SCGE Platform - Investigational New Drug (IND) Templates

Common Technical Document (CTD)

The eCTD contains an electronic table of contents also referred to as a backbone that manages all the metadata for an application. This backbone is broken down into five modules. Documents are placed appropriately into modules, which are graphically presented as the CTD Triangle.

Module 1 references regional information such as forms, cover letter, labeling, and investigational brochures.

Module 2 references summaries such as quality, clinical, and non-clinical summaries.

Module 3 references quality information.

Module 4 references non-clinical information.

Module 5 references clinical information.

Comprehensive Table of Contents Headings and Hierarchy

General Resources for Formatting an IND

Individual Templates

FDA Submission Elements

Module1 is for administrative information and prescribing information, and should contain documents that are specific to each region; for example, application forms or the proposed label for use in the region.

Section				Section Name	Required for submission		Submission Timing	Guidance	Regulation	Templates	Examples
					Initial IND	Marketing
Module1
1.1				Forms	Yes			Content and Format of INDs for Phase 1 Studies
	1.1.1			Form FDA 1571: Investigational New Drug Application (IND)	Yes		Include with every submission (initial IND AND all amendments)	Instructions for Forms: FDA 1571, FDA 1572, FDA 3674	Form FDA 1571	FDA Form 1571	Module 1.1 Instructions for FDA Forms 1571, 1572, 3674
	1.1.2			Form FDA 356h: Application to Market a New Drug, Biologic, or an Antibiotic Drug for Human Use	No	Yes	Include with marketing applications only		Applications for Biologics Licenses; Procedures for Filing Form FDA 356h Form FDA 356h
	1.1.3			Form FDA 3397: User Fee Cover Sheet	No	Yes	Include with marketing applications only
	1.1.4			Form FDA 2252: Transmittal of Annual Reports for Drugs and Biologics for Human Use	No	Yes	Include with marketing applications only		Form FDA 2252
	1.1.5			Form FDA 2253: Transmittal of Advertisements and Promotional Labeling for Drugs and Biologics for Human Use	No	Yes	Include with marketing applications, CDER INDs only		Form FDA 2253 Form FDA 2253
	1.1.6			Form FDA 2567: Transmittal of Labels and Circulars	No	Yes	Include with marketing applications only
	1.1.7			Form FDA 3674: Certification of Compliance	Yes		Include with initial IND and IND amendments (if applicable)	Civil Money Penalties Relating to the ClinicalTrials.gov Data Bank Instructions for Form FDA 3674 Instructions for Forms: FDA 1571, FDA 1572, FDA 3674	Form FDA 3674 Violations relating to the submission of certifications and/or clinical trial information	FDA Form 3674	Module 1.1 Instructions for FDA Forms 1571, 1572, 3674
	1.1.8			Form FDA 3792: Biosimilar User Fee Cover Sheet (BsUFA)	No	Yes	Required for biosimilars or generic drug applications only
	1.1.9			Form FDA 3794: Generic Drug User Fee Cover Sheet (GDUFA)	No	Yes	Required for biosimilars or generic drug applications only
	1.1.10			Form 1572: Statement of Investigator	Maybe		Include with initial IND and IND amendments (if applicable)	Instructions for Forms: FDA 1571, FDA 1572, FDA 3674 Statement of Investigator Frequently Asked Questions	Obtaining Information from the Investigator — Form 1572 Protocol amendments — New investigator	FDA Form 1572	Module 1.1 Instructions for FDA Forms 1571, 1572, 3674
1.2				Cover Letter	Yes				Content and Format of Information Amendment
1.3				Administrative information	Yes
	1.3.1			Contact/Sponsor/Applicant Information	Yes		Initial IND only			Module 1.3.1: Contact Information for Sponsor
		1.3.1.1		Change of Address or Corporate Name	No		Include in IND amendment (if applicable)
		1.3.1.2		Change in Contact/Agent	No		Include in IND amendment (if applicable)
		1.3.1.3		Change in Sponsor	No		Include in IND amendment (if applicable)
		1.3.1.4		Transfer of Obligation	No		Include in IND amendment (if applicable)		Transfer of Obligations to a Contract Research Organization Transfer of Obligations to a Contract Research Organization
		1.3.1.5		Change in Ownership of an Application	No	Yes	Include with marketing applications only		Change in Ownership of an Application
	1.3.2			Field Copy Certification	No	Yes	Include with marketing applications only		Field Copy Certification
	1.3.3			Debarment Certification	No	Yes	Include with marketing applications only	Submitting Debarment Certification Statements	Civil Money Penalties for Actions in Connection with Debarred Persons Debarment Certification
	1.3.4			Financial certification and disclosure	No	Yes	Include with marketing applications only	Financial Disclosure by Clinical Investigators	Financial certification and disclosure Financial Disclosure By Clinical Investigators
	1.3.5			Patent and exclusivity	No	Yes
		1.3.5.1		Patent Information	No	Yes	Include with marketing applications only		Patent Information Patent Information
		1.3.5.2		Patent Certification	No	Yes	Include with marketing applications only		Patent Certification
		1.3.5.3		Exclusivity request	No	Yes	Include with marketing applications only	Reference Product Exclusivity for Biological Products Filed Under Section 351(a) of the PHS Act
	1.3.6			Tropical disease priority review voucher	No		Not applicable to gene therapies
1.4				References	Maybe				Master Files in Biologics License Applications
	1.4.1			Letter of authorization	Maybe		Include in initial IND or amendments (if applicable)
