2015 - 2016
GLOBAL PARTICIPATION IN CLINICAL TRIALS REPORT
www.fda.govJULY 2017
Global Participation in Clinical Trials Report2015-2016 JULY 2017
1
Table of Contents
Introduction .................................................................................................................2
Welcome to the FDA’s Center for Drug Evaluation
and Research’s (CDER’s) Drug Trial Snapshots: ................................................................................................... 2
Global Clinical Trials Report ....................................................................................................................................... 2
Geography ...................................................................................................................4
Where are Participants From? ................................................................................................................................... 4
Trial Participants by Country ..................................................................................................................................... 6
How does Global Participation Change by Year? ............................................................................................... 8
How many CDER NME Approvals had Foreign Data? ....................................................................................... 9
Trial Participants by Year Approved, Therapeutic Area, and Geography ................................................... 10
Gender ..........................................................................................................................11
How does Gender Participation differ by Geographic Location? ................................................................ 11
Percent Women among Trial Participants by Country ..................................................................................... 12
Of the Women Trial Participants in Cardiovascular (CVD) Trials,
what was the Racial composition? ......................................................................................................................... 14
Race ..............................................................................................................................15
How does Racial Participation differ by Geographic Location? ................................................................... 15
Black/AA Clinical Trial Participation in the United States ................................................................................ 16
Participation of Black/AAs in Clinical Trials for Oncology, Cardiology, and Psychiatry ........................ 24
Global Participation in Clinical Trials Report
2
Introduction
Welcome to the FDA’s Center for Drug Evaluation and Research’s (CDER’s) Drug Trial Snapshots: Global Clinical Trials Report
Within the past two decades, clinical research has become more global and complex, with clinical trials increasingly being conducted in countries outside the United States. Every year, CDER approves a number of novel drugs based upon review of safety and efficiency measures in sponsor submitted clinical trial data from participants all over the world. Although the Center oversees approval of drugs to be marketed within the United States, these innovative new products come from drug development programs spanning multiple therapeutic areas, hundreds of clinical trial sites, and tens of thousands of people globally.
In recent years, there has been growing interest in stratifying different characteristics of clinical trial participants in an attempt to better understand variability in drug response. As part of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA 907), the U.S. Congress required the U.S. Food and Drug Administration (FDA) to report on the diversity of participants in clinical trials and the extent to which safety and effectiveness data is based on demographic factors such as sex, age, and race. The Center has responded with the Drug Trials Snapshots program to increase transparency of the demographic data of participants in pivotal clinical trials.
2015-2016
3
Janet Woodcock, M.D. Director, Center for Drug Evaluation and Research (CDER)
John Whyte, M.D., M.P.H.Director, Professional Affairs and Stakeholder Engagement (PASE)
Marsha B. HendersonAssistant Commissioner of Women’s Health,FDA’s Office of Women’s Health (OWH)
Complementing our recent two-year Drug Trials Snapshots Summary Report, this special report provides additional information on clinical trial participants by site location over the same two-year time frame (2015-16). Partnering with the FDA’s Office of Women’s Health (OWH), we combined site location data with demographic data of participants at those sites and summarize our results in three sections: Participants by Geography, Gender by Location, and Race by Location.
Drug development has become a global enterprise that relies on data from patients all over the world. We hope this information can provide more insight into the modern drug development paradigm and how new medicines are approved for human use is derived from humans around the globe.
Global Participation in Clinical Trials Report
4
Geography
Where are Participants From?The country contributing the most clinical trial participants was the United States. Compared to the population of the entire world (7.4 Billion), the US (0.35 Billion) makes up a little more than 4% of the world population.
Trial Participants by US vs Non-US
69%
Total Participants = 131,749
31%
.35 Billion PeopleUnited States
2015-2016
5
Geography
Where are Participants From?The country contributing the most clinical trial participants was the United States. Compared to the population of the entire world (7.4 Billion), the US (0.35 Billion) makes up a little more than 4% of the world population.
Population of US vs Non-US
United States
Rest of World
Total People = 7,403,227,777
96%
4%
7.4 Billion PeopleRest of World
Global Participation in Clinical Trials Report
6
Trial Participants by CountryThe top ten countries enrolling clinical trial participants are the United States (40,835), Russia (6,875), Germany (5,802), Poland (5,170), Czechia (4,858), Canada (4,748), India (3,841), Ukraine (3,551), France (3,040), and Hungary (2,887).
