Submission-Level Information A total of 6 data elements, shown in Table 1, pertain to the entire submission. Most relate to regulatory status. Table 1: Submission-Level Information [NOTE: Yellow highlight indicates a proposed change (new variable, variable name, variable label) from the draft 7 November 2012 structure] Variable Index Variable Name Variable Label Type Controlled Terms or Format Notes or Description Sample Value Comments CDISC Source (S=SDTM, A=ADaM) 5 IND IND Number Num 6 digit identifier Investigational New Drug (IND) application number. If study not performed under IND, leave blank. 010010 No existing Controlled Terminology for TSPARM/TSPARMCD. S/TS new TSPARM/TSPARMCD 7 NDA NDA Number Num 6 digit identifier FDA new drug application (NDA) number, if available/applicable. If not applicable, leave blank. 021212 No existing Controlled Terminology for TSPARM/TSPARMCD. S/TS new TSPARM/TSPARMCD 8 BLA BLA Number Num 6 digit identifier FDA identification number for biologics license application, if available/applicable. If not applicable, leave blank. 123456 No existing Controlled Terminology for TSPARM/TSPARMCD. S/TS new TSPARM/TSPARMCD 3 SPONCNT Sponsor Count Num Integer Total count of sponsors throughout the study. If there was a change in the sponsor while the study was ongoing, with sponsors as defined in 21 CFR 312.3, enter an integer indicating the total count of sponsors. If there was no change in the sponsor while the study was ongoing, enter “1”. 1 No existing Controlled Terminology for TSPARM/TSPARMCD. S/TS new TSPARM/TSPARMCD
4 SPONNAME Sponsor Name Char String Full name of the sponsor organization conducting the study at the time of study completion, as defined in 21CFR 312.3(a). If the sponsor name exceeds 200 characters, provide the abbreviated sponsor name from Clinicaltrials.gov and document this in the Notes or Description section in the define file (Clinical Site Data Elements Summary). Drug Co, Inc. S/TSPARM where TSPARMCD=’SPONSOR’ 9 SUPPNUM Supplemental Number Num Integer Serial number for supplemental application, if applicable. If no information is available, leave blank. 4 No existing Controlled Terminology for TSPARM/TSPARMCD. S/TS new TSPARM/TSPARMCD
Study-Level Information A total of 5 data elements, shown in Table 2, pertain to the study. Variable STUDYID is a potential index variable and key that allows for integration with other datasets. Table 2: Study-Level Information [NOTE: Yellow highlight indicates a proposed change (new variable, variable name, variable label) from the draft 7 November 2012 structure] Variable Index Variable Name Variable Label Type Controlled Terms or Format Notes or Description Sample Value Comments CDISC Source (S=SDTM, A=ADaM) 1 STUDYID Study Identifier Char String Study or trial identification number. ABC-123 S/TS.STUDYID 2 STUDYTL Study Title Char String Title of the study as listed in the clinical study report (limit 200 characters). If the title exceeds 200 characters, provide the abbreviated title from Clinicaltrials.gov and document this in the Notes or Description section in the define file (Clinical Site Data Elements Summary). Double blind, randomized, placebo controlled clinical study on the influence of drug X on indication Y S/TS.TSPARMCD=’TITLE’ 11 ARM Description of Planned Treatment Arm Char String Plain text label for the name given to an Arm or treatment group as referenced in the clinical study report (limit 200 characters). Active name and dose (e.g., Active 25mg), Comparator product name (e.g., Drug x), or Placebo S/TA.ARM