	1.4.2			Statement of right of reference	Maybe		Include in initial IND or amendments (if applicable)
	1.4.3			List of authorized persons to incorporate by reference	Maybe		Include in initial IND or amendments (if applicable)
	1.4.4			Cross reference to previously submitted information	Maybe		Include in initial IND or amendments (if applicable)
1.5				Application Status	No
	1.5.1			Withdrawal of an IND	No		Include in IND amendment (if applicable)		Withdrawal of an IND
	1.5.2			Inactivation request	No		Include in IND amendment (if applicable)		Administrative Actions — Inactive Status Inactive Status
	1.5.3			Reactivation request	No		Include in IND amendment (if applicable)		Reactivation Request
	1.5.4			Reinstatement request	No		Include in IND amendment (if applicable)
	1.5.5			Withdrawal of an unapproved BLA, NDA, ANDA, or Supplement	No	Yes	Include with marketing applications only
	1.5.6			Withdrawal of listed drug	No	Yes	Include with marketing applications only
	1.5.7			Withdrawal of approval of an application or revocation of license	No	Yes	Include with marketing applications only		Withdrawal from Sale of Approved Biological Products
1.6				Meetings	Maybe				Administrative Actions — Meetings Early Consultation — End-of-Phase 1 Meetings Meetings and correspondence
	1.6.1			Meeting request	Maybe		Initial IND or IND amendments (if applicable)
	1.6.2			Meeting background materials	Maybe		Initial IND or IND amendments (if applicable)
	1.6.3			Correspondence regarding meetings	Maybe		Initial IND or IND amendments (if applicable)
1.7				Fast Track	Maybe
	1.7.1			Fast track designation request	Maybe		Any submission prior to marketing application	Expedited Programs for Serious Conditions		Module 1.7.1: Fast Track Designation Request
	1.7.2			Fast track designation withdrawal request	No		IND amendment only (if applicable)
	1.7.3			Rolling review request	No	Yes	Include with marketing applications only
	1.7.4			Correspondence regarding fast track/rolling review	No	Yes	Include with marketing applications only
1.8				Special protocol assessment request	No			Special Protocol Assessment
	1.8.1			Clinical study	No		Any submission prior to marketing application
	1.8.2			Carcinogenicity study	No		Any submission prior to marketing application
	1.8.3			Stability study	No		Any submission prior to marketing application
	1.8.4			Animal efficacy study for approval under the animal rule	No		Any submission prior to marketing application
1.9				Pediatric administrative information	No			Guidance on Pediatric Study Plans Pediatric Labeling Information PREA Compliance	Labeling Requirements — Pediatric Studies Pediatric Research Equity Act of 2003
	1.9.1			Request for waiver of pediatric studies	No		Any submission prior to marketing application		Requests for Waiver of Pediatric Studies
	1.9.2			Request for deferral of pediatric studies	No		Any submission prior to marketing application		Requests for Deferral of Pediatric Studies
	1.9.3			Request for pediatric exclusivity determination	No		Any submission prior to marketing application
	1.9.4			Proposed pediatric study request and amendments	No		Any submission prior to marketing application
	1.9.6			Other correspondence regarding pediatric exclusivity or study plans	No		Any submission prior to marketing application
1.10				Dispute Resolution	Maybe			Instructions for Formal Dispute Resolution Process	Administrative Actions — Dispute Resolution
	1.10.1			Request for dispute resolution	Maybe		Initial IND or IND amendments (if applicable)
	1.10.2			Correspondence related to dispute resolution	Maybe		Initial IND or IND amendments (if applicable)
1.11				Information Amendment: Information not Covered under Modules 2 to 5	No				Information Amendments
	1.11.1			Quality information amendment	No		IND amendment only (if applicable)
	1.11.2			Nonclinical/Safety information amendment	No		IND amendment only (if applicable)
	1.11.3			Clinical/Efficacy information amendment	No		IND amendment only (if applicable)
	1.11.4			Multiple module information amendment	No		IND amendment only (if applicable)
1.12				Other correspondence	Maybe
	1.12.1			Pre IND correspondence	Maybe		Initial IND (if applicable)		Early Consultation — Pre-IND Meetings
	1.12.2			Request to charge for clinical trial	Maybe		Initial IND or IND amendments (if applicable)	Charging for Investigational Drugs Under an IND	Charging for Investigational Drugs Under an IND	Module 1.12.2: Request to Charge for a Clinical Trial
	1.12.3			Request to charge for expanded access	Maybe		Initial IND or IND amendments (if applicable)	Charging for Investigational Drugs Under an IND	Charging for Investigational Drugs Under an IND	Module 1.12.3: Request to Charge for Expanded Access
	1.12.4			Request for comments and advice	Maybe		Initial IND or IND amendments (if applicable)		Administrative Actions — Comments and Advice on an IND
	1.12.5			Request for a waiver	Maybe		Initial IND or IND amendments (if applicable)	Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications Process for Submitting a Waiver to FDA	Waiver of labeling requirements Waiver requests Waivers of FDA Requirements for Foreign Clinical Trials Not Conducted Under an IND	Module 1.12.5: Request for a Waiver
	1.12.6			Exception from informed consent for emergency research	No		Not applicable to gene therapies	Exception from Informed Consent Requirements for Emergency Research	Exception from Informed Consent Requirements for Emergency Research IND Requirements involving exception from informed consent