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10K
20K
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10%
20%
30%
Perc
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5,80
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5,17
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4,85
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4,74
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3,84
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3,55
1
3,04
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2,88
7
2,87
8
2,87
2
2,70
3
2,37
5
2,36
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2,31
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2,25
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2,25
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2,07
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2,03
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1,97
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1,68
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1,57
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1,56
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1,40
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1,37
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1,36
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928
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964
557
531
529
524
489
463
386
376
334
329
266
264
233
205
177
157
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129
105
103
101
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81 65 62 50 30 27 26 26 1784 2 17
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4.40
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3.92
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3.69
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3.60
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2.92
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2.70
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2.31
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2.19
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2.18
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2.18
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1.80
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1.79
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1.76
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1.71
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1.71
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1.57
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1.54
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1.28
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1.19
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1.06
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1.04
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0.78
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0.73
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0.71
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0.70
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0.56
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0.49
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5.22
%
0.42
%
0.40
%
0.40
%
0.40
%
0.37
%
0.35
%
0.29
%
0.29
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0.25
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0.25
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0.20
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0.20
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0.18
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0.16
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0.13
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0.12
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0.12
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0.10
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0.08
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0.08
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0.06
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0.02
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0.02
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0.01
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0.01
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0.00
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0.00
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0.44
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2015-2016
7
Trial Participants by CountryThe top ten countries enrolling clinical trial participants are the United States (40,835), Russia (6,875), Germany (5,802), Poland (5,170), Czechia (4,858), Canada (4,748), India (3,841), Ukraine (3,551), France (3,040), and Hungary (2,887).
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1,68
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1,57
4
1,56
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81 65 62 50 30 27 26 26 1784 2 17
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9%
4.40
%
3.92
%
3.69
%
3.60
%
2.92
%
2.70
%
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%
2.19
%
2.18
%
2.18
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%
1.79
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1.76
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1.71
%
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%
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United States
Rest of World
Global Participation in Clinical Trials Report
8
How does Global Participation Change by Year?Participation in clinical trials varies by year. In calendar year 2015, there were 105,808 participants in pivotal clinical trials. In calendar year 2016, there were 25,941 participants in pivotal clinical trials.
Calendar Year (2016)Total Participation = 25,941
Calendar Year (2015)Total Participation = 105,808
Rest of World76,033 Participants
71.86%
United States29,775 Participants
28.14%
Rest of World14,881 Participants
57.36%
United States11,060 Participants
42.64%
2015-2016
9
YEAR NME Approvals (N) NMEs (%) WITH ANY PARTICIPANTS OUTSIDE US
2015 45 42 (93%)
2016 21* 15 (71%)
How many CDER NME Approvals had Foreign Data?In 2015, CDER approved 45 novel drugs, either as new molecular entities (NMEs) under New Drug Applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs).
In 2016, CDER approved 22 novel drugs, either as new molecular entities (NMEs) under New Drug Applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs).
*Site location data was not available for one new molecular entity
Global Participation in Clinical Trials Report
10
Trial Participants by Year Approved, Therapeutic Area, and GeographyMore insight into pivotal trial participants are provided below by year approved, therapeutic area, and geography.
United States Rest of World
0K 10K 20K 30K 40K 50K 0K 10K 20K 30K 40K 50K
2015
Oncology
Anesthesiology
Cardiovascular Disease
Dermatology
Endocrinology, Diabetes, and Metabolism
Gastroenterology
Hematology
Infectious Disease
Medical Genetics
Nephrology
Neurology
Obstetrics and Gynecology
Pulmonary Disease
Psychiatry
Rheumatology
Diagnostics - Medical Imaging
Ophthalmology
2016
Oncology
Anesthesiology
Cardiovascular Disease
Dermatology
Endocrinology, Diabetes, and Metabolism
Gastroenterology
Hematology
Infectious Disease
Medical Genetics
Nephrology
Neurology
Obstetrics and Gynecology
Pulmonary Disease
Psychiatry
Rheumatology
Diagnostics - Medical Imaging
Ophthalmology
1,345
2,689
9,288
1,235
5,449
3,000
1,161
1,377
3,157
749
256
22
42
5
3,057
2,860
2,249
724
665
612
588
174
99
32
11,830
47,245
2,945
1,831
5,429
2,006
1,689
1,571
103
221
391
118
273
381
2,533
4,063
1,046
3,245
3,550
329
113
2
United States Rest of World
0K 10K 20K 30K 40K 50K 0K 10K 20K 30K 40K 50K
2015
Oncology
Anesthesiology
Cardiovascular Disease
Dermatology
Endocrinology, Diabetes, and Metabolism
Gastroenterology
Hematology
Infectious Disease
Medical Genetics
Nephrology
Neurology
Obstetrics and Gynecology
Pulmonary Disease
Psychiatry
Rheumatology
Diagnostics - Medical Imaging
Ophthalmology
2016
Oncology
Anesthesiology
Cardiovascular Disease
Dermatology
Endocrinology, Diabetes, and Metabolism
Gastroenterology
Hematology
Infectious Disease
Medical Genetics
Nephrology
Neurology
Obstetrics and Gynecology
Pulmonary Disease
Psychiatry
Rheumatology
Diagnostics - Medical Imaging
Ophthalmology
1,345
2,689
9,288
1,235
5,449
3,000
1,161
1,377
3,157
749
256
22
42
5
3,057
2,860
2,249
724
665
612
588
174
99
32
11,830
47,245
2,945
1,831
5,429
2,006
1,689
1,571
103
221
391
118
273
381
2,533
4,063
1,046
3,245
3,550
329
113
2
2015-2016
11
Gender
How does Gender Participation differ by Geographic Location?Gender composition at non-US sites was majority male, whereas gender composition at US sites was closer to 50:50.
