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Mental Health
PRESENTED BY AMERICA’S BIOPHARMACEUTICAL RESEARCH COMPANIES
Over the past half century, biopharma- Facts About Mental Health in the
United States ..................................8
ceutical research has helped alleviate the
Medicines in Development Chart ...... 11
burden and improve the quality of life
e
tan renia
De tism
on
Glossary ....................................... 24
An D
ty
dic /
tiv
Ad Use
H
ssi
AD
Drug Development/
Au
h
pre
ce
61.5 MILLION
AMERICANS
NIMH’s practical guidelines have documented the sometimes
limited benefits of current treatments and the need for a new
generation of mental health medicines.
MENTAL
gets are ever approved for patients. And although advances
in our understanding of mental disorders and how to treat
them have allowed America’s biopharmaceutical companies
and other partners in the collaborative ecosystem to con-
ILLNESSES
duct cutting-edge research, additional scientific research is
needed to reduce the destructive toll of these disorders and
allow more people to lead healthier, more productive lives.
Challenges in Mental
Health Research
COST THE
U.S. $317 BILLION ANNUALLY
in lost wages, health care and disability
Development of new and effective treatments for mental
disorders has been hindered by many factors, including a Source: NIMH.
MENTAL
Although research into mental disorders can be very diffi-
cult, there are some promising medicines in the pipeline that
are looking at new ways to treat many of these disorders.
ADHD Schizophrenia
1 in 20
Several medicines in development for schizophrenia are
AFFECTS inhibitors of a subtype of phosphodiesterase (PDE), a key
21
A medicine in development for major depressive disorder in
patients who have not responded to current treatments com-
bines an opioid modulator with an opioid stimulator. Opioid MILLION
stimulators have been shown to help in the treatment of AMERICAN ADULTS
depression, but they can also be highly addictive. By coupling
the stimulant with an opioid receptor blocker, the addictive Source: NIMH.
ANXIETY
Director, National Institute of Mental Health
DISORDERS
AFFECT
academic-industry research collaboration dedicated to
finding new methods for developing drugs for schizophre-
40 MILLION
AMERICAN ADULTS
nia and depression. NEWMEDS is based in Europe but
involves many U.S. industry partners. Ultimately, the goal
of NEWMEDS is to develop new approaches for shorter
Source: NIMH.
and more efficient trials of new medications, with clinical
trials that may require fewer patients and give faster results.
YMPTOMS
disease pathology and developing new, effective treatments.
DISORGANIZED PSYCHOSIS
O
Many of the challenges in mental health research reflect the
THOUGHT PROCESS HALLUCINATIONS reality that we are still learning how the human brain works.
DIAGNOSISCOGNITIVE PSYCHOLOGICAL
TREATMENTGEN
SCHIZOPHRENIA
BRAIN CHEMISTRY DELUSIONS
Also, research efforts are complicated by the fact that many
disorders fall under the broad umbrella of mental health,
ENETIC
• The Army Study to Assess Risk and Resilience in Ser- MENTAL HEALTH
vicemembers (Army STARRS) is an unparalleled research
study on suicide prevention—a critical area of research
for the Army, other military personnel and the general
population. The 5-year study, one of the largest and most
complex studies ever administered by NIMH, is being
conducted in collaboration with select academic research
teams.
Anxiety Disorders 15
Attention-Deficit
Hyperactivity/Disorder 15
Autism Spectrum
Disorders 6
Bipolar Disorders 5
Application
Depression 29 Submitted
Phase III
Eating Disorders 2 Phase II
Schizophrenia 36 Phase I
Substance Use 20
Disorders
Tic Disorders 3
Other Disorders 2
• Although most children with ADHD have normal or • Major depressive disorder is the leading cause of dis-
above-normal intelligence, 40 percent to 60 percent ability among Americans ages 15-44. It affects nearly
have serious learning difficulties.4 15 million U.S. adults.
• Children and adolescents with ADHD are more likely Eating Disorders1
than children without the disorder to suffer from other
• In their lifetime, an estimated 0.6 percent of the U.S. adult
mental disorders.4
population will suffer from anorexia, 1.0 percent from
Autism Spectrum Disorders5 bulimia, and 2.8 percent from a binge-eating disorder.