	1.12.7			Public disclosure statement for exception from informed consent for emergency research	No		Not applicable to gene therapies
	1.12.8			Correspondence regarding exception from informed consent for research	No		Not applicable to gene therapies
	1.12.9			Notification of discontinuation of clinical trial	No		IND amendment only (if applicable)
	1.12.10			Generic drug enforcement act statement	No		Not applicable to gene therapies
	1.12.11			ANDA basis for submission statement	No		Not applicable to gene therapies	ANDA Submissions — Content and Format Referencing Approved Drugs in ANDA Submissions
	1.12.12			Comparison of generic drug and reference listed drug	No		Not applicable to gene therapies	ANDA Submissions — Content and Format
	1.12.13			Request for waiver for in vivo studies	No		Not applicable to gene therapies
	1.12.14			Environmental analysis	Yes		Required in initial IND and marketing application	Content and Format of INDs for Phase 1 Studies Environmental Assessments for Gene Therapies, Vectored Vaccines, and Related Recombinant Viral or Microbial Products	Environmental Impact Considerations — Human drugs and biologics IND Content and Format — Environmental Analysis Requirements	Module 1.12.14: Environmental Analysis
	1.12.15			Request for waiver of in vivo bioavailability studies	No		Not applicable to gene therapies		Criteria for Waiver of Evidence of In Vivo Bioavailability or Bioequivalence
	1.12.16			Field alert reports	No		IND amendment only (if applicable)	Field Alert Report Submission: Questions and Answers
	1.12.17			Orphan drug designation	Maybe		Initial IND or IND amendments (if applicable)		Content and Format of a Request for Orphan Drug Designation
	1.12.18			Regenerative medicine advanced therapy (RMAT) designation	No		IND amendments (if applicable)	Expedited Programs for Regenerative Medicine Therapies for Serious Conditions
1.13				Annual Report	No				Changes to an Approved BLA — Annual Reports Changes to an Approved BLA — General Considerations
	1.13.1			Summary for nonclinical studies	No		IND amendments (annual reports only and if applicable)		Annual Reports — Preclinical Studies
	1.13.2			Summary of clinical pharmacology information	No		IND amendments (annual reports only and if applicable)		Annual Reports — Clinical Pharmacology
	1.13.3			Summary of safety information	No		IND amendments (annual reports only and if applicable)		Annual Reports — Safety Information
	1.13.4			Summary of labeling changes	No		IND amendments (annual reports only and if applicable)		Changes to an Approved BLA — Labeling Changes in Annual Reports
	1.13.5			Summary of manufacturing changes	No		IND amendments (annual reports only and if applicable)		Annual Reports — Manufacturing or microbiological changes
	1.13.6			Summary of microbiological changes	No		IND amendments (annual reports only and if applicable)		Annual Reports — Manufacturing or microbiological changes
	1.13.7			Summary of other significant new information	No		IND amendments (annual reports only and if applicable)		Protocol amendments — Changes in a Protocol Protocol amendments — Content and Format Protocol amendments — New investigator Protocol amendments — New protocol
	1.13.8			Individual study information	No		IND amendments (annual reports only and if applicable)		Annual Reports — Individual Study Information
	1.13.9			General investigational plan	No		IND amendments (annual reports only and if applicable)		Annual Reports — General Investigational Plan
	1.13.10			Foreign marketing	No		IND amendments (annual reports only and if applicable)		Annual Reports — Foreign Marketing
	1.13.11			Distribution data	No		IND amendments (annual reports only and if applicable)
	1.13.12			Status of postmarketing study commitments and requirements	No	Yes	Marketing application amendments (annual reports only and if applicable)		Annual Progress Reports of Postmarketing Studies
	1.13.13			Status of other postmarketing studies and requirements	No	Yes	Marketing application amendments (annual reports only and if applicable)		Annual Progress Reports of Postmarketing Studies
	1.13.14			Log of outstanding regulatory business	No		IND amendments (annual reports only and if applicable)		Annual Reports — Outstanding Regulatory Business
	1.13.15			Development safety update report (DSUR)	No		IND amendments (annual reports only)	DSUR Guidance
1.14				Labeling	Yes				Biological Products — Labeling Standards Changes to an Approved BLA — Labeling Changes Date of Manufacture for Biological Products
	1.14.1			Draft Labeling	No	Yes
		1.14.1.1		Draft carton and container labels	No	Yes
		1.14.1.2		Annotated draft labeling text	No	Yes
		1.14.1.3		Draft labeling text	No	Yes
		1.14.1.4		Label comprehension studies	No	Yes	Only if required by FDA
		1.14.1.5		Labeling History	No	Yes
	1.14.2			Final labeling	No	Yes			Exemptions from adequate directions for use General Labeling Provisions — Location of Expiration Date General Labeling Provisions — Lot Numbers General Labeling Provisions — National Drug Code numbers General Labeling Provisions — Statement of Ingredients Requirements on content and format of labeling for human prescription drug and biological products Specific requirements on content and format of labeling for human prescription drugs and biological products Statement of dosage
		1.14.2.1		Final carton or container labels	No	Yes	Marketing applications only
		1.14.2.2		Final package insert (package inserts, patient information, Medication guides)	No	Yes	Marketing applications only
