one mind, traumatic brain injury research and CDISC Standards
Posted October 23, 2014
CDISC NEWSLETTER – October 2014 — Traumatic Brain Injury (TBI) is a growing global public health and medical problem. The World Health Organization (WHO) estimates that TBI will surpass many diseases as the major cause of death and disability by the year 2020. According to C Bryan-Hancock and J Harrison (2013), “the global incidence rate of TBI is estimated at 200 per 100 000 people per year; however, this rate is uncertain and a likely underestimate”. TBI not only affects patients, but also is a significant burden on caregivers and family members, as well as society as a whole. TBI is a complex syndrome; different types of trauma can produce varying types of injuries and symptoms. According to the Centers for Disease Control the most common causes of TBI are falls, unknown causes, unintentional blunt trauma, motor vehicle accidents and assaults. These injuries can range from diffuse damage, not visible on imaging, to hematomas and skull fractures.
TBI symptoms can present as mild, moderate, or severe. The NINDS website lists the symptoms of the various degrees of TBI. Depending on the injury, treatment required may be minimal or may include interventions such as medications, emergency surgery or surgery years after the initial impact/injury. Treatments currently utilized include physical therapy, speech therapy, recreation therapy, occupational therapy and vision therapy. To date, there are no FDA approved medications to treat the cumulative symptoms of TBI.
The International TBI Community is conducting several ongoing multi-center prospective, observational studies involving thousands of patients in the United States and Europe. The aims of these studies, TRACK-TBI and CENTER-TBI, include data sharing and the development of repositories for neuroimaging, proteomic, and genetic biomarkers. TBI research is extremely challenging due to fact that the disease can manifest so differently across patients. Current TBI studies are primarily focused on comparative effectiveness research. The goals of these studies are to compare different interventions and strategies to prevent, diagnose, treat and monitor health conditions. This can guide patients, providers, and decision-makers in choosing interventions that are most effective for individual patients under specific circumstances.
One Mind™ plays a leading role in the research, funding, marketing and public awareness of brain injury, and seeks to “accelerate the development and implementation of improved diagnostics, treatments, and cures for all affected by brain illness and injury.” Recently One Mind highlighted the importance of “Connecting the Dots”, where One Mind is building on the efforts of the TBI research community, National Institutes of Health (NIH), and the Department of Defense (DoD) to develop TBI Common Data Elements (TBI-CDEs). Financial support from One Mind is helping to fund conversion of TBI-CDEs to internationally accepted CDISC standards, a format approved by the US Food & Drug Administration (FDA). This work is expected to reduce the time required for FDA acceptance of research findings and accelerate the pace at which new diagnostics and treatments reach the patient.
Each year One Mind brings together top scientists, philanthropists, pharmaceutical and healthcare leaders, and government decision-makers to explore ways to accelerate treatments and cures for brain disease and injury such as TBI. The 3rd Annual One Mind Summit (May 13-15, Washington DC) was focused on accelerating open science and addressing barriers and incentives to sharing data in medical research. The goals were to examine how current systems for recording, storing and sharing data could be reengineered to make them more useful for sharing and for integration into meta-analysis and other ‘big data’ projects that can advance knowledge. Given the importance of data standards in this ecosystem, CDISC received a special invitation to participate in the summit.
The One Mind Summit kicked off with a CEO Update from General Peter Chiarelli, U.S. Army (Retired), Chief Executive Office, One Mind. General Chiarelli will give the opening keynote at the upcoming CDISC International Interchange on 12 November.
The One Mind Summit included 130 participants as well as renowned keynote speakers and expert panelists, including:
- Honorable Patrick J. Kennedy, Co-founder, One Mind
- Honorable Chaka Fattah, Congressman, U.S. House of Representatives
- Dr. Michel Goldman, Executive Director, Innovative Medicines Initiative
- James Heywood, Chairman & Co-Founder, PatientsLikeMe
- Dr. Geoff Manley, Professor and Vice Chairman of Neurological Surgery, UCSF
- Leaders from across the National Institutes of Health…Dr. Christopher Austin (NIH), Dr. Shai Silberberg (NINDS), Dr. Thomas Insel (NIMH) & Dr. Philip Bourne (NIH)
Bron Kisler (CDISC VP, Strategic Initiatives) spoke on one of the panels – Research Funders-Grant Criteria and Grant Governance.
This provided an opportunity to speak to a new audience about CDISC; highlight progress in developing therapeutic area data standards; as well as experiences working collaboratively with other organizations on data sharing initiatives:
- Partnership with the Critical Path Institute
- CFAST (Coalition for Accelerating Standards and Therapies) and progress on therapeutic area data standards
- FDA therapeutic area priorities for standardization, including TBI and Post Traumatic Stress
- Partnership with the National Cancer Institute Enterprise Vocabulary Services
- Experiences working with the PKD Foundation and successful integration of PKD legacy data toward identification of a new biomarker
- Coalition Against Major Diseases and accelerating drug development for patients with chronic neurodegenerative diseases (e.g. Alzheimer’s & Parkinson’s Disease) through the sharing and integration of pre-competitive clinical trials data
- The vision of the National Institute of Neurological Disorders & Stroke toward data standardization, and experiences working collaboratively on Parkinson’s Disease, Multiple Sclerosis and Traumatic Brain Injury
- The Innovative Medicines Initiative consortium eTRIKS (European Translational Information and Knowledge Management Services), using CDISC standards and the TranSMART platform
Bron made three commitments on behalf of CDISC: (1) development of a TBI version 1.0 data standard before the One Mind Summit in 2015; (2) work with One Mind leadership to further expand collaboration around TBI e.g CFAST, FDA; and (3) through the IMI eTRIKS project, work toward alignment of CDISC standards and the tranSMART platform, which is also being implemented by One Mind.
In his closing remarks, General Chiarelli (Retired) reiterated the importance of TBI data standards, galvanizing commitment and progress toward development of a TBI Data Standard, funded by One Mind. This will result in the publication of the first set of global standards for Traumatic Brain Injury. CDISC is working closely with TBI researchers and NINDS.
TBI v1 deliverables - Translating TBI-CDEs into CDISC Standards
CDISC plans to release the first version of the TBI Therapeutic Area User Guide (TAUG-TBI) in the second quarter of 2015. This TAUG-TBI v1 will be focused on the most commonly collected data used across TBI trials. These standards will facilitate clinical information about the brain injury, imaging and neurological findings, and patient outcomes. This User Guide will also include SDTM and CDASH metadata, CDASH annotated CRFs, and SDTM examples. Accompanying these standards will be a set of standardized terminology and Clinical Outcome Assessment (COA) Supplements to the SDTMIG for approximately twenty-five of the most commonly used instruments.
Version 1.0 of the TAUG-TBI is scheduled to be released in Q2 2015. The public review version of the document should be available in late Q1 2015 or early Q2 2015 and will be announced on the CDISC website.
C Bryan-Hancock, J Harrison. The global burden of traumatic brain injury: preliminary results from the Global Burden of Disease Project. Inj Prev 2010;16:Suppl 1 A17 doi:10.1136/ip.2010.029215.61
About One Mind
One Mind is an independent, 501(c)(3) non-profit organization dedicated to benefiting all affected by brain illness and injury through fostering fundamental changes that will radically accelerate the development and implementation of improved diagnostics, treatments, and cures; while eliminating the stigma. One Mind believes in open science principles and creates global public-private partnerships between governmental, corporate, scientific, and philanthropic communities. Visit us at www.onemind.org or follow us via Twitter or Facebook.
About Cavendish Global
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