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Lupus community panel proposals for optimising clinical trials: 2018

Formidable impediments stand in the way of treatment development for lupus. These include the unwieldy size of current trials, international competition for scarce patients, complex outcome measures and a poor understanding of these outcomes in the world at large. The heterogeneity of the disease it...

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Autores principales: Merrill, Joan T, Manzi, Susan, Aranow, Cynthia, Askenase, Anca, Bruce, Ian, Chakravarty, Eliza, Chong, Ben, Costenbader, Karen, Dall’Era, Maria, Ginzler, Ellen, Hanrahan, Leslie, Kalunian, Ken, Merola, Joseph, Raymond, Sandra, Rovin, Brad, Saxena, Amit, Werth, Victoria P
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5894527/
https://www.ncbi.nlm.nih.gov/pubmed/29657738
http://dx.doi.org/10.1136/lupus-2018-000258
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author Merrill, Joan T
Manzi, Susan
Aranow, Cynthia
Askenase, Anca
Bruce, Ian
Chakravarty, Eliza
Chong, Ben
Costenbader, Karen
Dall’Era, Maria
Ginzler, Ellen
Hanrahan, Leslie
Kalunian, Ken
Merola, Joseph
Raymond, Sandra
Rovin, Brad
Saxena, Amit
Werth, Victoria P
author_facet Merrill, Joan T
Manzi, Susan
Aranow, Cynthia
Askenase, Anca
Bruce, Ian
Chakravarty, Eliza
Chong, Ben
Costenbader, Karen
Dall’Era, Maria
Ginzler, Ellen
Hanrahan, Leslie
Kalunian, Ken
Merola, Joseph
Raymond, Sandra
Rovin, Brad
Saxena, Amit
Werth, Victoria P
author_sort Merrill, Joan T
collection PubMed
description Formidable impediments stand in the way of treatment development for lupus. These include the unwieldy size of current trials, international competition for scarce patients, complex outcome measures and a poor understanding of these outcomes in the world at large. The heterogeneity of the disease itself coupled to superimposition of variegated background polypharmacy has created enough immunological noise to virtually ensure the failure of lupus treatment trials, leaving an understandable suspicion that at least some of the results in testing failed drugs over the years may not have been negative, but merely uninterpretable. The authors have consulted with many clinical trial investigators, biopharmaceutical developers and stakeholders from government and voluntary sectors. This paper examines the available evidence that supports workable trial designs and proposes approaches to improve the odds of completing interpretable treatment development programs for lupus.
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spelling pubmed-58945272018-04-13 Lupus community panel proposals for optimising clinical trials: 2018 Merrill, Joan T Manzi, Susan Aranow, Cynthia Askenase, Anca Bruce, Ian Chakravarty, Eliza Chong, Ben Costenbader, Karen Dall’Era, Maria Ginzler, Ellen Hanrahan, Leslie Kalunian, Ken Merola, Joseph Raymond, Sandra Rovin, Brad Saxena, Amit Werth, Victoria P Lupus Sci Med Review Formidable impediments stand in the way of treatment development for lupus. These include the unwieldy size of current trials, international competition for scarce patients, complex outcome measures and a poor understanding of these outcomes in the world at large. The heterogeneity of the disease itself coupled to superimposition of variegated background polypharmacy has created enough immunological noise to virtually ensure the failure of lupus treatment trials, leaving an understandable suspicion that at least some of the results in testing failed drugs over the years may not have been negative, but merely uninterpretable. The authors have consulted with many clinical trial investigators, biopharmaceutical developers and stakeholders from government and voluntary sectors. This paper examines the available evidence that supports workable trial designs and proposes approaches to improve the odds of completing interpretable treatment development programs for lupus. BMJ Publishing Group 2018-03-23 /pmc/articles/PMC5894527/ /pubmed/29657738 http://dx.doi.org/10.1136/lupus-2018-000258 Text en © Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2018. All rights reserved. No commercial use is permitted unless otherwise expressly granted. This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
spellingShingle Review
Merrill, Joan T
Manzi, Susan
Aranow, Cynthia
Askenase, Anca
Bruce, Ian
Chakravarty, Eliza
Chong, Ben
Costenbader, Karen
Dall’Era, Maria
Ginzler, Ellen
Hanrahan, Leslie
Kalunian, Ken
Merola, Joseph
Raymond, Sandra
Rovin, Brad
Saxena, Amit
Werth, Victoria P
Lupus community panel proposals for optimising clinical trials: 2018
title Lupus community panel proposals for optimising clinical trials: 2018
title_full Lupus community panel proposals for optimising clinical trials: 2018
title_fullStr Lupus community panel proposals for optimising clinical trials: 2018
title_full_unstemmed Lupus community panel proposals for optimising clinical trials: 2018
title_short Lupus community panel proposals for optimising clinical trials: 2018
title_sort lupus community panel proposals for optimising clinical trials: 2018
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5894527/
https://www.ncbi.nlm.nih.gov/pubmed/29657738
http://dx.doi.org/10.1136/lupus-2018-000258
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