Drugs

Drug Trials Snapshots Summary Report (2015 and 2016)

Jan 1, 2015 - Dec 31, 2016

Introduction

Every year, CDER approves a number of novel drugs based upon review of safety and efficiency measures from sponsor submitted clinical trial data. Participation in these clinical trials has varied greatly, with some trials having fewer than ten patients to others including several thousand. In recent years, the representation of certain subgroups such as women and people of racial minority groups, has become of greater interest to the general public. 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. Recognizing the lack of easily accessible information about participation in drug trials, CDER piloted a new transparency initiative called the Drug Trials Snapshots.

Snapshots are data posted online in a standardized format after approval of a novel drug that is either a New Molecular Entity (NME) or original biologic (BLA) product. They show who participated in the pivotal clinical trials used to approve the drug and stratify the data by sex, race, and age subgroups. Further, the Snapshots provide statements on whether there were any observed differences in safety and efficacy by demographic subgroups at the time of approval. Since January 2015, CDER has been publishing a Drug Trials Snapshots for each novel drug approved within a month of the official approval date.

Our Summary Reports are another commitment to enhancing transparency and better understanding of the drug development process. The report summarizes the first two years of the Drug Trials Snapshots program and is broken down by calendar years 2016 and 2015. Each calendar year provides an overall average of each demographic group followed by a more detailed summary table of the percent representation of sex, race, and age per clinical trial. Since the launch of the Drug Trials Snapshots, over a quarter million people have visited the website.

We hope this information is helpful to promote dialogue on the appropriate representation of different subgroups in clinical trials and welcome your feedback.

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)

Drug Trials Snapshots Report (2016)

2016 Summary Statistics
(Jan 1, 2016 - Dec 19, 2016)

In 2016, CDER-approved 221 novel drugs, either as new molecular entities (NMEs) under New Drug Applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs). Overall, 31,468 patients participated in these trials. Subpopulation demographics are presented below:

Figure 1. Demographic Subgroups in 2016

 WOMENAFRICAN AMERICAN ASIAN WHITEOTHER*AGE
65 AND OLDER
AVERAGE48%7%11%76%7%21%

* The percentages of the categories “American Indian or Alaska Native (AI/AN),” “Native Hawaiian or Other Pacific Islander (NH/OPI),” and “Unknown/Unreported” were small enough that we combined them into the “Other” category for the purposes of this review.
1Novel Drug Approvals for 2016

Therapeutic Areas 2016

More insight into demographics for all 22 CDER-approved NMEs are provided below in Table 1.

Table 1.  All Approvals (2016)

BRAND NAMEINDICATIONWOMENAFRICAN AMERICANASIANWHITEOTHERAGE
65 and OLDER
ADLYXIN§

treatment of type 2 diabetes mellitus

52%3%32%64%2%19%
ADLYXIN§§

treatment of type 2 diabetes mellitus

31%4%13%75%8%34%
ANTHIM

For the treatment of inhalational anthrax

46%28%1%69%2%9%
AXUMIN

Detection of prostate cancer recurrence

0%4%<1%31%64%66%
BRIVIACT

Treatment of partial-onset seizures

49%3%12%74%11%2%
CINQUAIR

For the treatment of a specific type of severe asthma (called eosinophilic phenotype asthma)

62%12%8%73%8%6%
DEFITELIO

Treatment of hepatic veno-occlusive disease (VOD)

45%6%4%71%19%0%
EPCLUSA

Treatment of chronic Hepatitis C virus genotypes 1, 2, 3, 4, 5 or 6 infection.

