Potential Area of European Leadership in Medicines Development

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Potential Area of European Leadership in
Medicines Development The Biotechnology
case Johan Vanhemelrijck EuropaBio EFGCP Annual
Conference Brussels, 1 February 2006
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Biotechnology ? A Creative technology based on
using the New Knowledge of Life Sciences to
Research, Develop and Use new services and
products across all sectors Therapies,
Agriculture and Food, Diagnostics,
Forensics, Inspections, Chemicals, Energy,
Textile
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Biotechnology The Invisible Revolution
Corn is corn Soya is soya
BioPlastic is plastic BioDiesel is diesel.

• Products are products whatever the production
  technology.

Medicines are medicines
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US Versus EU
-- 1976 Companies -- 94000 employees - 35000 in
RD -- 6 billion in RD -- 750 million in
Venture Capital -- 1 billion in debt financing
2003 figures .
-- 1830 Companies -- 172,400 employees -- 16.4
billion in RD -- 2.1 billion in Venture
Capital -- 4.3 billion in debt financing
Source Critical I
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Arts and skills of the bio-entrepreneurMinimize
entrepreneurial risk, maximize societal value
Political risk or 4th Hurdle
Research risks
IPR risks
VALUE
Financial risks
Regulatory risks
Market risks
RISK
TIME LINE
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Expected Results

• Biotechnology to be the basis for the knowledge
  based economy
• Biotechnology to create jobs and wealth
• Biotechnology to cure and prevent diseases.
• Biotechnology to increase survival
• Biotechnology to improve patients quality of
  life
• Biotechnology to help the environment (KYOTO)
• Biotechnology to feed the world
• Biotechnology to return to the plant based
  economy
• Biotechnology to optimise renewable resources
• Biotechnology to be competitive

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Innovation or Stagnation the US response
The medical product development process is no
longer able to keep pace with basic scientific
innovation. Only a concerted effort to apply the
new biomedical science to medical product
development will succeed in modernizing the
critical path.
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Healthcare Biotech Progress

• Causative agents
• Real cure
• Less side effects
• Wound healing
• Genetic tests
• Genetic deficiencies
• Disease mechanisms
• Rare Diseases
• Etc

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• Where is Biotech in Healthcare
• To Replace Old Medicines
• Insulin
• Growth Hormone
• To avoid the risk of accidental Contamination
• 2. To Improve on the Spot Diagnosis
• Elisa
• Genetic Testing
• Patient Selection

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• Biotech in Healthcare
• To produce Large Molecules
• ? Can only be produced by living cells
• ? Are essential for Humans
• Can be produced in Yeasts, Bacteria, Animals,
  Plants
• In sufficient quantities
• At the best attainable quality
• To avoid the risk of Accidental Contamination
• To Understand Diseases, Deficiencies and Nature
  Mistakes .. up to the level of DNA
• More Understandings leads to more Ways to
  tackle the problems

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• Biotech in Healthcare
• 5. To have a Cure instead of Alleviation of
  Symptoms
• Advanced Cells and Tissue Therapies
• ? Cells Producing Insulin
• ? Heart Rejuvenation
• 6. To improve Predictability of Efficacy
• ? System Biology
• ? Reactions of Human cells in culture..
• To improve Predictability of Toxicity
• ? Tests on Human cell / tissue cultures
• Resulting in RRR Experimental Animals?

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• Biotech in Healthcare
• To improve Targeting of Medicinal Products
• To aid in Production Quality assurance and
  Control
• 10. To Repair DNA Deficiencies?
• DNA or RNA Computers

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The European Strategy on Life Sciences and
Biotechnology

• Excellent
• European
• Visionary
• Pragmatic
• Concrete actions what who when
• Respecting society values
• Respecting regulatory oversight and principles
• Based on facts
• Taking up the world challenges, also the
  developing world
• Seeking coherence
• Based on dialogue
• In the whole of Europe, across policies, sectors
  and actors
• And has been endorsed by the European Commission,
  the European Parliament and the heads of state of
  all Member States of the European Union

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What is needed?

• Full and complete implementation of the European
  life sciences and Biotechnology Strategy across
  Europe

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European Biotech Companies Impact on growth
(products)
less products in development
Lower RD budgets in Europe, means
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Are We Ready to Be Leading?

• Is the Healthcare Legislation in EU including
  Member States?
• Classical Advanced
• ? Clear Yes No
• ? Providing predictable outcome Yes?
  Not yet
• ? Result oriented Yes Not yet
• ? Provide access to the medicines? Not always
  or Not yet
• always not everywhere
• ? Patient Oriented Not really Not yet
• Is the Registration System Adapted to the new
  approaches?

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Are We Ready to Be Leading?

• Is Research Stimulated?
• ? IMI FP 7
• Cells Tissues
• Is Research Inhibited
• ? Stem Cells in certain Areas
• ? Ethical Discussions

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Are We Ready to Be Leading?

• Is Entrepreneurship and innovation stimulated and
  sustained ?
• ? SMEs YIC FP 7
• CIP
• Are innovative products welcomed or suspect?
• ? Use of Commitology as vote on science?
• ? 4th Hurdle versus access and reward?

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Healthcare Manifesto Mission To promote a
patient-oriented, innovative and dynamic
economic and regulatory environment for
healthcare biotechnology in Europe.
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European Biotech Industry delivers

• New medicines 20 of todays medicines are
  biotech and 50 of medicines in the pipeline are
  biotech
• New strategies against unmet medical needs.
• New plants for the benefit of the environment,
  the consumer, the farmer, the white
  biotechnology, the patient.
• New hope for patients, through improved
  diagnostic tests
• New hope for Europe through diagnostics for BSE
• New hope for Europe through elimination!!! of
  Rabies.
• New knowledge on the cause of diseases

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Biotechnology The Invisible RevolutionIndustry
expectation Coherence of Policies!!!!

• Predictable rules stimulate research.
• Robust product evaluation and approval stimulate
  entrepreneurship.
• Policy support for reducing the cost of RD
• Policy support reducing the time to market.
• Policy support for increasing the market access.
• Policy support for life saving and real cures.
• Policy support of new therapy avenues for rare
  diseases.
• Policy support of YIC/YLC investments.

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EMEA Opens arms to biotech SMEs

• 100 reduction on administrative assistance
• 90 fee reductions on scientific advice and
  inspection
• Fee deferrals after authorisation
• No fee if no success conditional on scientific
  advice
• EMEA Buddy system

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Innovation for the benefit of patients

Human Insulin First application of Biotech in
medicine Recombinant human growth hormone First
Synthetic human growth hormone with no risk of
CJD infection Recombinant interferon beta
Enabled real progress in treating Multiple
Sclerosis Recombinant erythropoietin
(EPO) Revolutionized the treatment of anemia,
eliminating viral infections including AIDS and
Hepatitis B or C Monoclonal antibodies for the
treatment of cancer Development of drugs
targeting specific types of cancer cells
Herceptin (Trastuzumab) for breast cancer
.
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Our Corporate Members
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Our Corporate Members
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Biotechnology for a quality of life in a
sustainable society
www.europabio.org