Contains Nonbinding Recommendations 

Collection of Race and Ethnicity Data
 

in Clinical Trials
 

Guidance for Industry and 

Food and Drug Administration Staff
 

Document issued on October 26, 2016 

For questions about this document, contact the FDA Office of Minority Health at 240-402-5084 or 

omh@fda.hhs.gov. 

U.S. Department of Health and Human Services (HHS)
 

Food and Drug Administration (FDA)
 

Office of the Commissioner (OC)
 

Office of Minority Health (OMH)
 

Office of Women’s Health (OWH) 

Center for Drug Evaluation and Research (CDER)
 

Center for Biologics Evaluation and Research (CBER)
 

Center for Devices and Radiologic Health (CDRH)
 

October 2016
 

Clinical Medical
 
 

 

 

 

Contains Nonbinding Recommendations 

Preface
 

Public Comment
 

You may submit electronic comments and suggestions at any time for Agency consideration to 

http://www.regulations.gov. Submit written comments to the Division of Dockets Management, 

Food and Drug Administration, 5630 Fishers Lane, Room 1061, (HFA-305), Rockville, MD 20852. 

Identify all comments with the docket number FDA-2016-D-3561. Comments may not be acted 

upon by the Agency until the document is next revised or updated. 

Additional Copies 

Additional copies are available from the Internet. You may also send an e-mail request to 

omh@fda.hhs.gov to receive a copy of the guidance. 

Additional copies are available from the Office of The Commissioner by written request to the 

Office of Minority Health, 10903 New Hampshire Ave., WO1, Room 3331A, Silver Spring, MD 

20993-0002, by calling 240-402-5084, or by email, omh@fda.hhs.gov. 

U.S. Department of Health and Human Services (HHS)
 

Food and Drug Administration (FDA)
 

Office of the Commissioner (OC)
 

Office of Minority Health (OMH)
 

Office of Women’s Health (OWH) 

Center for Drug Evaluation and Research (CDER)
 

Center for Biologics Evaluation and Research (CBER)
 

Center for Devices and Radiologic Health (CDRH)
 

October 2016
 

Clinical Medical
 
 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Contains Nonbinding Recommendations 

Table of Contents 

I. INTRODUCTION ................................................................................................................................. 1
 

II. SCOPE ................................................................................................................................................... 2
 

III. BACKGROUND ................................................................................................................................... 4
 

A.	 Department of Health and Human Services Report and Guidance ........................................................ 4
 

B.	 ICH E5 - Guidance on Ethnic Factors in the Acceptability of Foreign Clinical Data ...........................5
 

C.	 National Institutes of Health Initiatives, Revitalization Act .................................................................. 5
 

D.	 FDA Regulations, Guidances, and Section 907 of the Food and Drug Administration Safety and 

Innovation Act of 2012 (FDASIA) ........................................................................................................ 6
 

Relevance of Population Subgroup Studies .................................................................................. 7
 

IV. COLLECTING RACE AND ETHNICITY DATA IN CLINICAL TRIALS ....................................... 9
 

A.	 Two-Question Format ............................................................................................................................ 9
 

B.	 Self-Reporting........................................................................................................................................ 9
 

C.	 Ethnicity................................................................................................................................................. 9
 

D.	 Race ..................................................................................................................................................... 10
 

E.	 Use of More Detailed Racial and Ethnic Categories ........................................................................... 11
 

F.	 Use of the term “nonwhite” ................................................................................................................. 12
 

V. PRESENTATION OF CLINICAL TRIAL RACE AND ETHNICITY DATA .................................. 12
 

VI. REFERENCES .................................................................................................................................... 13
 

VII.	 BIBLIOGRAPHY................................................................................................................................ 13
 

HHS Policy and Reports .............................................................................................................................. 13
 

NIH Policies, Reports, and Resources ......................................................................................................... 14
 

FDA Regulations, Reports, and Legislation ................................................................................................ 14
 

FDA Guidances for Industry........................................................................................................................ 14
 

ICH Guidances............................................................................................................................................. 15
 
 

 

 

Contains Nonbinding Recommendations 

Other Sources............................................................................................................................................... 16
 

NIH PHS Cumulative Inclusion Enrollment Report Form .......................................................................... 16
 
 

 

 

 

Collection of Race and Ethnicity Data
 

in Clinical Trials
 

Guidance for Industry and Food and
 

Drug Administration Staff1
 

This guidance represents the current thinking of the Food and Drug Administration (FDA or 

Agency) on this topic. It does not establish any rights for any person and is not binding on FDA 

or the public. You can use an alternative approach if it satisfies the requirements of the applicable 

statutes and regulations. To discuss an alternative approach, please contact the relevant FDA 

review division for your medical product. 

I. INTRODUCTION 

The purpose of this guidance is to provide FDA expectations for and recommendations on 

use of a standardized approach for collecting and reporting race and ethnicity data in 

submissions for clinical trials for FDA regulated medical products conducted in the United 

States and abroad. Using standard terminology for age, sex, gender, race, and ethnicity helps 

ensure that subpopulation data is collected consistently. The recommended standardized 

2 approach is based on the Office of Management and Budget (OMB) Directive 15 and 

3 developed in accordance with section 4302 of the Affordable Care Act , the HHS 

Implementation Guidance on Data Collection Standards for Race, Ethnicity, Sex, Primary 

4 Language, and Disability Status , and the Food and Drug Administration Safety and 

5 6 Innovation Act (FDASIA) Section 907 Action Plan . This guidance lists the OMB 

1 This guidance has been developed by the Agency-wide Race and Ethnicity Working Group from the Office of the 

Commissioner (OC), the Office of Minority Heath (OMH), the Office of Women’s Health (OWH), the Office of 

Public Health Strategy and Analysis (OPHSA), the Center for Biologics Evaluation and Research (CBER), the Center 

for Drug Evaluation and Research (CDER), and the Center for Devices and Radiological Health (CDRH) of the Food 

and Drug Administration (FDA). 2	 

 Office of Management and Budget (OMB) Directive No. 15 Revisions to the Standards for the Classification of 

