2015-2016 Global Participation in Clinical Trials Report · Global Participation in Clinical Trials...

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.

https://www.fda.gov/Drugs/InformationOnDrugs/ucm412998.htm

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.

https://www.fda.gov/downloads/Drugs/InformationOnDrugs/UCM541327.pdf




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


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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30%

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40,8

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5,80

2

5,17

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4,85

8

4,74

8

3,84

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3,55

1

3,04

0

2,88

7

2,87

8

2,87

2

2,70

3

2,37

5

2,36

4

2,31

6

2,25

5

2,25

4

2,07

0

2,03

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1,97

7

1,68

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1,57

4

1,56

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1,40

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1,37

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1,36

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1,31

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928

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964

557

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529

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205

177

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129

105

103

101

583

81 65 62 50 30 27 26 26 1784 2 17

30.9

9%

4.40

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3.92

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3.69

%

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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2.05

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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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1.50

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1.28

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1.19

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1.19

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1.06

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1.04

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1.04

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%

0.78

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0.73

%

0.71

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0.70

%

0.56

%

0.49

%

5.22

%

0.42

%

0.40

%

0.40

%

0.40

%

0.37

%

0.35

%

0.29

%

0.29

%

0.25

%

0.25

%

0.20

%

0.20

%

0.18

%

0.16

%

0.13

%

0.12

%

0.12

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0.10

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0.08

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0.08

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0.08

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0.06

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0.06

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0.05

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0.05

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0.02

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0.02

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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

%

0.44

%

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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1,57

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3.92

%

3.69

%

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%

2.92

%

2.70

%

2.31

%

2.19

%

2.18

%

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1.79

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1.76

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1.71

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%

0.44

%

United States

Rest of World


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


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


Dermatology


Gastroenterology

Hematology

Infectious Disease

Medical Genetics

Nephrology

Neurology


Pulmonary Disease

Psychiatry

Rheumatology


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


Dermatology


Gastroenterology

Hematology

Infectious Disease

Medical Genetics

Nephrology

Neurology


Pulmonary Disease

Psychiatry

Rheumatology


Ophthalmology

2016

Oncology

Anesthesiology


Dermatology


Gastroenterology

Hematology

Infectious Disease

Medical Genetics

Nephrology

Neurology


Pulmonary Disease

Psychiatry

Rheumatology


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


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.


53.1%

United Kingdom36.9%

United States49.1%

New Zealand30.2%

South Africa45.2%

South Korea45.8%

Philippines37.4%

Macedonia41.6%


27.4%

Argentina35.8%

Lithuania22.4%

Australia41.9%

Thailand45.0%

Lebanon75.8%


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


2015-2016

13


53.1%

United Kingdom36.9%

United States49.1%

New Zealand30.2%

South Africa45.2%

South Korea45.8%

Philippines37.4%

Macedonia41.6%


27.4%

Argentina35.8%

Lithuania22.4%

Australia41.9%

Thailand45.0%

Lebanon75.8%


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



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


Missing

Global

Rest of the World United States


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%


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


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%


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


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.




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


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.




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


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


Some Other Race

75.82%4,405

24.18%1,405

Onocology

97.26% 7,480

2.74%211



97.50% 55,118

2.50%1,415



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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