us-fda-guidance-4ac79178dc57388a3d5e4c092876760b•Providing Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications Guidance for Industry
Providing Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications Guidance for Industry
us-fda-guidance-4ac79178dc57388a3d5e4c092876760bProviding Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications Guidance for IndustryGuidance
Administrative guidance, not enacted legislation. This captured publisher edition preserves the listed document and attachments; later developments are not independently consolidated.
- Docket Number:
- FDA-2014-N-1006
- Issued by:
- Guidance Issuing OfficeCenter for Drug Evaluation and ResearchCenter for Biologics Evaluation and Research
Under section 745A(a) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), at least 24 months after the issuance of a final guidance document in which the Food and Drug Administration (FDA or Agency) has specified the electronic format for submitting submission types to the Agency, such content must be submitted electronically and in the format specified by FDA. This guidance describes how sponsors and applicants must organize the content that they submit to the Agency electronically for all submission types under section 745A(a) of the FD&C Act. This guidance also references several technical specification documents and the electronic common technical document (eCTD) Technical Conformance Guide, which provide additional details regarding the organization of content for electronic submissions.
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58687461fnlrv8.docx 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) September 2024 Electronic Submissions Revision 8
Providing Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications Guidance for Industry Additional copies are available from: Office of Communications, Division of Drug Information Center for Drug Evaluation and Research Food and Drug Administration 10001 New Hampshire Ave., Hillandale Bldg., 4 th Floor Silver Spring, MD 20993-0002 Phone: 855-543-3784 or 301-796-3400; Fax: 301-431-6353 Email: druginfo@fda.hhs.gov https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs and/or Office of Communication, Outreach and Development Center for Biologics Evaluation and Research Food and Drug Administration 10903 New Hampshire Ave., Bldg. 71, Room 3128 Silver Spring, MD 20993-0002 Phone: 800-835-4709 or 240-402-8010 Email: ocod@fda.hhs.gov https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances 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) September 2024 Electronic Submissions Revision 8
RELATED DOCUMENTS Technical specifications associated with this guidance are provided as separate documents and are updated periodically. A list of documents cited within this guidance are provided at the end of this document. For a complete list of all documents and supportive files needed to submit electronically, refer to the eCTD web page at https://www.fda.gov/drugs/electronic-regulatory-submission- and-review/electronic-common-technical-document-ectd. REVISION HISTORY DATE S UMMARY OF R EVISIONS April 2017 Update to Guidance Section I. Introduction • Added paragraph describing rationale for changing timetable for required master file submissions in eCTD from 24 months to 36 months Section III.B. Timetable for Implementation of Electronic Submission Requirements • Updated section to reflect that the requirement for master files to be filed electronically takes effect 36 months after May 5, 2015 • Updated example of timetable to reflect actual timetable for the implementation of the electronic submissions requirement April 2018 Update to Guidance Section I. Introduction • Added paragraph describing rationale for extending timetable for Type III drug master file submissions in eCTD for an additional 12 months Section III.A. Types of Submissions That Must Adhere to the Electronic Submission Requirement Described in This Guidance • Revised paragraph to reflect change in nomenclature of “biologic product files (BPFs)” to “other master files relevant to a biological product” Section III.B. Timetable for Implementation of Electronic Submission Requirements • Updated section to reflect that the requirement for Type III drug master files to be filed electronically takes place 48 months after May 5, 2015 Updated example of timetable to reflect actual timetable for the implementation of the electronic submissions requirement
DATE S UMMARY OF R EVISIONS January 2019 Update to Guidance Section I. Introduction • Added paragraph describing rationale for extending timetable for Type III drug master file submissions in eCTD for an additional 12 months Section III.B. Timetable for Implementation of Electronic Submission Requirements • Updated section to reflect that the requirement for Type III drug master files to be filed electronically takes place 60 months after May 5, 2015 • Updated example of timetable to reflect actual timetable for the implementation of the electronic submissions requirement February 2020 Update to Guidance Section I. Introduction • Added paragraph describing the addition of exemptions and waivers from complying with eCTD requirements Section III.C. Types of Submissions That Are Exempted From the eCTD Requirement Described in This Guidance • Updated section to include exemption for Type III drug master files Section III.D. Types of Submissions That May Qualify for a Long-Term Waiver From the eCTD Requirement Described in This Guidance • Added section to include waiver criteria for certain PET drug INDs, NDAs, ANDAs, and BLAs, and waiver criteria for certain Type II DMFs Section III.E. Types of Submissions That May Qualify for a Short-Term Waiver From the eCTD Requirement Described in This Guidance • Added section to include the criteria to qualify for a waiver and the instructions on how to submit a request for a short-term waiver September 2024 General • Updated hyperlinks throughout guidance Section III.F. The eCTD Specifications Section III.H. Submission Structure: Granularity, Files, and Folders Section III.J. Document Life Cycle Section III.L. Datasets and Study Information ⦁ Updated sections (III.F, H, J, and L) to include eCTD v4.0
D ATE S UMMARY OF R EVISIONS Section IV. Technical Specification Documents Referenced in This Guidance ⦁ Updated section to include eCTD v4.0-related documents