		1.14.2.3		Final labeling text	No	Yes	Marketing applications only
	1.14.3			Listed Drug Labeling	No	Yes
		1.14.3.1		Annotated comparison with listed drug	No	Yes	Included with generic drug applications
		1.14.3.2		Approved labeling text for listed drug	No	Yes	Included with generic drug applications
		1.14.3.3		Labeling text for reference listed drug	No	Yes	Included with generic drug applications
	1.14.4			Investigational drug labeling	Yes
		1.14.4.1		Investigational brochure	Maybe		Initial IND or IND amendments (if applicable)	Content and Format of INDs for Phase 1 Studies	IND Content and Format — Investigator's Brochure	BGTC Investigator's Brochure Template Module 1.14.4.1: Investigator's Brochure	Example Text (Elpida Therapeutics)
		1.14.4.2		Investigational drug labeling	Yes		Required in initial IND application or in amendments if changes are made	Content and Format of INDs for Phase 1 Studies	IND Content and Format — Labeling Labeling of an investigational new drug	Module 1.14.4.2: Investigational Drug Labeling
	1.14.5			Foreign labeling	No		Marketing applications (if applicable)
	1.14.6			Product labeling for 2253 submissions	No		Include with marketing applications, CDER INDs only	Promotional Labeling and Advertising Materials for Human Prescription Drugs	Prescription Drug Advertising Prescription Drug Marketing
1.15				Promotional material [promotional-material-audience-type]	No			Pediatric Labeling Information Promotional Labeling and Advertising Materials for Human Prescription Drugs Television Ad Review Program	Prescription Drug Advertising Prescription Drug Marketing
	1.15.1			Correspondence relating to promotional materials	No	Yes	Include in marketing application (if applicable)
		1.15.1.1		Request for advisory comments on launch materials	No	Yes	Include in marketing application (if applicable)
		1.15.1.2		Request for advisory comments on non-launch materials	No	Yes	Include in marketing application (if applicable)
		1.15.1.3		Presubmission of launch promotional materials for accelerated approval products	No	Yes	Include with marketing applications that are seeking accelerated approval designation (prior to launch)
		1.15.1.4		Presubmission of non-launch promotional materials for accelerated approval products	No	Yes	Include with marketing applications that are seeking accelerated approval designation (after launch phase)
		1.15.1.5		Pre-dissemination review of television ads	No	Yes	Include in marketing application (if applicable)
		1.15.1.6		Response to untitled letter or warning letter	No	Yes	Include in marketing application (if applicable)
		1.15.1.7		Response to information request	No	Yes	Include in marketing application (if applicable)
		1.15.1.8		Correspondence accompanying materials previously missing or rejected	No	Yes	Include in marketing application (if applicable)
		1.15.1.9		Withdrawal request	No	Yes	Include in marketing application (if applicable)
		1.15.1.10		Submission of annotated references	No	Yes	Include in marketing application (if applicable)
		1.15.1.11		General correspondence	No	Yes	Include in marketing application (if applicable)
	1.15.2			Materials attribute = [promotional-material-doc-type]	No	Yes	Include in marketing application (if applicable)
		1.15.2.1		Material = [promotional-material-type, material-id, issue-date]	No	Yes	Include in marketing application (if applicable)
			1.15.2.1.1	Clean version	No	Yes	Include in marketing application (if applicable)
			1.15.2.1.2	Annotated version	No	Yes	Include in marketing application (if applicable)
			1.15.2.1.3	Annotated labeling version	No	Yes	Include in marketing application (if applicable)
			1.15.2.1.4	Annotated references	No	Yes	Include in marketing application (if applicable)
1.16				Risk management plan	No
	1.16.1			Risk Management (non-REMS)	No		Not required, but FDA highly encourages sponsors to proactively manage known or anticipated risks
	1.16.2			Risk Evaluation and Mitigation Strategy (REMS)	No			REMS Assessment: Planning and Reporting	Risk Evaluation and Mitigation Strategies
		1.16.2.1		Final REMS	No	Yes	Include in marketing application, postmarketing amendment
		1.16.2.2		Draft REMS	No	Yes	Include in marketing application, postmarketing amendment
		1.16.2.3		REMS Assessment	No	Yes	Include in marketing application, postmarketing amendment
		1.16.2.4		REMS Assessment Methodology	No	Yes	Include in marketing application, postmarketing amendment
		1.16.2.5		REMS Correspondence	No	Yes	Include in marketing application, postmarketing amendment
		1.16.2.6		REMS Modification History	No	Yes	Include in marketing application, postmarketing amendment
1.17				Postmarketing studies	No			Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products
	1.17.1			Correspondence regarding postmarketing commitments	No	Yes	Marketing applications only
	1.17.2			Correspondence regarding postmarketing requirements	No	Yes	Marketing applications only
1.18				Proprietary names	No
	1.18.1			Proprietary names	No		At any point up to and including marketing application	Nonproprietary Naming of Biological Products	Selection of established name for new chemical entitites
	1.18.2			Biological Proper Name Suffix	No		At any point up to and including marketing application
1.19				Pre-EUA and EUA	No
1.20				General investigational plan for initial IND	Yes			Content and Format of INDs for Phase 1 Studies INDs Prepared and Submitted by Sponsor-Investigators	IND Content and Format — Introductory Statement and General Investigational Plan	Module 1.20: General Investigational Plan

