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 U.S. Food and Drug Administration (FDA or the Agency) has specified the electronic format for submitting certain submission types to the Agency, such content must be submitted electronically and in the format specified by FDA. FDA’s guidance for industry Providing Regulatory Submissions in Electronic Format — Standardized Study Data (December 2014 [original]; October 2020 [revision 1]; June 2021 [revision 2]) implements and describes these electronic submission requirements under section 745A(a) of the FD&C Act for clinical and nonclinical study data, specifies the types of submissions to which these requirements apply, and specifies timetables for the implementation of the requirements.
On March 11, 2020, FDA published a Federal Register notice that announced that FDA’s support of the Clinical Data Interchange Standards Consortium (CDISC) for Study Data Tabulation Model version 1.8 (SDTM v1.8), and CDISC Standard for Exchange of Nonclinical Data Implementation Guide—Animal Rule version 1.0 (SENDIG–AR v1.0) began on March 15, 2020. That document also announced that these new standards would be required in submissions for studies that start after March 15, 2022. That document omitted the 36-month implementation period for certain INDs as required by FDA’s guidance for industry Providing Regulatory Submissions in Electronic Format–Standardized Study Data. On June 10, 2021, FDA published a Federal Register notice that corrected that error and clarified that that these new standards would be required for studies initiated after March 15, 2022, for new drug applications (NDAs), abbreviated new drug applications (ANDAs), and biologics license applications (BLAs) and for studies initiated after March 15, 2023, for certain investigational new drug applications (INDs) submitted to the Center for Drug Evaluation and Research (CDER) for products being developed under the regulations commonly known as the Animal Rule (see 21 CFR 314.600-650 for drugs and 21 CFR 601.90-95 for biological products). Currently, only CDER will require the use of these Animal Rule data standards (i.e., SDTM v1.8 and SENDIG-AR v1.0).
Specifically, Standard Exchange for Nonclinical Data (SEND) datasets are required for any nonclinical natural history or efficacy study initiated after March 15, 2022, for NDAs, ANDAs, and BLAs and for any nonclinical natural history or efficacy study initiated after March 15, 2023, for certain INDs that are submitted to CDER and for which the CDER review division expects a full tabulation of data (i.e., line listings of the results for each individual animal) to support detailed review. Although not required, FDA also recommends that sponsors submit SEND datasets for such studies that were initiated before March 15, 2022, and March 15, 2023, as applicable. In addition, FDA recommends SEND datasets for such studies that are submitted to pre-INDs and FDA’s Animal Model Qualification Program.
Application-specific questions about which natural history and efficacy studies should include full tabulations of data and datasets should be discussed with the appropriate CDER review division as early as possible during product development. Questions about natural history studies that will be submitted to an animal model qualification package should be discussed with the Animal Model Qualification Program (contact CDERAnimalModelQualification@fda.hhs.gov).
Additional information about data standards is available at FDA’s Study Data Standards Resources web page. For additional information on how FDA interprets and intends to implement the electronic submission requirements of section 745A(a) of the FD&C Act, see FDA’s guidance for industry Providing Regulatory Submissions in Electronic Format – Submissions Under Section 745A(a) of the Federal Food, Drug, and Cosmetic Act (December 2014). For general data standards inquiries for CDER, contact cder-edata@fda.hhs.gov.
For further information about FDA’s expectations for the reports of nonclinical natural history and efficacy studies submitted to support Animal Rule applications (exclusive of the data standards) see section IV.D of FDA’s guidance for industry Product Development Under the Animal Rule (October 2015).
