Transcript Molecular Biology Background of Biotechnology in Public

Biopharmaceuticals and
agrobioproducts:
a North – South conflict?
November 16-17, 2006, Warsaw
The lecture:
new
inventive
commercial
IPR in BIOTECHNOLOGY
means:
CONNECTION of science, technology and industry;
 Globalisation;
 Innovations, property and profit;
 Highly skilled researchers and workers in
industry;
 Productivity, growth and structural changes;
 Information and communication policy.

SECTORS in ECONOMY
Agriculture
 Industry
 Health

=
=
=
green biotech
white biotech
red biotech
violet
=
legislation (IPR)
 In summary:
Contribution of biotechnology to the entire

BIOeconomy
VAC
 value added chain
=
 science & technology
+
 law & IPR
+
 society & perception

Aims of genetic engineering:
identification of
specific property as a
gene;
 transfer and/or
modification of a
gene;
 regeneration of a
modified organism.

commercialisation
[legislation,
biosafety, public
perception]
Bioproducts in national and global
economy:
1. Biomedicine
–
–
–
–
Biopharmaceuticals,
Molecular markers,
Targeted therapies,
Diagnosis.
2. Food & feed
– Functional food
– Nutraceutics
3. Plants for industry
– biomaterials
– bioenergy
– bioreactors
Biotechnology is green:


Essential for sustainable future of our society:
Renewable resources
(“green” bio-resources: biomass, bioenergy, biomaterials)

Clean production [recycling is possible]

The transition from ChemistryBio-based chemistry
one of the essential leads for the transition to renewable
bio-resources
opportunities

Genomics and modern (Bio)Technology offers
excellent tools for major improvements and
innovations for the transition of our strong
petro-based chemistry to
bio-based chemistry

The use of bio-waste (“biomass”) offers a
way of turning environmental burden into
environmental advantage

A report by the USDA national statistics service states that the U.S. has
increased the acreage of soybean, corn, and cotton this year. Biotech
soybean was the most popular choice amongst farmers, accounting
for 89% of the total of all soybeans grown in the U.S. The statistics
released include:
* 66.6 million acres of biotech soy were planted in ’06, up from 62.76
million in 2005.
* 12.6 million acres of biotech cotton were planted, accounting for
83% of all cotton grown.
* 48.4 million acres of biotech corn were planted, accounting for 61%
of all corn planted, and 6 million acres more than in 2005.
Additional information is available at:
http://usda.mannlib.cornell.edu/usda/nass/Acre//2000s/2006/Acre-06-30-2006 .pdf
These can be converted to any
desired product(s)
Shopping List
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Fruit pie
Biscuits
Bread rolls
Chicken soup (tinned) (for vegetarians: vegetable soup)
Chocolate bar
Cornflakes
Crisps
Margarine
Mayonnaise
Pasta
Ready meal Asian food
Readily prepared schnitzel coated with breadcrumbs
(for vegetarians: corn burger)
Spaghetti sauce (instant)
Sandwich spread
Vanilla ice cream
biopharmaceuticals
& biomarket trends

