Standardized Study Data: An Update

Presented at theDCDISC Meeting

Ron Fitzmartin, PhD, MBAOffice of Strategic ProgramsCenter for Drug Evaluation and ResearchU.S. Food and Drug Administration 25 April 2014

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Disclaimer

The views and opinions presented here represent those of the speaker and should not be considered

to represent advice or guidance on behalf of the Food and Drug Administration.

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Steps Toward the Submission of Electronic Standardized Study Data

Data Standards @ the Foundation of Review

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TraceabilityData Quality

Regulatory Review

Data Standards Protocol Data Capture CSR Submission

Validation

Predictability

Com

mon

Tools

Com

munication

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Fiscal Year 2013:Number and Percent of NDAs with

Study Data Submissions in CDISC SDTM*

NDAs# of

Submissions % with

CDISC SDTM DataNew Unique NDAs** 98 61%

All NDAs 223 55%

* One or more explicitly stated SDTM studies (or study data structure that resembled SDTM).

**New unique NDAs with data submissions (not previously submitted).Source: Office of Business Informatics

A Glimpse at Progress:Standardized Study Data in FDA

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Data Standards Governance

Automated ReviewEnvironment

Communication

Support GuidesData Standards Catalog

Help

Web Pages

eDataTeam

* examples

External Collaborations*

CSC

Project Operations

Strategy, Policy & Guidance

PDUFA V Goals – Section

XII

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“…develop

standardized clinical

data terminology

through open

standards

development

organizations (i.e.,

CDISC)”

“… periodically publish

final guidance specifying

the completed data

standards, formats,

and terminologies that

sponsors must use to

submit data in

applications.”

FDASIA Re-authorized

PDUFA*

*PDUFA: Prescription Drug User Fee Act

Draft Binding Guidances &Tech Guide

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Public Comment Period Ends: 7 May 2014

What is Binding Guidance?

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Binding Guidance =

Guidance = • Recommends,• Suggests, • Encourages

• Requires

Do it! Binding Guidance

has a bite!

“Please”

• Electronic submission of standardized study data required for: – Certain INDs– NDAs– ANDAs– Certain BLAs

• Including amendments, supplements, and reports

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eStudy Data Guidance

Study Data Tech Conformance Guide

11http://www.fda.gov/forindustry/datastandards/studydatastandards/default.htm

• Guide supplements the eStudy guidance.

• Provides technical recommendations / specifications for “e” submission of standardized data.

• Consolidates Common Issues and Study Data Specs + much more.

Data Standards Catalog

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eStudy Data Guidance

Dates Requirement

Begins and Ends

Therapeutic Area (TA)Standards Project Plan

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• Adopt or adapt existing TA data elements

• Harmonize with healthcare standards, as possible

• Implementation Guidance

• Standardized Study Data will become Binding

• Collaborate with SDOs & TA Experts

• Ensure Public Comment

• Identify efficacy endpoints

• Manageable increments

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FDA TA Standards Objectives

Express Requirements in Sustainable

Standards

Open and Transparent

Process

Establish FDA’s

requirements

Implement in Guidance to Industry

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Collaboration on TA Standards Development

Academia Government

IndustryClinical / Medical

Societiesjoint

partnership

active participants

Coalition for Accelerating Standards & Therapies

FDA’s Role in the TA Project Stages

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• Scientific & Technical Input• Planning / Prioritization• Initial Expert Review

• FDA Division Expert Review

• FDA Testing / Acceptance

• Guidance

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FDA’s TA Efficacy Requirements Process

Plan & Scope with the Review Division

Efficacy Requirements

Mapping & Report

Acceptance Testing of

CDISC Published TA

Tech Guide &

Data Standards Catalog

Communicate Results to

CFAST

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• Multiple Sclerosis• Diabetes• QT Studies• Traumatic Brain

Injury• Parkinson’s**• Virology**• Major Depressive

Disorder

• Schizophrenia• Influenza• COPD• Pain**• Alzheimer’s**

• Tuberculosis**• Polycystic Kidney

Disease*• Asthma*

• Rheumatoid Arthritis

• Diabetic Nephropathy

• Renal Transplant• Osteoporosis

• Hepatitis C• Oncology-

Breast• Lipid Lowering

• Complicated intra-abdominal infection

• Complicated skin infections

• ADHD• Irritable Bowel Disorder• Anticonvulsants• Comp Urinary Infection

• Anticoagulants for Atrial Fibulation

• Prevention of Pregnancy• Treatment of Erectile

Dysfunction• Acne

Early Look @ Status*of TA Initiative

*Approved-Ongoing & Planned Projects or Published as of April 2014; **Published by CDISC

0102030405060708090

100 ~58 TAs%

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Thank You

Ronald.Fitzmartin@fda.hhs.govand the Data Standards Team