TABLE OF CONTENTS I. INTRODUCTION............................................................................................................. 1 II. BACKGROUND ............................................................................................................... 2 III. REQUIREMENT TO SUBMIT ELECTRONICALLY UNDER THIS GUIDANCE3 A. Types of Submissions That Must Adhere to the Electronic Submission Requirement Described in This Guidance ................................................................................................................... 3 B. Timetable for Implementation of Electronic Submission Requirements.................................. 4 C. Types of Submissions Exempted From the eCTD Requirement Described in This Guidance6 D. Certain Positron Emission Tomography Drugs and Type II DMF Submissions That May Qualify for a Waiver From the eCTD Requirement Described in This Guidance ........................... 6 1. Certain Positron Emission Tomography Drug Submissions ........................................................... 7 2. Certain Type II DMF Submissions .................................................................................................. 8 3. Where to Submit Waiver Requests ................................................................................................... 8 4. FDA Response to Waiver Requests .................................................................................................. 9 E. Types of Submissions That May Qualify for a Short-Term Waiver From the eCTD Requirement Described in This Guidance ........................................................................................... 9 1. Content of Waiver Requests ........................................................................................................... 10 2. Where to Submit Waiver Requests ................................................................................................. 10 3. FDA Response to Waiver Requests ................................................................................................ 11 F. The eCTD Specifications ............................................................................................................. 11 G. Pre-Submission Considerations .................................................................................................. 12 H. Submission Structure: Granularity, Files, and Folders .......................................................... 12 I. File Formats and Versions .......................................................................................................... 13 J. Document Life Cycle.................................................................................................................... 14 K. Summary of Clinical Efficacy and Summary of Clinical Safety.............................................. 14 L. Datasets and Study Information ................................................................................................. 14 M. Transmitting Electronic Submissions ........................................................................................ 15 N. FDA Forms ................................................................................................................................... 15 O. Restrictions on Submission of Paper Copies ............................................................................. 15 P. Receipt Date.................................................................................................................................. 15 IV. TECHNICAL SPECIFICATION DOCUMENTS REFERENCED IN THIS GUIDANCE ................................................................................................................................. 16 V. RELATED REFERENCES ........................................................................................... 17
1 Providing Regulatory Submissions in Electronic Format — Certain 1 Human Pharmaceutical Product1 Applications and Related 2 Submissions Using the eCTD Specifications 3 Guidance for Industry2 4 5 6 I. INTRODUCTION 7 8 Under section 745A(a) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), at least 9 24 months after the issuance of a final guidance document in which the Food and Drug 10 Administration (FDA or Agency) has specified the electronic format for submitting submission 11 types to the Agency, such content must be submitted electronically and in the format specified by 12 FDA.3,4 This guidance describes how sponsors and applicants must organize the content that 13 they submit to the Agency electronically for all submission types under section 745A(a) of the 14 FD&C Act. This guidance also references several technical specification documents5 and the 15 Electronic Common Technical Document Conformance (eCTD) Guide, which provide additional 16 details regarding the organization of content for electronic submissions.6 17 18 This guidance implements the electronic submission requirements of section 745A(a) of the 19 FD&C Act for the electronic format of the content submitted in new drug applications (NDAs), 20 abbreviated new drug applications (ANDAs), certain biologics license applications (BLAs), and 21 1 The term human pharmaceutical product, as used in this guidance, includes any product intended for human use that meets the definition of drug and does not also meet the definition of device under the Federal Food, Drug, and Cosmetic Act (FD&C Act), including both (1) drugs approved under the FD&C Act and (2) biological products approved under the Public Health Service Act (PHS Act). Similarly, for the purposes of this guidance, unless otherwise specified, the term drug refers to human prescription drugs, including those that are licensed as biological products (biologics). 2 This guidance has been prepared by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). 3 See 21 U.S.C. 379k–1. 4 For additional information on how FDA interprets and intends to implement the electronic submission requirements of section 745A(a) of the FD&C Act, please see the guidance for industry Providing Regulatory Submissions in Electronic Format — Submissions Under Section 745A(a) of the Federal Food, Drug, and Cosmetic Act (December 2014). We update guidances periodically. For the most recent version of a guidance, check the FDA guidance web page at https://www.fda.gov/regulatory-information/search-fda-guidance-documents. 5 For instance, to reflect the evolving nature of the technology and the experience of those using this technology, the electronic common technical document (eCTD) technical specifications are being provided as separate documents in connection with this guidance. These associated specifications will be updated periodically. For the most recent versions of related technical specifications (CDER and CBER), check the eCTD web page at https://www.fda.gov/drugs/electronic-regulatory-submission-and-review/electronic-common-technical-document- ectd. 6 For the most recent version of the eCTD Technical Conformance Guide, check the eCTD Resources web page at https://www.fda.gov/drugs/electronic-regulatory-submission-and-review/ectd-resources.