Federal Data on Race and Ethnicity (October 30, 1997), available at 

https://www.whitehouse.gov/omb/fedreg_1997standards 3 Patient Protection and Affordable Care Act, Public Law 111-148, Section 4302 (42 U.S.C. § 300kk) (March 23, 

2010), available at https://www.gpo.gov/fdsys/pkg/CREC-2009-11-19/pdf/CREC-2009-11-19-pt1-PgS11607-

3.pdf#page=127 4 HHS Implementation Guidance on Data Collection Standards for Race, Ethnicity, Sex, Primary Language, and 

 Disability Status (October 31, 2011), available at https://aspe.hhs.gov/sites/default/files/pdf/76331/index.pdf 5	 

 Food and Drug Administration Safety and Innovation Act (FDASIA), Public Law 112-144 (July 9, 2012), available at 

https://www.congress.gov/bill/112th-congress/senate-bill/3187 
 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Contains Nonbinding Recommendations 

categories for race and ethnicity and describes FDA's reasons for recommending the use of 

these categories in medical product (drugs, biologics, and devices) applications. In addition, 

this guidance recommends a format for collection of race and ethnicity clinical trial data that 

are submitted in standardized data sets per the Study Data Tabulation Model7

, in the 

electronic Common Technical Document (eCTD)8

, and as specified in FDA’s Guidance on 

providing regulatory submissions in electronic format9 . 

This document is intended to provide guidance on: 

	 Meeting the requirements set forth in the 1998 final rule10 regarding presentation of 

demographic data on investigational new drug (IND) applications and new drug 

applications (NDAs) (known as the “Demographic Rule”) and collection of race and 

ethnicity data in biologics license applications (BLAs) and medical device applications 

	 Addressing the FDA Safety and Innovation Act, Section 907 Action Plan6 to improve 

demographic subgroup gaps in data quality 

FDA's guidance documents, including this guidance, do not establish legally enforceable 

responsibilities. Instead, guidances describe the Agency's current thinking on a topic and 

should be viewed only as recommendations, unless specific regulatory or statutory 

requirements are cited. The use of the word should in Agency guidances means that something 

is suggested or recommended, but not required. 

II. SCOPE 

This document is intended to provide guidance on the collection of race and ethnicity in 

clinical trials. This guidance provides clarifying recommendations on the two step collection 

process of race and ethnicity data and has been developed in support of the FDASIA 907 

Action Plan to improve the completeness and quality of demographic subgroup data. This 

guidance replaces the 2005 FDA guidance on Collection of Race and Ethnicity in Clinical 

Trials11 . 

6	 FDA Action Plan To Enhance The Collection and Availability of Demographic Subgroup Data (August 2014) , 

available at http://www.fda.gov/downloads/RegulatoryInformation/Lefislation/SignificantAmendmentstothe 

FDCAct/FDASIA/UCM410474.pdf 7	 CDISC standardized Study Data Tabulation Model (SDTM), Analysis Data Model (ADaM), Operational Data Model 

(ODM), http://www.cdisc.org/system/files/members/standard/study_data_tabulation_model_v1_4.pdf 8	 Providing Regulatory Submissions in Electronic Format – Certain Human Pharmaceutical Product Applications and 

Related Submissions Using the eCTD Specifications: Guidance for Industry (May 2015), available at 

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/FormsSubmissionRequirements/ElectronicSubmissions/uc 

m153574.htm 9	 Providing Regulatory Submissions In Electronic Format - Standardized Study Data: Guidance for Industry 

(December 2014), available at http://www.fda.gov/downloads/Drugs/.../Guidances/UCM292334.pdf 10 63 FR 6854 (February 11, 1998) (codified at 21 CFR 312.33(a)(2) and 21 CFR 314.50(d)(5)) , 

https://www.gpo.gov/fdsys/pkg/FR-1998-02-11/pdf/98-3422.pdf 11 Collection of Race and Ethnicity in Clinical Trials (September 2005), 

http://www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm126396.pdf 

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For drugs, the Demographic Rule requires IND holders to tabulate in their annual report the 

number of participants enrolled in clinical trials by age, race, and gender12 and requires 

sponsors of NDAs to include summaries of effectiveness and safety data for important 

demographic subgroups, including racial subgroups13. FDA also strongly recommends the 

collection and reporting of ethnicity data (Hispanic-Latino/not Hispanic-Latino) consistent 

with OMB standards and guidelines2 . 

This guidance is also intended to help applicants in preparing BLAs. 

For medical devices, FDA also recommends application sponsors collect ethnicity and race data 

in accordance with the OMB recommendations2, the information collection standards discussed 

in this guidance document and, when finalized, the Center for Devices and Radiologic Health 

(CDRH) and Center for Biologics Evaluation and Research (CBER) Age/Race/Ethnicity draft 

guidance document14 . 

FDA expectations are that sponsors enroll participants who reflect the demographics for 

13 clinically relevant populations with regard to age, gender, race, and ethnicity . A plan to 

address inclusion of clinically relevant subpopulations should be submitted for discussion to 

the Agency at the earliest phase of development and, for drugs and biologics, no later than the 

end of the phase 2 meeting. Inadequate participation and/or data analyses from clinically 

relevant subpopulations can lead to insufficient information pertaining to medical product 

safety and effectiveness for product labeling. Patient characteristics such as age, sex, gender, 

geographic location (e.g. rural), emotional, physical, sensory, and cognitive capabilities can 

often be important variables when evaluating medical product safety and efficacy. However, 

these will not be addressed within this guidance. 