Global
Rest of the World United States
Total Participants = 131,747* *Gender missing for 2 US participants
Total Participants = 90,914 Total Participants = 40,833
57%
60%51%
43%
40%49%
Males
Females
Global Participation in Clinical Trials Report
12
United Arab Emirates100.0%Dominican Republic
53.1%
United Kingdom36.9%
United States49.1%
New Zealand30.2%
South Africa45.2%
South Korea45.8%
Philippines37.4%
Macedonia41.6%
Venezuela26.9% Singapore
27.4%
Argentina35.8%
Lithuania22.4%
Australia41.9%
Thailand45.0%
Lebanon75.8%
Portugal30.1%Morocco85.7%
Panama13.3%
Georgia26.0%
Sweden33.6%
Ukraine37.8%
Norway29.5%Canada
45.2%
Iceland32.4%
Mexico55.0%
Brazil41.8%
India34.3%
Russia42.1%
Chile47.2%
Peru48.0%
China40.7%
25.00 60.00
Percentage ofWomen Partcipation
Percent Women among Trial Participants by CountryGlobally, 43% of trial participants are women. Overall, the female:male ratio was between 30:70 and 45:55, consistent with global participation.
United Arab Emirates100.0%Dominican Republic
53.1%
United Kingdom36.9%
United States49.1%
New Zealand30.2%
South Africa45.2%
South Korea45.8%
Philippines37.4%
Macedonia41.6%
Venezuela26.9% Singapore
27.4%
Argentina35.8%
Lithuania22.4%
Australia41.9%
Thailand45.0%
Lebanon75.8%
Portugal30.1%Morocco85.7%
Panama13.3%
Georgia26.0%
Sweden33.6%
Ukraine37.8%
Norway29.5%Canada
45.2%
Iceland32.4%
Mexico55.0%
Brazil41.8%
India34.3%
Russia42.1%
Chile47.2%
Peru48.0%
China40.7%
25.00 60.00
Percentage ofWomen Partcipation
2015-2016
13
United Arab Emirates100.0%Dominican Republic
53.1%
United Kingdom36.9%
United States49.1%
New Zealand30.2%
South Africa45.2%
South Korea45.8%
Philippines37.4%
Macedonia41.6%
Venezuela26.9% Singapore
27.4%
Argentina35.8%
Lithuania22.4%
Australia41.9%
Thailand45.0%
Lebanon75.8%
Portugal30.1%Morocco85.7%
Panama13.3%
Georgia26.0%
Sweden33.6%
Ukraine37.8%
Norway29.5%Canada
45.2%
Iceland32.4%
Mexico55.0%
Brazil41.8%
India34.3%
Russia42.1%
Chile47.2%
Peru48.0%
China40.7%
25.00 60.00
Percentage ofWomen Partcipation
Global Participation in Clinical Trials Report
14
Of the Women Trial Participants in Cardiovascular (CVD) Trials, what was the Racial composition? More insight into the races of women participating in cardiovascular trials is provided in the figures below.
White
Other
Asian
Black or African American
Women Participants in CVD Trials = 18,960
78.7%
12.7%5.4%
3.2%
100%
90%
80%
70%
60%
50%
40%
30%
20%
10%
0%8,006
Afib Stroke and Embolism
3,387Heart
Failure
3,524Deep Vein
Thrombosis and Pulm. Embolism
923Pulm. Arterial Hypertension
3,120Myocardial Infarction
82%
5%
12% 2% 5% 5% 3%
75%
8%
12% 22%22%
67%
7%
63%
12%
93%
1%
4%3%
2015-2016
15
2.1%
Race
How does Racial Participation differ by Geographic Location?The majority of Asian trial participants were at non-US sites. The representation of Black or African American participants at US sites is similar to the US general population, which is 13% Black or African American (2011-2015 Census).