2 certain investigational new drug applications (INDs) to the Center for Drug Evaluation and 22 Research (CDER) or to the Center for Biologics Evaluation and Research (CBER). See section 23 III.A of this guidance for more information regarding required submission types. Submissions 24 that are not submitted electronically and electronic submissions that are not in a format that FDA 25 can process, review, and archive will not be filed or received unless they have an exemption or 26 waiver from the electronic submission requirements. 27 28 The revised guidance was issued on May 5, 2015, and provided a timetable of 24 months after 29 issuance of the final guidance for the initial implementation of the electronic submission 30 requirement for NDAs, ANDAs, BLAs, and master files (May 5, 2017) and 36 months for 31 commercial INDs (May 5, 2018). The timetable indicated that NDAs, BLAs, ANDAs, and 32 master files were to be submitted electronically in eCTD format starting on May 5, 2017 (May 5, 33 2018, for commercial INDs). Subsequently, in April 2017, in response to industry comments 34 and internal review, FDA extended the implementation date for drug master files (DMFs) to 35 36 months (to May 5, 2018) (revision 4), and in April 2018, FDA extended the implementation 36 date for Type III DMFs to 48 months (May 5, 2019) (revision 5). After issuing revision 5, FDA 37 determined that many of the concerns expressed in industry comments and confirmed by internal 38 review remained. Therefore, the Agency revised this guidance to further extend the 39 implementation date for Type III DMFs until May 5, 2020 (revision 6) . In February 2020, the 40 Agency issued revision 7 to include exemptions for Type III DMFs. In addition, revision 7 41 included criteria identifying the types of submissions that may qualify for a long-term or a short- 42 term waiver from eCTD submission requirements and how to submit a waiver request. 43 44 This revision (revision 8) modifies previous versions by updating hyperlinks throughout; 45 updating language to include eCTD v4.0 in sections III.F, H, J, and L; and updating eCTD v4.0- 46 related documents in section IV. 47 48 49 II. BACKGROUND 50 51 In section 745A(a) of the FD&C Act, Congress granted explicit authorization to FDA to 52 implement the statutory electronic submission requirements in guidance and required that FDA 53 “shall” issue such guidance. Accordingly, as indicated by the words must or required, this 54 document is not subject to the usual restrictions in FDA’s good guidance practice (GGP) 55 regulations, such as the requirement that guidances not establish legally enforceable 56 responsibilities (see 21 CFR 10.115(d); see also the guidance for industry Providing Regulatory 57 Submissions in Electronic Format — Submissions Under Section 745A(a) of the Federal Food, 58 Drug, and Cosmetic Act (December 2014) (the 745A(a) Implementation guidance)). 59 60 To comply with the GGP regulations and make sure that regulated entities and the public 61 understand that guidance documents are nonbinding, FDA guidances ordinarily contain standard 62 language explaining that guidance documents should be viewed only as recommendations unless 63 specific regulatory or statutory requirements are cited. FDA is not including this standard 64 language in this guidance because it is not an accurate description of all the effects of this 65 guidance. Insofar as this document specifies the format for electronic submissions or provides 66
3 “criteria for waivers of and exemptions from” the requirements of section 745A(a) of the FD&C 67 Act, it will have binding effect. 68 69 70 III. REQUIREMENT TO SUBMIT ELECTRONICALLY UNDER THIS GUIDANCE 71 72 As of May 5, 2017, sponsors and applicants must submit the content for which an electronic 73 format for submission is specified in this guidance in such electronic format unless the 74 submission is exempted or waived. In other words, such submissions must be consistent with the 75 requirements set forth below in this section. 76 77 A. Types of Submissions That Must Adhere to the Electronic Submission 78 Requirement Described in This Guidance 79 80 Section 745A(a) of the FD&C Act applies to submissions under section 505(b), (i), or (j) of the 81 FD&C Act and under section 351(a) or (k) of the Public Health Service (PHS) Act. These 82 include the following submission types: 83 84 • Certain investigational new drug applications (INDs)7,8 85 • New drug applications (NDAs) 86 • Abbreviated new drug applications (ANDAs) 87 • Certain biologics license applications (BLAs)9,10 88 7 This guidance is not applicable to investigational new drug applications (INDs) for devices that are regulated by CBER as biological products under section 351 of the PHS Act and that also require the submission of an IND before the submission of a biologics license application (BLA). Although a discussion of which devices CBER regulates as biological products is outside the scope of this guidance, as a general matter, this category of INDs would include investigational devices that are used to screen blood donations for certain transfusion-transmissible infections and to test human cells, tissues, or cellular or tissue-based products to make a donor-eligibility determination. These submissions are subject to the requirements under section 745A(b) of the FD&C Act. See the guidance for industry and FDA staff eCopy Program for Medical Device Submissions (April 2020), which implements the electronic copy provisions of section 745A(b) for medical device submissions to FDA. 8 This guidance is not applicable to noncommercial INDs. 9 This guidance is not applicable to those devices that are regulated by CBER as biological products under section 351 of the PHS Act, including those that do not require the submission of an IND before the submission of a BLA. Although a discussion of which devices CBER regulates as biological products under section 351 of the PHS Act is outside the scope of this guidance, as a general matter, this category would include devices that are used to screen blood donations for certain transfusion-transmissible infections and reagents used in determining donor/recipient compatibility in transfusion medicine. These submissions are subject to the requirements under section 745A(b) of the FD&C Act. See the guidance for industry and FDA staff eCopy Program for Medical Device Submissions (April 2020). 10 Specifically, this guidance is not applicable to submissions for blood and blood components, including Source Plasma.
4 Section 745A(a) also applies to all subsequent submissions, including amendments, supplements, 89 and reports, to the submission types identified above.11,12 90 91 FDA considers master files to be submissions to an NDA, ANDA, BLA, or IND and therefore to 92 fall within the scope of requirements set forth in section 745A(a). These include new DMFs (21 93 CFR 314.420) and other master files relevant to a biological product (21 CFR 601.51)13 and any 94 amendments to or annual reports on previously submitted DMFs or other master files relevant to 95 a biological product. This guidance also applies to submissions for drug/device combination 96 products filed pursuant to section 505 of the FD&C Act or subsection (a) or (k) of section 351 of 97 the PHS Act. 98 99 A submission that is not in the electronic format(s) described in this guidance will not be filed or 100 received unless it has an exemption or waiver for the electronic submission requirements (see 101 sections III.C, III.D, and III.E) with respect to that submission. 102 103 Under section 745A(a)(3) of the FD&C Act, the electronic submission requirements do not apply 104 to submissions described in section 561 of the FD&C Act (e.g., expanded access INDs and 105 protocols for individual patients, including for emergency use; expanded access INDs and 106 protocols for intermediate-sized patient populations; and expanded access treatment INDs and 107 protocols). FDA will continue to accept submissions under section 561 in alternative formats 108 (e.g., portable document format (PDF) files following the common technical document (CTD) 109 organization).14 110 111 B. Timetable for Implementation of Electronic Submission Requirements 112 113 The requirement to submit NDAs, ANDAs, and BLAs electronically became effective 24 months 114 after May 5, 2015 (i.e., May 5, 2017). The requirement for INDs and master files, other than 115 11 Although certain postmarketing safety report submissions fall within the scope of section 745A(a) of the FD&C Act, FDA has separate regulations that require postmarketing safety reports to be submitted in electronic format (see 21 CFR 310.305, 314.80, 314.98. 600.80, and 600.81 and section 760 of the FD&C Act) and has issued related non- binding guidance on these postmarketing safety reports. Accordingly, FDA has not issued guidance under section 745A with respect to electronic format for postmarketing safety reports. For recommendations with respect to submissions related to postmarketing safety reports under §§ 310.305, 314.80, 314.98. 600.80, and 600.81 or under section 760 of the FD&C Act, see the guidance for industry Providing Submissions in Electronic Format — Postmarketing Safety Reports (April 2022). FDA may consider, at a future date, whether to include information in guidance pertaining to submission of postmarketing safety reports in electronic format under section 745A(a) of the FD&C Act. 12 For further information about IND safety reports, see 21 CFR 312.32 and the guidance for industry Safety Reporting Requirements for INDs and BA/BE Studies (December 2012). Additional information may also be found in the guidance for industry Providing Regulatory Submissions in Electronic Format: IND Safety Reports (April 2024). 13 For the purposes of this guidance, the term DMF refers to both drug master files and master files relevant to biological products. 14 See the ICH guidance for industry M4 Organization of the Common Technical Document for the Registration of Pharmaceuticals for Human Use (October 2017).