This guidance does not address the level of participation of racial and ethnic groups in clinical 

trials. For questions related to the level of participation or the size of a trial, sponsors should 

12 The terms sex and gender have been used interchangeably in some FDA documents. However, according to a 2001 

consensus report from the Institute of Medicine (Institute of Medicine, Committee on Understanding the Biology of 

Sex and Gender Differences. Exploring the Biological Contributions to Human Health: Does Sex Matter?, National 

Academy of Sciences, 2001), the terms have distinct definitions which should be used consistently to describe 

research results. Sex refers to the classification of living things, generally as male or female according to their 

reproductive organs and functions assigned by chromosomal complement. Gender refers to a person’s selfrepresentation as male or female, or how that person is responded to by social institutions based on the individual’s 

gender presentation. Gender is rooted in biology, and shaped by environment and experience. Because of underlying 

differences in the statutes and regulations referenced in this policy, the terms “gender” and “sex” have both been used 

in this document in accordance with the source material referenced. 13 21 CFR § 312.33(a)(2) (April 1, 2015). See also 21 CFR § 314.50(d)(5)(v) and (vi)(a) regarding demographic data 

submission in NDAs, available at http://www.ecfr.gov/cgi-bin/textidx?SID=41aa69ac1cd29cf7eb1c3b24d764f310 

&mc=true&node=se21.5.314_150&rgn=div8 14 Draft Guidance: Evaluation and Reporting of Age, Race, and Ethnicity Data in Medical Device Clinical Studies (June 

2016), available at http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/ 

GuidanceDocuments/UCM507278.pdf. Draft guidances are included for completeness only. As draft documents, 

they are not intended to be implemented until published in final form. 

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consult with the review division of the appropriate centers and office15 16 17for guidance on 

clinical trial design and demographic sub-groups prior to the start of a trial. 

III. BACKGROUND 

Over recent decades the Agency’s views, as well as those of the medical community, have evolved 

regarding the collection of race and ethnicity information in clinical studies. 

Prior to developing the recommendations set forth in this guidance, FDA publicly sought input 

from a variety of experts and stakeholders regarding the study and evaluation of age, race, and 

ethnicity in clinical studies for medical products. On April 1, 2014, FDA convened a public 

18 19 hearing for feedback on the findings of the FDASIA 907 Report, to obtain input on the issues 

and challenges associated with the collection, analysis, and availability of demographic subgroup 

data (i.e., age, sex, race, and ethnicity) in applications for approval of FDA‐regulated medical 

18 20 products. FDA also opened a public docket for further input . On April 9, 2015 and December 2, 

21 2015 , various government agencies, physician professional societies, and patient advocacy groups 

participated in public workshops to discuss strategies for ensuring diversity, inclusion, and 

meaningful participation in clinical trials. This guidance reflects the concerns and 

recommendations generated in these and other public fora. The following is a brief history of the 

adoption of the OMB categories for reporting of race and ethnicity data by the Department of 

Health and Human Services (HHS), the National Institutes of Health (NIH) and FDA. 

A. Department of Health and Human Services Report and 

Guidance
 

In 1999, HHS issued a report, Improving the Collection and Use of Racial and Ethnic Data 

in HHS22 . The report describes HHS policy on collecting and reporting data on race and 

15 Center for Devices and Radiological Health Organization, 

http://www.fda.gov/AboutFDA/CentersOffices/OrganizationCharts/ucm347835.htm 16 CBER Offices and Divisions, 

http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CBER/ucm122875.htm 17 CDER Offices and Divisions, 

http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm075128.htm 18 Notice of Public Hearing: Action Plan for the Collection, Analysis, and Availability of Demographic Subgroup Data 

in Applications for Approval of Food and Drug Administration-Regulated Medical Products, 79 FR 42, 12134 

(March 4, 2014), available at https://www.federalregister.gov/articles/2014/03/04/2014-04625/action-plan-for-thecollection-analysis-and-availability-of-demographic-subgroup-data-in 

19 http://www.fda.gov/downloads/RegulatoryInformation/Legislation/SignificantAmendmentstotheFDCAct/FDASIA/U 

CM397391.pdf (April 1, 2014) 20 Institute of Medicine Workshop: Strategies for Ensuring Diversity, Inclusion, and Meaningful Participation in 

Clinical Trials (April 9, 2015), available at 

http://www.nationalacademies.org/hmd/Activities/SelectPops/HealthDisparities/2015-APR-09.aspx 21 FDA Public Meeting: Clinical Trials-Assessing Safety and Efficacy for Diverse Populations, 80 FR 210, 66909 

(October 30, 2015), available at https://www.federalregister.gov/articles/2015/10/30/2015-27728/clinical-trialsassessing-safety-and-efficacy-for-diverse-populations-public-meeting-request-for 22 Improving the Collection and Use of Racial and Ethnic Data in HHS (December 1, 1999), available at 

https://aspe.hhs.gov/report/improving-collection-and-use-racial-and-ethnic-data-hhs 

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ethnicity for HHS programs. The report asks for the inclusion of race and ethnicity 

categories in HHS funded and sponsored data collection and reporting systems in all HHS 

programs, including in both health and human services. The policy was developed to (1) 

help monitor HHS programs, (2) determine whether Federal funds are being used in a 

nondiscriminatory manner, and (3) promote the availability of standard race and ethnicity 

data across various agencies to facilitate HHS responses to major health and human 

4 services issues. This policy, updated in 2011 , clearly states that the minimum standard 

2 categories in OMB Policy Directive 15 should be used when collecting and reporting data 

23 in HHS data systems or reporting HHS funded statistics . 

B. ICH E5 - Guidance on Ethnic Factors in the Acceptability of 

Foreign Clinical Data 

In 1999, as part of an international effort by the United States and others to harmonize 

technical requirements for pharmaceutical drug development and regulation (the 

International Conference on Harmonization (ICH)), the FDA published a guidance entitled 

E5 Guidance on Ethnic Factors in the Acceptability of Foreign Clinical Data24 (63 FR 

31790, June 10, 1998), that described how clinical data collected in one region could be 

used in the registration or approval of a drug or biological product in another region, taking 

into account the influence of ethnic factors. The E5 guidance defines ethnic factors that 

affect response in terms of both intrinsic and extrinsic issues. Because differences in ethnic 

factors have the potential to affect responses in some subpopulations, the E5 guidance 

provides a general framework for evaluating medicines with regard to their sensitivity to 

ethnic factors. In particular, the Question and Answer Addendum to E5 introduces the 

multi-regional clinical trial study design as one means to evaluate treatment response 

heterogeneity and extrapolation. Multi-regional clinical trials may present special situations 

for the collection and self-reporting of race and ethnicity. 

