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 Womens 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 Womens 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
  
1
 
Drug Administration Staff  
 
    
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 
approach is based on  the Office of Management and Budget (OMB)  Directive 152  and 
developed in accordance  with section 4302 of the Affordable Care  Act3,  the HHS 
Implementation Guidance on Data Collection Standards for Race, Ethnicity, Sex, Primary  
Language, and Disability Status4, and the Food and  Drug  Administration  Safety  and 
Innovation Act5  (FDASIA) Section 907  Action  Plan6 .   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  Womens  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 FDAs 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  
2  
  Contains  Nonbinding  Recommendations  
 
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  
clinically relevant populations with regard to  age, gender, race,  and ethnicity13.  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 persons  self-
representation  as male or  female,  or  how  that person  is  responded  to  by  social institutions  based  on  the individuals  
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.  
3  
  Contains  Nonbinding  Recommendations  
 
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 Agencys 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  
hearing18  for feedback19 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 FDAregulated medical 
products.   FDA  also opened a public docket for further input18. On April 9, 201520  and December  2,  
201521, 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-the-
collection-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-trials-
assessing-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  
4  
  Contains  Nonbinding  Recommendations  
 
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 
services issues. This policy, updated in 20114, clearly  states that the minimum standard 
categories in OMB Policy  Directive 152  should be used when collecting  and reporting data 
in HHS data systems or reporting  HHS funded statistics23.     
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  
In 1993, the National Institutes of Health (NIH) Revitalization Act25  directed NIH to 
establish guidelines for including women and minorities in NIH-sponsored clinical 
research26.   In 2001, NIH was directed27 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  
5  
  Contains  Nonbinding  Recommendations  
 
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 FDAs 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  
6  
  Contains  Nonbinding  Recommendations  
 
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 subjects 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.  
7  
  Contains  Nonbinding  Recommendations  
 
in skin structure and physiology can affect response to dermatologic and topically  
applied products (Taylor 2002)35.  Mortality rates of patients on dialysis have been 
shown to differ across race and ethnicity  groups36.  Several studies have  also  shown 
that Blacks respond less well  to several classes of antihypertensive agents (beta 
blockers and angiotensin converting enzyme  (ACE) inhibitors) (Exner 200137  and 
Yancy 200138). 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 
heritage  (Xie 2001)40.  The incidence of anticonvulsant carbamazepine41-induced 
Stevens-Johnson syndrome-toxic epidermal necrolysis has shown a strong 
association with HLA-B*1502  (Chung 200442), an allele that is highly prevalent in 
Asian populations (particularly Chinese) when compared to non-Asian populations  
(Hung 200643and  Alfirevic 200644).  
 
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  
8  
  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 subjects 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 self-
reported 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  
  Contains  Nonbinding  Recommendations  
 
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   
These categories roll up to the 
c.  ____Yes,  Puerto  Rican   
Hispanic or  Latino category of 
d.  ____Yes, Cuban  
the OMB standard  
e.  ____Yes,  Another  Hispanic, Latino/a  or  Spanish origin  
 
Race  Data  Standard   
What  is  your  race?  (One or  more categories  may  be  selected)  
 
a.  ____White   
These categories are part of the 
b. ____Black or  African American   
current OMB standard  
c.  ____American  Indian or  Alaska  Native     
d.  ____Asian  Indian   
e.  ____Chinese   
f.  ____  Filipino   
These categories roll up to the Asian 
g. ____Japanese    
category of the OMB standard  
h.  ____Korean  
i.  ____  Vietnamese   
j.  ____  Other  Asian    
k.  ____Native  Hawaiian  
l.  ____  Guamanian  or Chamorro    These categories roll-up to the Native Hawaiian or  
m.  ___  Samoan    Other Pacific Islander category of the OMB standard  
n. ____Other  Pacific Islander              
 
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 
based on the Demographic Rule10  13  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  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).  
 
12  
  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 Angiotensin- 
Converting-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