title: Common data elements for clinical research in Friedreich's ataxia.
abstract: To reduce study start-up time, increase data sharing, and assist investigators conducting clinical studies, the National Institute of Neurological Disorders and Stroke embarked on an initiative to create common data elements for neuroscience clinical research. The Common Data Element Team developed general common data elements, which are commonly collected in clinical studies regardless of therapeutic area, such as demographics. In the present project, we applied such approaches to data collection in Friedreich's ataxia (FRDA), a neurological disorder that involves multiple organ systems. To develop FRDA common data elements, FRDA experts formed a working group and subgroups to define elements in the following: ataxia and performance measures; biomarkers; cardiac and other clinical outcomes; and demographics, laboratory tests, and medical history. The basic development process included identification of international experts in FRDA clinical research, meeting by teleconference to develop a draft of standardized common data elements recommendations, vetting of recommendations across the subgroups, and dissemination of recommendations to the research community for public comment. The full recommendations were published online in September 2011 at http://www.commondataelements.ninds.nih.gov/FA.aspx. The subgroups' recommendations are classified as core, supplemental, or exploratory. Template case report forms were created for many of the core tests. The present set of data elements should ideally lead to decreased initiation time for clinical research studies and greater ability to compare and analyze data across studies. Their incorporation into new, ongoing studies will be assessed in an ongoing fashion to define their utility in FRDA.
authors.affiliation: Department of Neurology, University of Pennsylvania and the Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA. lynchd@mail.med.upenn.edu
authors.forename: David R
authors.initials: DR
authors.lastname: Lynch
authors.forename: Massimo
authors.initials: M
authors.lastname: Pandolfo
authors.forename: Jorg B
authors.initials: JB
authors.lastname: Schulz
authors.forename: Susan
authors.initials: S
authors.lastname: Perlman
authors.forename: Martin B
authors.initials: MB
authors.lastname: Delatycki
authors.forename: R Mark
authors.initials: RM
authors.lastname: Payne
authors.forename: Robert
authors.initials: R
authors.lastname: Shaddy
authors.forename: Kenneth H
authors.initials: KH
authors.lastname: Fischbeck
authors.forename: Jennifer
authors.initials: J
authors.lastname: Farmer
authors.forename: Paul
authors.initials: P
authors.lastname: Kantor
authors.forename: Subha V
authors.initials: SV
authors.lastname: Raman
authors.forename: Lisa
authors.initials: L
authors.lastname: Hunegs
authors.forename: Joanne
authors.initials: J
authors.lastname: Odenkirchen
authors.forename: Kristy
authors.initials: K
authors.lastname: Miller
authors.forename: Petra
authors.initials: P
authors.lastname: Kaufmann
mesh_terms: D000046:Academic Medical Centers; D015415:Biomarkers; D035843:Biomedical Research / Q000706:statistics & numerical data*; D003625:Data Collection; D003627:Data Interpretation, Statistical; D016208:Databases, Factual; D003710:Demography; D005621:Friedreich Ataxia / Q000145:classification / Q000175:diagnosis / Q000628:therapy*; D006331:Heart Diseases / Q000209:etiology; D006801:Humans; D007391:International Cooperation; D055825:National Institute of Neurological Disorders and Stroke (U.S.); D009316:National Institutes of Health (U.S.); D009460:Neurologic Examination; D009462:Neurology / Q000706:statistics & numerical data; D011597:Psychomotor Performance / Q000502:physiology; D012015:Reference Standards; D013685:Telecommunications; D009626:Terminology as Topic; D016896:Treatment Outcome; D014481:United States
chemical_list: D015415:Biomarkers