38%6%7%85%2%11%
EUCRISA

To treat mild to moderate eczema (atopic dermatitis) in patients two years of age and older

56%28%5%61%6%0%
EXONDYS 51#

Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

0%0%8%92%0%0%
EXONDYS 51##

Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

0%0%0%100%0%0%
LARTRUVO

Treatment of soft tissue sarcoma

56%8%3%86%2%32%
NETSPOT

For detection of a specific type of tumors called somatostatin receptor positive neuro-endocrine tumors (NETs)

52%N/AN/AN/A100%N/A
NUPLAZID

Treatment of hallucinations and delusions in patients with Parkinson’s disease

36%1%5%91%3%82%
OCALIVA

Treatment of primary biliary cholangitis in adults

91%1%1%94%3%19%
RUBRACA

Treatment of women with certain type of advanced ovarian cancer

100%2%6%80%12%42%
SPINRAZA

Spinal muscular atrophy (SMA)

55%2%6%86%6%0%
TALTZ

Treatment of moderate to severe plaque psoriasis in adults

32%2%4%93%1%7%
TECENTRIQ

Treatment of a type of bladder cancer called urothelial carcinoma

78%2%2%91%5%59%
VENCLEXTA

Treatment of chronic lymphocytic leukemia (CLL)

31%3%<1%94%3%58%
XIIDRA

Treatment of the signs and symptoms of dry eye disease

76%9%4%85%2%37%
ZEPATIER

Treatment of chronic Hepatis C genotypes 1 or 4 infection

39%15%9%75%1%11%
ZINBRYTA

Treatment of relapsing forms of multiple sclerosis (MS)

67%1%3%91%5%0%
ZINPLAVA

Decreasing the risk of Clostridium difficile infection recurrence

57%5%8%85%2%62%

§ Clinical Trial of Type 2 DM patients
§§ Clinical Trial of Type 2 DM patients who recently had a heart attack

# Baseline Demographics of Trials 1 and 2
## Baseline Demographics of Trial 3

Drug Trials Snapshots Report (2015)

2015 Summary Statistics
(Jan 1, 2015 - Dec 31, 2015)
In calendar year 2015, CDER approved 452 novel drugs, either as new molecular entities (NMEs) under New Drug Applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs). Overall, 105,826 patients participated in these trials.  Subpopulation demographics are presented below:

Figure 2. Demographic Subgroups in 2015
 

 WOMENAFRICAN AMERICANASIANWHITEOTHER*AGE
65 AND OLDER
AGE
75 AND OLDER**
AGE
80 AND OLDER**
AVERAGE40%5%12%79%4%37%15%6%

* The percentages of the categories “American Indian or Alaska Native (AI/AN),” “Native Hawaiian or Other Pacific Islander (NH/OPI),” and
“Unknown/Unreported” were small enough that we combined them into the “ Other” category for the purposes of this report.
**These particular subgroups were calculated as part of a Geriatrics Report and are not a regular feature of the Drug Trial Snapshots

2To be consistent with the 2015 CDER Novel Drugs Report, three Drug Trials Snapshots were removed: Stiolto Respimat, Ryzodeg and Vistogard.  Novel Drug Approvals for 2015  

All Approvals 2015

More insight into demographics for all 45 CDER-approved novel drugs are provided below in Table 2.

Table 2. All Drug Approvals (2015)

BRAND NAMEINDICATIONWOMENAFRICAN AMERICANASIANWHITEOTHERAGE
65 and OLDER
AGE
75 and OLDER
AGE
80 and OLDER
ADDYI

Treatment of acquired, generalized hypoactive sexual desire disorder (HSDD) in premenopausal women

100%8%1%89%2%0%0%0%
ALECENSA

For the treatment of metastatic non-small cell lung cancer

55%2%18%74%7%14%4%<1%
ARISTADA

Treatment of schizophrenia

32%40%13%47%<1%0%0%0%
AVYCAZ

Treatment of complicated intra-abdominal infection (abbreviated as cIAI)

26%<1%27%60%12%11%8%4%
AVYCAZ

Treatment of complicated urinary tract infection (abbreviated as cUTI)

74%5%10%60%25%17%4%3%
BRIDION

For the reversal of the effects of certain neuromuscular blocking agents

52%3%12%84%<1%29%<1%<1%
CHOLBAM

For treatment of bile acid synthesis disorders due to single enzyme defects

43%11%7%61%20%N/AN/AN/A
CHOLBAM

For treatment of peroxisomal disorders, including Zellweger spectrum disorders

33%0%0%83%17%N/AN/AN/A
CORLANOR

To reduce hospitalization from worsening heart failure.