Market: 2005 - 602 bln USD
(+7,7% from 2004)
– 2010 - 842 bln USD
Participation in the market:
North America + EU + Japan = 82,3%
Generics:
USA
40%
Germany
60%
Poland
90%
New biopharmaceuticals:
nervous system + cardiovascular + metabolism
therapy  HORMONES
The leader in biotechnology: AMGEN
(12,5 bln USD 2005)
12000
rok 2003 (31,5 mld USD)
10000
rok 2010 (52,9 mld USD)
mln USD
8000
6000
4000
2000
0
EPO
IFN
INS
CSF
BF
HT
F/PI
ENZ
IL
PA
GF
Fig. 5. 2003 sales value and 2010 sales forecast of individual
biopharmaceuticals on global market of recombinant proteins [37]
(EPO - erythropoietin; IFN - interferons; INS - insulin; CSF – colony stimulating
factors; BF – blood coagulation factors; HT – hormone therapeutics; F/PI – fusion
proteins/protein inhibitors; ENZ - enzymes; IL - interleukins; PA – tissue plasminogen
activators; GF – growth factors)
Acc. P. Borowicz, M. Jarecka
Biotechnologia 2005
Bioeconomy - how to describe it?
 The question:
What are the basic indicators of biotech
contribution to productivity and overall
economy?
 The answer:
Patents; but: internationally comparable
indicators are not available
Track to receive the
biotechnological invention
Track to receive the
biotechnological invention
discovery
Track to receive the
biotechnological invention
discovery
aim
Track to receive the
biotechnological invention
discovery
aim
invention
patent
Track to receive the
biotechnological invention
discovery
aim
invention
technology
Track to receive the
biotechnological invention
discovery
aim
inven- technology
tion
optimalization
Track to receive the
biotechnological invention
discovery
aim
invention
technology
optimalization
preclinical
tests
(animals)
In case of medicament
Track to receive the
biotechnological invention
discovery
aim
invention
technology
optimalization
preclinical clinical
tests
tests
(animals) I, II, III
In case of medicament
Track to receive the
biotechnological invention
discovery
aim
invention
technology
optimalization
preclinical clinical
Registests
tests
(animals) I, II, III tration
In case of medicament
Track to receive the biotechnological invention
– inculding costs
discovery
aim
invention
5%
technology
optimalization
preclinical
clinical
tests
Registests
(animals)
I, II, III tration
25%
70%
In case of medicament
Scientific Risk
of Research Activities
Discovery and Development of a Successful Medicine
YEARS
15
14
13
12
11
10
9
8
7
6
5
4
3
2
1
0
PHASES
INTRODUCTION/
REGISTRATION
DEVELOPMENT
POST-MARKETING SURVEILLANCE
1
2
2-5
III
CLINICAL TEST (HUMANS)
10 - 20
II
I
5 - 10
BASIC
RESEARCH
IV
PRECLINICAL TEST (ANIMALS)
3,000 - 10,000
QUANTITY OF SUBSTANCES
Source: Based on PhRMA analysis, updated for data per Tufts Center for the Study of Drug Development (CSDD) database
SYNTHESIS,
EXAMINATION &
SCREENING
Patent protection strategy
2. Method of
preparation
for example
after 10 years
Pharmaceutical form
(for example oral),
after 20 years
- II medical indication
compound A
method of preparation
pharmaceutical composition
I medical indication
Pharmaceutical form - compositions
(for example iniection),- pharmaceutical products
- forms
for example
after 15 years
next preparation
methods
New
pharmaceuticals and generics
Pharmaceutical
industry
NEW [BIO]
PHARMACEUTICALS
GENERICS
Patents
SPC
Waiting for patent
expiration
EXCLUSIVITY
COMPETITION
Suplementary Protection Certificate (SPC)
 for pharmaceuticals and plant protection
products
• these regulations allow the owners of patents
involving innovative drugs and plant
protection products to extend the patent
protection for even up to five years.
Bolar Amendment in CE
The Bolar Amendment permits generic firms to
make use of originating firms’ otherwise
protected clinical data before the relevant patent
have expired.
 In contrast, regulatory regimes and enabling
legislations in many other developed nations are
complex and tend to inhibit the rapid marketing of
generic drugs.

Consequence: unfair commercial use

Generic drug registration is used/relied on data of
original drug before the relevant patent has expired.
PHARMACEUTICALS
originals
generics
Submit undisclosed
(safety and efficacy data)
Submit bioequivalent studies
Aspects:










the balance between
[costs – losses – profits];
transfer of knowledge
[North to South???, one-way direction]
fostering innovation;
preservation of national heritage and biodiversity;
regional specificity;
local needs for highly qualified staff;
functioning of current licensing practice;
cost of licensing vs price of patenting;
availability of licensing;
blocking [false] patents.
INNOVATION
-specific parameters vs.
value of innovation
Priority,
 Affordability,
 Quality,
 Sustainability,
 Uncertainty,
 Monopoly rights,
 Stimulation of research,
 Avoiding duplication.