5 Type III DMFs, to be filed electronically became effective 36 months after May 5, 2015 (i.e., 116 May 5, 2018). For all of these submission types, if you do not have an exemption or waiver, you 117 must electronically submit any amendments, supplements, and reports in eCTD format, even if 118 the original submission was submitted to FDA in non-eCTD format before implementation of the 119 electronic submission requirements. 120 121 The timetable for the initial implementation of the electronic submission requirement is shown in 122 italics below. Table 1 summarizes the timetable. 123 124 On May 5, 2015, FDA issued the final guidance for industry on Providing Regulatory 125 Submissions in Electronic Format — Certain Human Pharmaceutical Product 126 Applications and Related Submissions Using the eCTD Specifications. Submission types 127 NDA, ANDA, and BLA must be submitted in eCTD format beginning May 5, 2017. IND 128 submissions and master files, other than Type III DMFs, must be submitted in eCTD 129 format beginning May 5, 2018. 130 131 Table 1: Timetable for the Initial Implementation of the Electronic Submission 132 Requirement 133 134 Submission Type Final eCTD Guidance Posted on FDA Website (yyyy-mm-dd) Date Requirement Begins (yyyy-mm-dd) NDA ANDA BLA 2015-05-05 2017-05-05 Commercial IND Master Files Other Than Type III DMFs 2015-05-05 2018-05-05 135 Additional information regarding submissions pertaining to promotional materials made to the 136 Office of Prescription Drug Promotion in CDER and to the Advertising and Promotional 137 Labeling Branch in CBER is described in a separate guidance, which also provides the timetable 138 for implementation of those submissions in electronic format.15 139 140 15 See the guidance for industry Providing Regulatory Submissions in Electronic and Non-Electronic Format — Promotional Labeling and Advertising for Human Prescription Drugs (April 2022).
6 C. Types of Submissions Exempted From the eCTD Requirement Described in 141 This Guidance 142 143 Section 745A(a)(2) of the FD&C Act allows FDA to establish exemptions from the electronic 144 submission requirements. Accordingly, FDA has exempted the following from the eCTD 145 requirements under section 745A(a)(2):16 146 147 1. All submissions to noncommercial INDs.17 For the purposes of this guidance, the term 148 noncommercial IND refers to an IND for a product that is not intended for commercial 149 distribution; this exemption includes research and investigator-sponsored INDs. 150 151 2. All Type III DMF submissions.18 152 153 Although these specific submissions will be exempt from filing in eCTD format as described in 154 this guidance, FDA still encourages applicants to send submissions in an alternative electronic 155 format (e.g., PDF files following the CTD structure). 156 157 D. Certain Positron Emission Tomography Drugs and Type II DMF 158 Submissions That May Qualify for a Waiver From the eCTD Requirement 159 Described in This Guidance 160 161 Section 745A(a)(2) authorizes FDA to establish criteria for waivers from its electronic 162 submission requirements. Accordingly, FDA may grant a long-term waiver from the eCTD 163 requirements under section 745A(a)(2)19 in the following circumstances: 164 165 16 See section III.B of the 745A(a) Implementation guidance (Providing Regulatory Submissions in Electronic Format — Submissions Under Section 745A(a) of the Federal Food, Drug, and Cosmetic Act). Noncommercial IND submissions are not required to submit a request for this exemption. 17 Noncommercial IND submissions are not required to submit a request for this exemption. Although INDs covered under section 561 of the FD&C Act might be referred to as a type of noncommercial IND, they have been statutorily excepted from the scope of section 745A(a). As a result, they need not submit in eCTD format, albeit for a different reason than the submissions exempted here. See section III.A of this guidance for information on the types of INDs covered under section 561 of the FD&C Act. 18 Type III DMFs are submitted to the Agency to provide information regarding packaging or packaging materials in support of NDAs, ANDAs, or BLAs. The DMF web page is accessible at https://www.fda.gov/drugs/developmentapprovalprocess/formssubmissionrequirements/drugmasterfilesdmfs/default. htm. 19 See section III.C of the 745A(a) Implementation guidance.
