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For many years, vaccines have been used
to successfully prevent devastating infec-
tious diseases such as smallpox, measles
and polio. According to data from the
U.S. Centers for Disease Control and
Prevention (CDC), 10 infectious diseases
have been at least 90 percent eradicated
in the United States thanks to vaccines.
This has protected millions of children
and families from needless illness.
These public health triumphs illustrate the
major contributions that vaccines have
made in saving countless lives around
the world. In the past several years,
through our growing understanding of
the molecular underpinnings of disease
and technological advances, many new
vaccines have been developed, including
one against human papillomavirus (HPV)
infections that can lead to cervical cancer,
a vaccine to guard against the anthrax
virus before exposure, and a vaccine to
prevent pneumococcal infections in high-
risk populations.
But vaccines are not only for preventing
infectious diseases, some help the body
fight a range of illnesses by activating
the immune system to recognize and at-
tack disease. In 2010, a new cancer vac-
cine for the treatment of prostate cancer
was approved in the United States, and
many more immunotherapeutic vaccines
are in development.
Today, biopharmaceutical research com-
panies are developing 271 vaccines for
infectious diseases, cancer, neurological
disorders, allergies and other diseases.
Among the projects in development are:
• A therapeutic vaccine for HIV infection
intended to delay disease progression.
• A monoclonal antibody vaccine that
targets both pandemic and seasonal
influenza.
• A genetically-modified vaccine designed
for the treatment of pancreatic cancer.
• An irradiated vaccine for protection
against malaria.
The development and regulatory path
that these vaccine candidates face is
complex. As with the development of all
drugs, the majority of vaccines must pre-
vail through years of clinical testing before
they can be approved for use by the gen-
eral public. However, advances in other
scientific fields, such as genomics and
manufacturing technologies, are becom-
ing increasingly useful in the development
of new vaccines. The continued efforts
of researchers within biopharmaceutical
companies and across the ecosystem,
who are pursuing new techniques and
strategies in vaccine development, cre-
ate tremendous opportunities to protect
against many more life-threatening
diseases in the future.
Nearly 300 Vaccines Are in Development;
Research Focuses on Prevention and Treatment
MeDiciNes iN DeVeloPMeNT
Vaccines
A Report on the Prevention and Treatment of Disease Through Vaccines
presented by america’s biopharmaceutical research companies
Vaccines in Development*
contents
Innovative Vaccines in the Pipeline .................2
Preventative Vaccines: Positive Impact ...........3
Scientific Advances in Vaccine Development ....4
Vaccine Successes in the United States ........6
Vaccines in Development .......................... 10
Glossary ...................................................33
Drug Development/Approval Process ........36
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Phase III
Phase II
Phase I
*Some vaccines are listed in more than one category.
Medicines in Development Vaccines 20132
innovative Vaccines in the Pipeline
Building on the transformational successes to date, the
future of vaccines—both for the prevention and treatment
of disease—offers great hope for improving and preserving
public health in the United States and across the globe. The
271 medicines in development span a wide array of diseases,
and employ exciting new scientific and technical knowledge.
Here are a few examples of the promising vaccines in the
pipeline:
Malaria—A malaria vaccine in development has shown to
be 100 percent effective in early clinical trials in preventing
the transmission of the disease from infected mosquitoes to
humans. The vaccine builds on initial knowledge gained in
the 1970s, where researchers demonstrated that long-term
protection against malaria was possible when volunteers
received thousands of bites from radiated infected mosqui-
toes. The vaccine in development uses a weakened form of
the whole sporozoite, a life-stage of the parasite Plasmo-
dium falciparnum that causes malaria. This weakened form
of the parasite is enough to cause an immune response, but
not cause the disease, thus leading to protection against any
future malarial bite infection.
cervical cancer—A live, attenuated Listeria monocyto-
genes (LM)-based immunotherapy is in development for
the treatment of women who already have cervical cancer
as a result of infection by the human papillomavirus (HPV).
The vaccine targets the HPV gene E7, which is responsible
for the transformation of HPV-infected cells into dysplastic
or malignant tissue. The vaccine is engineered to secrete a
fusion protein that instructs the body’s immune system to
destroy the tumors.