14 DISCSTUD Number of Subject Discons from Study Num Integer Number of subjects in the safety population who discontinued from the study. 5 This pertains to subjects that were enrolled after informed consent was obtained; could capture in ADaM.ADSL.EOSSTT if data were collected. 15 DISCTRT Number of Subject Discons from Study Treatment Num Integer Number of subjects in the safety population who discontinued from the study treatment. 10 Could capture in ADaM.ADSL if data were collected. 18 TRTEFFR Treatment Efficacy Result Num Floating Point Summary statistic for each primary efficacy endpoint by treatment arm at a given site. 0, 0.25, 1, 100 Variable on which treatments are compared according to the SAP; e.g., change from baseline, time to event. 19 TRTEFFS Treatment Efficacy Result Standard Deviation Num Floating Point Standard deviation of the efficacy result (TRTEFFR) for each primary endpoint by treatment arm at a given site. If N=1, set to 0. 0.065 20 SITEEFFE Site-Specific Efficacy Effect Size Num Floating Point Site effect size with the same representation as reported for the primary efficacy analysis. 0, 0.25, 1, 100 The pre-specified primary comparison is the effect size desired here. 21 SITEEFFS Site-Specific Efficacy Effect Size Standard Deviation Num Floating Point Standard deviation of the efficacy effect size (SITEEFFE). If N=1, set to 0. 0.065
28 LASTNAME Investigator Last Name Char String Last name of the principal clinical investigator as it appears on the Form FDA 1572 or the signed investigator agreement. At sites where the clinical investigator has changed during the course of the study, the most recent clinical investigator should be listed. Doe Only the principal clinical investigator is needed here. May be able to capture in ADaM by parsing SDTM DM.INVNAM. 29 FRSTNAME Investigator First Name Char String First name of the principal clinical investigator as it appears on the Form FDA 1572 or the signed investigator agreement. John Only the principal clinical investigator is needed here. May be able to capture in ADaM by parsing SDTM DM.INVNAM. 30 MINITIAL Investigator Middle Initial Char String Middle initial of the principal clinical investigator, if any, as it appears on the Form FDA 1572 or the signed investigator agreement. M Only the principal clinical investigator is needed here. May be able to capture in ADaM by parsing SDTM DM.INVNAM. 31 PHONE Investigator Phone Number Char String Phone number of the principal clinical investigator. Include country code for non-US numbers. 32 FAX Investigator Fax Number Char String Fax number of the principal clinical investigator. Include country code for non-US numbers. If not available, leave blank. 33 EMAIL Investigator Email Address Char String Email address of the principal clinical investigator.
34 COUNTRY Country Char ISO 3-letter ISO 3166 country code in which the site is located. USA S/DM.COUNTRY or A/ADSL.COUNTRY 35 STATE State Char String Unabbreviated state or province in which the site is located. If not applicable, enter NA. Maryland 36 CITY City Char String Unabbreviated city, county, or village in which the site is located. Silver Spring 37 POSTAL Postal Code Char String Postal code in which the site is located. If not applicable, enter NA. 20850 38 STREET Street Address Char String Street address and office number at which the site is located. 1 Main St. 39 STREET1 Street Address Char String Street address and office number at which the site is located. Use this field when Variable STREET does not permit sufficient space to fully describe street address and office number at which the site is located. Suite 100 Note that our Working Group is proposing that variables DOMAIN and FINLMAX be removed from the FDA’s draft technical specifications dated 7 November 2012.
SAE (Non-Serious AEs) DEATH (Deaths) PROTVIOL (Protocol Violations) For the other 18 data elements (46%) shown below, such as investigator contact information, most are included in the eCTD submission but are not in a format amenable to data analysis. Although technology exists to mine the xml backbone of the eCTD for information, it is not clear that this would be a more efficient process than obtaining it by more traditional methods. Issues are that some of the information may not be known at the time CRT datasets are finalized (e.g., NDA or BLA number), while other information may be known (e.g., investigator contact information), potentially resulting in some xml backbone mining being done by the FDA and some by the sponsor. UNDERIND (Under IND) TRTEFFR (Treatment Efficacy Result) TRTEFFS (Treatment Efficacy Std Dev) SITEEFFE (Site Efficacy Effect Size) SITEEFFS (Site Efficacy Effect Size Std Dev) FINLDISC (Financial Disclosure Amt.) LASTNAME (Last Name) FIRSTNAME (First Name) MINITIAL (Middle Initial) PHONE (Phone) FAX (FAX) EMAIL (Email Address) COUNTRY (Country) STATE (State) CITY (City) POSTAL (Postal Code) STREET (Street Address) STREET1 (Street Address Cont’d) A tabular representation of this information is shown below: CDISC Applicability of Data Elements Variable Category Variable Count Fraction of Total (39) Explicitly accommodated by CDISC 7 18% Accommodated by CDISC if extensible Controlled Terminology used 5 13% Can easily derive from SDTM/ADaM and capture in ADaM 9 23% No existing path 18 46%