7 1. Certain Positron Emission Tomography Drug Submissions 166 167 The requirement to comply with the eCTD requirement for certain positron emission tomography 168 (PET20) IND, NDA, ANDA, or BLA submissions could adversely impact the development and 169 availability of PET drugs. FDA may grant a waiver to a PET drug sponsor or applicant intending 170 to submit an IND, NDA, ANDA, or BLA if all of the following apply: 171 172 (a) The applicant produces PET drugs at a single PET drug facility. 173 174 (b) PET drugs are the only FDA-regulated drug products (other than noncommercial drug 175 or biological products) manufactured or produced by the sponsor or applicant. 176 177 (c) The sponsor or applicant explains that because it meets the criteria in (a) and (b) 178 above, it cannot achieve compliance with eCTD requirements. 179 180 A waiver request should be sent to FDA before submitting the document(s) for which this 181 waiver is claimed,21 with an explanation regarding why the sponsor or applicant’s 182 compliance with the requirement cannot be achieved, including that the sponsor or 183 applicant is representing that (a) through (c) above are met22 and a description of the 184 proposed alternative submission format23 the sponsor or applicant will be using during the 185 duration of the waiver (e.g., PDF files following the CTD structure). 186 187 The information provided in the waiver request may be verified through inspection or through a 188 records request in lieu of an inspection. 189 190 20 PET is a medical imaging method that produces a computerized image (scan) using a unique type of radiopharmaceutical. A PET drug is a radioactive drug characterized by spontaneous disintegration of unstable nuclei by the emission of positrons and is used for providing dual photon positron emission tomographic diagnostic images (21 CFR 212.1). PET drugs are distinct among radiopharmaceuticals because of their unique production methods, and many are characterized by their short half-lives (some as short as 20 minutes). Many PET drug production facilities are close in proximity to the patients to whom the drugs are administered, and the production of the drug is on demand. 21 Sponsors and applicants should request a pre-assigned application number before submitting a waiver request. 22 See section 745A(a) of FD&C Act and 21 CFR 312.10 and 314.90(a)(1). 23 If submission in eCTD format is not possible, FDA still encourages applicants to send submissions electronically in an alternative electronic format (e.g., PDF files following the CTD structure).
8 2. Certain Type II DMF Submissions 191 192 Holders of certain Type II DMFs24 that solely support an application for a PET drug or a 193 noncommercial IND application may also qualify for a waiver. FDA recognizes that the holders 194 of these Type II DMFs may be distinct from the holder of the application(s) in question. FDA 195 may grant a waiver to a holder intending to submit a Type II DMF if the Type II DMF holder 196 explains that it cannot achieve compliance with eCTD requirements because one of the following 197 applies: 198 199 (a) The Type II DMF is intended to support an application for a PET drug (i.e., IND, 200 NDA, ANDA, or BLA) and contains information regarding radiolabeled drug 201 products or production of PET radionuclides, and the Type II DMF holder is an 202 academic institution, government (state or federal) entity, or a nonprofit25 research 203 organization. 204 205 OR 206 207 (b) The Type II DMF is solely used to support a noncommercial IND application, and the 208 Type II DMF holder is an academic institution, government (state or federal) entity, 209 or a nonprofit research organization. 210 211 A waiver request should be sent to FDA before submitting the document(s) for which this 212 waiver is claimed,26 with an explanation regarding why the sponsor or applicant’s 213 compliance with the eCTD requirement cannot be achieved (i.e., that the sponsor or 214 applicant is representing that (a) or (b) above is met), including a description of the 215 proposed alternative submission format27 the sponsor or applicant will be using during the 216 duration of the waiver (e.g., PDF files following the CTD structure). 217 218 The information provided on the waiver request may be verified through inspection or through a 219 records request in lieu of an inspection. 220 221 3. Where to Submit Waiver Requests 222 223 Waiver requests for qualifying PET drugs or Type II DMFs should be submitted in one of the 224 following ways: 225 226 24 Type II DMFs are submitted to the Agency to support drug applications to make quality information available for Agency evaluation of the quality of active pharmaceutical ingredients and drug products used in investigational studies. 25 For the purposes of this guidance, a nonprofit is a charitable organization recognized as tax-exempt under section 501(c)(3) of the United States Internal Revenue Code of 1986 (Title 26 of the United States Code). 26 Sponsors and applicants should request a pre-assigned application number before submitting a waiver request. 27 See footnote 25.
9 • CDER: 227 - Email to esub@fda.hhs.gov 228 229 • CBER 230 - Email to esubprep@fda.hhs.gov 231 232 The waiver request should reference all products that are to be covered by the waiver. The 233 waiver request should be clearly titled “LONG-TERM WAIVER REQUEST — eCTD 234 REQUIREMENTS” in bold capital letters at the top of the first page of the submission. 235 236 4. FDA Response to Waiver Requests 237 238 FDA reviews waiver requests on a case-by-case basis. FDA will generally respond to the 239 requestor28 in writing, stating whether the waiver is granted or denied and whether the proposed 240 alternative submission format is acceptable. Long-term waivers from the requirement to 241 submit in eCTD format, if granted, will be valid for five (5) years from the date the waiver 242 is granted, will apply only to the requestor, and will not be transferrable to another 243 sponsor or applicant. Sponsors or applicants may reapply to recertify their eligibility for this 244 waiver up to 6 months before the waiver expiration date, using the same process as described in 245 section III.D.3 of this guidance. If the criteria are no longer met at the time of recertification, the 246 waiver will not be granted. 247 248 If FDA grants a waiver, the requestor should include a statement in the cover letter of each 249 subsequent submission indicating that an eCTD submission waiver has been granted by 250 FDA, including the dates for the waiver. 251 252 Although these specific submissions may receive waivers from eCTD format requirements as 253 described in this guidance, FDA still encourages applicants to send submissions electronically in 254 an alternative electronic format (e.g., PDF files following the CTD structure).29 255 256 E. Types of Submissions That May Qualify for a Short-Term Waiver From the 257 eCTD Requirement Described in This Guidance 258 259 Section 745A(a)(2) of the FD&C Act authorizes the Agency to set forth criteria for waivers from 260 the requirements of electronic submissions. FDA will grant short-term waivers from the eCTD 261 requirement only in unique and rare circumstances and for a limited duration. Companies 262 experiencing technical difficulties with transmission of their electronic submissions to FDA 263 should consult FDA for technical assistance rather than submitting a waiver request. FDA may 264 grant temporary waivers of the requirement for eCTD submission if one or more of the following 265 events or circumstances exist: 266 267 28 To follow up with the company, FDA will generally contact the individual who submitted the waiver request unless an alternate contact person is provided. 29 See footnote 25.
