A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis

Understanding patient clinical progression is a key gateway to planning effective clinical trials and ultimately enabling bringing treatments to patients in need. In a rare disease like amyotrophic lateral sclerosis (ALS), studies of disease natural history critically depend on collaboration between...

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Autores principales:	Suzanne F. Cook, Thomas Rhodes, Courtney Schlusser, Steve Han, Chao Chen, Neta Zach, Venkatesha Murthy, Shreya Davé
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	amyotrophic lateral sclerosis real-world data rare disease registries biobanks Neurology. Diseases of the nervous system RC346-429
Acceso en línea:	https://doaj.org/article/67fe608696cf4764bc9d225c3674bd2f
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spelling	oai:doaj.org-article:67fe608696cf4764bc9d225c3674bd2f2021-11-08T05:16:06ZA Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis1664-229510.3389/fneur.2021.770001https://doaj.org/article/67fe608696cf4764bc9d225c3674bd2f2021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fneur.2021.770001/fullhttps://doaj.org/toc/1664-2295Understanding patient clinical progression is a key gateway to planning effective clinical trials and ultimately enabling bringing treatments to patients in need. In a rare disease like amyotrophic lateral sclerosis (ALS), studies of disease natural history critically depend on collaboration between clinical centers, regions, and countries to enable creation of platforms to allow patients, caregivers, clinicians, and researchers to come together and more fully understand the condition. Rare disease registries and collaborative platforms such as those developed in ALS collect real-world data (RWD) in standardized formats, including clinical and biological specimen data used to evaluate risk factors and natural history of disease, treatment patterns and clinical (ClinROs) and patient- reported outcomes (PROs) and validate novel endpoints. Importantly, these data support the development of new therapeutics by supporting the evaluation of feasibility and design of clinical trials and offer valuable information on real-world disease trajectory and outcomes outside of the clinical trial setting for comparative purposes. RWD may help to accelerate therapy development by identifying and validating outcome measures and disease subpopulations. RWD can also make potential contributions to the evaluation of the safety and effectiveness of new indications for approved products and to satisfy post-approval regulatory and market access requirements. There is a lack of amalgamated information on available registries, databases, and other sources of real-world data on ALS; thus, a global review of all available resources was warranted. This targeted review identifies and describes ALS registries, biobanks and collaborative research networks that are collecting and synthesizing RWD for the purposes of increasing patient awareness and advancing scientific knowledge with the hope of expediting future development of new therapies.Suzanne F. CookThomas RhodesCourtney SchlusserSteve HanChao ChenNeta ZachVenkatesha MurthyShreya DavéFrontiers Media S.A.articleamyotrophic lateral sclerosisreal-world datarare diseaseregistriesbiobanksNeurology. Diseases of the nervous systemRC346-429ENFrontiers in Neurology, Vol 12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	amyotrophic lateral sclerosis real-world data rare disease registries biobanks Neurology. Diseases of the nervous system RC346-429
spellingShingle	amyotrophic lateral sclerosis real-world data rare disease registries biobanks Neurology. Diseases of the nervous system RC346-429 Suzanne F. Cook Thomas Rhodes Courtney Schlusser Steve Han Chao Chen Neta Zach Venkatesha Murthy Shreya Davé A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis
description	Understanding patient clinical progression is a key gateway to planning effective clinical trials and ultimately enabling bringing treatments to patients in need. In a rare disease like amyotrophic lateral sclerosis (ALS), studies of disease natural history critically depend on collaboration between clinical centers, regions, and countries to enable creation of platforms to allow patients, caregivers, clinicians, and researchers to come together and more fully understand the condition. Rare disease registries and collaborative platforms such as those developed in ALS collect real-world data (RWD) in standardized formats, including clinical and biological specimen data used to evaluate risk factors and natural history of disease, treatment patterns and clinical (ClinROs) and patient- reported outcomes (PROs) and validate novel endpoints. Importantly, these data support the development of new therapeutics by supporting the evaluation of feasibility and design of clinical trials and offer valuable information on real-world disease trajectory and outcomes outside of the clinical trial setting for comparative purposes. RWD may help to accelerate therapy development by identifying and validating outcome measures and disease subpopulations. RWD can also make potential contributions to the evaluation of the safety and effectiveness of new indications for approved products and to satisfy post-approval regulatory and market access requirements. There is a lack of amalgamated information on available registries, databases, and other sources of real-world data on ALS; thus, a global review of all available resources was warranted. This targeted review identifies and describes ALS registries, biobanks and collaborative research networks that are collecting and synthesizing RWD for the purposes of increasing patient awareness and advancing scientific knowledge with the hope of expediting future development of new therapies.
format	article
author	Suzanne F. Cook Thomas Rhodes Courtney Schlusser Steve Han Chao Chen Neta Zach Venkatesha Murthy Shreya Davé
author_facet	Suzanne F. Cook Thomas Rhodes Courtney Schlusser Steve Han Chao Chen Neta Zach Venkatesha Murthy Shreya Davé
author_sort	Suzanne F. Cook
title	A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis
title_short	A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis
title_full	A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis
title_fullStr	A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis
title_full_unstemmed	A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis
title_sort	descriptive review of global real world evidence efforts to advance drug discovery and clinical development in amyotrophic lateral sclerosis
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/67fe608696cf4764bc9d225c3674bd2f
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A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis

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