C. National Institutes of Health Initiatives, Revitalization Act 

25 In 1993, the National Institutes of Health (NIH) Revitalization Act directed NIH to 

establish guidelines for including women and minorities in NIH-sponsored clinical 

26 27 research . In 2001, NIH was directed to ensure that women and minorities were 

included as participants, unless their exclusion was justified due to circumstances specified 

by NIH guidelines. Furthermore, clinical trials were to be designed and carried out in a 

23 On September 21, 2016, HHS issued the final rule on Clinical Trials Registration and Results Information 

Submission (42 CFR Part 11). When fully implemented, the final rule will require the submission of race and 

ethnicity information with summary results information, if it is collected during the trial. Available at 

https://www.gpo.gov/fdsys/pkg/FR-2016-09-21/pdf/2016-22129.pdf 24 E5 Guidance on Ethnic Factors in the Acceptability of Foreign Clinical Data, available at 

http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E5_R1/Step4/E5_R1__Guideline. 

pdf 25 NIH Revitalization Act of 1993, (PL 103-43) (June 10, 1993), available at https://grants.nih.gov/grants/olaw/pl103-

43.pdf 26 NIH Revitalization Act of 1993, (PL 103-43), Subtitle B (June 10, 1993), available at 

http://orwh.od.nih.gov/about/pdf/NIH-Revitalization-Act-1993.pdf 27 NIH Policy and Guidelines on The Inclusion of Women and Minorities as Subjects in Clinical Research (October 

2001), available at https://grants.nih.gov/grants/funding/women_min/guidelines_amended_10_2001.htm 

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manner that would elicit information across genders and diverse racial and ethnic 

subgroups to examine differential effects on such groups. 

NIH guidelines stipulate that when proposing a phase 3 clinical trial, evidence must be 

reviewed to establish whether or not there are potentially clinically important sex- and 

racial/ethnic-based differences in the anticipated effects of the intervention. If previous 

studies support the existence of significant differences, the primary questions and design of 

the trial must specifically accommodate this. For example, if men and women are thought 

to respond differently to an intervention, then the phase 3 clinical trial must be designed to 

answer two separate primary questions, one for men, and the other for women. When prior 

studies support no significant differences for either sex/gender or racial/ethnic subgroups 

with a given intervention, then sex/gender and racial/ethnic status will not be required as 

subject selection criteria, although the inclusion and analysis by gender and racial/ethnic 

groups is strongly encouraged. When prior studies neither support nor negate significant 

differences, then the design of the phase 3 clinical trial will be required to include sufficient 

and appropriate entry of sex/gender and racial/ethnic participants so that valid analysis of 

the intervention effects can be performed. However, the trial will not be required to provide 

high statistical power for these comparisons. 

D. FDA Regulations, Guidances, and Section 907 of the Food and 

Drug Administration Safety and Innovation Act of 2012 

(FDASIA) 

In 1997, OMB issued its revised recommendations for the collection and use of race and 

ethnicity data by Federal agencies (Policy Directive 15)2 

. OMB stated that its race and 

ethnicity categories were not anthropologically or scientifically-based designations, but 

instead were categories that described the sociocultural construct of our society. 

As outlined in FDA’s 2005 Guidance on Collection of Race and Ethnicity in Clinical 

Trials11, FDA recommended the use of the standardized OMB race and ethnicity categories 

for data collection in clinical trials for two reasons. First, the use of the recommended OMB 

categories will help ensure consistency in demographic subset analyses in applications 

submitted to FDA13 and in data collected by other government agencies. Second, 

consistency in these categories may make the demographic subset analysis more useful in 

evaluating potential differences in the safety and effectiveness of medical products among 

population subgroups. To assess potential subgroup differences in a meaningful way, it is 

important to use uniform, standard methods of defining racial and ethnic subgroups. 

FDA regulations require sponsors of Investigational New Drugs (INDs)28 to report the total 

number of subjects initially planned for inclusion in the study; the number entered into the 

study to date, tabulated by age group, gender, and race; the number whose participation in 

the study was completed as planned; and the number who dropped out of the study for any 

28 21 CFR § 312.33(a)(2), available at 

https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=312.33 

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reason. FDA regulations for NDAs require sponsors to present a summary of safety and 

effectiveness data by demographic subgroups (age, sex, race, and other subgroups as 

appropriate)29, as well as an analysis of whether modifications of dose or dosage intervals 

are needed for specific subgroups30 . FDA recommends the identification of a subject’s race 

and/or ethnicity in such summaries. 

Section 907 of FDASIA directed the Agency to develop a report31 “addressing the extent to 

which clinical trial participation and the inclusion of safety and effectiveness data by 

demographic subgroups, including sex, age, race, and ethnicity, is included in applications 

submitted to the Food and Drug Administration.” Section 907 also directed FDA to develop 

an action plan outlining “recommendations for improving the completeness and quality of 

analyses of data on demographic subgroups in summaries of product safety and 

effectiveness data and in labeling; on the inclusion of such data, or the lack of availability of 

such data, in labeling; and on improving the public availability of such data to patients, 

health care providers, and researchers.” In support of that Action Plan, FDA has developed 

this guidance as one of the actions to improve the completeness and quality of demographic 

subgroup data 

Relevance of Population Subgroup Studies 

Differences in response to medical products have already been observed in racially 

and ethnically distinct subgroups of the U.S. population32. These differences may be 

attributable to intrinsic factors (e.g., genetics, metabolism, elimination), extrinsic 

factors (e.g., diet, environmental exposure, sociocultural issues), or interactions 

between these factors (Huang 2008)33 . 