• In 2010, about 1 in 68 8-year-olds were identified with • Women are much more likely than men to develop an
autism spectrum disorder (ASD).* The 2010 estimate is eating disorder. They are three times as likely to experi-
roughly 30 percent higher than the estimate for 2008 ence anorexia (0.9 percent of women vs. 0.3 percent of
(1 in 88), 60 percent higher than the estimate for 2006 men) and bulimia (1.5 percent of women vs. 0.5 percent
(1 in 110), and 120 percent higher than the estimates of men) during their lives. They are also 75 percent more
for 2002 and 2000 (1 in 150). (The exact cause of the likely to have a binge-eating disorder (3.5 percent of
increase is unknown, but researchers believe some of it women vs. 2.0 percent of men).
may be due to the way children are identified, diagnosed,
and served in their local communities.) Schizophrenia
• Approximately 2.4 million American adults age 18 and
• Boys were almost five times more likely to be identified
older have schizophrenia in a given year. It affects men
with ASD than girls in 2010. About 1 in 42 boys and 1 in
and women with equal frequency. Schizophrenia often
189 girls were identified with ASD.
first appears in men in their late teens or early twenties.
Depression1 In contrast, women are generally affected in their twen-
ties or early thirties.1
• Mood disorders, which include major depressive dis-
order, dysthymic disorder, and bipolar disorder, affect • The appearance of schizophrenic symptoms before age
nearly 21 million adults, or about 9.5 percent of the U.S. 12 is rare—less than one-sixtieth as common as the
population age 18 and older in a given year. adult-onset type. Neurodevelopmental damage seems to
be greater in childhood schizophrenia than in the adult-
• Bipolar disorder affects approximately 5.7 million Ameri-
onset type.5
can adults, or about 2.6 percent of the U.S. population
age 18 and older in a given year. The median age of Substance Use/Addictive Disorders
onset for bipolar disorder is 25 years.
• An estimated 17 million Americans have an alcohol use
• Depression can strike anyone regardless of age, ethnic disorder—a medical term that includes both alcoholism
background, socioeconomic status, or gender; however, and harmful drinking that does not reach the level of
studies have found that depression is about twice as dependence.6
common in women as in men. In any given one year
• Each year in the United States, nearly 80,000 people die
period, depressive illnesses affect more than 12 million
from alcohol-related causes, making it the third leading
women and more than 6 million men.
preventable cause of death in our country.6
* Estimates are based on information collected from the health and special education records of 8-year-old children living in areas of 11 different states. They
do not represent the entire population of children in the United States.
• Estimates of the total overall costs of substance abuse 6. National Institute on Alcohol Abuse and Alcoholism,
in the United States, including productivity and health- www.niaaa.nih.gov
and crime-related costs, exceed $600 billion annually. 7. National Institute on Drug Abuse, www.drugabuse.gov
That includes approximately $193 billion for illicit drugs, 8. Substance Abuse and Mental Health Services Adminis-
$193 billion for tobacco, and $235 billion for alcohol.7 tration, www.samhsa.gov
Anxiety Disorders
Product Name Sponsor Indication Development Phase*
*For more information about a specific medicine or company in the report, please use the website provided.
Anxiety Disorders
Product Name Sponsor Indication Development Phase
Attention-Deficit/Hyperactivity Disorder
Product Name Sponsor Indication Development Phase
Attention-Deficit/Hyperactivity Disorder
Product Name Sponsor Indication Development Phase
Bipolar Disorders
Product Name Sponsor Indication Development Phase
Depression
Product Name Sponsor Indication Development Phase
armodafinil Teva Pharmaceutical major depressive disorder associated Phase III completed
North Wales, PA with bipolar 1 disorder www.tevapharm.com
(see also eating disorders)
Depression
Product Name Sponsor Indication Development Phase
Depression
Product Name Sponsor Indication Development Phase
Eating Disorders
Product Name Sponsor Indication Development Phase
Schizophrenia
Product Name Sponsor Indication Development Phase
Schizophrenia
Product Name Sponsor Indication Development Phase
Schizophrenia
Product Name Sponsor Indication Development Phase
Schizophrenia
Product Name Sponsor Indication Development Phase
Bunavail™ BioDelivery Sciences International opioid use disorder, opioid abuse application submitted
buprenorphine/naloxone Raleigh, NC www.bdsi.com
transmucosal
TIC Disorders
Product Name Sponsor Indication Development Phase
Other Disorders
Product Name Sponsor Indication Development Phase
The content of this report has been obtained through public, government and industry sources, and the Adis “R&D Insight”
database based on the latest information. Report current as of April 28, 2014. The medicines in this report include medi-
cines being developed by U.S.-based companies conducting trials in the United States and abroad, PhRMA-member compa-
nies conducting trials in the United States and abroad, and foreign companies conducting clinical trials in the United States.