*Other includes American Indian/Alaskan Native (AI/AN), Native Hawaiian/Other Pacific Islander (NH/OPI), and Others
White
Other
Asian
Black or African American
Missing
Global
Rest of the World United States
Total Participants = 131,749
Total Participants = 90,914 Total Participants = 40,835
78.6%
11.8%3.8%
5.4%0.4%
77.6% 81.0%
16.1%14.5%4.5%
2.3%
1.3%0.4%
0.2%
Global Participation in Clinical Trials Report
16
Black or African American (Black/AA) Clinical Trial Participation in the United StatesThe next series of maps provides more insight into where the majority of Black or African American (Black/AA) participants live compared to where Black/AAs participated in clinical trials at the US Zip-Code level.
Where do Black or African Americans Live?
2017 Black/African American Population
0 to 6
6 to 32
32 to 240
240 to 1,410
1,410 to 84,000
2015-2016
17
Global Participation in Clinical Trials Report
18
This map shows where Black/AAs participated in clinical trials. Red icons represent zip codes in which at least one Black/AA participated in a clinical trial.
Where do Black or African American participate in Clinical Trials? (Based on NMEs from 2015-16)
Partcipants Percentage
Black/AAs 5,913 14.48%Some Other Race 34,922 85.52%
2017 Black/African American Population
0 to 6
6 to 32
32 to 240
240 to 1,410
1,410 to 84,000
Race of ParticipantBlack/AA
2015-2016
19
Global Participation in Clinical Trials Report
20
Where do non-Black or African American participate in Clinical Trials? (Based on NMEs from 2015-16)
This map shows where non-Black/AAs participated in clinical trials. Blue icons represent zip codes in which non-Black/AAs participated in trials.
Partcipants Percentage
Black/AAs 5,913 14.48%Some Other Race 34,922 85.52%
2017 Black/African American Population
0 to 6
6 to 32
32 to 240
240 to 1,410
1,410 to 84,000
Race of ParticipantSome Other Race
2015-2016
21
Global Participation in Clinical Trials Report
22
Where do Black/AAs participate in Clinical Trials compared to Non-Black/AA Races? (Based on NMEs from 2015-16)
This map shows where Black/AAs participate in clinical trials compared to Non-Black/AA races. Roughly comparing the sites with Black/AA participants and sites with non-Black/AA participants, it seems to suggest clinical trials in the US are being conducted where Black/AAs live.
Partcipants Percentage
Black/AAs 5,913 14.48%Some Other Race 34,922 85.52%
2017 Black/African American Population
0 to 6
6 to 32
32 to 240
240 to 1,410
1,410 to 84,000
Race of ParticipantBlack/AA
Some Other Race
2015-2016
23
Global Participation in Clinical Trials Report
24
Participation of Black/AAs in Clinical Trials for Oncology, Cardiology, and PsychiatryMore insight into the participation rates of Black/AAs in clinical trials for Oncology, Cardiology, and Psychiatry are provided in the figures below.
Psychiatry
Black or African American
Some Other Race
75.82%4,405
24.18%1,405
Onocology
97.26% 7,480
2.74%211
Total Participants = 7,691
Cardiovascular Disease
97.50% 55,118
2.50%1,415
Total Participants = 92,329
Total Participants = 5,810
Global Participation in Clinical Trials Project Team MembersWe would like to acknowledge the hard work of the following offices and people in the creation of this
Global Participation in Clinical Trials Report:
Office of Women’s Health (OWH)
Marjorie Jenkins, MD, MEHP, FACP
OWH Medical Director and Expert
Ruth Geller, MS
OWH ORISE Fellow
Office of Translational Sciences (OTS)
ShaAvhrée Buckman-Garner, MD, PhD, FAAP
Director, Office of Translational Sciences
Khadijatou Njimoluh, MD
OTS ORISE Fellow
Salvatore Pepe, PharmD, MS
Program Coordinator
Professional Affairs and Stakeholder Engagement (PASE)
Milena Lolic, MD
Medical Officer
Junyang Wang, MSc
Clinical Analyst
U.S. Food and Drug Administration10903 New Hampshire Ave. Silver Spring, MD 20993www.fda.gov
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