An FDA review of drug approvals between 2008 and 2013 found that approximately 

one-fifth of new drugs demonstrated some differences in exposure and/or response 

across racial/ethnic groups (Ramamoorthy 2015)34 . For example, racial differences 

29 21 CFR § 314.50(d)(5)(v) and (vi)(a) (October 2015). See also Integrated Summary of Effectiveness Guidance for 

Industry, available at http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/ 

guidances/ucm079803.pdf 30 Under 21 CFR 314.101(d)(3), the Agency may refuse to file an NDA if it is incomplete because it does not contain 

information required by 21 CFR 314.50. Thus, if there is an inadequate evaluation for safety and/or effectiveness of 

the population intended to use the drug, including pertinent subsets, such as gender, age, and racial subsets the 

Agency may refuse to file the application. See FDA's Manual of Policies and Procedures, Office of New Drugs, Good 

Review Practice: Refuse to File (October 10, 2013), available at http://www.fda.gov/downloads/AboutFDA/ 

CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProcedures/UCM370948.pdf 31 FDA Report Collection, Analysis, and Availability of Demographic Subgroup Data for FDA-Approved Medical 

Products (August 2013), available at http://www.fda.gov/downloads/RegulatoryInformation/Legislation/ 

SignificantAmendmentstotheFDCAct/FDASIA/UCM365544.pdf 32 In fact, in June of 2005, FDA approved BiDil, the first drug approved by the Agency to treat a disease in patients 

identified by race. The drug was approved for the treatment of heart failure in Black patients. The sponsor 

conducted two trials in the general population that failed to show a benefit, but suggested a benefit of BiDil in Black 

patients. The company then studied the drug in 1,050 self-identified Black patients and it was shown to be safe and 

effective. 33 Huang SM & Temple R, 2008, “Is this the drug or dose for you? Impact and consideration of ethnic factors in global 

drug development, regulatory review, and clinical practice,” Clin Pharmacol Ther, 84: 287 -294 34 Ramamoorthy A, Pacanowski MA, Bull, J, and Zhang L, 2015, "Racial/ethnic differences in drug disposition and 

response: review of recently approved drugs," Clin Pharmacol Ther, 97: 263-273. 

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in skin structure and physiology can affect response to dermatologic and topically 

35 applied products (Taylor 2002) . Mortality rates of patients on dialysis have been 

36 shown to differ across race and ethnicity groups . Several studies have also shown 

that Blacks respond less well to several classes of antihypertensive agents (beta 

37 blockers and angiotensin converting enzyme (ACE) inhibitors) (Exner 2001 and 

38 Yancy 2001 ). For cytochrome P450 2D6 (CYP2D6), an important enzyme that 

metabolizes drugs belonging to a variety of therapeutic areas such as antidepressants, 

antipsychotics, and beta blockers, the frequency of poor metabolizers is higher in 

Whites39 (7-10%) and Blacks or African Americans (3-8%) than in persons of Asian 

40 41 heritage (Xie 2001) . The incidence of anticonvulsant carbamazepine -induced 

Stevens-Johnson syndrome-toxic epidermal necrolysis has shown a strong 

42 association with HLA-B*1502 (Chung 2004 ), an allele that is highly prevalent in 

Asian populations (particularly Chinese) when compared to non-Asian populations 

43 44 (Hung 2006 and Alfirevic 2006 ). 

Collecting data on race and/or ethnicity is critical to identifying population-specific 

signals. As illustrated above, genetic studies may explain the basis for observed 

differences in pharmacokinetics, efficacy, or safety across racial or ethnic subgroups, 

and FDA has recommended collection of DNA samples in clinical trials for such 

purposes 45. FDA has also published guidance on enrichment strategies for selecting 

participants for clinical trials based on prognostic or predicative biomarkers (such as 

genotype)46 . 

35 Taylor S, 2002, “Skin of Color: Biology, Structure, Function, and Implications for Dermatologic Disease,” Journal 

of American Academy of Dermatology, 46: S41-62 36 Yan, Guofen, et al., 2013, "The relationship of age, race, and ethnicity with survival in dialysis patients," Clinical 

Journal of the American Society of Nephrology, 8.6: 953-961. 37 Exner D, Dries D, Donamski M, and Cohn J, 2001, “Lesser Response to Angiotensin Converting-Enzyme Inhibitor 

Therapy in Black as Compared with White Patients With Left Ventricular Dysfunction,” N Engl J Med, 344: 1351-

1357 38 Yancy C, Fowler M, Colucci W, Gilbert E, Bristow M, et al., 2001, “Race and the Response to Adrenergic Blockade 

with Carvedilol in Patients with Chronic Heart Failure,” N Engl J Med, 344: 1358-1365. 

39 The terms used in this guidance to describe the various racial and ethnic groups are those used by OMB 40 Xie H, Kim R, Wood A, and Stein C, 2001, “Molecular Basis of Ethnic Differences in Drug Disposition and 

Response,” Annu Rev Pharmacol Toxicol, 41: 815-850. 41 Carbamazepine postmarket drug safety and information (December 12, 2007), available at 

http://www.fda.gov/Drugs/DrugSafety/PostmarketDrugSafetyInformationforPatientsandProviders/ucm124718.htm 42 Chung, WH, Hung, SI, Hong, HS, Hsih, MS, Yang, LC, et al, 2004, “A Marker for Stevens-Johnson Syndrome,” 

Nature, 428: 486. 43 Hung SI, Chung WH, Jee SH, Chen WC, Chang YT, et al, 2006, “Genetic Susceptibility to Carbamazepine -Induced 

Cutaneous Adverse Drug Reactions,” Pharmacogenetics and Genomics, 16: 297-306 44 Alfirevic A, Jorgensen AL, Williamson PR, Chadwick DW, Park BK, Pirmohamed M. 2006, “HLA -B locus in 

Caucasian patients with carbamazepine hypersensitivity,” Pharmacogenomics, 7:813-818 45 Guidance for Industry: Clinical Pharmacogenomics: Premarket Evaluation in Early-Phase Clinical Studies and 

Recommendations for Labeling (January 2013), available at 

http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm337169.pdf 46 Draft Guidance: Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological 

Products (December 2012), available at 

http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm332181.pdf 

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Contains Nonbinding Recommendations 

IV. COLLECTING RACE AND ETHNICITY DATA IN 

CLINICAL TRIALS 

The classifications discussed below provide a minimum standard for maintaining, collecting, and 

presenting data on race and ethnicity for Federal reporting purposes. Consistent with OMB 

Policy Directive 15, the categories in this classification are social-political constructs and should 

not be interpreted as being scientific or anthropological in nature. They are not to be used as 

determinants of eligibility for participation in any Federal program. The standards have been 

developed to provide a common framework for uniformity and consistency in the collection and 

use of data on race and ethnicity by Federal agencies. 