HIV Infection—A therapeutic vaccine in development is tar-
geting the low-mutating (conserved) parts from the protein
p24 of the HIV virus. The vaccine consists of four peptides
that are modified to increase the immune response against
the conserved parts of the p24 protein. A sustained immune
response against the p24 protein has shown to be associ-
ated with delayed disease progression.
Milestones in Vaccine Development
first therapeutic
cancer vaccine (U.S.)
avian flu vaccine
egg-free vaccine
nasal flu vaccine
hepatitis B vaccine
polio vaccine
rabies vaccine
1796
1885
1952
1981
2003
2007
2010
2013
first vaccine ever developed
is for smallpox
Key issues
Medicines in Development Vaccines 2013 3
influenza—A monoclonal antibody (mAb) vaccine in
development targets both pandemic and severe seasonal
influenza A virus infections. The mAb vaccine, made from
recombinant human antibodies from human B-cell cultures,
specifically targets the M2 protein of the virus, which is es-
sential for the influenza virus to function normally.
Pancreatic cancer–A potential treatment for pancreatic
cancer is a combination of two therapeutic vaccines. The
treatment combines a Listeria-based vaccine that has
been engineered to express the tumor-associated antigen
mesothelin and allogeneic pancreatic cancer cells that are
genetically-modified to secrete the immune-stimulant,
granulocyte-macrophage colony stimulating factor (GM-CSF).
The cells are irradiated to prevent further cell growth
although they stay metabolically active. Sequential adminis-
tration of the vaccines in animal studies have demonstrated
enhanced tumor-specific T-cell and anti-tumor responses.
smoking cessation–One in a new class of targeted vac-
cines in development induces an antigen-specific immune
activation for smoking cessation and relapse prevention.
It is made from biocompatible and biodegradable materials
and is a fully synthetic nanoparticle vaccine engineered to
mimic the properties of natural pathogens to elicit an im-
mune response.
Preventative Vaccines:
Positive impact to Health and society
Vaccines are one of the most profound achievements of bio-
medical science and public health. Spanning more than 200
years of research and development, 10 infectious diseases
have been at least 90 percent eradicated in the United States
thanks to vaccines. This has protected millions of children
and families from avoidable illness.
The prevention of disease has an enormous impact on the
health of individuals, but also the health of communities and
research & hope awards
On September 11, 2013, PhRMA, along with distinguished members of the medical, patient, and provider
communities, presented the second annual Research & Hope Awards. This year’s program highlighted the
history, progress and promising future of vaccines and immunization. PhRMA proudly presented the following
awards:
the research & hope award for academic or public research in Vaccine development
Presented to individuals or a team from academic or government institutions for outstanding research in the area of vaccine
development.
2013 award presented to: Douglas R. Lowy, M.D., and John T. Schiller, Ph.D., National Cancer Institute
the research & hope award for biopharmaceutical industry research in Vaccine development
Presented to individuals or team from a biopharmaceutical company for outstanding research in the area of vaccine
development.
2013 award presented to: GlaxoSmithKline Malaria Vaccine Team
the research & hope award for patient and community health
Presented to an individual or organization that has had a significant impact educating parents, health care providers or
students about the importance of childhood immunizations and facilitating increased vaccinations in their community.
2013 award presented to: Linda Y. Fu, M.D., Children’s National Medical Center
To learn more about the dedicated researchers honored with the 2013 Research & Hope Awards, please
visit www.phrma.org/awards.
Key issues
“More than two-thirds of new vaccines
developed in the last 25 years have been
developed from the United States.”
Source: PhRMA 2013 Vaccine Fact Book
Medicines in Development Vaccines 20134
broadening protection against shingles
• Shingles is caused by the same virus (varicella-zoster virus) that causes chickenpox in childhood. In
patients that develop shingles, it can lay dormant in certain nerves for years and flare-up in adults
causing severe pain and blisters that can last for a very long time. Shingles tends to affect older people
and those with weakened immune systems. A vaccine approved in 2006 for people age 60 and older
was recently approved to include people 50-59 years of age. About 200,000 people between the ages of
50-59 get shingles each year.
protecting newborns from pertussis
• Pertussis (whooping cough) is a very contagious bacterial disease and one of the most commonly
occurring vaccine-preventable diseases in the United States. In recent years, reported pertussis cases
have been on the rise—there were more than 40,000 reported cases in 2012—and the Advisory
Committee on Immunization Practices (ACIP) now recommends that all pregnant women receive the
Tdap vaccine (tetanus toxoid, reduced diphtheria toxoid, and acellular pertussis vaccine). Vaccinating
women during pregnancy will stimulate maternal anti-pertussis antibodies that should pass to the
newborn providing protection against pertussis until they are old enough to be vaccinated themselves.