Appendix 2: Proposed Revised FDA Draft Technical Specifications Variable Index Variable Name Variable Label Type Controlled Terms or Format Notes or Description Sample Value 1 STUDYID Study Identifier Char String Study or trial identification number. ABC-123 2 STUDYTL Study Title Char String Title of the study as listed in the clinical study report (limit 200 characters). If the title exceeds 200 characters, provide the abbreviated title from Clinicaltrials.gov and document this in the Notes or Description section in the define file (Clinical Site Data Elements Summary). Double blind, randomized placebo controlled clinical study on the influence of drug X on indication Y 3 SPONCNT Sponsor Count Num Integer Total count of sponsors throughout the study. If there was a change in the sponsor while the study was ongoing, with sponsors as defined in 21 CFR 312.3, enter an integer indicating the total count of sponsors. If there was no change in the sponsor while the study was ongoing, enter “1”. 1 4 SPONNAME Sponsor Name Char String Full name of the sponsor organization conducting the study at the time of study completion, as defined in 21 CFR 312.3(a). If the sponsor name exceeds 200 characters, provide the abbreviated sponsor name from Clinicaltrials.gov and document this in the Notes or Description section in the define file (Clinical Site Data Elements Summary). Drug Co, Inc. 5 IND IND Number Num 6 digit identifier Investigational New Drug (IND) application number. If study not performed under IND, leave blank. 010010 6 UNDERIND Under IND Char String Value should equal “Y” if study at the site was conducted under an IND (i.e., a Form FDA 1572 was signed by the investigator) and “N” if the study was not conducted under an IND at the site (i.e., a Form FDA 1572 was not signed by the investigator). Y 7 NDA NDA Number Num 6 digit identifier FDA new drug application (NDA) number, if available/applicable. If not applicable, leave blank. 021212 8 BLA BLA Number Num 6 digit identifier FDA identification number for biologics license application, if available/applicable. If not applicable, leave blank. 123456
2 Change from Baseline Continuous 0.74 0.0861 0.60 0.1502 ∙ 0 2 0 1 < $25,000 Doe John 3 Percent Responders Binary 0.14 0.0694 0.34 0.1405 ∙ 2 2 0 1 < $25,000 Doe John 4 Change from Baseline Continuous 0.14 0.0699 0.60 0.1502 ∙ 2 2 0 1 < $25,000 Doe John 0 Percent Responders Binary 0.48 0.1042 0.33 0.1427 ∙ 3 2 1 0 ≥ $25,0000 Washington George 1 Change from Baseline Continuous 0.67 0.0983 0.52 0.1515 ∙ 3 2 1 0 ≥ $25,0000 Washington George 4 Percent Responders Binary 0.14 0.0694 0.33 0.1427 ∙ 0 2 0 3 ≥ $25,0000 Washington George 3 Change from Baseline Continuous 0.14 0.0700 0.52 0.1515 ∙ 0 2 0 3 ≥ $25,0000 Washington George 2 Percent Responders Binary 0.54 0.0959 0.35 0.1448 ∙ 2 2 0 1 ≥ $25,0000 Jefferson Thomas 0 Change from Baseline Continuous 0.65 0.0931 0.43 0.1485 ∙ 2 2 0 1 ≥ $25,0000 Jefferson Thomas 5 Percent Responders Binary 0.19 0.0769 0.35 0.1448 ∙ 3 6 0 0 ≥ $25,0000 Jefferson Thomas 6 Change from Baseline Continuous 0.19 0.0769 0.43 0.1485 ∙ 3 6 0 0 ≥ $25,0000 Jefferson Thomas 1 Percent Responders Binary 0.46 0.0977 0.34 0.1275 ∙ 4 1 0 0 unknown Lincoln Abraham 0 Change from Baseline Continuous 0.71 0.0891 0.5545 0.1397 ∙ 4 1 0 0 unknown Lincoln Abraham 2 Percent Responders Binary 0.12 0.0625 0.34 0.1275 ∙ 1 2 0 1 unknown Lincoln Abraham 1 Change from Baseline Continuous 0.15 0.0694 0.5545 0.1397 ∙ 1 2 0 1 unknown Lincoln Abraham
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