The recommendations in this section reflect the Agency's commitment to and expectations
 

for more consistent demographic subgroup data collection. For studies conducted both 

inside and outside the United States, the Agency recommends the following process, which
 

is based on the current OMB Directive for collecting racial and ethnic data. These
 

recommendations are also consistent with the format for collecting race and ethnicity data
 

set forth in NIH guidance47 .
 

A. Two-Question Format 

In order to be consistent with OMB and other recommended best practices, FDA 

recommends using the two-question format for requesting race and ethnicity information, 

with the ethnicity question preceding the question about race. Example: 

Question 1 (answer first): Do you consider yourself Hispanic/Latino or not 

Hispanic/Latino? 

Question 2 (answer second): Which of the following five racial designations best 

describes you? More than one choice is acceptable. 

B. Self-Reporting 

FDA recommends that trial participants self-report race and ethnicity information and 

those individuals be permitted to designate a multiracial identity. When the collection of 

self-reported designations is not feasible (e.g., because of the subject’s inability to 

respond), we recommend that the information be requested from a first-degree relative or 

other knowledgeable source. Race and ethnicity should not be assigned by the study team 

conducting the trial. 

C. Ethnicity 

For ethnicity, we recommend the following minimum choices be offered: 

47 NIH Policy and Guidelines on The Inclusion of Women and Minorities as Subjects in Clinical Research (Amended, 

October 2001), available at https://grants.nih.gov/grants/funding/women_min/guidelines_amended_10_2001.htm 

9 
 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Contains Nonbinding Recommendations 

Hispanic or Latino: A person of Cuban, Mexican, Puerto Rican, South or Central 

American, or other Spanish culture or origin, regardless of race. The term, “Spanish origin,” 

can be used in addition to “Hispanic or Latino.” 

Not Hispanic or Latino 

D. Race 

For race, we recommend the following minimum choices be offered: 

American Indian or Alaska Native: A person having origins in any of the original 

peoples of North and South America (including Central America), and who maintains 

tribal affiliation or community attachment. 

Asian: A person having origins in any of the original peoples of the Far East, Southeast 

Asia, or the Indian subcontinent, including, for example, Cambodia, China, India, Japan, 

Korea, Malaysia, Pakistan, the Philippine Islands, Thailand, and Vietnam. 

Black or African American: A person having origins in any of the black racial 

groups of Africa. Terms such as “Haitian” or “Negro” can be used in addition to 

“Black or African American.” 

Native Hawaiian or Other Pacific Islander: A person having origins in any of the 

original peoples of Hawaii, Guam, Samoa, or other Pacific Islands. 

White: A person having origins in any of the original peoples of Europe, the Middle East, 

or North Africa. 

We recommend offering an option of selecting one or more racial designations or 

additional subgroup designations. Recommended forms for the instruction accompanying 

the multiple response questions are “Mark one or more” and “Select one or more.” 

Sponsors should report the number of respondents in each racial category who selfreported as Hispanic or Latino. When aggregate data are presented, data producers should 

provide the number of respondents who marked (or selected) only one category, separately 

for each of the five racial categories. In addition to these numbers, data producers are 

encouraged to provide the detailed distributions, including all possible combinations of 

multiple responses to the race question. If data on multiple responses are collapsed, at a 

minimum the total number of respondents reporting “more than one race” shall be made 

available (see attached NIH PHS Inclusion Enrollment Report form48) 

48 NIH PHS Inclusion Enrollment Report Form (March 25, 2015), available at 

http://grants.nih.gov/grants/forms/inclusion-enrollment-report.pdf 

10 
 

 

 

 

 

 

 

 

 

 

 

E. Use of More Detailed Racial and Ethnic Categories 

In certain situations, as recommended in OMB Policy Directive 15, more detailed race and 

ethnicity information may be desired. For example, for clinical trials conducted outside 

the United States, FDA recognizes that the recommended categories for race and ethnicity 

were developed in the United States and that these categories may not adequately describe 

racial and ethnic groups in foreign countries. Furthermore, White can reflect origins in 

Europe, the Middle East, or North Africa; Asian can reflect origins from areas ranging 

from India to Japan. 

In situations where appropriate, FDA recommends using more detailed categories by 

geographic region to provide sponsors the flexibility to adequately characterize race and 

ethnicity. As outlined in the 2011 HHS Implementation Guidance on Data Collection 

Standards for Race, Ethnicity, Sex, Primary Language, and Disability Status4

, if additional 

granularity or more detailed characterizations of race or ethnicity are collected to enhance 

understanding of the trial participants, FDA recommends these characterizations be 

traceable to the five minimum designations for race and two designations for ethnicity 

listed in sections D and C above. Example (from the above referenced 2011 HHS 

Guidance4

): 