Module 2 contains the CTD summaries and should begin with a general introduction to the drug, including its pharmacological class, mode of action and proposed clinical use. Module 2 should also provide the overall summary of the 'quality' information provided, the non-clinical overview and the clinical overview, as well as the non-clinical written summaries and the tabulated summaries, and the clinical summary.

Section			Section Name	Required for submission		Submission Timing	Guidance	Regulation	Templates	Examples
				Initial IND	Marketing
Module2
2.2			Introduction to Summary	Y			Content and Format of INDs for Phase 1 Studies	IND Content and Format — Introductory Statement and General Investigational Plan	m2.2_introduction.docx
2.3			Quality Overall Summary	Y					m2.3_quality_overall_summary.docx
	2.3.I		Introduction	Y		Initial IND application, revise as necessary			m2.3_qos_introduction.docx
	2.3.S		Drug Substance [substance (O), manufacturer (O)]	Y					m2.3_qos_drug_substance.docx
	2.3.P		Drug Product [product (O), dosage form (O)]	Y					m2.3_qos_drug_product.docx
	2.3.A		Appendices
		2.3.A.1	Facilities and Equipment [facility (O)]	Y		Initial IND application, revise as necessary		Facilities, Equipment, Animals and Care Good Laboratory Practice for Nonclinical Laboratory Studies — Equipment Good Laboratory Practice for Nonclinical Laboratory Studies — Facilities Quailty Assurance Unit	m2.3.A.1_facilities_equipment.docx
		2.3.A.2	Adventitious Agents Safety Evaluation [component (O)]	Y		Initial IND application, revise as necessary			m2.3.A.2_adventitious_agents.docx
		2.3.A.3	Excipients	M		Initial IND application, revise as necessary			m2.3.A.3_novel_excipients.docx
	2.3.R		Regional Information	M		Initial IND application, revise as necessary			m2.3.R_regional_information.docx
2.4			Nonclinical Overview	Y			Nonclinical Overview and Nonclinical Summaries of Module 2		m2.4_nonclinical_overview.docx
2.5			Clinical Overview	M			ICH Guideline on Efficacy		m2.5_clinical_overview.docx
2.6			Nonclinical Written and Tabulated Summaries				Nonclinical Overview and Nonclinical Summaries of Module 2
	2.6.1		Introduction	Y		Initial IND application, revise as necessary			m2.6.1_introduction.docx
	2.6.2		Pharmacology Written Summary	Y		Initial IND application, revise as necessary			m2.6.2_pharmacology_written_summary.docx
	2.6.3		Pharmacology Tabulated Summary	Y		Initial IND application, revise as necessary			m2.6.3_pharmacology_tabulated_summary.docx	Pharmacology Tabulated Summaries-Example Tables
	2.6.4		Pharmacokinetic Written Summary	M		Initial IND application, revise as necessary			m2.6.4_pharmacokinetics_written_summary.docx
	2.6.5		Pharmacokinetic Tabulated Summary	M		Initial IND application, revise as necessary			m2.6.5_pharmacokinetics_tabulated_summary.docx	Pharmacokinetics Tabulated Summaries-Example Tables
	2.6.6		Toxicology Written Summary	Y		Initial IND application, revise as necessary			m2.6.6_toxicology_written_summary.docx
	2.6.7		Toxicology Tabulated Summary	Y		Initial IND application, revise as necessary			m2.6.7_toxicology_tabulated_summary.docx	Toxicology Tabulated Summaries-Example Tables Toxicology Tabulated Summaries-Example Tables
2.7			Clinical Summary				ICH Guideline on Efficacy
	2.7.1		Summary of Biopharmaceutic Studies and Associated Analytical Methods	M		As early as initial IND application (see note), completed by marketing application			m2.7.1_summary_biopharmaceutic_analytical.docx	Example Text from GSK (Dolutegravir, GSK1349572)
	2.7.2		Summary of Clinical Pharmacology Studies	M		As early as initial IND application (see note), completed by marketing application			m2.7.2_summary_clinical_pharm.docx	Example Text from GSK (Dolutegravir, GSK1349572)
	2.7.3		Summary of Clinical Efficacy [indication]	M		As early as initial IND application (see note), completed by marketing application			m2.7.3_summary_clinical_efficacy.docx	Example Text from GSK (Dolutegravir, GSK1349572)
	2.7.4		Summary of Clinical Safety	M		As early as initial IND application (see note), completed by marketing application			m2.7.4_summary_ clinical_safety.docx	Example Text from GSK (Dolutegravir, GSK1349572)
	2.7.5		References - List of References	M		As early as initial IND application (see note), completed by marketing application			m2.7.5_references.docx	Example Text from GSK (Dolutegravir, GSK1349572)
	2.7.6		Synopses of Individual Studies	M		As early as initial IND application (see note), completed by marketing application				Example Text from GSK (Dolutegravir, GSK1349572)

