Biotechnology can be defined as the use of
living organisms (such as bacteria), biological processes or biological systems
to create a desired end-result or end-product.
Primary markets for biotechnology include: 1) Agriculture, where genetically-modified
seeds are now in wide use in many nations.
These seeds deliver plants that have can much higher crop yields per
acre, and often have qualities such as disease-resistance, resistance to
herbicides and drought-resistance.
2)
The manufacture of enzymes, including enzymes used in food processing (such as
the making of certain dairy products) and in converting organic matter into
ethanol for fuel.
3) Pharmaceuticals,
where biotechnology creates such therapies as antibodies, interleukins and
vaccines based on living organisms (as opposed to the chemical compounds that
make up traditional drugs) that can target specific cellular conditions, often
with dramatic results (such as the drug Keytruda that famously fought brain
cancer for former U.S.
President Jimmy Carter).
Biotechnology is a modern word that describes a
very old science.
For example,
bio-enzymes have always been essential in the production of cheese.
The modern difference is that much of the
world's cheese production today utilizes a bio-engineered version of an enzyme
called microbial chymosin.
This chymosin
is made by cloning natural genes into useful bacteria.
Another example: For thousands of years, mankind has used
naturally-occurring microbes to convert fruit juices into wine.
Plunkett Research estimates that combined
biotech revenues for publicly-held
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Biotechnology can be defined as the use of
living organisms (such as bacteria), biological processes or biological systems
to create a desired end-result or end-product.
Primary markets for biotechnology include: 1) Agriculture, where genetically-modified
seeds are now in wide use in many nations.
These seeds deliver plants that have can much higher crop yields per
acre, and often have qualities such as disease-resistance, resistance to
herbicides and drought-resistance. 2)
The manufacture of enzymes, including enzymes used in food processing (such as
the making of certain dairy products) and in converting organic matter into
ethanol for fuel. 3) Pharmaceuticals,
where biotechnology creates such therapies as antibodies, interleukins and
vaccines based on living organisms (as opposed to the chemical compounds that
make up traditional drugs) that can target specific cellular conditions, often
with dramatic results (such as the drug Keytruda that famously fought brain
cancer for former U.S. President Jimmy Carter).
Biotechnology is a modern word that describes a
very old science. For example,
bio-enzymes have always been essential in the production of cheese. The modern difference is that much of the
world’s cheese production today utilizes a bio-engineered version of an enzyme
called microbial chymosin. This chymosin
is made by cloning natural genes into useful bacteria. Another example: For thousands of years, mankind has used
naturally-occurring microbes to convert fruit juices into wine.
Plunkett Research estimates that combined
biotech revenues for publicly-held firms headquartered in the U.S. and E.U.
will be $176 billion during 2018, while the U.S. firms’ portion will be $135
billion. Genetically-engineered drugs,
or “biotech” drugs, represent roughly 11% of the total global prescription
drugs market. The U.S. Centers for
Medicare & Medicaid Services (CMS) forecast called for prescription drug
purchases in the U.S. to total about $344.5 billion during 2018, representing
about $1,050 per capita. That projected
total is up from only $200 billion in 2005 and a mere $40 billion in 1990.
Estimates of the size of the drugs market vary
by source, but it is generally accepted that the global prescription drugs market
was more than $1 trillion in 2017.
Plunkett Research estimates the global pharmaceuticals market at $1.2
trillion for 2019. By 2027, American
drug purchases alone may top $576.7 billion, according to the CMS, thanks to a
rapidly aging U.S. population, increased access to insurance and the continued
introduction of expensive new drugs.
Advanced generations of drugs developed through
biotechnology continually enter the marketplace. The results may be very promising for
patients, as a technology-driven tipping point of medical care is approaching
where drugs that target specific genes and proteins may eventually become
widespread. However, it continues to be
difficult and expensive to introduce a new drug in the U.S.
According to FDA figures, 42 new molecular
entities (NMEs) and new biotech drugs (BLAs) were approved in the U.S. during
2018. These NMEs are novel, new active
substances that are categorized differently from “NDAs” or New Drug
Applications. NDAs may seek approval for
drugs based on combinations of substances that have been approved in the
past. Also, a large number of generic
drug applications are being approved each year.
That is, an application to manufacture a drug that was created as a
brand name, and has now lost its patent so that competing firms may seek FDA
approval to manufacture it.
Dozens of exciting new, biotech drugs that
target specific genes are seeking regulatory approval. Many of these drugs are for the treatment of
specific forms of cancer. In a few
instances, doctors are making treatment decisions based on a patient’s personal
genetic makeup. (This strategy is often
referred to as personalized medicine.)
New breakthroughs in genetically targeted drugs occur regularly. An exciting drug for certain patients who
suffer from the skin cancer known as melanoma is a good example. Zelboraf, developed by drug firms Roche
Holding and Daiichi Sankyo, will dramatically aid melanoma patients who are
shown through genetic tests to have a mutated gene called BRAF. In trials, about 50% of such patients saw
their tumors shrink, compared to only 5.5% of patients who received traditional
chemotherapy.
New Drug Application Categories
Applications for drug approval by the
FDA fall under the following categories:
BLA
(Biologics License Application): An
application for approval of a drug synthesized from living organisms. That is, they are drugs created using
biotechnology. Such drugs are sometimes
referred to as biopharmaceuticals.