Ethnicity Data Standard 

Are you Hispanic, Latino/a, or of Spanish origin? (One or more categories may be selected) 

a. ____No, not of Hispanic, Latino/a, or Spanish origin 

b. ____Yes, Mexican, Mexican American, Chicano/a 

c. ____Yes, Puerto Rican 

d. ____Yes, Cuban 

e. ____Yes, Another Hispanic, Latino/a or Spanish origin 

These categories roll up to the 

Hispanic or Latino category of 

the OMB standard 

Race Data Standard 

What is your race? (One or more categories may be selected) 

a. ____White 

b. ____Black or African American 

c. ____American Indian or Alaska Native 

d. ____Asian Indian 

e. ____Chinese 

f. ____ Filipino 

g. ____Japanese 

h. ____Korean 

i. ____ Vietnamese 

j. ____ Other Asian 

k. ____Native Hawaiian 

l. ____ Guamanian or Chamorro

m. ___ Samoan 

n. ____Other Pacific Islander 

These categories are part of the 

current OMB standard 

These categories roll up to the Asian 

category of the OMB standard 

These categories roll-up to the Native Hawaiian or 

Other Pacific Islander category of the OMB standard 

Contains Nonbinding Recommendations 

11 
 

 

 

 

 

 

 

 

 

 

 

 

Contains Nonbinding Recommendations 

Where concerns exist in the representation of race or ethnicity categories, sponsors 

are encouraged to discuss the race or ethnicity issues with the appropriate review 

division. 

F. Use of the term “nonwhite” 

The term “nonwhite” is not acceptable for use in the presentation of Federal Government 

data. It should not be used in publication or text of any report. 

V. PRESENTATION OF CLINICAL TRIAL RACE AND 

ETHNICITY DATA 

For INDs, NDAs, and BLAs, we recommend the submission of tabulated demographic data 

10 13 based on the Demographic Rule for all clinical trials using the characterizations of race 

and ethnicity described in this guidance. Beginning in May 2017, CDER and CBER will 

require marketing applications to be submitted electronically49 . CDER and CBER use the 

eCTD as the standard for their electronic applications. When submitting an electronic 

application, presentation of demographic data is described in ICH M4E eCTD Guidance 

(section 2.7.4.1.3 and table 2.7.4.2)50, which suggests a tabular display of demographic 

characteristics (e.g., age, sex, race) by treatment group (e.g., active drug, placebo)51. With 

regard to the description of race and ethnicity, the categories that are suggested previously in 

this document (Section IV) provide more detail than those suggested in the ICH M4E eCTD 

guidance. FDA recommends that sponsors provide the level of detail described in Section IV 

of this guidance. For relevant device submissions, we recommend following the guidance 

presented here and, when finalized, the recommendations in the draft guidance documents 

Evaluation and Reporting of Age, Race, and Ethnicity Data in Medical Device Clinical 

Studies14 . 

49 Providing Regulatory Submissions in Electronic Format – Certain Human Pharmaceutical Product Applications and 

Related Submissions Using the eCTD Specifications: Guidance for Industry (May 2015), available at 

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM333969.pdf 50 Revision of M4E Guideline on Enhancing the Format and Structure of Benefit-Risk Information on ICH Efficacy – 

M4E(R2) (June 15, 2016), available at 

http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/CTD/M4E_R2_Efficacy/M4E_R2__Step_4.pdf 51

12 

The ICH M4 eCTD document suggests specific kinds of demographic information to be collected as a part of a 

clinical trial, but does not provide rigid specifications on how the data should be presented, noting, for example, that 

“if relative exposure of demographic groups in the controlled trials differ from the overall exposure, it may be useful 

to provide separate tables.” Choices of how best to summarize demographic data depend on the nature of the data to 

be conveyed. For some trials, it may be useful to show the distribution of one demographic characteristic within a 

second demographic (e.g., the age distribution of men and women enrolled in a set of controlled trials). 
 

 

 

 

 

 

 

 

 

 

 

 

 

Contains Nonbinding Recommendations 

VI. REFERENCES
	

Alfirevic A, Jorgensen AL, Williamson PR, Chadwick DW, Park BK, Pirmohamed M. 2006, 

“HLA-B locus in Caucasian patients with carbamazepine hypersensitivity,”
	

Pharmacogenomics, 7:813-818
 

Chung, WH, Hung, SI, Hong, HS, Hsih, MS, Yang, LC, et al, 2004, “A Marker for 

Stevens-Johnson Syndrome,” Nature, 428: 486.
 

Exner D, Dries D, Donamski M, and Cohn J, 2001, “Lesser Response to AngiotensinConverting-Enzyme Inhibitor Therapy in Black as Compared with White Patients 

With Left Ventricular Dysfunction,” N Engl J Med, 344: 1351-1357.
 

Huang SM & Temple R, 2008, “Is this the drug or dose for you? Impact and consideration of 

ethnic factors in global drug development, regulatory review, and clinical practice,” Clin 

Pharmacol Ther, 84: 287-294 

Hung SI, Chung WH, Jee SH, Chen WC, Chang YT, et al, 2006, “Genetic Susceptibility 

to Carbamazepine-Induced Cutaneous Adverse Drug Reactions,” Pharmacogenetics and 

Genomics, 16: 297-306. 

Ramamoorthy A, Pacanowski MA, Bull, J, and Zhang L, 2015, "Racial/ethnic differences 

in drug disposition and response: review of recently approved drugs," Clin Pharmacol 

Ther, 97: 263-273.
 

Taylor S, 2002, “Skin of Color: Biology, Structure, Function, and Implications for
 

Dermatologic Disease,” Journal of American Academy of Dermatology, 46: S41-62.
 

Xie H, Kim R, Wood A, and Stein C, 2001, “Molecular Basis of Ethnic Differences in Drug
	

Disposition and Response,” Annu Rev Pharmacol Toxicol, 41: 815-850.
 

Yan, Guofen, et al., 2013, "The relationship of age, race, and ethnicity with survival in dialysis 

patients," Clinical Journal of the American Society of Nephrology, 8.6: 953-961. 

Yancy C, Fowler M, Colucci W, Gilbert E, Bristow M, et al., 2001, “Race and the Response to 

Adrenergic Blockade with Carvedilol in Patients with Chronic Heart Failure,” N Engl J 

Med, 344: 1358-1365. 