IPR actors:










[1]
Private sector
Public sector
Finance and banking
Busines & economy
Science & research & development
Research at academia & industry
Industry at agriculture, pharmacy, food and feed, etc.
Management
Legislation
Non-governmental organisation
IPR actors
[2]
 Scientists
 Managers
 Business people
 Greens
 Lay people
INVENTION
Patents:
 new
 inventive
 commercial
Art. 10 Art. 24 p.w.p (Poland)
Art. 52.1 EPC
in biotech:
a) product of biology,
b) containing biologics,
c) biological method.
Generics
 Essential similarity
 Biosimilar (but not identical)
 the same chemical formula (?)
 the same bioavailability (?)
 the same bioeffect (?)
 simplified registration procedure
 low cost
Different Levels of Added Value
Research-based Industry
Employment
Trade balance
Research
560,000 (incl. 88,000 R&D) in 2002
- 114,800 within German Market
€28,000 million in 2001 (up from €5,200
million in 1985)
- 35 NCE (New Chemical Entites) global
launched in 2000 (5 global annual
„blockbusters”)
- 36 NCE (New Chemical Entites) global
launched in 2001
Development
costs
Development
time
Development
risk
Generics
manufacturer
85,000 (mostly CEE)
? (negative)
no discovery or
advanced
€895 millions and growing
less than €1 million
10-13 years
2-3 years
significant: one in 5-10,000
zero risk, pick most
successful
Protein structure
1. Amino acid sequence
2. Linear
3. Three dimensional
4. Interactions
Interferon Beta
MW 19'000D
Denaturation and
Breakdown
Oligonucleotides complementary to
L-rRNA
To differentiate
Biosimilars and Generics
•Manufacturing Process;
•Molecular Properties;
•Safety;
•Efficacy
Safety Considerations
For Biotech Medicines, each product has a unique
safety profile dependent on its:
• Mechanism of action
• Unique manufacturing process
• Composition (by-products and impurities)
Aspects of novelty [1]:
 Research
 Commercialization
 Simplicity covered by “omics and logics”
 Monopoly
vs public goods
 Biodiversity vs monoculture of agriculture
Aspects of novelty [2]; solutions:
 Patent pool [collaborative mechanism];
 Clearing-house mechanism;
 Fostering collaboration.
 The necessary factors to be included:
technical, business, financial,
legal, public perception,
anti-trust concerns.
Patent pool
[collaborative mechanism]



The term patent pool is commonly used to refer to a joint patent licensing
program that consists of a number of companies who have decided to offer a
joint license for their patents that are essential to the practice of a defined
specification.
An Essential Patent is a patent that contains one or more
claims that are unavoidably and necessarily infringed by
the implementation of a specification for which it is
essential.
Competition authorities have indicated
that the pro-competitive benefits of patent pools-of allowing a
broader group of companies to have access to Essential Patents
efficiently through a joint licensing program-may be diluted by
including patents that are not Essential Patents.
www.oecd.org
Clearing-house mechanism

Expertise in managing information and technology varies
enormously from country to country. For this reason, the Convention on Biological Diversity [in
1992] has established a "Clearing-House Mechanism" to ensure that all governments have access
to the information and technologies they need for their work on biodiversity.
The network of national focal points for the mechanism is
being established to address matters relating to technical and scientific
cooperation. The Parties have recently emphasized the need to strengthen the
role of these focal points. Building a network of non-governmental
organizations and other institutions working on biodiversity could
contribute to this goal. Establishing National, Regional, Subregional and
Thematic Clearing-House Focal Points for specific topics could also help.
The clearing-house is based on the philosophy that broad participation
and easy access must be a top priority. Its database can therefore be tapped
through both traditional and electronic means of communication. Special
efforts are made to ensure the participation of indigenous communities,
whose unique knowledge and expertise are so important.
The Clearing-House is coordinated by the Executive Secretary and
overseen and guided by an Informal Advisory Committee (IAC) set up by the
Parties to the Convention.
www.oecd.org
Foster collaboration

Series of laws use patent ownership to foster
collaboration between parties in the research
and development enterprise. Patents protect the
inventor’s investments in generating the
knowledge that is the basis for innovation and
serve as an incentive to the commercialization of
new ideas. As R&D has become more expensive,
ownership of title to inventions has been used as
a means to encourage collaborative work among
different players in the research enterprise.
[Wendy H. Schacht]
Problems
example: patent trolls
 Patent Act:
... The right to exclude others ... from making,
using, offering for sale, or selling the
invention ...
 Patent trolls:
patent holders who do not practice the
invention but instead use patents primarily
for obtaining license fees.
Quo vadis biotechnologiae?
Differentiation:
North
vs.
 Rich
vs.
 Developed
vs.
 Members
vs.
 Technology reach
 Science based
 Modern drugs
 Home cooking

