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Medicines in Development
Biologics
presented by america’s biopharmaceutical research companies
Overview
Biologics In Development Biologics Research Promises to
By Product Category and
Development Phase Bolster the Future of Medicine
America’s biopharmaceutical research Antisense drugs interfere with the commu-
companies are using biological process- nication process that tells a cell to produce
338
es to develop 907 medicines and vac- an unwanted protein. Vaccines, particularly,
cines targeting more than 100 diseases. therapeutic cancer vaccines, are another
Application Many biologics are made from a variety example of how the immune system is
Submitted
of natural sources—human, animal or being harnessed to fight disease.
Phase III microorganisms. Like small-molecule The biologics now in development make
drugs, some biologics are intended to use of a range of new technologies to
Phase II
treat diseases and medical conditions. harness scientific progress. For example,
Phase I
Other biologics are used to prevent or among the medicines in the pipeline are:
diagnose disease. Examples of biological
250 products include but are not limited to: • A genetically-modified virus-based
vaccine to treat melanoma.
• monoclonal antibodies
•A
monoclonal antibody for the treat-
• vaccines, including therapeutic ment of asthma.
vaccines
•A
n antisense therapy for the treatment
• blood and blood products for transfu- of leukemia.
sion and/or manufacturing into other
products •A
recombinant fusion protein to treat
type 2 diabetes.
• gene therapies
For more information on these products
• cell therapies and others, please see page 4 and our
The medicines discussed in this report accompanying backgrounder.
are either in human clinical trials or The 907 biologics in development
under review by the U.S. Food and Drug promise to push the frontiers of science
Administration (FDA). and bring new treatments to patients for
93
These medicines often represent cut- our most challenging diseases. See our
81
ting-edge research in which the latest report for a full list of the medicines and
69 scientific discoveries are translated vaccines in development.
into novel therapies that provide new This overview discusses the science
46 treatment options for patients. Increased behind biologics, potential medicines of
understanding of the molecular and the future, the need for continued invest-
30 genetic bases of disease has opened up ment in research and development, and
the development of a range of targeted the policies and regulations that help
treatments. For instance, monoclonal make such innovation possible.
antibodies (mAbs) are proteins that help
the immune system identify and bind
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The Human Genome Advancing Biomedical Science
Inside every human body are about 25,000 genes—each Over the past decade, a wave of scientific advances and new
responsible for a specific protein. A person’s genes tell their technologies have dramatically changed how medicines are
body to produce all the enzymes, hormones, antibodies and discovered. Greater knowledge of how diseases work at the
other proteins needed to make the body function. If one of genetic and molecular level has allowed researchers to pursue
the genes is missing or defective, the body will not have the new targets for therapy and better predict how certain bio-
proteins it needs to function properly and may have proteins pharmaceuticals will affect specific subpopulations of patients.
that actually cause disease. The modern tools of biotechnol-
ogy—coupled with computer technology’s ability to analyze • Bioinformatics—Bioinformatics use systems and mathe-
massive amounts of data quickly—help biopharmaceutical matical models to advance the scientific understanding of
scientists determine which genes or proteins are defective living systems. At its simplest level, bioinformatics involves
and are being used to develop new treatments across a range the creation and maintenance of biological databases,
of therapeutic areas. including DNA sequences. Bioinformatics also includes
calculation tools. These tools can decipher the molecular
pathways of disease, find patterns in the way genes re-
Manufacturing Complexities spond to drugs, interpret the three-dimensional structure
Most biologics are very complex molecules and cannot be of important proteins, and enable the computer-aided
fully characterized by existing science. For this reason, they design of new drugs.
often are characterized by their manufacturing processes.
• B
iomarkers—Every disease leaves a signature of molecular
Given the complexity of many biologics, the manufacturing “biomarkers” in our body—genes that turn on and off or
processes are likewise complex and very sensitive. Slight proteins released into the bloodstream. Biomarkers mea-
changes in temperature or other factors can impact the final sured in blood and other samples can tell us the state of
product and affect how it works in patients. Changes in the our health and how we might respond to treatment. They
manufacturing process or facility may require clinical studies are powerful tools that can detect certain diseases at their
to demonstrate safety, purity and potency. earliest stages before symptoms appear, when they are
most treatable. The identification of biomarkers is the first
step in developing a personalized medicine.
• M
olecular Targeting—The idea behind molecular targeting
is to design drugs that specifically attack the molecular
pathways that cause disease, without disrupting the nor-
mal functions in our cells and tissues.
• N
anotechnology—You can’t see it, but soon it will be every-
where. Nanotechnology is the science of building microscop-
ic devices at the molecular and atomic levels. In medicine,
biologic molecule
l aspirin molecule nanotechnology may also be used to help diagnose and
treat diseases. For example, tiny gold-coated “nanoshells”
could act like smart bombs, zeroing in on a tumor, entering
Monoclonal Antibodies: cancer cells, and lying in wait until an infrared beam or ra-
Targeted Cancer Therapy dio wave signals the particles to release an intense, deadly
dose of heat energy that destroys the cancer cells.
