Abstract
Objective: PRO-ACT platform, a de-facto reference knowledgebase with 30+ published papers, several disease models and staging systems, contains clinical trials selection biased data.
Similar platform to collaboratively share observational datasets for global large-scale phenotyping, disease progression models development and validation, linking clinical data to bio-, image-banks, WGS files, and omics, and for identifying disease sub-populations is essential to bring ALS/MND into Age of Precision Medicine and Big Data
Background: Develop common approaches to PALS’ data harmonization, integration, de-identification, sharing and distribution, and incentives for Data Contributors by introducing The Pooled Resource Open-Access Clinical Research (PRO-ACE) platform to facilitates:
- Structured and secure integration of retrospective/prospective clinical/clinical research information
- Harmonization, de-identification, aggregation, storage and open-access data distribution for secondary analyses.
Design/Methods:
- Harmonized DUAs, CDEs, SOPs plus NeuroGUID technology lead to accelerated studies’ review, and deployment, while preserving PALS’ privacy
- Standardized ICF language facilitates information aggregation/sharing
- Data from ERB/IRB approved observational studies, retrospective clinical assessments, population registries and clinical datasets are eligible for inclusion
- NeuroBANK™, patient-centric prospective clinical research environment is one of many PRO-ACE feeder platforms
- Curated de-identified and harmonized datasets merged into PRO-ACE Dataset are available for:
- Ad-hoc online analyses with NeuroCHARTS™ technology
- Open-access distribution for secondary analyses
- Data Access Committee reviews access requests
- Data Contributors become PRO-ACE Consortium members with benefits ranging from first-access rights to new data to securing authorship to papers
Results: 2,500+ longitudinal PALS’ records from observational studies available for analyses with hundred being added monthly.
PRO-ACE platform:
- Allows aggregation of existing publicly- and privately-collected datasets from multiple sources including Natural History studies, retrospective clinical assessments and other existing clinical datasets, providing an invaluable open-access resource for accelerating discovery
- Is the first platform for global collaboration in a single disease, with data devoid of placebo effects and clinical trial selection bias, thus being ideal for disease modeling and predictive algorithms validation
- Sandbox for developing clinical research collaborations and sharing incentives
Disclosure: Dr. Sherman has nothing to disclose. Dr. Berry has nothing to disclose. Dr. Gotkine has nothing to disclose. Dr. Glass has nothing to disclose. Dr. Lunetta has nothing to disclose. Dr. Floeter has nothing to disclose. Dr. Traynor has nothing to disclose. Dr. Katsovskiy has nothing to disclose. Dr. Kharakozova has nothing to disclose. Dr. Korin has nothing to disclose. Dr. Podesta has nothing to disclose. Dr. Sinani has nothing to disclose. Dr. Tarasenko has nothing to disclose. Dr. Cudkowicz has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Cytokinetics, Eli Lilly & Company, Orion, Biohaven, and MT Pharma. Dr. Cudkowicz has received personal compensation in an editorial capacity for JAMA Neurology.
Disputes & Debates: Rapid online correspondence
REQUIREMENTS
If you are uploading a letter concerning an article:
You must have updated your disclosures within six months: http://submit.neurology.org
Your co-authors must send a completed Publishing Agreement Form to Neurology Staff (not necessary for the lead/corresponding author as the form below will suffice) before you upload your comment.
If you are responding to a comment that was written about an article you originally authored:
You (and co-authors) do not need to fill out forms or check disclosures as author forms are still valid
and apply to letter.
Submission specifications:
- Submissions must be < 200 words with < 5 references. Reference 1 must be the article on which you are commenting.
- Submissions should not have more than 5 authors. (Exception: original author replies can include all original authors of the article)
- Submit only on articles published within 6 months of issue date.
- Do not be redundant. Read any comments already posted on the article prior to submission.
- Submitted comments are subject to editing and editor review prior to posting.