Module 3 contains information on quality topics

Section				Section Name	Required for submission		Submission Timing	Guidance	Regulation	Templates
					Initial IND	Marketing
Module3
3.2				Body of Data				CGMP for Phase 1 Investigational Drugs CMC Information for Gene Therapy INDs CMC Information for Phase 2 and Phase 3 Studies Comparability Protocols for Postapproval Changes to CMC Information Content and Format of INDs for Phase 1 Studies Quality Systems Approach to Pharmaceutical CGMP Regulations Test Procedures and Acceptance Criteria for Biotechnological/Biological Products	IND Content and Format — Chemistry, Manufacturing, and Control Information
	3.2.S			Drug Substance [name, manufacturer]				Good Manufacturing Practice Guidance for Active Pharmaceutical Ingredients	Cultures IND Content and Format — Drug Substance
		3.2.S.1		General Information	Y		Initial IND or IND amendments (if applicable)		Identity	m3.2.s.1_general_information.docx
		3.2.S.2		Manufacture						m3.2.s.2_manufacture.docx
			3.2.S.2.1	Manufacturer(s)	Y		Initial IND or IND amendments (if applicable)	Contract Manufacturing Arrangements for Drugs: Quality Agreements
			3.2.S.2.2	Description of Manufacturing Process and Process Controls	Y		Initial IND or IND amendments (if applicable)
			3.2.S.2.3	Control of Materials	Y		Initial IND or IND amendments (if applicable)
			3.2.S.2.4	Controls of Critical Steps and Intermediates	Y		Initial IND or IND amendments (if applicable)
			3.2.S.2.5	Process Validation and/or Evaluation	Y		Initial IND or IND amendments (if applicable)	Process Validation: General Principles and Practices
			3.2.S.2.6	Manufacturing Process Development	Y		Initial IND or IND amendments (if applicable)
		3.2.S.3		Characterization						m3.2.s.3_characterization.docx
			3.2.S.3.1	Elucidation of Structure and Other Characteristics	Y		Initial IND or IND amendments (if applicable)		Identity
			3.2.S.3.2	Impurities	Y		Initial IND or IND amendments (if applicable)		Purity
		3.2.S.4		Control of Drug Substance					Potency	m3.2.s.4_control_drug_substance.docx
			3.2.S.4.1	Specification	Y		Initial IND or IND amendments (if applicable)		Identity
			3.2.S.4.2	Analytical Procedures	Y		Initial IND or IND amendments (if applicable)
			3.2.S.4.3	Validation of Analytical Procedures	M		Include in initial IND if requested, update in annual reports	VALIDATION OF ANALYTICAL PROCEDURES Q2(R2) Validation of Analytical Procedures: Methodology
			3.2.S.4.4	Batch Analyses	Y		Initial IND or IND amendments (if applicable)
			3.2.S.4.5	Justification of Specification	Y		Initial IND or IND amendments (if applicable)
		3.2.S.5		Reference Standards or Materials	Y		Initial IND or IND amendments (if applicable)			m3.2.s.5_reference_standards.docx
		3.2.S.6		Container Closure Systems	Y		Initial IND or IND amendments (if applicable)		Containers and Closures	m3.2.s.6_container_closure_system.docx
		3.2.S.7		Stability				Evaluation of Stability Data Stability Testing of Biological Products Stability Testing of New Drug Substances and Products		m3.2.s.7_stability.docx
			3.2.S.7.1	Stability Summary and Conclusions	Y		Initial IND or IND amendments (if applicable)
			3.2.S.7.2	Post Approval Stability Protocol and Stability Commitment	N	Y	Prior to or with marketing application
			3.2.S.7.3	Stability Data	M		Include initial data if applicable, update in annual reports
	3.2.P			Drug Product [name, dosage form, manufacturer]					IND Content and Format — Drug product
		3.2.P.1		Description and Composition of the Drug Product	Y		Initial IND, update as needed		Identity	m3.2.p.1_description_composition.docx
		3.2.P.2		Pharmaceutical Development						m3.2.p.2_pharmaceutical_development.docx
		3.2.P.3		Manufacture				cGMP Requirements for Combination Products	Applicability of CGMP regulations for investigational drugs	m3.2.p.3_manufacture.docx
			3.2.P.3.1	Manufacturer(s)	Y		Initial IND, update as needed
			3.2.P.3.2	Batch Formula	Y		Initial IND, update as needed
			3.2.P.3.3	Description of Manufacturing Process and Process Controls	Y		Initial IND, update as needed
			3.2.P.3.4	Controls of Critical Steps and Intermediates	Y		Initial IND, update as needed
			3.2.P.3.5	Process Validation and/or Evaluation	N		Required prior to initiation of Phase 3 manufacturing
		3.2.P.4		Control of Excipients				FDA Policy on Human Blood Components for Manufacturing		m3.2.p.4_control_excipients.docx
			3.2.P.4.1	Specification(s)	Y		Initial IND, update as needed		Identity
			3.2.P.4.2	Analytical Procedures	Y		Initial IND, update as needed
			3.2.P.4.3	Validation of Analytical Procedures	M		Include in initial IND if requested, update in annual reports	VALIDATION OF ANALYTICAL PROCEDURES Q2(R2)
			3.2.P.4.4	Justification of Specifications	Y		Initial IND, update as needed
			3.2.P.4.5	Excipients of Human or Animal Origin	M		Initial IND, update as needed
			3.2.P.4.6	Novel Excipients	M		Initial IND, update as needed
		3.2.P.5		Control of Drug Product					Potency	m3.2.p.5_control_product.docx
			3.2.P.5.1	Specification(s)	Y		Initial IND, update as needed		Identity
			3.2.P.5.2	Analytical Procedures	Y		Initial IND, update as needed
			3.2.P.5.3	Validation of Analytical Procedures	Y		Include in initial IND if requested, update in annual reports	VALIDATION OF ANALYTICAL PROCEDURES Q2(R2)
			3.2.P.5.4	Batch Analyses	Y		Initial IND, update as needed
			3.2.P.5.5	Characterization of Impurities	Y		Initial IND, update as needed		Purity
			3.2.P.5.6	Justification of Specification(s)	Y		Initial IND, update as needed
		3.2.P.6		Reference Standards or Materials	Y		Initial IND, update as needed			m3.2.p.6_reference_standards.docx
		3.2.P.7		Container Closure System	Y		Initial IND, update as needed		Containers and Closures	m3.2.p.7_container_closure_system.docx
		3.2.P.8		Stability						m3.2.p.8_stability.docx
			3.2.P.8.1	Stability Summary and Conclusion	Y		Initial IND, update as needed
			3.2.P.8.2	Post-approval Stability Protocol and Stability Commitment	N	Y	Prior to or with marketing application
			3.2.P.8.3	Stability Data	Y		Initial IND, update as needed
	3.2.A			Appendices
		3.2.A.1		Facilities and Equipment [name, manufacturer]	Y		Initial IND, update as needed		Facilities, Equipment, Animals and Care Good Laboratory Practice for Nonclinical Laboratory Studies — Equipment Good Laboratory Practice for Nonclinical Laboratory Studies — Facilities Quailty Assurance Unit	m3.2.a.1_facilities_equipment.docx
		3.2.A.2		Adventitious Agents Safety Evaluation [name, dosage form, manufacturer]	Y		Initial IND, update as needed		Sterility Sterility	m3.2.a.2_adventitious_agents.docx
		3.2.A.3		Novel Excipients	Y		Initial IND, update as needed	Nonclinical Studies for the Safety Evaluation of Pharmaceutical Excipients		m3.2.a.3_novel_excipients.docx
	3.2.R			Regional Information					Batch Records Distribution Reports Lot Release Testing	m3.2.r_regional_information.docx
3.3				Literature References				CMC Information for Gene Therapy INDs		m3.3_references.docx