South
poor
undeveloped
non-members
vs. biodiversity reach
vs. common knowledge
vs. traditional medicine
vs. nutraceutics
SOLUTION:



Shall we give the fish?
or
shall we teach the
fishing?
=
a [gift of ] modern
products
or
access to the technology

Teaching the fishing:
how to do it?,
who should do it?
–=
• More questions
than answers.
Intellectual Property Blacklist –
„Watch LIST” of the United States Trade
Representantive (USTR)
Intellectual property rights legislation is issued
at the national level, but World Trade Organization
member states must adhere to the requirements
of the WTO Agreement on Trade-Related Aspects
of Intellectual Property Rights (TRIPS).
 The 2005 ”Special 301 Report” examines in detail
the adequacy and effectiveness of IPR protection
in 90 countries.

Watch LIST
Poland
- some progress in improving IPR protection;
- deficiencies in patent protection for pharmaceuticals, especially
lack of coordination between the Health Ministry and the Polish
Patent agency that would prevent the registration of unauthorised
patent-infringing products.
Hungary
- has made improvements to its IPR regime in the past years;
- does not provide any coordination between its health regulatory
agency and its patent authority to prevent the marketing
registrations of patent-infringing products.
Slovakia
-does not provide a coordination mechanism between its health
regulatory agency and its patent authority to prevent the registration
of unauthorised patent-infringing products
- Slovakia continues to store sensitive registration data on the
premises of a generic drug manufacture.
Europe and piracy
• Product piracy is responsible for
– 70.000 working places less each year
– Damage to the German economy alone: € 20 – 25 bln each
year
– € 500 bln yearly turnover in fakes (10% of world trade)
• Role of Customs Authorities:
– fight against counterfeiting
– performing the best possible controls and goods
– help prevent consumption of unsafe products
Source: Wirtschafts Woche, Nr. 27, June 30.2005
Prices of generics in 2003
2,5
2,2
Kilograms
2
1,5
1,1
1
1
0,7
0,5
0
Prices for generics in
EU15 are 15-50% higher
than in the US
 In Japan – prices of
generics are more
than double the U.S.
prices
 in Poland – prices are
lower than in the US

DE
PL
J
US
Source: U.S. Department of Commerce calculations based on data from
IMS Health, IMS MIDAS ™, Q4/2003 and the Center for Medicare
and medicaid Services (CMS)]
Utilization of generic drugs in 2003 as a
percentage of total consumption
90
80
70
%
86
85,6
PL
US
67,9
60,5
60
50
40
30
20
13
10
0
DE
UK
JP
Source: U.S. Department of Commerce calculations based on data of 29 molecules from IMS Health, IMS MIDAS ™, Q4/2003
Public perception
Patents and patenting = controversial issue.
We need:
Counter-balancing, in terms of information, various forms
of information through international network.
SOLUTION:

Instead of
recognising
differentiation we
should work on
harmonisation and
unification of IPR
system on world
arena.

In the today world of
“global village”
co-operation of all
actors on
international arena
will give all of us the
chance for synergy
effect.
Why Resistance to IP Protection in
Central Europe?
• Tradition of weak protection of property in CE
– good business with lack of IP
• Resistance to change
– survival of pre-existing alliances
– distortion caused by conflict of interest not eliminated
• IP not perceived as opportunity
– fear
– lack of positive experience with IP
• Tradition of low economic freedom
– low tradition of rewarding based on merits
– low tradition of valuing human capital
Developing new products

The ability of innovative industries to continue to
develope new products depends largely upon two
factors:
• a strong and effective intellectual property system
• the capacity to market new products effectively during
the period of time when the exclusive IPR exist.
Without intellectual property:
• no value created,
• no investment,
• no venture capital,
• no trade.
Effective IP Enforcement through
Cooperation
Effective IP enforcement requires sophisticated
interaction
 Cooperation between:

– legal community, academia and scientists and
creators
– patent offices to promote IP and facilitate usage
– venture capitalist, investors and scientists,
creators of IP
– courts, publication and discussion of reasoning
– enforcement authorities – prosecutors, police,
customs authorities
THANK YOU
... I am ready to answer questions.