The information in this report may not be comprehensive. For more specific information about a particular product, contact
the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available
on PhRMA’s website.
A publication of PhRMA’s Communications & Public Affairs Department (202) 835-3460
www.phrma.org | www.innovation.org | www.pparx.org
Provided as a public service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.
Copyright © 2014 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper
credit is given.
Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004
anxiety disorders—Anxiety is a normal combination of different symptom types. high blood pressure. They also experi-
reaction to stress and can be benefi- Those with mostly inattentive symptoms ence guilt, shame, and distress about
cial in some situations, but for some are sometimes said to have attention their binge-eating, which can lead to
people anxiety may become excessive. deficit disorder (ADD). They tend to be more binge-eating.
While people suffering may realize their less disruptive and are more likely not to
bipolar disorder—Also known as manic-
anxiety is too much, they may also be diagnosed with ADHD.
depressive illness, a brain disorder that
have difficulty controlling it, and it may
autism spectrum disorder (ASD)—ASD causes unusual shifts in mood, energy,
negatively affect their day-to-day living.
is a developmental disability that can activity levels, and the ability to carry
The wide variety of anxiety disorders
cause significant social, communica- out day-to-day tasks. Symptoms of
includes generalized anxiety disorder
tion and behavioral challenges. People bipolar disorder are severe. They are dif-
(GAD), obsessive-compulsive disorder
with ASD may communicate, interact, ferent from the normal ups and downs
(OCD), panic disorder, post-traumatic
behave, and learn in ways that are that everyone goes through from time
stress disorder (PTSD), and social
different from most other people. The to time. People with bipolar disorder
phobia. Collectively, they are among the
learning, thinking, and problem-solving experience unusually intense emotional
most common mental health disorders
abilities of people with ASD can range states that occur in distinct periods
experienced by Americans.
from gifted to severely challenged. called “mood episodes.” Each mood epi-
application submitted—An application Some people with ASD need a lot of sode represents a drastic change from
for marketing has been submitted by help in their daily lives; others need less. a person’s usual mood and behavior.
the company to the U.S. Food and Drug People with ASD often have problems An overly joyful or overexcited state is
Administration (FDA). with social, emotional, and communica- called a manic episode, and an ex-
tion skills. They might repeat certain tremely sad or hopeless state is called a
attention deficit/hyperactivity disor- behaviors and might not want change depressive episode. Sometimes, a mood
der (ADHD)—ADHD is a problem of in their daily activities. Many people episode includes symptoms of both
not being able to focus, being overac- with ASD also have different ways of mania and depression, called a mixed
tive, not being able control behavior, learning, paying attention, or reacting to state. People with bipolar disorder also
or a combination of these symptoms. things. Signs of ASD begin during early may be explosive and irritable during
For those problems to be diagnosed as childhood and typically last throughout a mood episode. Extreme changes in
ADHD, they must be out of the normal a person’s life. A diagnosis of ASD now energy, activity, sleep, and behavior
range for a person’s age and develop- includes several conditions that used to go along with those changes in mood.
ment. ADHD usually begins in childhood be diagnosed separately: autistic disor- Bipolar disorder symptoms can result
but may continue into the adult years. der, pervasive developmental disorder in damaged relationships, poor job or
It is the most commonly diagnosed not otherwise specified, and Asperger’s school performance, and even suicide.