24%1%8%89%2%38%11%3%
COSENTYX

Treatment of moderate to severe plaque psoriasis in adults who do not respond well to medication applied directly to the skin

30%1%22%68%9%8%1%<1%
COTELLIC

Part of combination treatment melanoma

42%N/AN/A93%7%27%9%3%
CRESEMBA

Treatment of invasive aspergillosis

40%<1%21%78%<1%24%5%<1%
CRESEMBA

Treatment of invasive mucormycosis

19%11%22%68%0%14%8%0%
DAKLINZA

Treatment of chronic Hepatitis C genotype 3 infection

41%4%5%90%1%7%0%0%
DARZALEX

Treatment of multiple myeloma

46%10%6%76%8%45%10%3%
EMPLICITI

Treatment of multiple myeloma

40%4%10%84%2%57%20%7%
ENTRESTO

Treatment of heart failure

22%5%18%66%11%49%19%7%
FARYDAK

Treatment of multiple myeloma

48%3%33%63%1%35%5%0%
GENVOYA†

Complete regimen for the treatment of HIV-1 in adults and children 12 years of age and older.

48%83%17%0%0%0%0%0%
GENVOYA‡

Complete regimen for the treatment of HIV-1 in adults and children 12 years of age and older.

13%23%9%61%7%3%<1%<1%
IBRANCE

Treatment of a specific form of advanced breast cancer called ER-positive, HER2-negative (ER+/HER-) breast cancer in women who have gone through menopause (post-menopausal)

100%1%6%90%3%46%9%3%
KANUMA(i)

Treatment of Lysosomal Acid Lipase (LAL) deficiency

44%11%11%44%33%0%0%0%
KANUMA(ii)

Treatment of Lysosomal Acid Lipase (LAL) deficiency

50%2%5%83%11%0%0%0%
KENGREAL

For prevention of coronary artery blood clot formation in patients undergoing PCI

28%3%3%94%<1%48%18%8%
KYBELLA

Treatment for double chin

85%8%2%87%3%1%0%0%
LENVIMA

Treatment of progressive, differentiated thyroid cancer (DTC) that can no longer be treated with radioactive iodine

49%2%18%79%<1%40%10%4%
LONSURF

Treatment of advanced colorectal cancer

39%1%35%58%7%44%8%1%
NATPARA

For control of hypocalcemia along with calcium and vitamin D in adults with hypoparathyroidism

79%<1%2%96%2%6%<1%<1%
NINLARO

Treatment of multiple myeloma

43%2%9%85%5%58%2%<1%
NUCALA

For the treatment of a specific type of severe asthma (called eosinophilic phenotype asthma)

59%3%11%85%<1%9%10%3%
ODOMZO

Treatment of locally advanced basal cell carcinoma

37%<1%0%94%6%54%34%23%
ORKAMBI

Treatment of cystic fibrosis

49%0%0%99%1%0%0%0%
PORTRAZZA

For the treatment of metastatic squamous non-small cell lung cancer

17%1%8%84%8%39%4%1%
PRALUENT

Treatment of certain patients with high cholesterol

40%4%3%90%3%32%6%<1%
PRAXBIND

Reversal of the anticoagulant effects of Pradaxa during emergency situations or when there is a need to reverse its blood-thinning effects.

