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Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations
Over the past 15 years, three new classes of drugs, glucagon‐like peptide‐1 (GLP‐1) receptor agonists, dipeptidyl peptidase 4 (DPP‐4) inhibitors and sodium glucose cotransporter‐2 (SGLT‐2) inhibitors have been approved to treat type 2 diabetes based on effects on glycemic control. Although large ran...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Blackwell Publishing Ltd
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6220800/ https://www.ncbi.nlm.nih.gov/pubmed/30294953 http://dx.doi.org/10.1111/dom.13453 |
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author | Mol, Peter G. M. Thompson, Aliza Heerspink, Hiddo J. L. Leufkens, Hubert G. M. |
author_facet | Mol, Peter G. M. Thompson, Aliza Heerspink, Hiddo J. L. Leufkens, Hubert G. M. |
author_sort | Mol, Peter G. M. |
collection | PubMed |
description | Over the past 15 years, three new classes of drugs, glucagon‐like peptide‐1 (GLP‐1) receptor agonists, dipeptidyl peptidase 4 (DPP‐4) inhibitors and sodium glucose cotransporter‐2 (SGLT‐2) inhibitors have been approved to treat type 2 diabetes based on effects on glycemic control. Although large randomized controlled trials have played an important role in characterizing the efficacy and safety of these agents on a population level, questions remain about how best to individualize therapy and target the “right” medicine to the “right” patient. In contrast, few medicines have been approved to treat diabetic kidney disease and initiatives have been launched on both sides of the Atlantic to facilitate the development of effective personalized medicines for the treatment of diabetic kidney disease. Increasingly, “omics,” imaging and other biomarkers will be used to match patients with therapies to which they are likely to respond best. This review addresses regulatory considerations related to precision medicine, draws lessons learned from other therapeutic areas and discusses efforts undertaken by the European (EMA) and United States (FDA) to facilitate the development of such therapies. Moving forward, an integrated approach that makes use of predictive preclinical models, innovative trial designs, observational “real‐world” data and novel statistical methodologies will likely be needed to complement inherently smaller RCTs conducted in more selected populations. Patient involvement will also be critical. Regulatory agencies are ready to engage in such approaches. |
format | Online Article Text |
id | pubmed-6220800 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | Blackwell Publishing Ltd |
record_format | MEDLINE/PubMed |
spelling | pubmed-62208002018-11-13 Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations Mol, Peter G. M. Thompson, Aliza Heerspink, Hiddo J. L. Leufkens, Hubert G. M. Diabetes Obes Metab Precision Medicine in Diabetic Kidney Disease. Guest Editor: Hiddo J. L. Heerspink Over the past 15 years, three new classes of drugs, glucagon‐like peptide‐1 (GLP‐1) receptor agonists, dipeptidyl peptidase 4 (DPP‐4) inhibitors and sodium glucose cotransporter‐2 (SGLT‐2) inhibitors have been approved to treat type 2 diabetes based on effects on glycemic control. Although large randomized controlled trials have played an important role in characterizing the efficacy and safety of these agents on a population level, questions remain about how best to individualize therapy and target the “right” medicine to the “right” patient. In contrast, few medicines have been approved to treat diabetic kidney disease and initiatives have been launched on both sides of the Atlantic to facilitate the development of effective personalized medicines for the treatment of diabetic kidney disease. Increasingly, “omics,” imaging and other biomarkers will be used to match patients with therapies to which they are likely to respond best. This review addresses regulatory considerations related to precision medicine, draws lessons learned from other therapeutic areas and discusses efforts undertaken by the European (EMA) and United States (FDA) to facilitate the development of such therapies. Moving forward, an integrated approach that makes use of predictive preclinical models, innovative trial designs, observational “real‐world” data and novel statistical methodologies will likely be needed to complement inherently smaller RCTs conducted in more selected populations. Patient involvement will also be critical. Regulatory agencies are ready to engage in such approaches. Blackwell Publishing Ltd 2018-10-07 2018-10 /pmc/articles/PMC6220800/ /pubmed/30294953 http://dx.doi.org/10.1111/dom.13453 Text en © 2018 The Authors. Diabetes, Obesity and Metabolism published by John Wiley & Sons 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 | Precision Medicine in Diabetic Kidney Disease. Guest Editor: Hiddo J. L. Heerspink Mol, Peter G. M. Thompson, Aliza Heerspink, Hiddo J. L. Leufkens, Hubert G. M. Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations |
title | Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations |
title_full | Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations |
title_fullStr | Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations |
title_full_unstemmed | Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations |
title_short | Precision medicine in diabetes and diabetic kidney disease: Regulatory considerations |
title_sort | precision medicine in diabetes and diabetic kidney disease: regulatory considerations |
topic | Precision Medicine in Diabetic Kidney Disease. Guest Editor: Hiddo J. L. Heerspink |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6220800/ https://www.ncbi.nlm.nih.gov/pubmed/30294953 http://dx.doi.org/10.1111/dom.13453 |
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