behavioral disorder in children. ADHD syndrome. Those conditions are now all But bipolar disorder can be treated, and
is diagnosed much more often in boys called autism spectrum disorder. people with the illness can lead full and
than in girls. A combination of genes productive lives.
and environmental factors likely plays a binge-eating disorder—With binge-eat-
role in the development of the condi- ing disorder a person loses control over depression—Everyone occasionally feels
tion. Symptoms of ADHD fall into three his or her eating. Unlike bulimia nervosa, blue or sad, but those feelings are usu-
groups: not being able to focus (inatten- periods of binge-eating are not fol- ally short-lived and pass within a couple
tiveness); being extremely active (hyper- lowed by purging, excessive exercise, or of days. Depression, however, interferes
activity); and not being able to control fasting. As a result, people with binge- with daily life and causes pain for both
behavior (impulsivity). Some people with eating disorder often are overweight or the sufferers and those who care about
ADHD have mainly inattentive symp- obese. People with binge-eating disor- them. Depression is a common but seri-
toms. Some have mainly hyperactive der who are obese are at higher risk for ous illness. Major depression and persis-
and impulsive symptoms. Others have a developing cardiovascular disease and tent depressive disorder are among the
several forms of depressive disorders. where none exists or providing a therapy an overwhelming urge to repeat certain
Major depression causes severe symp- that may be potentially superior to exist- rituals or behaviors called compulsions.
toms that interfere with a person’s abil- ing therapy. Once a drug receives Fast People with OCD can’t control those
ity to work, sleep, study, eat, and enjoy Track designation, early and frequent obsessions and compulsions—the rituals
life. An episode can occur only once in communication between the FDA and a usually end up controlling them. For
a person’s lifetime, but more often, a drug company is encouraged throughout example, if people are obsessed with
person has several episodes. Persistent the entire drug development and review germs or dirt, they may develop a com-
depressive disorder causes a depressed process. The frequency of communica- pulsion to wash their hands over and
mood that lasts for at least 2 years. tion ensures that questions and issues over again. If they develop an obsession
A person diagnosed with persistent are resolved quickly, often leading to with intruders, they may lock and relock
depressive disorder may have episodes earlier drug approval and access by their doors many times before going
of major depression along with periods patients. to bed. Performing such rituals is not
of less severe symptoms, but symp- pleasurable. At best, it produces tempo-
generalized anxiety disorder (GAD)—
toms must last for 2 years. People with rary relief from the anxiety created by
While everyone worries about things
depressive illnesses do not all experi- obsessive thoughts. People with OCD
like health, money, or family problems,
ence the same symptoms. The severity, perform their rituals even though doing
people with GAD are extremely worried
frequency, and duration of symptoms so interferes with daily life, and they find
about those problems and many other
vary depending on the individual and the repetition distressing. Although most
things, even when there is little or no
his or her particular illness. A few of the adults with OCD recognize that what
reason to worry about them. They are
many signs and symptoms of depression they are doing is senseless, some adults
very anxious about just getting through
are: persistent sad, anxious, or “empty” and most children may not realize that
the day, thinking that things will always
feelings; feelings of hopelessness or pes- their behavior is out of the ordinary.
go badly. At times, worrying keeps
simism; fatigue and decreased energy;
people with GAD from doing everyday Orphan Drug—A drug to treat a dis-
difficulty concentrating, remembering
tasks. They can’t relax, startle easily, and ease that has a patient population of
details, and making decisions; overeating,
have difficulty concentrating. Often they 200,000 or less in the United States, or
or appetite loss; and thoughts of suicide,
have trouble falling asleep or staying a disease that has a patient population
or suicide attempts.
asleep. Physical symptoms that often of more than 200,000 and a develop-
Fast Track—A process designed to accompany the anxiety include fatigue, ment cost that will not be recovered
facilitate the development and expedite headaches, muscle tension, muscle from sales in the United States.
the review of drugs to treat serious aches, difficulty swallowing, trembling,
panic disorder—People with panic dis-
diseases and fill an unmet medical need. twitching, irritability, sweating, nausea,
order have sudden and repeated attacks
The status is assigned by the U.S. Food lightheadedness, having to go to the
of fear that last for several minutes.
and Drug Administration (FDA). The bathroom frequently, feeling out of
Sometimes symptoms may last longer
purpose of this process is to get impor- breath, and hot flashes.
and are called panic attacks. Panic
tant new drugs to the patient earlier.