10 • Extraordinary events or circumstances occur that are beyond the control of the submitter 268 that justify a waiver, including but not limited to, natural disasters that impact computer 269 operations. 270 271 • An unplanned long-term internet disruption or other unplanned event occurs that would 272 preclude the sponsor from submitting in eCTD format (e.g., malware attacks). 273 274 • The sponsor or applicant intends to request a withdrawal of an application that has not yet 275 converted to eCTD format. 276 277 • The sponsor or applicant submitted a request for withdrawal and has not yet received 278 FDA’s acknowledgement of the withdrawal. 279 280 1. Content of Waiver Requests 281 282 The sponsor or applicant’s request to waive the eCTD electronic format requirement must 283 include all of the following as supporting documentation to justify the waiver:30 284 285 (a) A description of the circumstances or event — including the anticipated duration 286 of the circumstance or event — giving rise to the need for a waiver 287 288 (b) The requested duration of the waiver 289 290 (c) A description of the proposed alternative submission format31 the sponsor or 291 applicant will be using for the duration of the waiver 292 293 The request should reference all products that are to be covered by the waiver. The waiver 294 request should be clearly titled “WAIVER REQUEST — eCTD REQUIREMENTS” in bold 295 capital letters at the top of the first page of the submission. 296 297 Please submit waiver requests before filing a submission to which the waiver is claimed. 298 299 2. Where to Submit Waiver Requests 300 301 Waiver requests for NDAs, BLAs, ANDAs, DMFs, and commercial INDs may be sent to FDA 302 through the following means: 303 304 • CDER 305 - Email to esub@fda.hhs.gov 306 307 • CBER 308 - Email to esubprep@fda.hhs.gov 309 30 See 21 CFR 312.10(a)(3) and 314.90(a)(3). 31 See footnote 25.
11 310 3. FDA Response to Waiver Requests 311 312 FDA reviews waiver requests on a case-by-case basis. FDA will generally respond to the 313 requestor32 in writing, stating whether the waiver is granted or denied. If the waiver is granted, 314 FDA will also generally include in its response letter a description of the alternate submission 315 method(s) the Agency intends to accept and the time frame for the waiver. Waivers of the 316 requirement to submit in eCTD format, if granted, will be temporary, will apply only to 317 the requestor, and will not be transferrable to another sponsor. If FDA grants a waiver, 318 the requestor should include a statement in the cover letter of subsequent submissions 319 indicating that an eCTD submission waiver has been granted by FDA, including the dates 320 for the waiver. 321 322 Although these specific submissions may receive waivers from eCTD format requirements as 323 described in this guidance, FDA still encourages applicants to send submissions electronically in 324 an alternative electronic format (e.g., PDF files following the CTD structure33). 325 326 F. The eCTD Specifications 327 328 You must submit electronic submissions using a version of eCTD currently supported by FDA. 329 The versions of eCTD currently supported are specified in the Data Standards Catalog (available at 330 https://www.fda.gov/industry/fda-data-standards-advisory-board/study-data-standards-resources) 331 and is further described in the following technical specification documents: 332 333 International Council for Harmonisation (ICH) eCTD Version 3.2.2: 334 335 • ICH Electronic Common Technical Document Specification 336 337 • ICH eCTD Backbone File Specification for Study Tagging Files 338 339 • FDA eCTD Backbone Files Specification for Module 1 340 341 ICH eCTD Version 4.0: 342 343 • ICH eCTD v4.0 Implementation Guide 344 345 • FDA eCTD v4.0 Module 1 Implementation Guide 346 347 Additional technical specification documents are cited throughout this guidance. For a complete 348 list of required technical supportive files (e.g., style sheets and valid values) that you will need in 349 order to submit in the eCTD format, refer to the eCTD web page at 350 32 To follow up with the company, FDA will generally contact the individual who submitted the waiver request unless an alternate contact person is provided. 33 See footnote 25.
12 https://www.fda.gov/drugs/electronic-regulatory-submission-and-review/electronic-common- 351 technical-document-ectd. 352 353 G. Pre-Submission Considerations 354 355 Before making the first electronic submission to an application, you must obtain a pre-assigned 356 application number by contacting the appropriate Center. Information regarding how to obtain a 357 pre-assigned application number may be found on the eCTD web page at 358 https://www.fda.gov/drugs/electronic-regulatory-submission-and-review/submit-using-ectd. 359 360 H. Submission Structure: Granularity, Files, and Folders 361 362 Document granularity, or the level for which the submission content is broken out into separate 363 files, must be consistent with the Granularity Document found in the ICH guidance for industry 364 M4 Organization of the Common Technical Document for the Registration of Pharmaceuticals 365 for Human Use (October 2017) unless otherwise specified in the ICH M2 technical specification 366 eCTD IWG Question and Answer and Specification Change Request Document. 367 368 When submitting documents electronically using ICH eCTD version 3.2.2, with a few 369 exceptions, the eCTD specification maps the CTD headings to extensible markup language 370 (XML) elements.34 When submitting documents electronically using ICH eCTD version 4.0, the 371 eCTD specification maps the CTD headings to Health Level 7 (HL7) Regulated Product 372 Submission (RPS) Version 3 schema. The specification indicates that each element (heading) is 373 optional and that multiple document references (eCTD leaf or Context of Use elements) can be 374 created under each heading. 375 376 You must also follow the FDA eCTD technical specification The Comprehensive Table of 377 Contents Headings and Hierarchy for the comprehensive list of headings and hierarchy and a 378 section mapping the headings to their respective regulations.35 Because this is a comprehensive 379 list, not all headings are applicable to all submissions or submission types. 380 381 Files pertaining to each module must be placed in the appropriate folder (e.g., m1 – m5). The 382 terms folder and subfolder, as used in this guidance, are intended to be synonymous with 383 directory and subdirectory. The main submission, regional administrative folders, and certain 384 subfolders must have specific names. 385 386 You must use only letters, numbers, hyphens, or underscores in the folder and file names and not 387 blank spaces or special characters. When naming folders and files, the length of the entire path 388 must not exceed 150 characters. Empty folders and files must not be included in the submission. 389 34 For example, in Module 3, lower-level headings subordinate to 3.2.P.2 (e.g., 3.2.P.2.1, 3.2.P.2.1.1) are not mapped to an XML element. Consequently, leaf element files relating to, for example, 3.2.P.2.1, 3.2.P.2.1.1, either must be submitted as multiple leafs under the parent 3.2.P.2 element (heading) or combined into larger files and submitted at the 3.2.P.2 heading level. 35 See eCTD v3.2.2 or v4.0 submission standards at https://www.fda.gov/drugs/electronic-regulatory-submission- and-review/electronic-common-technical-document-ectd for corresponding version of The Comprehensive Table of Contents Headings and Hierarchy for eCTD.