Prior to this recommendation, only pregnant women who never received the Tdap vaccine received the
vaccine.
Key issues
Recent successes in Vaccine Use
economies by reducing health care costs and avoiding lost pro-
ductivity. Preventative vaccines are given to individuals, but see
their greatest benefit when entire populations are immunized.
When a high level of vaccination is achieved in a community
with an effective vaccine, disease transmission can be success-
fully interrupted. When disease transmission is interrupted,
even those who did not receive the vaccine, or those who did
not receive immunity from the vaccine, benefit from vaccination
and will be protected from the disease. This is known as herd
immunity—where sufficient immunization coverage prevents
the transmission of the disease to an otherwise susceptible
population. This is especially important for the young, the
elderly and those with compromised immune systems.
Because vaccines are given to healthy people, including chil-
dren, there can be concerns over the possible risks associated
with them. While some negative reactions after vaccination
can be attributable to the vaccine itself, many are unrelated.
Numerous research studies have shown that the risk of not
immunizing a community against certain communicable dis-
eases is far greater than the perceived risk of the vaccination.
Vaccines are highly studied before they are used in the gen-
eral population and highly regulated after approval. Biophar-
maceutical researchers are focused on continuing to improve
the safety and delivery of vaccines for healthy communities.
Today, there are 204 active clinical trials for vaccines, in-
cluding 107 that have not yet started recruiting patients or
are just now seeking volunteers to participate and another
97 that are active, but not recruiting new patients. Those
interested in obtaining more information about these vaccine
clinical trials, go to www.clinicaltrials.gov, the clinical tests
database of the National Institutes of Health.
scientific Advances in Vaccine
Development
Using promising new scientific approaches, researchers are
building on the successful history of vaccination against
infectious diseases. For example, advances in areas such as
genomics are enabling researchers to develop therapeutic
vaccines including immunotherapies for some types of cancer
and other diseases and conditions. In addition, vaccines today
are not limited to injectables—new delivery methods include
nasal sprays, powders and transdermal applications, among
others. Some recent examples of scientific advances include:
Therapeutic cancer Vaccines
There are varied approaches to creating a vaccine to treat
cancer—they can boost the body’s immune response to fight
Medicines in Development Vaccines 2013 5
Key issues
prevention
is better than a cure!
VACCINATION
polio
measles
rubella
pneumonia
cancer
hepatitis B
hepatitis A
smallpoxERADICATED
TARGETED FOR ELIMINATION
TARGETED FOR ELIMINATION
TARGETED FOR ELIMINATION
NOW PREVENTABLE
caused by viruses NOW PREVENTABLE
NOW PREVENTABLE
NOW PREVENTABLE
Source: PhRMA 2013 Vaccine Fact Book
cancer, they can stop the cancer from growing or slow its
growth, and can assist the immune system to locate and kill
the cancer. Two immunotherapy vaccines have been recently
approved by the Food and Drug Administration, one for the
treatment of prostate cancer and the other for melanoma.
Unraveling the Mystery of Deadly Bacteria
Scientists have identified the reason some humans are par-
ticularly susceptible to infection by the bacterium Staphylo-
coccus aureus or staph—which kills about 100,000 Ameri-
cans each year. Researchers at Vanderbilt University recently
reported that genetic variations in human hemoglobin can
make certain people resistant to staph infections.
Vaccine combinations to Prevent and
Treat HiV infection
Recent research provides insight into how immune response
provides protection against HIV infection. In a preclinical
study, novel vaccine combinations were found to provide
partial protection against HIV infection and some combina-
tions substantially reduced the viral load after infection.
