Data Standards
The data standards for the Neuromuscular Diseases were formed by unique Working Groups: Neuromuscular Diseases (NMD), Congenital Muscular Dystrophy (CMD), Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (DMD/BMD), Facioscapulohumeral muscular dystrophy (FSHD), Myasthenia Gravis (MG), Myotonic Dystrophy (MMD), and Spinal Muscular Atrophy (SMA).
Please view additional tabs for more information about the development of these CDEs.
Organized by domains and sub-domains, often used in clinical studies, data standards include:
- CDEs — Classified as Core, Supplemental–Highly Recommended, Supplemental, or Exploratory
- CRF Modules — Template forms that logically organize CDEs for data collection
- Guidance Documents — Provide further information about the CDEs
- Instrument Notice of Copyright (NOC) Documents — Include pertinent information on recommended instrument (Instrument Notice of Copyright Information)
Neuromuscular Diseases Start-up Resource Listing: All Core and Supplemental–Highly Recommended CDEs recommended for Neuromuscular Diseases study start-up
Neuromuscular Diseases Highlight Summary: Overview of all Neuromuscular Diseases-specific CDE recommendations as they appear on the website.
Click Expand All to view the CDEs associated with the CRF modules, organized by domain and subdomain.
Overview
The first set of Common Data Elements (CDEs) for Neuromuscular Diseases was developed in 2011.
To create a comprehensive set of CDEs for Neuromuscular Diseases, the NINDS formed the following unique Working Groups: Neuromuscular Diseases (NMD), Myasthenia Gravis (MG), Spinal Muscular Atrophy (SMA), and Duchenne/Becker Muscular Dystrophy (DMD/BMD).
The NMD CDE Working Group’s goal was to identify elements that would be useful across multiple types of NMD Clinical Studies. To achieve this objective, the NMD Working Group was divided into two focus areas – Adult studies and Pediatric studies. In both the Adult and Pediatric areas, subgroups were formed to focus on defining data elements in the domains of:
- Biomarkers/Genetics/Imaging
- Cardiac/Pulmonary
- Clinical Outcomes
- Cognitive/Behavioral/Psychological Assessments
- Demographics
While the Adult and Pediatric Working Groups initially worked separately, their age-specific NMD recommendations were later combined to form Version 1.0 of the NMD CDEs.
Additionally, the NINDS formed Working Groups to develop recommendations specific for Spinal Muscular Atrophy (SMA), Myasthenia Gravis (MG), and Duchenne and Becker Muscular Dystrophy (DMD/BMD). These three Working Groups were responsible for reviewing the NMD CDEs and forming disease-specific recommendations to supplement the overarching Neuromuscular Diseases CDEs.
Prior to these NMD Working Groups, a Congenital Muscular Dystrophy (CMD) Working Group was developed in 2010 focusing on CDEs for a natural history study in CMD. These CDEs are not incorporated with the NMD Diseases CDEs but are available as a zip file on the CMD Overview page.
Roster
The National Institute of Neurological Disorders and Stroke (NINDS) has formed an Oversight Committee to direct the updating and further development of Neuromuscular Diseases CDEs.
The Neuromuscular Diseases CDE Committee was formed first and focused on a set of general CDE recommendations to be utilized in clinical research across all neuromuscular diseases. Additional CDE Working Groups were subsequently formed to develop disease-specific recommendations for:
- Myasthenia Gravis (MG)
- Spinal Muscular Atrophy (SMA)
- Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (DMD/BMD)
- Congenital Muscular Dystrophy (CMD)
All three Working Groups (MG, SMA, and DMD/BMD) were charged with reviewing the combined Adult and Pediatric NMD CDEs to determine the additional elements needed for an MG, SMA, or DMD/BMD specific study.
All Working Group members actively developed the CDEs for their specific disease areas/subgroups and had an opportunity to review and comment on the draft recommendations internally prior to public review and posting. The NMD, MG, SMA, and DMD/BMD CDE Working Groups are supported by the NINDS CDE Team. The complete rosters for each Working Group and the rosters by NMD Subgroup are shown below. The Neuromuscular Diseases CDE Oversight Committee was created in 2013.
Publications
A publication related to the NMD CDE initiative is in development. The citation for this publication will be included on this site as soon as it is available.
Updates
Please see attached revision history document. Please contact NINDSCDE@emmes.com if you require further information or have any questions about the revision history.