NCE—New chemical entity. attacks are characterized by a fear of
Fast Track addresses a broad range of
disaster or of losing control even when
serious diseases. In general, determining obsessive-compulsive disorder (OCD)— there is no real danger. A person may
factors for whether a drug receives Fast People with OCD feel the need to also have a strong physical reaction dur-
Track include whether the drug will af- check things repeatedly or have certain ing a panic attack. It may feel like having
fect factors such as survival, day-to-day thoughts or perform routines and rituals a heart attack. Panic attacks can occur
functioning, or the likelihood that the over and over. The thoughts and rituals at any time, and many people with panic
disease, if left untreated, will progress associated with OCD cause distress and disorder worry about and dread the
from a less severe condition to a more get in the way of daily life. The frequent possibility of having another attack. A
serious one. Filling an unmet medical upsetting thoughts are called obsessions. person with panic disorder may become
need is defined as providing a therapy To try to control them, a person will feel discouraged and feel ashamed because
he or she cannot carry out normal rou- physical harm. The person who develops increased impulsive behaviors (e.g.,
tines like going to the grocery store or PTSD may have been the one who was sexual activities, drug and alcohol abuse,
driving. Having panic disorder can also harmed, the harm may have happened gambling, or spending large amounts
interfere with school or work. to a loved one, or the person may have of money). Many of the strategies used
witnessed a harmful event that hap- to treat both schizophrenia and affec-
Phase 0—First-in-human trials con- pened to loved ones or strangers. PTSD tive conditions can be employed for this
ducted in accordance with FDA’s 2006 was first brought to public attention in condition, including antipsychotic and
guidance on exploratory Investigational relation to war veterans, but it can result mood stabilizing medications, family
New Drug (IND) studies designed to from a variety of traumatic incidents, involvement, psychosocial strategies,
speed development of promising drugs such as a mugging, rape, being kid- self-care peer support, psychotherapy
by establishing early whether the tested napped or held captive, child abuse, and integrated care for co-occurring
compound behaves in humans as was major accidents, or natural disasters substance abuse (when appropriate).
anticipated from preclinical studies. such as floods or earthquakes.
schizophrenia—A chronic, severe, and
Phase I—Researchers test the drug in a schizoaffective disorder—A serious disabling brain disorder that has affected
small group of people, usually between mental illness that affects about one people throughout history. About 1
20 and 80 healthy adult volunteers, to in 100 people. Schizoaffective disor- percent of Americans have the illness.
evaluate its initial safety and tolerability der as a diagnostic entity has features People with the disorder may hear
profile, determine a safe dosage range, that resemble both schizophrenia and voices other people don’t hear. They
and identify potential side effects. also serious mood (affective) symp- may believe other people are reading
Phase II—The drug is given to volunteer toms. A person who has schizoaffec- their minds, controlling their thoughts,
patients, usually between 100 and 300, tive disorder will experience delusions, or plotting to harm them. That can
to determine whether the drug is effec- hallucinations, other symptoms that terrify people with the illness and make
tive, identify an optimal dose, and to are characteristic of schizophrenia, and them withdrawn or extremely agitated.
evaluate further its short-term safety. significant disturbances in their mood People with schizophrenia may not make
(e.g., affective symptoms). People who sense when they talk. They may sit for
Phase III—The drug is given to a larger, experience more than two weeks of hours without moving or talking. Some-
more diverse patient population, often psychotic symptoms in the absence times people with schizophrenia seem
involving between 1,000 and 3,000 of severe mood disturbances—and perfectly fine until they talk about what
patients (but sometimes many more then have symptoms of either depres- they are really thinking. Families and
thousands), to generate statistically sion or bipolar disorder—may have society are affected by schizophrenia,
significant evidence to confirm its safety schizoaffective disorder. Schizoaffective too. Many people with schizophrenia
and effectiveness. Phase III studies are disorder is thought to be between the have difficulty holding a job or caring
the longest studies and usually take bipolar and schizophrenia diagnoses for themselves, so they rely on others
place in multiple sites around the world. as it has features of both. Depressive for help. Cognitive symptoms are subtle
symptoms associated with schizoaffec- and may be difficult to recognize as part
post-traumatic stress disorder (PTSD)—
tive disorder can include—but are not of the disorder. Often, they are detected
When in danger, it’s natural to feel
limited to—hopelessness, helplessness, only when other tests are performed.