47%<1%7%85%7%90%60%44%
REPATHA††

Treatment of certain patients with high cholesterol

50%5%9%84%2%28%3%<1%
REPATHA‡‡

Treatment of certain patients with high cholesterol

49%0%4%90%6%0%0%0%
REXULTI

Treatment of schizophrenia

37%24%7%63%6%<1%0%0%
REXULTI

Treatment of major depressive disorder

69%12%<1%85%2%<1%0%0%
SAVAYSA

Reduction of risk of venous thromboembolism (VTE) in patients with previous VTE

43%4%21%70%5%33%13%6%
SAVAYSA

Prevention of stroke in patients with atrial fibrillation

38%1%14%81%4%74%40%17%
STRENSIQ(^)

Treatment of perinatal, infantile and juvenile-onset hypophosphatasia (HPP)

52%0%9%81%10%0%0%0%
STRENSIQ(^^)

Treatment of perinatal, infantile and juvenile-onset hypophosphatasia (HPP)

50%0%0%95%5%0%0%0%
TAGRISSO

Treatment of patients with advanced non-small cell lung cancer (NSCLC)

68%<1%60%36%3%45%13%4%
TRESIBA*

To improve glucose control in adults with diabetes mellitus

44%1%16%80%2%7%<1%<1%
TRESIBA**

To improve glucose control in adults with diabetes mellitus

44%7%21%70%2%24%3%<1%
UNITUXIN

Treatment of children with high-risk neuroblastoma

40%7%3%82%8%N/AN/AN/A
UPTRAVI

For the treatment of adults with pulmonary artery hypertension

80%2%21%75%2%18%1%<1%
VARUBI

To prevent delayed phase chemotherapy-induced nausea and vomiting

60%2%14%75%9%26%5%1%
VELTASSA

Treatment of hyperkalemia

39%<1%<1%99%<1%61%21%4%
VIBERZI

Treatment of irritable bowel syndrome with diarrhea

66%12%1%86%2%10%6%<1%
VRAYLAR

Treatment of schizophrenia

28%34%17%43%6%<1%0%0%
VRAYLAR

Treatment of bipolar disorder

41%25%24%49%2%<1%0%0%
XURIDEN

Treatment of patients with hereditary orotic aciduria

25%0%0%100%0%N/AN/AN/A
YONDELIS

Treatment of certain types of advances tissue sarcoma

70%12%4%76%8%22%3%<1%
ZURAMPIC

For lowering uric acid levels in the blood of adult patients with gout

4%12%6%78%5%13%3%<1%

† Clinical Trial of Children Only     
‡ Clinical Trial of Adults Only        
(i) Clinical Trial of Infants Only      
(ii) Clinical Trial of Children and Adults           
†† Clinical Trial of heterozygous familial hypercholesterolemia (HeFH) Patients 
‡‡ Clinical Trial of homozygous familial hypercholesterolemia (HoFH) Patients  
(^) Clinical Trial of Perinatal/Infantile-Onset HPP patients only          
(^^) Clinical Trial of Juvenile-Onset HPP patients only      
*Clinical Trial of Type 1 DM patients only       
**Clinical Trial of Type 2 DM patients only    

Therapeutic Areas 2015

For three groups of diseases (mental health, oncology, and cardiovascular health) there were multiple drugs approved in 2015. More insight in demographics for these approvals is provided below in Tables 3, 4, and 5.

Table 3. Mental Health Drug Approvals (2015)

BRAND NAMEINDICATIONWOMENAFRICAN AMERICANASIANWHITEOTHERAGE
65 and OLDER
AGE
75 and OLDER
AGE
80 and OLDER
ARISTADA

Schizophrenia

32%40%13%47%<1%0%0%0%
REXULTI

Schizophrenia

37%24%7%63%6%<1%0%0%
REXULTI

Major Depressive Disorder

69%12%<1%85%2%<1%0%0%
VRAYLAR

Schizophrenia

28%34%17%43%6%<1%0%0%
VRAYLAR

Bipolar disorder

41%25%24%49%2%<1%0%0%

Table 4.  Oncology  Drug Approvals (2015)

BRAND NAMEINDICATIONWOMENAFRICAN AMERICANASIANWHITEOTHERAGE
65 and OLDER
AGE
75 and OLDER
AGE
80 and OLDER
IBRANCE

Breast cancer

100%1%6%90%3%46%9%3%
YONDELIS

Advanced soft tissue sarcoma

70%12%4%76%8%22%3%<1%
TAGRISSO

Lung Cancer (T790M+, NSCLC)