Module 4 contains the nonclinical study reports

Section				Section Name	Required for submission		Submission Timing	Guidance	Regulation	Templates
					Initial IND	Marketing
Module4
4.2				Study Reports [study id_study Title]				Content and Format of INDs for Phase 1 Studies M3(R2) Nonclinical Safety Studies Preclinical Assessment of Investigational Cellular and Gene Therapy Products Providing Regulatory Submissions in Electronic Format—Standardized Study Data	Good Laboratory Practice for Nonclinical Laboratory Studies — Protocol for and Conduct of a Nonclinical Laboratory Study Good Laboratory Practice for Nonclinical Laboratory Studies — Records and Reports Good Laboratory Practice for Nonclinical Laboratory Studies — Test and Control Articles Good Laboratory Practice for Nonclinical Laboratory Studies — Testing Facilities Operation IND Content and Format — Pharmacology and Toxicology Information
	4.2.1			Pharmacology Studies	Y				IND Content and Format — Pharmacology and Drug Disposition	m4.2.1_pharm_study_report_template.docx
		4.2.1.1		Primary Pharmacodynamics	Y		As early as initial IND application, revise as necessary
		4.2.1.2		Secondary Pharmacodynamics	M		As early as initial IND application, revise as necessary
		4.2.1.3		Safety Pharmacology	M		As early as initial IND application, revise as necessary
		4.2.1.4		Pharmacodynamic Drug Interactions	M		As early as initial IND application, revise as necessary
	4.2.2			Pharmacokinetic Studies	Y				IND Content and Format — Pharmacology and Drug Disposition	m4.2.2_PK_study_report_template.docx
		4.2.2.1		Analytical Methods and Validation Reports	M		As early as initial IND application, revise as necessary
		4.2.2.2		Absorption	M		As early as initial IND application, revise as necessary
		4.2.2.3		Distribution	Y		As early as initial IND application, revise as necessary
		4.2.2.4		Metabolism	M		As early as initial IND application, revise as necessary
		4.2.2.5		Excretion	M		As early as initial IND application, revise as necessary
		4.2.2.6		Pharmacokinetic Drug Interactions	M		As early as initial IND application, revise as necessary
		4.2.2.7		Other Pharmacokinetic Studies	M		As early as initial IND application, revise as necessary
	4.2.3			Toxicology Studies	Y				IND Content and Format — Toxicology	m4.2.3_tox_study_report_template.docx
		4.2.3.1		Single-Dose Toxicity (in order by species, by route)	Y		As early as initial IND application, revise as necessary
		4.2.3.2		Repeat-Dose Toxicity (species, route, duration, including supportive toxicokinetics)	M		As early as initial IND application, revise as necessary
		4.2.3.3		Genotoxicity	M		As early as initial IND application, revise as necessary
			4.2.3.3.1	In vitro	M		As early as initial IND application, revise as necessary
			4.2.3.3.2	In vivo (including supportive toxicokinetics evaluations)	M		As early as initial IND application, revise as necessary
		4.2.3.4		Carcinogenicity	M		As early as initial IND application, revise as necessary
			4.2.3.4.1	Long-term studies	M		As early as initial IND application, revise as necessary
			4.2.3.4.2	Short-or medium-term studies	M		As early as initial IND application, revise as necessary
			4.2.3.4.3	Other Studies	M		As early as initial IND application, revise as necessary
		4.2.3.5		Reproductive and Developmental Toxicity	M		As early as initial IND application, revise as necessary
			4.2.3.5.1	Fertility and early embryonic development	M		As early as initial IND application, revise as necessary
			4.2.3.5.2	Embryofetal development	M		As early as initial IND application, revise as necessary
			4.2.3.5.3	Prenatal and postnatal development, including maternal function	M		As early as initial IND application, revise as necessary
			4.2.3.5.4	Studies in which the offspring are dosed and/or further evaluated	M		As early as initial IND application, revise as necessary
		4.2.3.6		Local Tolerance	M		As early as initial IND application, revise as necessary
		4.2.3.7		Other Toxicity Studies	M		As early as initial IND application, revise as necessary
			4.2.3.7.1	Antigenicity	M		As early as initial IND application, revise as necessary
			4.2.3.7.2	Immunotoxicity	M		As early as initial IND application, revise as necessary
			4.2.3.7.3	Mechanistic studies	M		As early as initial IND application, revise as necessary
			4.2.3.7.4	Dependence	M		As early as initial IND application, revise as necessary		IND Content and Format — Drug dependence and Abuse Potential
			4.2.3.7.5	Metabolites	M		As early as initial IND application, revise as necessary
			4.2.3.7.6	Impurities	M		As early as initial IND application, revise as necessary
			4.2.3.7.7	Other	M		As early as initial IND application, revise as necessary
4.3				Literature References						m4.3_references.docx