afraid. That fear triggers the “fight-or-
guilt, worthlessness, disrupted appetite, Cognitive symptoms include the follow-
flight” response as a healthy reaction
disturbed sleep, inability to concentrate, ing: poor “executive functioning” (the
meant to protect a person from harm.
and depressed mood (with or without ability to understand information and
But in PTSD, that reaction is changed
suicidal thoughts). Manic (bipolar) symp- use it to make decisions); trouble focus-
or damaged. People who have PTSD
toms associated with schizoaffective ing or paying attention; problems with
may feel stressed or frightened even
disorder can include increased energy, “working memory” (the ability to use in-
when they’re no longer in danger. PTSD
decreased sleep (or decreased need for formation immediately after learning it).
develops after a terrifying ordeal that
sleep), distractibility, fast speech, and Cognitive symptoms often make it hard
involved physical harm or the threat of
to lead a normal life and earn a living. substance use disorders—A term used harmful consequences. It is considered a
They can cause great emotional distress. to characterize illnesses associated with brain disease because drugs change the
Treatment helps relieve many symptoms drug use. There are two broad catego- brain—its structure and how it works.
of schizophrenia, but most people who ries: substance abuse and substance Those brain changes can be long lasting
have the disorder cope with symptoms dependence. Both are associated with and can lead to many harmful, often
throughout their lives. a maladaptive pattern of substance self-destructive, behaviors.
use that leads to clinically significant
social phobia (social anxiety disorder)— impairment. Drug abuse includes such
Tourette’s syndrome (TS)—A neurologi-
Social phobia is a strong fear of being symptoms as: failure to fulfill major
cal disorder characterized by repetitive,
judged by others and of being embar- role obligations; legal problems; use in
involuntary movements and vocaliza-
rassed. The fear can be so strong that situations that are physically hazardous;
tions called tics. The early symptoms of
it gets in the way of going to work or and continued use despite persistent
TS are typically noticed first in child-
school or doing other everyday things. social or interpersonal problems. The
hood, with the average onset between
Meeting new people or giving a public term dependence includes such symp-
the ages of 3 and 9. TS occurs in people
speech can make anyone nervous, but toms as: drug taking in larger amounts
from all ethnic groups; males are af-
people with social phobia worry about than intended; inability to cut down on
fected about three to four times more
such things for weeks before they hap- drug use; a great deal of time spent in
often than females. It is estimated that
pen. People with social phobia are afraid activities necessary to obtain the drug;
200,000 Americans have the most
of doing common things in front of and continued use despite knowledge
severe form of TS, and as many as 1
other people. For example, they might of health or social problems caused by
in 100 exhibit milder and less complex
be afraid to sign a check in front of a the drug. Dependence may or may not
symptoms such as chronic motor or
cashier at the grocery store, or they include “physical dependence,” defined
vocal tics. Although TS can be a chronic
might be afraid to eat or drink in front by withdrawal symptoms when drug use
condition with symptoms lasting a
of other people. Most people who have is abruptly ceased, and “tolerance,” the
lifetime, most people with the condition
social phobia know that they shouldn’t need for more of a drug to achieve a
experience their worst tic symptoms in
be as afraid as they are, but they can’t desired effect. The term “dependence”
their early teens, with improvement oc-
control their fear. For some people, can also refer to “addiction.” Addiction
curring in the late teens and continuing
social phobia is a problem only in certain is defined as a chronic, relapsing brain
into adulthood.
situations, while others have symptoms disease that is characterized by com-
in almost any social situation. pulsive drug seeking and use, despite
NDA SUBMIT T ED
IND SUBMIT T ED
NUMBER OF VOLUNTEERS