An approved monoclonal antibody (mAb) for the
treatment of cancer targets the epidermal growth • P
ersonalized Medicine—The sequencing of the human
factor (EGFR) that is linked to the growth and genome produced a “map” of the human genes in DNA.
development of many types of cancer. Originally This new genetic knowledge opens up the possibility of
the mAb was approved for the treatment of EGFR- developing “targeted” therapies for people with specific
expressing metastatic colorectal cancer. Additional gene sequences, and it can help physicians choose the
studies have found that the presence or absence of best treatments based on individual genetic, lifestyle
a certain gene mutation could predict the patients’ and environmental factors. Additionally, researchers are
response to treatment. Patients without the gene developing genetic tests that can tell if we are susceptible
mutation—about 65% of patients—are most likely to to certain diseases.
benefit from treatment.
2013 Report 3
Medicines in the Future MAbs were first explored as a therapeutic option as a result of
scientific breakthroughs that occurred in the mid-1970s and
Building on the impressive progress to date, the 907 medi-
early-1980s. By 2013, a total of 33 mAbs were approved in the
cines and vaccines listed in the PhRMA report, Biologic
United States. Because mAbs allow targeting of unhealthy
Medicines in Development, represent the next exciting
cells without harm to healthy cells, they have been particu-
frontier of biopharmaceutical research.
larly important in fighting cancer, and more recently, show
The report finds that the greatest amount of research is in great promise for autoimmune diseases, such as rheumatoid
monoclonal antibodies (mAbs), with 338 separate mAbs in arthritis. Vaccines have historically been used as a preventa-
development, and vaccines, with 250 vaccines in clinical tive tool in infectious diseases, such as pneumonia, HIV and
trials or under review at FDA. smallpox. But today, vaccines are also being used as therapies
for cancer and other diseases.
Antisense - 5 Antisense
Cell
therapy - 1
• A third-generation antisense medicine in development
Gene
therapy - 3 for the treatment of lymphoma inhibits production of a
specific protein which regulates many key genes import-
Monoclonal
antibodies - 22 ant in cancer growth—angiogenesis, cell metabolism,
cell proliferation, cell death and cell invasion. An overex-
pression of the protein in tumors results in resistance to
Vaccines - 134 Recombinant treatment. By reducing the amount of the protein in can-
proteins – 2
cer cells, the antisense medicine may be able to enhance
the effectiveness of current anticancer treatment.
Autoimmune Disorders 71
Blood Disorders 43
Cancer/Related Conditions 338
Cardiovascular Disease 58
Diabetes/Related Conditions 28
Digestive Disorders 26
Eye Conditions 25
Genetic Disorders 30
Infectious Diseases 176
Musculoskeletal Disorders 34
Neurologic Disorders 39
Respiratory Disorders 38
Skin Diseases 30
Transplantation 13
Other 58
2013 Report 5
Encouraging Continued
Biopharmaceutical Innovation The Future of Research Should Be
Protected by Adequate Incentives for
The development of new biologics is a long, complex and
Innovation; 12-Years of Data Protection is
costly endeavor. It takes about 10–15 years, on average, to
Critical for Patients
bring a medicine through the discovery and clinical trial
phases to patients, and the average R&D investment for each A biosimilar is similar to—but not the same as—an
new medicine is $1.2 billion, including the cost of failures. innovator biologic. A pathway for the approval of
biosimilars in the United States was included in the
Another report on medicines in the pipeline found that more
Patient Protection and Affordable Care Act of 2010.
than 5,000 potential new medicines—which may become
The pathway, which received broad bipartisan
available to U.S. patients—are in the pipeline globally—in
support in both chambers of Congress, struck an
large part funded by the more than $500 billion invested
appropriate balance between promoting increased
in research and development (R&D) since 2000 by PhRMA
competition and providing adequate incentives to
member companies. These promising candidates build on
support continued innovation of new treatments and
the more than 300 medicines that have been approved by
cures through a 12-year period of data protection.
the FDA in the last decade.
This is critical to spurring the investment in research
In order to realize the full potential of biologics, it is essential and development needed to seize the extraordinary
that the United States maintain strong policy and regulatory opportunities for medical advances against our most
environments that help foster the discovery and develop- costly and challenging diseases and the resulting
ment processes. jobs supported across the U.S. economy.
2013 Report 7
Pharmaceutical Research
Pharmaceutical and Manufacturers
Research of America
and Manufacturers of America
950 F Street, NW, Washington,
950 F Street, DC 20004
NW, Washington, DC 20004
www.phrma.org
www.phrma.org