Transcript Evolving Data Standards: Implications for Statistical Analysis and Review Stephen J. Ruberg, Ph.D. Eli Lilly & Company Sept 2004

Evolving Data Standards:
Implications for Statistical
Analysis and Review
Stephen J. Ruberg, Ph.D.
Eli Lilly & Company
Sept 2004
Outline
Background
The Language of Clinical Data
CDISC
•
•
CDISC Mission / Scope
The World of Standards
CDISC & HL7
•
•
Goals
Computable, Semantic Interoperability
Implications for Statistics
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2004 FDA/Industry Statistics Workshop
Company Confidential
Copyright © 2000 Eli Lilly and Company
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In the beginning ...
And the Lord said “Behold, they have all one
language…. Nothing that they now propose will be
impossible for them. Come, let us go down and there
confuse their language that they may not understand
one another’s speech.”
Genesis 11
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2004 FDA/Industry Statistics Workshop
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The World is Getting Smaller
What enables you to travel
the world and still be able
to use your credit card at
most ATMs?
Standardization of the
electronic banking world
This creates value for the
banks and the users
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Pharma 2010
•
IBM Business Consulting
Services analysis of the
pharma industry in 2010
•
New forms of data
management, integrated
databases and network of
communication channels to
share clinical data
Increased use of rolling
dossiers
Pervasive connectivity with
patients requires data
standards and interoperable
environments
•
•
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2004 FDA/Industry Statistics Workshop
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FDA News, Press Release
July 21, 2004
FDA Draft Guideline on Study Data Specifications
• Study Data Tabulation Model (SDTM) developed by
CDISC can be used for Clinical Trial Submission Data
•
“... Having the data presented in a standard structure
will improve FDA’s ability to evaluate the data and
help speed new discoveries to the public.” Lester M.
Crawford, Acting FDA Commissioner.
•
FDA is currently exploring regulatory approaches to
require the use of the STDM standard for regulatory
submissions.
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The Language of Clinical Trials
Language has two components
•
•
Structure: syntax, grammatical rules
Content: vocabulary
Vocabulary has attributes
•
•
•
Well-defined words; spelling
Nouns, verbs, etc.
Definition, meaning
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Syntax vs. Semantics
Time flies like an arrow.
Fruit flies like a banana.
The dog eats red meat.
The dog eats blue trees.
Give the patient pain medication.
Give the patient medication for pain.
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2004 FDA/Industry Statistics Workshop
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Copyright © 2000 Eli Lilly and Company
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CDISC Vision - Simply Stated
A future conversation on data interchange:
anyone: How do you want me to send the data?
everyone: We are using CDISC version 4.3.
anyone: I can do that.
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2004 FDA/Industry Statistics Workshop
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Copyright © 2000 Eli Lilly and Company
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CDISC Mission
CDISC is an open, multidisciplinary, non-profit
organization committed to the development of industry
standards to support the electronic acquisition,
exchange, submission and archiving of clinical trials
data and metadata for medical and biopharmaceutical
product development.
The mission of CDISC is to lead the development of
global, vendor-neutral, platform independent standards
to improve data quality and accelerate product
development in our industry.
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CDISC Mission
Extending to all aspect of pharmaceutical drug
development
•
All clinical trial data
• Protocol elements
• Stability studies
• Toxicology data
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CDISC in the “World of Standards”
International Conference on
Harmonization (ICH)
EFPIA
JPMA
EMEA
MHLW
KIKO
U.S. Dept. of Health and Human Services
(HHS)
PhRMA
U.S. FDA
Protocol Std
ADaM SDS ODM
MedDRA
NLM
Health Level 7 (HL7)
Reference
Information Model
RIM
ISO
LOINC
SNOMED
Clinical
Document
Architecture
eCTD
= Organization
LAB
NIH/NCI
DICOM
TC:
RCRIM
CDISC
CDC
= Dictionary,
Codelist
= Standard
= Model
= Document Standard,
or Architecture
CDISC/HL7 Goal
Research data at the point of clinical care.
Clinical care data at the point of research.
Every physician is a potential investigator.
Every patient is a potential trial participant.
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Challenge for Statistics
A world of computable, semantic interoperability
•
Access to electronic medical records
Analysis of data from controlled trials and naturalistic
settings
•
•
More data
More unstructured data
A few patients from many investigators
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2004 FDA/Industry Statistics Workshop
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Opportunities for Statistics
Adaptive randomization designs
Integratable data across
•
•
•
Studies
Compounds
Companies (e.g. FDA)
Rolling submissions
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Summary
Knowledge
Information
Integrated
Data
Automated
Tools
Standards
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