eCTD TECHNICAL CONFORMANCE GUIDE

1 ectd TECHNICAL CONFORMANCE GUIDE TECHNICAL Specifications Document This Document is incorporated by reference into the following Guidance Document(s): Guidance for Industry Providing Regulatory Submissions in Electronic Format Certain Human Pharmaceutical Product Applications and Related Submissions Using the ectd Specifications For questions regarding this TECHNICAL specifications document, contact CDER at or CBER at Department of Health and Human Services food and drug administration Center for drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER) July 2020 ectd TECHNICAL CONFORMANCE GUIDE July 2020 REVISION HISTORY DATE VERSION SUMMARY OF REVISIONS October 2015 I n it ia l Versio n September 2016 Updated/Clarified following sections.

2 Section (Tra nsit io nin g to e C TD Format an d Resubmissio n of Non- ectd Documents), a nd su bsections , , , Section ( ectd Leaf Titles) Section (Presubmissions) Section (Rolling Submission) Section (Cover Letter a nd Reviewer s GUIDE ) Section (I nfo rmation Amendments) Section (Letters of Authorization) Section (Field Co p y Certif icatio n) Section (Stu dy Repo rts) Section (Combining Multiple or Sections wit h Sim ila r M etadata) Section (Co mbinatio n Pro d ucts) References Removed section: Section (Datasets) Added sections: Section (Resubmissio n o f n on- ectd d o cuments ) Section (Websites, Electronic Interactive Programs, a nd Electronic Detail Aids) November 2017 Updated/Clarified following sections: Section (Transitioning from us-regional to us-regional ) Section (Resubmission of non- ectd d o cuments) Section ( ectd Life Cycle) Section (Cover Letter and Reviewer s GUIDE ) Section (Datasets) Section (M5 Stu dy Reports) Section (Periodic Safety Reports) Section (Datasets) Added sections: Section 0 (REMS) November 2018 Updated/Clarified following sections: Section ( ectd Submission Tracking and Lifecycle) Section (Study Tagging Files) Section (REMS) Added sections: (Regenerative Medicine Advanced Therapy Designation) December 2019 Updated/Clarified following sections: Section (Mo dule 4 - No n clin ica l) Section (Mo dule 5 - Clin ica l) Removed section: Section 3.

3 (Letters of Au t ho rizat ion) Added sections: 2 .9 (M a st er Files) (Pa tient Experience Da ta) DATE VERSION SUMMARY OF REVISIONS July 2020 Updated section: Section (REMS) i Table of Contents 1. BACKGRO UND ..1 PURPO S DO CUM ENT REV I SI O N AND RELATIONSHIP TO OTHER DO CUM ENTS ..2 2. GENERAL CONSIDERATIONS ..3 ectd ectd TRANS I TI O NI NG TO ectd FORMAT AND RESUBMISSION OF NON- ectd DO CUM ENTS ..3 Transitioning from Paper to ectd using us-regional Transitioning from Paper to ectd using us-regional ..3 Transitioning from us-regional to us-regional ..4 Resubmission of non- ectd ectd LE AF TI TLES ..4 ectd SUBMISSION TRACKI NG AND LIFE CYCLE ..5 PRESUBMISSIONS ..5 ROLLI NG STUDY TAGGI NG FI MAS TER FI L ES ..6 3. ORGANIZATION OF THE ectd .

4 7 MO DULE 1 ADM I NIS TRA TIV E INF O RM ATIO N AND PRES CRI BI NG INFORMATION ..7 FDA Regional ectd Backbone Files .. 7 Cover Letter and Reviewers GUIDE .. 7 Patient Experience Data ..8 Cross Referencing Previously Submitted Information that is not in ectd Format ..8 Advertisements and Promotional Labeling Material .. 10 Marketing Annual 11 Information 11 Field Copy Certification .. 12 Risk Evaluation and Mitigation Strategy (REMS) .. 12 Regenerative Medicine Advanced Therapy (RMAT) Designation .. 14 MO DULE 2 SUM MA RI ES .. 14 Bioequivalence Summary 14 MO DULE 3 QUA LI TY .. 14 L ot Distribution 14 Literature References .. 14 MO DULE 4 NO NCLI NI CAL .. 14 Study 14 Literature References .. 15 Datasets .. 15 MO DULE 5 CLINICAL.

