2019 China Interchange Beijing, China | 19-20 July 2019 · CDISC RWD Connect - Expert Advisory...
Transcript of 2019 China Interchange Beijing, China | 19-20 July 2019 · CDISC RWD Connect - Expert Advisory...
2019 China InterchangeBeijing, China | 19-20 July 2019
Rhonda FacileVp, Development Opportunities
19-20 September 2019
Agenda
• Standards Development Update
• Current Projects
• Tools to get started
• CDISC RWD Connect
Publishing Soon!
• ADaMIG v1.2
• CDASHIG 2.1
• CDASH Model v1.1
Standards Published Since the Last Year’s China Interchange
Foundational Standards
• SDTM v1.7
• SDTMIG v3.3
• SDTMIG-Medical Devices v1.1
• ADaM Conformance Rules v2.0
• Define.XML v2.1
Therapeutic Area Standards
• HIV
• PTSD
• Colorectal Cancer
• Huntington's Disease
• CDAD
• Lung Cancer
• Nutrition
Congratulations on the publication of the TAUG TCM CAD-Angina v1.0
https://www.cdisc.org/standards/therapeutic-areas/traditional-chinese-
medicine-coronary-artery-disease-angina
LOINC-CDISC Mapping
CDISC CT TEAM HAS COMPLETED THE LOINC-CDISC
LB MAPPING
CONTAINS OVER 2,000 LB
MAPPINGS
REVIEW BY THE REGENSTRIEF
INSTITUTE
SENT TO THE FDA LOINC WORKING
GROUP FOR INTERNAL REVIEW
MAPPING WILL BE RELEASED FOR
PUBLIC COMMENT
The Unified Code for Units of Measure (UCUM) Codelist Table
▪ A codelist table that allows users to toggle between UCUM and CDISC Unit codelists
▪ For each published CDISC submission value across all UNIT-based codelists in SDTM terminology, the mapping provides valid UCUM expressions associated with the unit concept
▪ Allows for a collected UCUM-compliant unit of measure to be easily matched to the appropriate CDISC submission value associated with that concept
Codetable Mapping Files
Temperature
F
C
cm
▪ Built to show explicit
relationships between
terms in different
codelists
▪ DS, CV, EG,
TU/TR/RS (Oncology)
Race/Ethnicity,
SC, TS, VS
What Are We Working on Now?
Foundational Standards in Development
Conformance Rules
SDTM v2.0SENDIG
v3.1.1ADaM
OCCDS v1.1
ADaM Data Structures for
IntegrationSDTM v3.4
SDTM Variable
Definitions
Multiple QRS and ADQRS Supplements
CDASH-SAE Supplement
v2.0
9#CDISCChina | #ClearDataClearImpact
Therapeutic Area Standards in Development
Type 1 Diabetes v1
Crohn's Disease v1
Acute Kidney
Injury v1Psoriasis v1
TCM Acupuncture
v1
Heart Failure v1
Pancreatic Cancer v1
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Publication of Known Issues
Errata
• Small errors such as typos that do not affect conformance
• Errors that affect conformance
Known Issues
• Inconsistencies within a CDISC standard
• Inconsistencies between standards
• Inconsistencies between a CDISC standard and regulatory agency advice
• Gaps in standards
Publication of Errata and Known Issues
Provide possible
solutions
Vetted and approved by
the CDISC Global
Governance Group
Will be made available in
real-time to the user
community
Curated Non-Standard Variable (NSV) Registry
Will be made available in
real-time to the
user community
Enables consistency
in the absence
of a standard variable
Will contain all non-
standard variables used in
CDISC Implementation
and User Guides
Vetted by the CDISC
Global Governance
Group
Consideration Documents for Regulatory Requirements
Race and ethnicity
Representation of SI and
conventional units
QRS logically
skipped items
PhUSE/CDISC Collaboration Primer
How to get started with CDISC
Linking of standards
Traceability
Compliance
CDISC Primer
SDTM Reorganization
Update outdated language
Move domain-specific variables
Make metadata consistent and separated
• Definitions will be added, and definition-like material will be removed from description column
Will be published in November 2020 as SDTM v2.0
Development of Principles
Guides and improves understanding of the standard
Facilitates decision making
Helps to optimize utilization of resources
Helps to manage change
Principles Hierarchy
CDISC Organization Principles
Standards Development High-Level Principles
Technical Team High-Level Principles
Cross Team and within Team Technical Implementation Principles and Rules
Accomplish the CDISC goals and mission without
promoting any individual vendor or organization
Manage scope with incremental development
Start with the end in mind
Domain variables based on the same root variable
have the same meaning in all domains
New! CDISC Knowledge Base
CDISC Knowledge
Base
An open, assessible, searchable
and user-friendly interface on
the CDISC Website to host new and
existing website content for
CDISC implementers
Knowledge Base – Components
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Coordinated with redeveloped
standards website pages
CDISC-generatedcontent - technical articles written by CDISC staff and
teams
Community-generated
content, curated by CDISC
Community questions, with curated CDISC team answers
Links to external useful content
Initial Launch end of October, new content added as it become available.
