonsmallcelllungcancerclinicaltrialspublishedbetween2000and2012aretrospectivecohortstudy AT expositohernandezjose trendsinendpointselectionandresultinterpretationinadvancednonsmallcelllungcancerclinicaltrialspublishedbetween2000and2012aretrospectivecohortstudy |