68%<1%60%36%3%45%13%4%
ALECENSA

Metastatic NSCLC

55%2%18%74%7%14%4%<1%
LENVIMA

Thyroid cancer

49%2%18%79%<1%40%10%4%
FARYDAK

Multiple Myeloma & other cancers

48%3%33%63%1%35%5%0%
DARZALEX

Multiple Myeloma

46%10%6%76%8%45%10%3%
NINLARO

Multiple Myeloma

43%2%9%85%5%58%2%<1%
COTELLIC

Melanoma

42%N/AN/A93%7%27%9%3%
EMPLICITI

Multiple Myeloma

40%4%10%84%2%57%20%7%
UNITUXIN

Neuroblastoma

40%7%3%82%8%N/AN/AN/A
LONSURF

Advanced metastatic colorectal cancer

39%1%35%58%7%44%8%1%
ODOMZO

Advanced basal cell carcinoma (BCC)

37%<1%0%94%6%54%34%23%
PORTRAZZA

Metastatic squamous non-small cell lung cancer (NSCLC)

17%1%8%84%8%39%4%1%

Table 5.  Cardiovascular  Drug Approvals (2015)

BRAND NAMEINDICATIONWOMENAFRICAN AMERICANASIANWHITEOTHERAGE
65 and OLDER
AGE
75 and OLDER
AGE
80 and OLDER
UPTRAVI

Pulmonary arterial hypertension

80%2%21%75%2%18%1%<1%
REPATHA*

Hypercholesterolemia

50%5%9%84%2%28%3%<1%
REPATHA**

Hypercholesterolemia

49%0%4%90%6%0%0%0%
PRAXBIND

Reversal of the anticoagulant effects of Pradaxa during emergency situations or when there is a need to reverse its blood-thinning effects.

47%<1%7%85%7%90%60%44%
SAVAYSA

Reduce  risk of pulmonary embolism in VTE patients

43%4%21%70%5%33%13%6%
PRALUENT

Hyperlipidemia

40%4%3%90%3%32%6%<1%
SAVAYSA

Reduce the risk of stroke in a Afib patients

38%1%14%81%4%74%40%17%
KENGREAL

Blood thinner following heart procedure

28%3%3%94%<1%48%18%8%
CORLANOR

Heart failure

24%1%8%89%2%38%11%3%
ENTRESTO

Heart failure

22%5%18%66%11%49%19%7%

* Clinical Trial of HeFH Patients
** Clinical Trial of HoFH Patients

Google Analytics Web Metrics

(Jan 1, 2015 – Dec 31, 2016)

The data below were calculated from Google Analytics and highlights the total number of page views, unique visitors, and average time spent on all Drug Trials Snapshots pages. 

Snapshots Website Highlights:

  • Over 235,946 people visited the site since launch of the website
  • These people generated 277,555 visits to the site (some came more than once)
  • Average of 10,000 visits per month
  • The average visit lasted 2:13 minutes
  • The top 5 Snapshots visited were Jublia, Lonsurf, Savaysa, Kybella, and Cosentyx

Visitors by Geographic Location:

The data below were calculated from Google Analytics, measuring Geographic Locations of visitors coming to the Drug Trials Snapshots.   

  • Almost 70% of visitors (115,407 sessions) are from the United States:
    • Top 5 Cities in the United States:
      • Silver Spring, MD (8%)
      • Seattle, WA (4%)
      • Denver, CO (2%)
      • Washington, DC (2%)
      • New York, NY (2%)

These data are based on a 0.15% subset of the entire traffic and reporting data of users coming to the Drug Trials Snapshots.  It is measured in sessions, a group of interactions that take place on your website within a given time frame. For example a single session can contain multiple screen or page views, events, social interactions, and ecommerce transactions. By default, a session lasts until there's 30 minutes of inactivity.

 

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