VII. BIBLIOGRAPHY 

HHS Policy and Reports 

13 
 

 

 

Contains Nonbinding Recommendations 

	 Policy Statement on Inclusion of Race and Ethnicity in DHHS Data Collection Activities 

(October 24, 1997) 

	 Improving the Collection and Use of Racial and Ethnic Data in HHS (December 1, 1999) 

	 US Department of HHS implementation guidance on data collections standards for race, 

ethnicity, sex, primary language, and disability status (October 31, 2011) 

NIH Policies, Reports, and Resources 

	 NIH Revitalization Act of 1993, Subtitle B (PL 103-43) (June 10, 1993) 

	 NIH policy on reporting race and ethnicity data: Subjects in clinical research
 

(August 8, 2001)
 

	 NIH Guidelines on the Inclusion of Women and Minorities as Subjects in Clinical 

Research (October 2001) 

	 Use of new inclusion management system required as of October 17, 2014 (October 2, 

2014) 

	 NIH PHS Inclusion Enrollment Report Form (March 25, 2015) 

	 NIH Policy Implementation Page: Inclusion of Women and Minorities as Participants 

in Research Involving Human Subjects 

FDA Regulations, Reports, and Legislation 

	 Food and Drug Administration Modernization Act of 1997 (Public Law 105-115, 

November 21, 1997)
 

	 Investigational New Drug Applications and New Drug Applications (21 CFR 312 and 

314, February 11, 1998) 

	 Food and Drug Administration Amendments Act (FDAAA) of 2007, Public Law 

No. 110-85 Section 901 of the Federal Food, Drug, and Cosmetic Act
 

(September 2009)
 

	 FDA Safety and Innovation Act (Public Law No. 112-114 (February 9, 2012) 

	 FDA Report Collection, Analysis, and Availability of Demographic Subgroup 

Data for FDA-Approved Medical Products (August 2013)
 

	 FDA Action Plan To Enhance The Collection and Availability of Demographic 

Subgroup Data (August 2014)
 

	 Food and Drug Administration (FDA), 1998, “Investigational New Drug Applications 

and New Drug Applications (21 CFR 314.50(b), April 1, 2015) 

FDA Guidances for Industry 

	 Guideline for the Study and Evaluation of Gender Differences in the Clinical Evaluation 

of Drugs (July 1993) 

	 General Considerations for the Clinical Evaluation of Drugs (February 1997) 

	 Guidance on IDE Policies and Procedures (January 1998) 

14 
Contains Nonbinding Recommendations 

 Format and Content of the Clinical and Statistical Sections of an Application (July 1998) 

 M4E: The CTD- Efficacy (August 2001) 

 Content and Format for Geriatric Labeling (October 2001) 

 Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data 

Analysis, and Impact on Dosing and Labeling (May 2003) 

 Collection of Race and Ethnicity Data in Clinical Trials (September 2005) 

 Clinical Studies Section of Labeling for Human Prescription Drugs and Biological 

Products - Content and Format (January 2006) 

 Adverse Reactions Section of Labeling for Human Prescription Drugs and Biological 

Products - Content and Format (January 2006) 

 Clinical Pharmacogenomics: Premarket Evaluation in Early-Phase Clinical Studies and 

Recommendations for Labeling (January 2013) 

 Design Considerations for Pivotal Clinical Investigations for Medical Devices 

(November 2013) 

 Evaluation of Sex-Specific Data in Medical Device Clinical Studies (August 2014) 

 General Clinical Pharmacology Considerations for Pediatric Studies for Drugs and 

Biological Product (December 2014) 

 Providing Regulatory Submissions In Electronic Format - Standardized Study Data 

(December 2014) 

 Providing Regulatory Submissions in Electronic Format —Certain Human 

Pharmaceutical Product Applications and Related Submissions Using the eCTD 

Specifications (May 2015) 

 Integrated Summary of Effectiveness Guidance for Industry (October 2015) 

 Human Immunodeficiency Virus-1 Infection: Developing Antiretroviral Drugs for 

Treatment (November 2015) 

 Study Data Technical Conformance Guide (March 2016) 

 Draft Guidance: Diabetes Mellitus: Developing Drugs and Therapeutic Biologics for 

Treatment and Prevention (February 2008) 

 Draft Guidance: Pharmacokinetics in Patients with Impaired Renal Function: Study 

Design, Data Analysis and Impact on Dosing and Labeling (March 2010) 

 Draft Guidance: Drug Interaction Studies — Study Design, Data Analysis, Implications, 

for Dosing and Labeling Recommendations (February 2012) 

 Draft Guidance: Enrichment Strategies for Clinical Trials to Support Approval of Human 

Drugs and Biological Products (December 2012) 

 Draft Guidance: Chronic Hepatitis C Virus Infection: Developing Direct-Acting Antiviral 

Drugs for Treatment (May 2016) 

 Draft Guidance: Evaluation and Reporting of Age, Race, and Ethnicity Data in Medical 

Device Clinical Studies (June 2016) 

ICH Guidances 

 ICH, E7 Studies in Support of Special Populations: Geriatrics (June 24, 1993) 

 ICH, E4 Dose Response Information to Support Drug Registration ( March 1994) 

 ICH, E5 Ethnic Factors in the Acceptability of Foreign Data (February 5, 1998) 

15 
Contains Nonbinding Recommendations 

	 ICH, E11 Clinical Investigation of Medicinal Products in the Pediatric Population (July 

20, 2000) 

	 ICH, M4 Common Technical Document for the Registration of Pharmaceuticals for 

Human Use (January 13, 2004) 

Other Sources 

	 General Accounting Office, FDA Needs to Ensure More Study of Gender 

Differences in Prescription Drug Testing, GAO/HFD-93-17 (October 

1992)
 

	 Office of Management and Budget: Standards for the Classification of Federal Data on 

Race and Ethnicity (June 9, 1994) 

	 Office of Management and Budget (OMB) Directive No. 15 Revisions to the Standards 

for the Classification of Federal Data on Race and Ethnicity (October 30, 1997) 

	 Best Pharmaceuticals Act for Children of 2002 (Public Law 107-109)
 

(February 4, 2002)
 

NIH PHS Cumulative Inclusion Enrollment Report Form 

16