NME (New
Molecular Entity):
A new chemical compound that has never before been approved for marketing in
any form in the U.S.
NDA (New
Drug Application):
An application requesting FDA approval, after completion of the all-important
Phase III Clinical Trials, to market a new drug for human use in the U.S. The drug may contain active ingredients that
were previously approved by the FDA.
Biosimilars
(generic biotech drugs): A term used to
describe generic versions of drugs that have been created using
biotechnology. Because biotech drugs
(“biologics”) are made from living cells, a generic version of a drug may not
be biochemically identical to the original branded version of the drug. Consequently, they are described as
“biosimilars” or “follow-on biologics” to set them apart.
In Europe, their manufacture and sale
has been allowed for some time under special guidelines. In February 2012, the FDA created guidelines
for biosimilars in the U.S.
Manufacturers are now able to rely to a large extent on the clinical
trials research previously conducted by the maker of the original version of
the drug.
Priority
Reviews: The FDA places some drug applications that
appear to promise “significant improvements” over existing drugs for priority
approval, with a goal of returning approval within six months.
Accelerated
Approval: A process at the FDA for reducing the
clinical trial length for drugs designed for certain serious or
life-threatening diseases.
Fast
Track Development: An enhanced process for
rapid approval of drugs that treat certain life-threatening or extremely
serious conditions. Fast Track is
independent of Priority Review and Accelerated Approval.
Personal genetic codes are becoming less
expensive and more widely attainable. Today,
the cost of decoding the most important sections of the human genome for an
individual patient has dropped dramatically.
Although total drug expenditures are currently
small in developing nations such as India, China and Brazil, they have
tremendous potential over the mid-term.
This means that major international drug makers will be expanding their
presence in these nations. However, it
also means that local drug manufacturers have tremendous incentive to invest in
domestic research and marketing.
The Coming BioIndustrial Era:
Some of the most exciting developments
in the world of technology today are occurring in the biotech sector. These include advances in agricultural
biotechnology, the convergence of nanotechnology and information technology
with biotech and breakthroughs in synthetic biotechnology.
The rapidly growing worldwide base of
biotechnology knowledge has the potential to create a new “bioindustrial
era.” For example, scientists’ ability
to capture refinable-oils from algae and other organisms (organisms that remove
carbon from the atmosphere as they grow) may eventually create a new source of
transportation fuel. Oil industry giant
ExxonMobil has backed research in this regard at Synthetic Genomics, Inc. with
hundreds of millions of dollars.
The use of enzymes in industrial
processes may enable us to bio-engineer a long list of highly desirable
substances at modest cost. The result
could easily be a lower carbon footprint for many industrial processes, less
industrial and residential waste to deal with and a significant increase in
yields in chemicals, coatings, food and other vital sectors. DuPont’s acquisition of global enzyme leader
Danisco is a good indicator of the looming era of bioindustrial advancements,
as DuPont made a $5.8 billion bet that it can help a vast variety of manufacturers
to achieve significant product enhancements and efficiencies.
Source: Plunkett Research, Ltd.
Significant ethical issues face the biotech
industry as it moves forward. They
include, for example, the ability to determine an individual’s likelihood to
develop a disease in the future, based on his or her genetic makeup today; the
potential to harvest replacement organs and tissues from animals or from cloned
human genetic material; and the ability to genetically alter the basic foods
that we eat. These are only a handful of
the powers of biotechnology that must be dealt with by society. Watch for intense, impassioned discussion of
such issues and a raft of governmental regulation as new technologies and
therapies emerge.
The biggest single issue may be privacy. Who should have access to your personal
genetic records? Where should they be
stored? How should they be accessed? Can you be denied employment or insurance
coverage due to your genetic makeup?
Global Factors Boosting Biotech Today:
1)
A
rapid aging of the population in nations including the EU, much of Asia and the
U.S., such as 70 million surviving Baby Boomers in America who are entering
senior years in rising numbers and require a growing level of health care. A significant portion of that care may be in
the form of biotech drugs.
2)
A
renewed, global focus on producing and stockpiling effective vaccines.
3)
Major
pharmaceuticals firms paying top prices to acquire young biotech drug companies
that own promising drugs.
4)
A
very significant market for genetically-engineered agricultural seeds
(“Agribio”), with farmers in dozens of nations planting genetically modified
seeds.
5)
Aggressive
investment in biotechnology research in Singapore, China and India, often with
government sponsorship—for example, Singapore’s massive Biopolis project.
6)
Very
promising research into synthetic biology.
7)
Dramatic
decreases in the cost of personal genetic studies, which can be a big boost to
personalized medicine.
8)
Highly
advanced biotech technologies known as gene therapies are slowly beginning to
prove their ability to cure patients.
9)
Rapid
growth in the overall prescription drug markets in developing nations.
10)
An
increased focus on the discovery and manufacture of new drugs (“orphan drugs”) that
impact rare diseases or relatively small portions of the population.
11)
The
advent of the genetic engineering process known as CRISPR, enabling scientists
to alter cells and repair defective cells.
This has profound potential in both human and animal health therapies.
12)
CRISPR
also has major potential to modify agricultural seeds and plants through gene
editing.
Source: Plunkett
Research, Ltd.
Internet Research Tip:
For the latest biotech developments
check out www.biospace.com , a private
sector portal for the biotech community, and www.bio.org ,
the web site of the highly regarded Biotechnology Industry Organization.