13 390 All documents in the electronic submission must be placed in a main submission folder and 391 named using the sequence number (which you must specify): 392 393 • eCTD version 3.2.2 submissions must use a 4-digit number with leading zeros. 394 • eCTD version 4.0 submissions must use whole numbers without leading zeros. 395 396 The eCTD backbone file for the submission must be placed in the main submission folder along 397 with the checksum file for the eCTD backbone file: 398 399 • The eCTD version 3.2.2 backbone file (index.xml) applies only to modules 2 through 5 400 and uses md5 checksum. 401 402 • The eCTD version 4.0 backbone file (submissionunit.xml) applies to all modules and 403 uses sha256 checksum. 404 405 Numbering for each subsequent submission to the same application is described in: 406 407 • eCTD version 3.2.2, FDA technical specification, section III.B of the eCTD Backbone 408 Files Specification for Module 1 409 410 • ICH eCTD version 4.0 Implementation Guide 411 412 Sequence numbers are used to differentiate between submissions within the same application and 413 need not correspond to the order in which they are received by FDA. It is not necessary for 414 sequence numbers and IND serial numbers to match for submissions to an IND. 415 416 Subfolders within each module are required to organize files in a submission. These subfolders 417 must be placed in the sequence number folder. Empty subfolders must not be included. When 418 submitting documents electronically using eCTD version 3.2.2, the util subfolder is required to 419 organize supporting eCTD technical files in the submission, as described in the ICH M2 420 technical specification Electronic Common Technical Document Specification. Other specific 421 folder names that are compliant with the eCTD version 3.2.2 format can be found in the same 422 document. When submitting documents electronically using eCTD version 4.0, the required 423 compliant subfolder names and supporting eCTD technical files are described in the ICH eCTD 424 Version 4.0 Implementation Guide. 425 426 I. File Formats and Versions 427 428 Files within an eCTD submission must adhere to the formats and versions specified in the 429 associated FDA technical specification Specifications for File Format Types Using eCTD 430 Specifications. PDF files submitted must adhere to the FDA technical specification Portable 431 Document Format (PDF) Specifications. 432 433
14 J. Document Life Cycle 434 435 If a document replaces a document previously submitted with an eCTD backbone file within the 436 same application, you must use the eCTD replace operation to indicate this, rather than 437 submitting the file as new. 438 439 When submitting documents electronically using ICH eCTD version 3.2.2, you must not indicate 440 that files are new if they are in fact replacing files already submitted. If you intend to remove a 441 file, you must use the delete operation. For instructions, see the ICH M2 technical specification 442 Electronic Common Technical Document Specification. 443 444 When submitting documents electronically using ICH eCTD version 4.0, if a document replaces 445 one or more previously submitted documents within the same application, you must use the 446 replacementOf element. You must not indicate that files are new if they are in fact replacing 447 files already submitted. If you intend to remove a file, you must use the suspended status code. 448 For instructions, see the ICH eCTD Version 4.0 Implementation Guide. 449 450 K. Summary of Clinical Efficacy and Summary of Clinical Safety 451 452 When submitting a Summary of Clinical Efficacy and/or a Summary of Clinical Safety, the 453 location of these documents within the eCTD must adhere to the guidance for industry Integrated 454 Summaries of Effectiveness and Safety: Location Within the Common Technical Document 455 (April 2009). 456 457 L. Datasets and Study Information 458 459 Datasets must only be provided in modules 3, 4, or 5 and not in modules 1 or 2. 460 461 When submitting documents electronically using ICH eCTD version 3.2.2 and providing study 462 information in either module 4 or 5, you must include the Study Tagging File (STF) described in 463 the associated ICH M2 technical specification eCTD Backbone File Specification for Study 464 Tagging Files (see section III.F above). Datasets must be referenced in an STF using the 465 appropriate STF file-tag describing the document’s contents. 466 467 When submitting documents electronically using ICH eCTD version 4.0 and providing study 468 information in either module 4 or 5, you must include the Keyword Definition for a Study Id and 469 Study Title described in the associated ICH eCTD version 4.0 Implementation Guide (see section 470 III.F above). Datasets must be referenced in the submission unit using the appropriate document 471 type keyword describing the document’s contents. 472 473 For further information regarding the submission of study data, see the guidance for industry 474 Providing Regulatory Submissions in Electronic Format — Standardized Study Data (June 475 2021). 476 477