New Methods for Vaccine Development
Live recombinant vaccines use an attenuated (weakened) virus
from one disease as the delivery vehicle for another infec-
tious disease. This approach is used to enhance the immune
response or is used when the virus being treated (such as
HIV) is too potent and would actually cause the disease.
DNA vaccines consist of DNA that is coded for a spe-
cific antigen—a protein to which the immune system will
respond. The DNA vaccine is injected into the body where
it produces the antigen and elicits an immune response
against the antigen.
New Delivery Methods
Researchers are running preclinical studies looking at
using a stomach bacterium to deliver vaccines through
food, like yogurt or drinkable liquids. Harmful genes are
removed from the bacterium and replaced with those
from the virus, such as influenza, to stimulate an immune
response in the individual.
Other new delivery methods include inhalable powders,
nasal sprays, a transdermal patch that contains several
microscopic needles that delivers the vaccine instead of
a syringe; and a vaccine that is not rendered ineffective if
stored at warmer temperatures, making it useful in areas
where cold storage is limited.
egg-free influenza Vaccines
Historically, vaccines against influenza were grown in fertilized
chicken eggs, leading to long development times, which make
it difficult to respond to public health emergencies such as the
“swine flu” pandemic in 2009 and ensure enough supply of
the seasonal flu vaccine each year. Egg-free vaccines are also
important in that they provide a viable treatment option to
those with egg allergies.
In 2012, the first cell-culture-derived vaccine was approved
to protect against seasonal influenza in adults. It is manufac-
tured using full-scale cell-culture technology, an alternative
to traditional egg-based production. Cell-culture technology
uses a mammalian cell line rather than chicken eggs to grow
virus strains.
This year the first 100 percent egg-free influenza vaccine was
approved for the prevention of influenza in adults. It is a novel
protein recombinant vaccine for the prevention of seasonal
influenza disease and is the first to be made in a 100 percent
egg-free system without growing influenza viruses, so it can
be made quickly and without the risk of infections.
Medicines in Development Vaccines 20136
Overview
• The current U.S. vaccination schedule for children be-
tween birth and age 6 recommends immunizations for
14 different diseases. Each disease for which vaccina-
tions are recommended can cause serious illness or death
in unvaccinated populations, and those diseases might
quickly begin to appear again if vaccination rates drop.
The United States has seen mumps outbreaks in recent
years since vaccination rates have dropped, with severe
complications and hospitalizations required for some
patients. And before the introduction of the Hib (Hae-
mophilus influenzae type b) vaccine, Hib meningitis
affected more than 12,000 American children annually,
killing 600 and leaving many others with seizures, deaf-
ness, and developmental disabilities. After introduction of
the vaccine, the number of deaths from Hib dropped to
fewer than 10 per year.
• Most vaccines prove to be highly effective. After receiving
the second dose of the MMR vaccine (measles, mumps
and rubella) or the stand-alone measles vaccine, 99.7
percent of vaccinated individuals are immune to measles.
The inactivated polio vaccine offers 99 percent effective-
ness after three doses. The varicella (chickenpox) vaccine
is between 85 percent and 90 percent effective in pre-
venting all varicella infections, but 100 percent effective in
preventing moderate and severe chicken pox.
• Measles infection causes encephalitis (inflammation of
the brain) for one in 1,000 infected individuals. Over-
all, measles infection kills two of every 1,000 infected
individuals. In contrast, the combination MMR (measles,
mumps and rubella) vaccine results in a severe allergic
reaction only once in every million vaccinated individu-
als, while preventing measles infection. The benefits of
vaccine-acquired immunity extraordinarily outweigh the
serious risks of infection.
• Before the varicella vaccine became available, chickenpox
infections resulted in 10,000 hospitalizations and caused
more than 100 deaths each year in the United States.
Even uncomplicated cases of chickenpox cause children to
miss a week or more of school, with a caregiver missing
work to care for the sick child.
Diseases Covered by Vaccines in the United States
• Diphtheria once was a major cause of illness and death
among children. The United States recorded 206,000
cases of diphtheria in 1921, resulting in 15,520 deaths (a
case-fatality ratio of 7.5 percent). Diphtheria death rates
range from about 20 percent for those under age
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