Public Review Period for Facioscapulohumeral Muscular Dystrophy CDEs: November 20,
2014 - January 16, 2015
The Facioscapulohumeral Muscular Dystrophy (FSHD) CDE Working Groups and the National
Institute of Neurological Disorders and Stroke (NINDS) CDE Team have released a
draft version (Pre-release Version 0.0) of the FSHD CDEs for public review.
After the public review period, the FSHD CDE Working Group will review and revise
the recommendations as needed. Version 1.0 of the FSHD recommendations will be posted
at the end of February 2015. Please note that changes are still
being incorporated and final documents will not be available until February 2015.
Facioscapulohumeral Muscular Dystrophy CDE Public Review
Facioscapulohumeral Muscular Dystrophy Common Data Element Public Review Package
The Pre-release Version 0.0 Facioscapulohumeral Muscular Dystrophy Public Review
package consists of one zip file containing the following documents:
- Case Report Form (CRF) Modules (each with a corresponding CDE Details spreadsheet,
in pdf format).
- CRF modules are template data collection forms which include the CDEs identified
and defined by the FSHD Working Group. The CRF modules were drafted by the NINDS
CDE Team using the CDEs identified by the FSHD Working Group and can be used as the
building blocks for study data collection forms.
- Instrument Recommendations Spreadsheets
- The Instrument Recommendations spreadsheet includes description and scoring information
on the instrument/scales for outcomes in FSHD .
- Guideline Documents (when applicable)
- Notice of Copyrights and instrument recommendations
- Including the following details:
- Name of Instrument
- Short description
- Comments/ Special Instructions
- Copyright Information
*Each CDE or instrument should be classified according to the definitions below:
General Core: A data element that is required
for all NINDS funded studies.
Disease Core: A data element that collects
essential information applicable to any disease-specific study, including all therapeutic
areas. The NINDS and its appointed working groups assign the disease “Core” classification
based on the current clinical research best practices. In each case, the disease
Core CDEs are a small subset of the available CDEs, where it is anticipated that
investigators will need to collect the disease Core CDEs on any type of study. These
are required for all disease-specific studies.
Disease Supplemental - Highly Recommended:
A data element which is essential based on certain conditions or study types in
clinical research studies. In most cases, these have been used and validated in
the disease area. These data elements are required for the specified disease condition,
study type or design.
Disease Supplemental: A data element which
is commonly collected in clinical research studies. Use depends upon the study design,
protocol or type of research involved. These are recommended, but not required,
Disease Exploratory: A data element that
requires further validation, but may fill current gaps in the CDEs and/or substitute
for an existing CDE once validation is complete. Such data elements show great promise,
but require further validation before they are ready for prime-time use in clinical
research studies. They are reasonable to use with the understanding that it has
limited validation in the target group.
Many of the CDEs will overlap across study types, which allows for comparisons and
meta-analysis across studies. Consistency of the data elements and the CDE formats
is kept in order to ensure the ability to transfer critical medical information
electronically from one center to another. This consistency also allows for continuity
across different disease areas. The goals of the NINDS CDE initiative are to increase
the efficiency and effectiveness of clinical research studies and clinical treatment,
increase data quality, facilitate data sharing, and help educate new clinical investigators
Please send all comments to NINDSCDE@emmes.com
or to Joanne Odenkirchen (firstname.lastname@example.org)
by January 16, 2015.
Prospective Assessments for Suicidal Ideation and Behavior
Investigators should review the FDA’s "Guidance for Industry: Suicidal Ideation and Behavior: Prospective Assessment of Occurrence in Clinical Trials” for the most up-to-date information about suicidal ideation and behavior.
One scale that FDA suggests is the Columbia Suicide Severity Rating Scale (C-SSRS) (available
The NINDS CDE Team has requested, and in many cases received, permission to post
proprietary instruments/scales recommended by the CDE Working Groups to this site.
When permission is granted, the documents are posted in the exact format they were
received in order to maintain the validity of the instrument. If approval is not
granted for the instrument to be reproduced on this site, with the author/copyright
holder’s permission, a link is posted to an external Web site where users can get
more information about the instrument. If you have difficulty accessing either the
proprietary instruments/scales or the external links, please contact the
NINDS CDE Project Officer, Joanne Odenkirchen.
- John T. Kissel, MD - Ohio State University, Columbus, OH, USA
- George W.A.M. Padberg, MD - Nijmegen, Netherlands
- Enzo Ricci, MD - Universita Cattolica del Sacro Cuore, Rome, Italy
- Sabrina Sacconi, MD - Centre Hopitalier Universitaire de Nice Sophia Antipolis, Nice, France
- Jeffrey Statland, MD - University of Kansas Medical Center, Kansas, USA
- Rabi Tawil, MD - University of Rochester Medical Center, Rochester, NY, USA
- Baziel G. M. van Engelen, MD - Neuromuscular Centre Nijmegen, Nijmegen, Netherlands
- Kathryn R. Wagner, MD, PhD - Kennedy Krieger Institute, Baltimore, MD, USA
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NIH Coordinating Team
- Robin Conwit, MD - National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
- Elizabeth McNeil, MD, MSc - National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
- Joanne Odenkirchen, MPH - NINDS CDE Project Officer, National Institutes of Health/National Institute of Neurological Disorders and Stroke (NIH/NINDS), Bethesda, Maryland, USA
- John Porter, PhD - National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
- Yaffa Rubenstein PhD - NCATS/NIH
- Tiina Urv, PhD - NICHD/NIH
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NINDS CDE Team
- Sherita Ala'I, MS - The EMMES Corporation, Rockville, MD, USA
- Marybeth Montoro, MPH, CPH - The EMMES Corporation, Rockville, MD, USA
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Note: Institutions for all individuals acknowledged in this section
were those they belonged to when they joined the Committee. All Committee
members were asked to voluntarily disclose all potential sources of bias in their
involvement with the NINDS CDE Project. The summary disclosure information is available
upon request by contacting NINDSCDE@emmes.com.