Module 5 contains the clinical study reports.

Section			Section Name	Required for submission		Submission Timing	Guidance	Regulation	Templates
				Initial IND	Marketing
Module5
5.2			Tabular Listing of All Clinical Studies	M					m5.2_tabulated_list_clinical_studies.docx
5.3			Clinical Study Reports and Related Information [study id_study Title]				Content and Format of INDs for Phase 1 Studies ICH Guideline on Efficacy Providing Regulatory Submissions in Electronic Format—Standardized Study Data Structure and Content of Clinical Study Reports E3	IND Content and Format — Previous Human Experience with the Investigational Drug
	5.3.1		Reports of Biopharmaceutic Studies					Bioavailability and Bioequivalence Requirements
		5.3.1.1	Bioavailability (BA) Study Reports	M		As early as initial IND application, revise as necessary
		5.3.1.2	Comparative BA and Bioequivalence (BE) Study Reports	M		As early as initial IND application, revise as necessary
		5.3.1.3	In Vitro-In Vivo Correlation Study Reports	M		As early as initial IND application, revise as necessary
		5.3.1.4	Reports of Bioanalytical and Analytical Methods for Human Studies	M		As early as initial IND application, revise as necessary
	5.3.2		Reports of Studies Pertinent to Pharmocokinetics using Human Biomaterials
		5.3.2.1	Plasma Protein Binding Study Reports	M		As early as initial IND application, revise as necessary
		5.3.2.2	Reports of Hepatic Metabolism and Drug Interaction Studies	M		As early as initial IND application, revise as necessary
		5.3.2.3	Reports of Studies Using Other Human Biomaterials	M		As early as initial IND application, revise as necessary
	5.3.3		Reports of Human Pharmacokinetic (PK) Studies
		5.3.3.1	Healthy Subject PK and Initial Tolerability Study Reports	N		Not applicable to gene therapies, not tested in healthy subjects
		5.3.3.2	Patient PK and Initial Tolerability Study Reports	M		As early as initial IND application, revise as necessary
		5.3.3.3	Intrinsic Factor PK Study Reports	M		As early as initial IND application, revise as necessary
		5.3.3.4	Extrinsic Factor PK Study Reports	M		As early as initial IND application, revise as necessary
		5.3.3.5	Population PK Study Reports	M		As early as initial IND application, revise as necessary
	5.3.4		Reports of Human Pharmacodynamic (PD) Studies
		5.3.4.1	Healthy Subject PD and PK/PD Study Reports	N		Not applicable to gene therapies, not tested in healthy subjects
		5.3.4.2	Patient PD and PK/PD Study Reports	M		As early as initial IND application, revise as necessary
	5.3.5		Reports of Efficacy and Safety Studies
		5.3.5.1	Study Reports and Related Information of Controlled Clinical Studies Pertinent to the Claimed Indication	M		As early as initial IND application, revise as necessary
		5.3.5.2	Study Reports and Related Information of Uncontrolled Clinical Studies	M		As early as initial IND application, revise as necessary
		5.3.5.3	Reports of Analyses of Data From More Than One Study
		5.3.5.4	Other Clinical Study Reports	M		As early as initial IND application, revise as necessary
	5.3.6		Reports of Postmarketing Experience	N		As early as initial IND application, revise as necessary	Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products	Information required for Individual Case Safety Reports (ICSRs) Postmarketing Reporting of Adverse Experiences Postmarketing reporting requirements — "Alert Reports"
	5.3.7		Case Report Forms and Individual Patient Listings	M		As early as initial IND application, revise as necessary
5.4			Literature References	Y					m5.4_references.docx

External Resources/Links

eCTD v4.0 Technical Conformance Guide (FDA) - Content current as of March 2025
Submit Using eCTD (FDA)
OTP Pre-IND Meetings (FDA)
OTP INTERACT Meeting (FDA)
Elpida Therapeutics (SPG50 Phase I/II Regulatory Documents)
BGTC Regulatory Templates

Contact SCGE

IND (Investigational New Drug) Application for gene therapy

Common Technical Document (CTD)

FDA Submission Elements

External Resources/Links