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Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective

The burden of Parkinson’s disease (PD) continues to grow at an unsustainable pace particularly given that it now represents the fastest growing brain disease. Despite seminal discoveries in genetics and pathogenesis, people living with PD oftentimes wait years to obtain an accurate diagnosis and hav...

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Autores principales: Stephenson, Diane, Badawy, Reham, Mathur, Soania, Tome, Maria, Rochester, Lynn
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8385507/
https://www.ncbi.nlm.nih.gov/pubmed/33579873
http://dx.doi.org/10.3233/JPD-202428
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author Stephenson, Diane
Badawy, Reham
Mathur, Soania
Tome, Maria
Rochester, Lynn
author_facet Stephenson, Diane
Badawy, Reham
Mathur, Soania
Tome, Maria
Rochester, Lynn
author_sort Stephenson, Diane
collection PubMed
description The burden of Parkinson’s disease (PD) continues to grow at an unsustainable pace particularly given that it now represents the fastest growing brain disease. Despite seminal discoveries in genetics and pathogenesis, people living with PD oftentimes wait years to obtain an accurate diagnosis and have no way to know their own prognostic fate once they do learn they have the disease. Currently, there is no objective biomarker to measure the onset, progression, and severity of PD along the disease continuum. Without such tools, the effectiveness of any given treatment, experimental or conventional cannot be measured. Such tools are urgently needed now more than ever given the rich number of new candidate therapies in the pipeline. Over the last decade, millions of dollars have been directed to identify biomarkers to inform progression of PD typically using molecular, fluid or imaging modalities. These efforts have produced novel insights in our understanding of PD including mechanistic targets, disease subtypes and imaging biomarkers. While we have learned a lot along the way, implementation of robust disease progression biomarkers as tools for quantifying changes in disease status or severity remains elusive. Biomarkers have improved health outcomes and led to accelerated drug approvals in key areas of unmet need such as oncology. Quantitative biomarker measures such as HbA1c a standard test for the monitoring of diabetes has impacted patient care and management, both for the healthcare professionals and the patient community. Such advances accelerate opportunities for early intervention including prevention of disease in high-risk individuals. In PD, progression markers are needed at all stages of the disease in order to catalyze drug development—this allows interventions aimed to halt or slow disease progression (very early) but also facilitates symptomatic treatments at moderate stages of the disease. Recently, attention has turned to the role of digital health technologies to complement the traditional modalities as they are relatively low cost, objective and scalable. Success in this endeavor would be transformative for clinical research and therapeutic development. Consequently, significant investment has led to a number of collaborative efforts to identify and validate suitable digital biomarkers of disease progression.
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spelling pubmed-83855072021-09-09 Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective Stephenson, Diane Badawy, Reham Mathur, Soania Tome, Maria Rochester, Lynn J Parkinsons Dis Review The burden of Parkinson’s disease (PD) continues to grow at an unsustainable pace particularly given that it now represents the fastest growing brain disease. Despite seminal discoveries in genetics and pathogenesis, people living with PD oftentimes wait years to obtain an accurate diagnosis and have no way to know their own prognostic fate once they do learn they have the disease. Currently, there is no objective biomarker to measure the onset, progression, and severity of PD along the disease continuum. Without such tools, the effectiveness of any given treatment, experimental or conventional cannot be measured. Such tools are urgently needed now more than ever given the rich number of new candidate therapies in the pipeline. Over the last decade, millions of dollars have been directed to identify biomarkers to inform progression of PD typically using molecular, fluid or imaging modalities. These efforts have produced novel insights in our understanding of PD including mechanistic targets, disease subtypes and imaging biomarkers. While we have learned a lot along the way, implementation of robust disease progression biomarkers as tools for quantifying changes in disease status or severity remains elusive. Biomarkers have improved health outcomes and led to accelerated drug approvals in key areas of unmet need such as oncology. Quantitative biomarker measures such as HbA1c a standard test for the monitoring of diabetes has impacted patient care and management, both for the healthcare professionals and the patient community. Such advances accelerate opportunities for early intervention including prevention of disease in high-risk individuals. In PD, progression markers are needed at all stages of the disease in order to catalyze drug development—this allows interventions aimed to halt or slow disease progression (very early) but also facilitates symptomatic treatments at moderate stages of the disease. Recently, attention has turned to the role of digital health technologies to complement the traditional modalities as they are relatively low cost, objective and scalable. Success in this endeavor would be transformative for clinical research and therapeutic development. Consequently, significant investment has led to a number of collaborative efforts to identify and validate suitable digital biomarkers of disease progression. IOS Press 2021-07-16 /pmc/articles/PMC8385507/ /pubmed/33579873 http://dx.doi.org/10.3233/JPD-202428 Text en © 2021 – The authors. Published by IOS Press https://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution Non-Commercial (CC BY-NC 4.0) License (https://creativecommons.org/licenses/by-nc/4.0/) , which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Stephenson, Diane
Badawy, Reham
Mathur, Soania
Tome, Maria
Rochester, Lynn
Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective
title Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective
title_full Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective
title_fullStr Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective
title_full_unstemmed Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective
title_short Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective
title_sort digital progression biomarkers as novel endpoints in clinical trials: a multistakeholder perspective
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8385507/
https://www.ncbi.nlm.nih.gov/pubmed/33579873
http://dx.doi.org/10.3233/JPD-202428
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