Cargando…

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...

Descripción completa

Detalles Bibliográficos
Autores principales: Cook, Suzanne F., Rhodes, Thomas, Schlusser, Courtney, Han, Steve, Chen, Chao, Zach, Neta, Murthy, Venkatesha, Davé, Shreya
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8606522/
https://www.ncbi.nlm.nih.gov/pubmed/34819914
http://dx.doi.org/10.3389/fneur.2021.770001
_version_ 1784602349578223616
author Cook, Suzanne F.
Rhodes, Thomas
Schlusser, Courtney
Han, Steve
Chen, Chao
Zach, Neta
Murthy, Venkatesha
Davé, Shreya
author_facet Cook, Suzanne F.
Rhodes, Thomas
Schlusser, Courtney
Han, Steve
Chen, Chao
Zach, Neta
Murthy, Venkatesha
Davé, Shreya
author_sort Cook, Suzanne F.
collection PubMed
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 Online
Article
Text
id pubmed-8606522
institution National Center for Biotechnology Information
language English
publishDate 2021
publisher Frontiers Media S.A.
record_format MEDLINE/PubMed
spelling pubmed-86065222021-11-23 A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis Cook, Suzanne F. Rhodes, Thomas Schlusser, Courtney Han, Steve Chen, Chao Zach, Neta Murthy, Venkatesha Davé, Shreya Front Neurol Neurology 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. Frontiers Media S.A. 2021-11-08 /pmc/articles/PMC8606522/ /pubmed/34819914 http://dx.doi.org/10.3389/fneur.2021.770001 Text en Copyright © 2021 Cook, Rhodes, Schlusser, Han, Chen, Zach, Murthy and Davé. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Neurology
Cook, Suzanne F.
Rhodes, Thomas
Schlusser, Courtney
Han, Steve
Chen, Chao
Zach, Neta
Murthy, Venkatesha
Davé, Shreya
A Descriptive Review of Global Real World Evidence Efforts to Advance Drug Discovery and Clinical Development in Amyotrophic Lateral Sclerosis
title 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_short 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
topic Neurology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8606522/
https://www.ncbi.nlm.nih.gov/pubmed/34819914
http://dx.doi.org/10.3389/fneur.2021.770001
work_keys_str_mv AT cooksuzannef adescriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT rhodesthomas adescriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT schlussercourtney adescriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT hansteve adescriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT chenchao adescriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT zachneta adescriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT murthyvenkatesha adescriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT daveshreya adescriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT cooksuzannef descriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT rhodesthomas descriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT schlussercourtney descriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT hansteve descriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT chenchao descriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT zachneta descriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT murthyvenkatesha descriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis
AT daveshreya descriptivereviewofglobalrealworldevidenceeffortstoadvancedrugdiscoveryandclinicaldevelopmentinamyotrophiclateralsclerosis