preparing fda submission data packages · · 2017-12-25preparing fda submission data packages...

Raising the Standard of Excellence

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Preparing FDA Submission Data Packages

Yuguang Zhao, MS

Senior Vice President, Programming and Development

Tiepu Liu, MD, PhD

President of Global Biometrics


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Contents

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............................................................................................................................................................................... 4

CDISC FDA: ............................................................................................................................................... 7

....................................................................................................................... 9

............................................................................................................................................. 11

SDSP - ............................................................................................................................... 11

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eCTD .................................................................................................................................................... 12

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Food and Drug Administration : FDA 2016 12 17

New Drug Applications : NDAs

Biologics License Applications : BLAs Abbreviated New Drug Application :

ANDAs Standard for Exchange of Nonclinical Data : SEND

Study Data Tabulation Model : SDTM Analysis Data Model :

ADaM CDISC Clinical Data Interchange Standards Consortium

2017 12 17 INDs

1

FDA

FDA

2

CDISC FDA

FDA FDA

3

CDISC

FDA

FDA


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1998 4 FDA Center for Drug Evaluation and Research : CDER

Providing Regulatory Submissions in

Electronic Format-NDAs 4 1997 9

NDA

1998 FDA CBER

BLA PLA ELA

5

1999 FDA CDER CBER Guidance for Industry Providing Regulatory Submissions in Electronic

Format — General Considerations 6

NDA BLA

FDA

Common Technical Document CTD CTD International

Conference on Harmonization ICH 7 ICH CTD 5

1 1 2 5


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1 8

ICH

CTD

eCTD

CDISC

CDISC

CDISC

9


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2004 7 FDA eCTD CDISC

2006 12 CDISC SDTM

2015 5 FDA Providing Regulatory Submissions in Electronic Format – Certain Human

Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications (revision 3) 10

FDA NDA BLA

ICH FDA CDISC


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CDISC FDA:

FDA CDISC

CDISC

SDS – Submission Data Standards

ADaM – Analysis Data Model

SEND – Standard for Exchange of Non-clinical Data

CDASH – Clinical Data Acquisition Standards Harmonization

FDA

“The Food and Drug Administration Safety and Innovation Act” FDA

2012 7

”The Prescription Drug User Fee Act : PDUFA V”

5 CDISC

FDA PDUFA 11

2014 12 FDA “Providing Regulatory Submissions in Electronic Format — Standardized Study Data

12” 4 Data Standard Catalog


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FDA 13

Data Standards Catalog

Study Data Technical Conformance Guide

FDA Specific SEND Validation Rules

FDA Specific SDTM Validation Rules

Data Standards Catalog

FDA FDA

Study Data Technical Conformance Guide Study Data Specifications document CDER Common

Data Standards Issues document

NDA

BLA RTF Refuse-To-File ANDAs RRS Refuse-To-Receive

24 36

IND NDA ANDA BLA FDA

2016 12 17

2017 12 17 NDA / BLA ANDA

RTF RTR

FDA ICH eCTD CDISC FDA


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SEND SDTM

ADaM

:

SEND Data Package SDTM Data Package ADaM Data Package acrf.pdf .xpt files Define.xml Study Data Reviewer’s Guide (nSDRG.pdf)

acrf.pdf .xpt files Define.xml Study Data Reviewer’s Guide (cSDRG.pdf)

.xpt files Define.xml Analysis Data Reviewer’s Guide (ADRG.pdf)

Study Data Technical Conformance Guide 14

:


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SDSP -

2014 12 FDA Study Data Standardization Plan: SDSP

SDSP

SDSP

FDA

SDSP FDA

.

The Pharmaceutical Users Software Exchange (PhUSE)

SDSP 15 FDA

CSR

SDTM ADaM CSR

Integrated Summaries of Safety and Efficacy (ISS/ISE)

SDTM ADaM ISS/ISE SDTM

ADaM WHO drug MedDRA


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FDA FDA

pre-IND IND

CDER CBER FDA 16 NDA

II pre-

NDA / BLA

eCTD

FDA

FDA

RTF RTR

SDSP

FDA

FDA CDISC

/

CDISC

17


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CDISC

75 70-90

12 2

FDA

FDA


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1 Providing Regulatory Submissions in Electronic Format — Submissions Under Section 745A(a) of the Federal

Food, Drug, and Cosmetic Act, Guidance for Industry, U.S. Department of Health and Human Services Food and

Drug Administration Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and

Research (CBER), December 2014.

2 US FDA Website: http://www.fda.gov/AboutFDA/WhatWeDo/, FDA Mission, accessed January 18, 2017.

3 Providing Regulatory Submissions in Electronic Format — Standardized Study Data, Draft Guidance. U.S.

Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and

Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological

Health (CDRH), February 2012.