5 15 Tabular Listing of All Clinical Studies .. 15 Study 16 Case Report Forms (CRFs) .. 16 Periodic Safety Reports .. 17 IND Safety Reports .. 17 Literature References .. 17 ii Datasets .. 17 4. ISSUES AND SOLUTIONS .. 18 ISSUE: COM BI NI NG MULTI PLE O R SECTIO NS WI TH SIM I LA R METADATA .. 18 ISSUE: CLI NI CAL STUDY REPO RT SUBMITTED IN INCO RRECT SECTI O N .. 18 ISSUE: NOT APPLICABLE (N/A) O R UNAS S I GNED FO LDERS I N MO DULE 4 O R 5 .. 18 ISSUE: MULTI PLE SIM I LA R STF STRUCTURES DISPLAYING IN MO DULE 4 O R 19 5. COMBINATION 20 REFERENCES .. 24 RELATED 25 Contains Nonbinding Recommendations 1 ectd TECHNICAL CONFORMANCE GUIDE This TECHNICAL specifications document represents the current thinking of the food and drug administration (FDA or Agency) on this topic.

6 It does not create any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. To discuss an alternative approach, con tact the FDA staff responsible for implementing this specifications document by email at or 1. Introduction Background This ectd TECHNICAL Conf ormance GUIDE ( GUIDE ) provides specifications, recommendations, and general considerations on how to submit electronic Common TECHNICAL Document ( ectd )-based electronic submissions to the Center for drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER). The GUIDE supplements the guidance for industry Providing Regulatory Submissions in Electronic Format Certain Human Pharmaceutical Product Applications and Related Submissions Using the ectd Specifications ( ectd Gu id ance).

7 1 The ectd Guidance implements the electronic submission requirements of section 745A(a) of the Federal food , drug , and Cosmetic Act (FD&C Act) with respect to electronic submissions f or certain investigational new drug applications (INDs); new drug applications (NDAs); abbreviated new drug applications (ANDAs); certain biologics license applications (BLAs); and master files submitted to CDER or CBER. These submissions may apply to combination products with CDER or CBER as the lead Purpose This GUIDE provides TECHNICAL recommendations to sponsors and applicants for the standardized electronic submission format of INDs, NDAs, ANDAs, BLAs, and master f iles. The GUIDE is intended to complement and promote interactions between sponsors and applicants and FDA s electronic submission support staff.

8 However, it is not intended to replace the need for sponsors and applicants to communicate directly with support staff regarding implementation approaches or issues relating to electronic submissions. Because of the inherent variability across studies and applications, it is dif f icult to identif y all issues that may occur related to the preparation and transmission of electronic submissions. Therefore, prior to submission, sponsors and applicants should discuss questions with the appropriate center s electronic submission support staff within the appropriate center CDER: or CBER: 1 A link to this document can be found on the FDA ectd website (h / ectd ) 2 See 2 1 CFR Pa rt 3 . Co m binatio n p ro ducts a re comprised o f a ny co mbinatio n o f a d ru g a nd a m ed ical device; a medical device and a biological product; a biological product and a drug ; or a drug , a medical device, and a biological product.

9 Combination products are assigned to a lead center for review; see 21 CFR Contains Nonbinding Recommendations 2 Document Revision and Control FDA issued an initial Federal Register notice announcing availability of this GUIDE for public comment on its contents. Future revisions will be posted directly on the ectd website3 and the revision history page of this document will contain sufficient information to indicate which sections of the GUIDE have been revised. Relationship to Other Documents This GUIDE integrates and updates information discussed pre viously in the ectd Guidance and other specifications documents (including Agency presentations). The examples of issues and concerns discussed in the GUIDE are intended as examples only of common issues, not an inclusive list of all possible issues.

10 This GUIDE should be considered a companion document to the following: Guidance to Industry Providing Regulatory Submissions in Electronic Format Certain Human Pharmaceutical Product Applications and Related Submissions Using the ectd Specif ications FDA ectd Website FDA Data Standards Catalog ectd Submission Standards 3 FDA ectd website ( ) Contains Nonbinding Recommendations 3 2. General Considerations ectd Publishing Submissions in the ectd format should be created following all applicable guidances and specifications available on our ectd website: ectd Samples Samples of eCTDs can be submitted for feedback on document placement, navigation, and effective use of metadata and Study Tagging Files (STFs). For ectd samples and instructions, please refer to our ectd Basics and Getting Started website located at: Transitioning to ectd Format and Resubmission of non- ectd Documents When transitioning to ectd format, you are not required to resubmit documents already submitted in paper or other electronic format.