Tools to Get Started
Conformance Rules
• CDISC authors rules that determine conformance with the standard by version of standard
• Regulatory agencies have their own rules specific to their needs, these may or may not be the same as CDISC authored conformance rules
• Historical development of rules• Standards were written before the rules which led to ambiguity
• The guides were specific to a team and standard
Future of Conformance Rules
RULES ARE NOW REQUIRED AT THE TIME OF PUBLICATION
OF ANY STANDARD
WORKING ON ESTABLISHING CROSS-TEAM RULES
DEVELOPMENT METHODS
SUPPORTED BY CDISC LIBRARY
Disclaimer: This is only a proposed draft snapshot.
Clear and transparent
conformance rules
Clear and transparent
associations (assumptions,
examples, etc.)
A catalog of cross-standard
conformance rules
Redesigned Conformance Rules
Curated Example Collection
• Examples exist across many Implementation and User Guides in PDFs
• Goal is to make examples accessible through CDISC Library
• Users will be able to select examples by biomedical concept, therapeutic area, observation class, domain etc.
• Curation to ensure examples remain consistent with one another
• Initial scope: Diabetes v1.0, Diabetic Kidney Disease v1.0, Type 1 Diabetes v1.0
Curated Examples
Disclaimer: This is only a proposed draft
snapshot.
CDISC RWD Connect
Current Landscape
For years, the pharmaceutical industry has been using CDISC data standards for new drug submissions to regulatory agencies. Currently, CDISC standards are required for electronic submission in the USA and Japan and are recommended in Europe and China.
In academic and public health settings, there are few recommended standards for health data, and many activities do not implement data standards.
Non-standardized data is collected in different formats and different terminology, which does not allow for the data to be consolidated, compared and shared.
This disconnect creates an evidence gap that slows scientific and public health advances.
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Potential Benefits of using of CDISC Standards
Bring structure and semantic clarity to collected data
Be able to make better use of the data, increase the value of their data
Encourage a culture of FAIR guiding principles1
Open the door for a myriad of opportunities for data sharing within your institution and with others
Safe and effective treatments for patients - faster
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1 Wilkinson, M, Dumontier, M, et. al. (2016). The FAIR Guiding Principles for scientific data management and
stewardship. Science Data 3, article number: 160018. Retrieved from https://www.nature.com/articles/sdata201618
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• Overarching Goals from the Blue Ribbon Commission:
• Listen to the academic community to understand the barriers to
implementing CDISC standards for RWD
• Develop a vision and strategy for the implementation of CDISC
standards in academic settings.
• Make the tools and guidance we agree are needed a reality
• Research Questions:
• What are the priorities, needs, and challenges of academics around
the use of data standards?
• How can CDISC lower the barriers to implementing CDISC
standards?
• What are the requirements for potential tools and educational
materials for implementation support?
CDISC RWD Connect
CDISC RWD Connect - Guiding Principles
Foster a better foundation for data sharing and support platform-agnostic data sharing
Standardize where possible first, focus on core cross-cutting concepts, then extend as appropriate
Encourage: free open source, or affordable, easy to use tools.
Help investigators to focus on the science, and not on standardization
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Research Activities
involving human health data (to produce generalizable knowledge)
Non-research Activitiesinvolving human health data
(not to produce generalizable knowledge)
Public health
activities
Routine
healthcare
services
Health data
self-monitoring/
self-gathering
Mobile and
wearables
Electronic
health records
Billing records
Public health
registries
Census and
vital events
Experimental
studies
Observational
studies
Household
surveys
National Health
Accounts
Regulated
studies
Non-
regulated
studies
CDISC Standards Today CDISC RWD Connect Broad Focus Area
Health claimsGenetic & other
databases
Real-World Data(non-experimental setting)
What is RWD?