15 M. Transmitting Electronic Submissions 478 479 The FDA Electronic Submissions Gateway (ESG)36 enables the secure submission of regulatory 480 information for review and is our preferred method of transmission. For all submissions that are 481 10 gigabytes (GB) or smaller, you must use the FDA ESG. 482 483 For submissions that are greater than 10 GB, refer to the FDA technical specification 484 Transmitting Electronic Submissions Using eCTD Specifications. 485 486 N. FDA Forms 487 488 Electronic submissions must include only FDA fillable forms (e.g., Form FDA 1571 or Form 489 FDA 356h) and electronic signatures to enable automated processing of the submission. FDA 490 forms are available at https://www.fda.gov/about-fda/reports-manuals-forms/forms. Scanned 491 images of FDA forms will not be accepted. 492 493 O. Restrictions on Submission of Paper Copies 494 495 When submitting in eCTD format, paper copies of the application, including review copies and 496 desk copies in paper, must not be submitted. The only exception to this is the submission of 497 paper copies of meeting briefing materials, when requested, as described in the guidances for 498 industry on formal meetings between the FDA and sponsors or applicants.37 499 P. Receipt Date 500 501 The receipt date for an electronic submission will be determined only after the submission has 502 passed a technical validation check to ensure that it can be opened, processed, and archived. The 503 submitter is responsible for monitoring their receipt pathway to determine whether a submission 504 has been rejected. Additional information on the validation of electronic submissions is 505 available in the FDA technical specification Specifications for eCTD Validation Criteria. 506 507 Additional information on receipt dates for electronic submissions is available in the guidance 508 for industry Providing Regulatory Submissions in Electronic Format — Receipt Dates (February 509 2014). 510 511 Contact Information: 512 513 For questions related to providing electronic submissions according to the recommendations in 514 this guidance, contact the Electronic Submission Support Team (ESUB) at esub@fda.hhs.gov for 515 submissions to CDER and at esubprep@fda.hhs.gov for submissions to CBER. Specific 516 36 Additional information concerning the FDA ESG is available at https://www.fda.gov/industry/electronic-submissions-gateway. 37 See also the following draft guidances: Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products (September 2023) and Formal Meetings Between the FDA and Sponsors or Applicants of BsUFA Products (August 2023). When finalized, these guidances will represent FDA’s current thinking on these topics.
16 questions pertaining to the content of applications should be directed to the appropriate review 517 division or office. 518 519 520 IV. TECHNICAL SPECIFICATION DOCUMENTS REFERENCED IN THIS 521 GUIDANCE 522 523 524 The following are technical specification documents referenced in this guidance (see section I). 525 526 For a complete list of the current technical supportive files that you will need in order to submit 527 in eCTD format, refer to the eCTD Submission Standards document located on FDA’s eCTD 528 web page at https://www.fda.gov/drugs/electronic-regulatory-submission-and-review/electronic- 529 common-technical-document-ectd. 530 531 532 1. Electronic Common Technical Document Specification - ICH M2 technical specification 533 534 2. The eCTD Backbone File Specification for Study Tagging Files - ICH M2 EWG 535 technical specification 536 537 3. eCTD Backbone Files Specification for Module 1 - FDA technical specification 538 539 4. eCTD IWG Question and Answer and Specification Change Request Document - ICH 540 M2 technical specification 541 542 5. FDA eCTD Comprehensive Table of Contents Headings and Hierarchy - FDA 543 technical specification 544 545 6. Specifications for File Format Types Using eCTD Specifications - FDA technical 546 specification 547 548 7. Portable Document Format (PDF) Specifications - FDA technical specification 549 550 8. Transmitting Electronic Submissions Using eCTD Specifications - FDA technical 551 specification, Transmission Specifications 552 553 9. Specifications for eCTD Validation Criteria - FDA technical specification, eCTD 554 Validation Specifications web page 555 556 10. eCTD v4.0 Implementation Guide - ICH M8 technical specification 557 558 11. Module 1 eCTD v4.0 Implementation Guide - FDA technical specification 559 560
17 V. RELATED REFERENCES 561 562 The guidance documents referenced below can be accessed via FDA’s guidance web page at 563 https://www.fda.gov/industry/fda-basics-industry/guidances. 564 565 1. Providing Regulatory Submissions in Electronic Format — Submissions Under Section 566 745A(a) of the Federal Food, Drug, and Cosmetic Act (December 2014) - FDA guidance for 567 industry 568 569 2. Providing Regulatory Submissions in Electronic Format — Standardized Study Data (June 570 2021) - FDA guidance for industry 571 572 3. Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products 573 (September 2023) - FDA draft guidance for industry 574 575 4. Formal Meetings Between the FDA and Sponsors or Applicants of BsUFA Products (August 576 2023) - FDA draft guidance for industry 577 578 5. Providing Submissions in Electronic Format – Postmarketing Safety Reports (April 2022) - 579 FDA guidance for industry 580 581 6. Providing Regulatory Submissions in Electronic Format — Receipt Dates (February 2014) - 582 FDA guidance for industry 583 584 7. M4 Organization of the Common Technical Document for the Registration of 585 Pharmaceuticals for Human Use (October 2017) - ICH guidance for industry 586 587 8. Integrated Summaries of Effectiveness and Safety: Location Within the Common Technical 588 Document (April 2009) - FDA guidance for industry 589 590 9. Reporting Requirements for INDs and BA/BE Studies (December 2012) - FDA guidance for 591 industry 592 593 10. Providing Regulatory Submissions in Electronic Format: IND Safety Reports (April 2024) - 594 FDA guidance for industry 595 596 11. Providing Regulatory Submissions in Electronic and Non-Electronic Format — Promotional 597 Labeling and Advertising for Human Prescription Drugs (April 2022) - FDA guidance for 598 industry 599 600 601 602 603 604 605
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