4 Guidance for Industry Providing Regulatory Submissions in Electronic Format — NDAs, Draft Guidance, U.S.


Research (CDER), April 1998

5 Electronic Submissions of a Biologics License Application (BLA), Product License Application (PLA)

/Establishment License Application (ELA) to the Center for Biologics Evaluation and Research (63 FR 29741,

6/1/98).

Electronic Submissions of Case Report Forms (CRFs), Case Report Tabulations(CRTs) and Data to the Center for

Biologics Evaluation and Research (63 FR 29739; 6/1/98).

Pilot Program for Electronic Investigational New Drug (eIND) Applications for Biological Products (63 FR 29740;

6/1/98).

Instructions for Submitting Electronic Lot Release Protocols to the Center for Biologics Evaluation and Research

(63 FR 29742; 6/98).

6 Guidance for Industry Providing Regulatory Submissions in Electronic Format — General Considerations, U.S.


Research (CDER), Center for Biologic Evaluation and Research (CBER), January 1999.

7 International Conference on Harmonization Website: http://www.ich.org/about/history.html, accessed,

January 18, 2017

8 International Conference on Harmonization Website: http://www.ich.org/products/ctd.html, accessed,

January 18, 2017.

9 Study Data Standards for Regulatory Submissions Position Statement, September 15, 2003, FDA Website:

http://www.fda.gov/ForIndustry/DataStandards/StudyDataStandards/ucm368613.htm, Position Statement,

accessed January 18, 2017.

10 Providing Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product

Applications and Related Submissions Using the eCTD Specifications Guidance for Industry, U.S. Department of

Health and Human Services Food and Drug Administration Center for Drug Evaluation and Research (CDER),

Center for Biologics Evaluation and Research (CBER), May 2015, Electronic Submissions, Revision 3.

11 Study Data Standards for Regulatory Submissions Position Statement, September 15, 2003, FDA Website:

http://www.fda.gov/ForIndustry/DataStandards/StudyDataStandards/ucm368613.htm, Position Statement,



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12 Providing Regulatory Submissions In Electronic Format — Standardized Study Data, Guidance for Industry,

U.S. Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and

Research (CDER), Center for Biologics Evaluation and Research (CBER), December 2014, Electronic

Submissions.

13 Study Data Standards Resources, FDA Website:

http://www.fda.gov/forindustry/datastandards/studydatastandards/default.htm, Important Notices, accessed

January 18, 2017.

14 Study Data Technical Conformance Guide, Technical Specifications Document, U.S. Department of Health

and Human Services Food and Drug Administration Center for Drug Evaluation and Research (CDER), Center for

Biologics Evaluation and Research (CBER), November 2016.

15 Study Data Standardization Plan, PhUSE Website:

http://www.phusewiki.org/wiki/index.php?title=Study_Data_Standardization_Plan_(SDSP), Project

Deliverables, accessed January 18, 2017.

16 CDER/CBER Study Data Standardization Plan Recommendations, FDA Website:

http://www.fda.gov/downloads/ForIndustry/DataStandards/StudyDataStandards/UCM447119.pdf, accessed

January 18, 2017.

17 2014 CDISC Business Case, Executive Summary, CDISC Website:

https://www.cdisc.org/system/files/all/article/PDF/2014%20Business%20Case_Executive%20Summary.pdf,



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Yuguang Zhao, MS

Senior Vice President of Programming and Development

Yuguang Zhao is the Senior Vice President of Programming and

Development at FMD K&L. Mr. Zhao is responsible for providing strategic

planning and corporate direction for continued growth of the company,

providing scientific leadership and resource planning for statistical support

on all projects, and ensuring the teams provide high quality statistical

programming support for Phase I, II, III, and IV clinical trials. He has over 19

years of experience in the pharmaceutical industry and has been very

active in industry working groups. He is used to be a member in the CDISC

SDS team and has received FDA Leveraging/Collaboration Award for the

participation of creation of SDTM and accompanying implementation guide

in 2005. He has co-led the CDISC/FDA data integration pilot. For questions

on preparing FDA Submission data packages, please contact Yuguang at:

[email protected].

Tiepu Liu, MD, PhD

President of Global Biometrics

Tiepu Liu, MD, PhD, is the President of Global Biometrics at FMD K&L. Dr.

Liu has served as Director of Biostatistics at PPD, Executive Director of

Statistics and Data Management at UBC, and a Senior Director of

Biostatistics at Graceway Pharmaceuticals and The Medicines Company,

before joining FMD K&L to lead the Biostatistics and Data Management

functions. Dr. Liu has published 100 scientific papers and more than 100

abstracts and presentations. He has served on numerous scientific review

committees and panels for the National Institute of Health (NIH) and other

government agencies. Dr. Liu has led numerous clinical development

projects, interacting with various regulatory agencies and Data Monitoring

Committees. He has been active in CDISC standards and CFAST initiative.

For questions on preparing FDA submission data packages, please contact

Tiepu at: [email protected].


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preparing fda submission data packages · · 2017-12-25preparing fda submission data packages...

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