Other sources of
patient-reported
outcomes
Case-control
studies
Cohort studies
Cross-sectional
studies
Other types of
studies
Research
registries
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Experimental
studies
Observational
studies
Routine health
services
Public health
activities
Self-monitoring or
self-gathering
activities
Research activities
Activities involving human health data
Non-research activities
Case Report Forms
from experimental
studies
Case Report Forms
from observational
studies
Electronic health
recordsFinancial records
Registries, census,
health surveys
Mobile, wearables,
genetic self-tests, etc
Sources of data
Main data source
Other data sources
Possible data sources
Real-World Data(non-experimental setting)
Activities involving human health data and their different data sources
CDISC RWD Connect - Overall Plan and Timeline
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Phase Timeline Activities
Phase 1 Q3 2019 –
Q4 2020
• Convene an international Expert Advisory Board
• Engage the Expert Advisory Board in discussion using the Delphi method
• Develop 2-3 use cases of that demonstrate the use CDISC standards in RWD
settings
• Draft a white paper/articles documenting methods, findings and vision, strategy
and policy recommendations for the use of standards in academic settings
• Collect requirements and raise funds for building tools for academics settings.
Phase 2 2021-2022
• Develop tools and training materials.
• Test and validate tools and training materials.
• Develop a special journal supplement on data standards for RWD with
contributions from the Expert Advisory Board and partners.
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Region Academic & Research Government
Americas
Harvard Medical School
Massachussets General Hospital
University of California at San Francisco
Duke University
University of Chicago
University Arkansas Medical School
George Washington University
RedCap
SAS Institute
Pan American Health Organization / World Health Organization
National Cancer Institute (NCI)
National Institutes of Health (NIH)
Food and Drug Administration (FDA)
Patient-Centered Outcomes Research Institute (PCORI)
Europe
Oxford University
Innovative Medicines Initiative (IMI)
Liverpool Clinical Trials Research Centre
European Medicines Agency (EMA)
Asia
Daegu University Medical School, South Korea
Institute of Health Data Infrastructure for All (IDIAL)
Nagoya University Medical Center
Sapporo Medical University
Nagoya University Medical Center
Pharmaceutical and Medical Devices Agency (PMDA)
Japan Agency for Medical Research and development (AMED)
National Institute of Public Health (Japan)
The Leona M. and Harry B. Helmsley Foundation (US)
Bill and Melinda Gates Foundation (US)
Wellcome Trust (UK)
Cohen’s Veterans Network (US)
CDISC RWD Connect - Expert Advisory Board
CDISC RWD Connect - Expert Advisory BoardThe Ask
Participate in a kick-off and final project meeting.
Participate in 3 rounds of qualitative Delphi surveys (modified).
Co-author publications (articles, white paper) on vision, strategy and policy recommendations for the use of standards in academic settings.
Assist in identifying potential tool development partners and fundraising sources.
Time needed: ~ 2-4 hours to answer the first Delphi round and 1-2 hours for second and third rounds, for a total time commitment of 4-8 hours.
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CDISC RWD Connect – The Delphi Process
Use Case 1 – CDISC for Public Health Programs
PAHO HEARTS Project• Exploring implementing CDASH core variables to treat
hypertension in the HEARTS project.
• Potential strategic partner for CDISC:
• Could open the doors to all countries in Latin America and the
Caribbean
• It is part of the United Nations and WHO
• If use case is successful, it could lead to official PAHO
recommendation of CDISC standards for Ministries of Health.
• Status:
✓ Face-to-face meeting (July 25)
✓ CDISC developed CRF visualization and metadata example
table and shared with PAHO (August 9)
✓ CDISC sent proposed work plan (August 9, PAHO is
currently reviewing)
• Outcome: development of a joint manuscript on the application of
CDISC standards for HEARTS public health program.
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Use Case 2 – CDISC for Observational Research Studies
Potential - Infectious Diseases Data Observatory (IDDO)
• IDDO generates new research evidence through data re-use.
• IDDO works with researchers based in disease-endemic countries to aggregate, standardize and analyze existing health data to answer questions that can only be addressed through collaborative analysis.
• The IDDO data-sharing platform accepts clinical, laboratory, observational, and epidemiological data on poverty-related and emerging diseases.
• IDDO is using SDTM as the standard for their platform (work in progress).
• Status:
• CDISC to reach out to IDDO about jointly drafting a manuscript on the experience of using CDISC for RWD.
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Use Case 3 – CDISC for Registry Data
Evaluating potential Use Cases
• Literature review - Registries used for patient enrollment (Incl/Exclcriteria)
• TID Registry
• Others
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Research
registries
Use Case 4 - FHIR
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• Article review:
• The use of HL7 FHIR as eSource to Pre-populate CDASH Case Report Forms using a CDISC ODM API.
• Use of Fast Healthcare Interoperability Resources (FHIR) in the Generation of Real World Evidence (RWE)
• Use of FHIR in Clinical Research: From Electronic Medical Records to Analysis
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Experimental
studies
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