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 & CompanySept 2004

23 Sept 20042004 FDA/Industry Statistics Workshop

Company ConfidentialCopyright © 2000 Eli Lilly and Company

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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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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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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

http://www.infomerchant.net/creditcardprocessing/accept_credit_cards.html



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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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FDA News, Press ReleaseJuly 21, 2004

• 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.

FDA Draft Guideline on Study Data Specifications

http://www.fda.gov/default.htm

http://www.fda.gov/default.htm

http://www.hhs.gov/



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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

The dog eats red meat.

The dog eats blue trees.

Give the patient pain medication.

Give the patient medication for pain.

Time flies like an arrow.

Fruit flies like a banana.



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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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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

CDISC in the “World of Standards”

SNOMED

MedDRA

= Document Standard, or Architecture

Protocol Std

ClinicalDocument

Architecture

DICOM

ADaM

International Conference on Harmonization (ICH) U.S. Dept. of Health and Human Services

(HHS)

Health Level 7 (HL7)

U.S. FDA

CDISC

TC:RCRIM

NIH/NCI NLM

EFPIA

EMEA MHLWKIKO

PhRMAJPMA

CDC

Reference Information Model

RIM

LAB

eCTD

LOINC

ISO

ODMSDS

= Organization= Dictionary, Codelist = Standard = Model



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CDISC/HL7 Goal

Every physician is a potential investigator.

Every patient is a potential trial participant.

Research data at the point of clinical care.

Clinical care data at the point of research.



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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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Opportunities for Statistics

Adaptive randomization designs

Integratable data across• Studies• Compounds• Companies (e.g. FDA)

Rolling submissions



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Summary

Standards

IntegratedData

Automated Tools

Information

Knowledge

Documents

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