Science, research and developmentEuropean Commission Dr Fergal DONNELLY Biotechnology and Applied Genomics Unit Research Directorate General European Commission

Science, research and development European Commission

Dr Fergal DONNELLYBiotechnology and Applied Genomics Unit

Research Directorate GeneralEuropean Commission

Application of knowledge and technologies in the field of genomics and biotechnology for health

LIFE SCIENCES, GENOMICS AND BIOTECHNOLOGY FOR HEALTH

Warsaw, POLAND, 22 6.2004


(Source: EFPIA)

• Employment : about 560,000 jobs in Europe, including 88,200 in R&D units

The pharmaceutical industry remains one of Europe’s

best performing high-technology growth sectors

• R&D investment: 17,500 million Euro in 2000 (up from 4,300 million Euro in 1985)

• Trade surplus: 25,200 million Euro in 2000 (up from 5,100 million Euro in 1985).



Pharmaceutical R&D expenditure in Europe, USA and Japan

(Euro Million), 1990-2001

0

5

10

15

20

25

30

1990 1995 2000 2001

Europe

USA

Japan8.000

5.342

2.810

10.958

9.078

5.221

17.495

21.631

7.495

18.800

26.384

N.A.

(Source: EFPIA)



Location of R&D spending by EU pharma companies (%)

0

10

20

30

40

50

60

70

80

1990 1995 1999

European Union

United States

Rest of the world

73

26

1

69

26

5

59

34

7

(Source: EFPIA)



Origin of the top 10 medicines by worldwide sales

0 2 4 6 8 10

2000

1992

Europe

Europe

USA

USA

(Source: EFPIA)



The Innovation Cycle in Drug Discovery

1. Europe is STRONGin Life Sciences.

2. Europe is WEAKin the market.

3. There is insufficient investment flowing to the critical development stages between basic research

and the market.

New R&D investment

TechnologicalDevelopment

Product and ProcessDevelopment

MarketResearch



“Towards a European research area” - COM 2000(6)

Proposes better framework conditions to foster integration of research activities and coordination of research and innovation policies

THE POLITICAL DRIVE

Lisbon Summit - March 2000

Sets a strategic goal for Europe to become the most competitive and dynamic knowledge-based economy in the world

Barcelona Summit - March 2002

Calls for adoption of the 6th Framework Programme by June 2002, agrees that research spending in the EU should approach 3% GDP

by 2010, with two thirds coming from business, stresses the importance of frontier technologies for future growth and welcomes the Commission's strategic vision for biotechnology and life sciences



Linking the ability to discover to the ability to produce



Improving the diagnostic and therapeutic arsenal for health care



Cell Factory Key ActionCell Factory Key ActionA strategy for Health and Drug DiscoveryA strategy for Health and Drug Discovery

New safermedicines faster

Preparing the regulatory

frame for new medicines

Promoting dialogue between industry,

academics, clinicians, patients, regulators

Financing R&D projects101 projects 1999-Sept.2001

200 Million Euros EU contribution




Drug discovery R&D projects

Cell Factory (Framework Programme 5: 1998-2002)

Number EC contribution of projects (in Million Euro)

Drug discovery 56 110.01

New therapeutic strategies 39 79.84

In vitro tests 6 10.49

TOTAL 101 200.34




Life Sciences, Genomics and Biotechnology for Health (FP6)L

ife

Sci

ence

s, G

enom

ics

&bi

otec

hnol

ogy

for

heal

th

• Advanced Genomics and its applications for health (1100 M€)

– Fundamental knowledge and basic tools for functional genomics in all organisms (Structural genomics; proteomics; comparative genomics; bioinformatics; Multidisciplinary genomics approaches to basic biological processes)

– Application of knowledge and technologies in the field of genomics and biotechnology for health (Technology platforms; support to start-up companies)

• Advanced Genomics and its applications for health (1100 M€)

– Fundamental knowledge and basic tools for functional genomics in all organisms (Structural genomics; proteomics; comparative genomics; bioinformatics; Multidisciplinary genomics approaches to basic biological processes)

– Application of knowledge and technologies in the field of genomics and biotechnology for health (Technology platforms; support to start-up companies)

•Combating major diseases (1155 M€)

– Application-oriented approach to medical genomics knowledge (Cardiovascular disease, diabetes and rare diseases ; resistance to antibiotics ; Studying the brain and combating diseases of the nervous

system ; Studying human development and the ageing process) – Broader biomedical approach (cancer, poverty-related infectious diseases)

•Combating major diseases (1155 M€)

– Application-oriented approach to medical genomics knowledge (Cardiovascular disease, diabetes and rare diseases ; resistance to antibiotics ; Studying the brain and combating diseases of the nervous

system ; Studying human development and the ageing process) – Broader biomedical approach (cancer, poverty-related infectious diseases)



(1.1.1.b)

“The strategic objective of this line is to foster the competitiveness of Europe’s biotechnology industry by exploiting the wealth of biological data produced by genomics and advances in biotechnology”.

Council Decision of 30/09/2002




111ib

Rational and accelerated development of new, safer and more effective drugs including pharmacogenomics approaches

Development of new diagnostics

Development of new in-vitro tests to replace animal experimentation

Development and testing of new preventive and therapeutic tools, such as somatic gene and cell therapies (in particular stem cell therapies, for example those on neurological and neuromuscular disorders) and immunotherapies

Innovative research in post-genomics, which has high potentialfor application



The topics in the Workprogramme must be contemplated against the background of:

Translational approach

Enhance the European biotechnology industry

Innovation in the health sector of the biotechnology industry, especially SMEs

Stimulation of entrepreneurship and opportunities through venture capital and the EIB

Identification of regulatory bottlenecks in the new applications for genomics

Anticipation of ethical implications

Council Decision of 30/09/2002


Science, research and development European CommissionNetworks of Excellence:

Objectives

To structure the EU research potential

by integrating research capacities• Joint research activities• Integrating activities• Spreading of excellence• Common management

Management Group

Eu

rop

ean

Com

mis

sion

Research team leader

Management Group

• Joint research activities

• Integrating activities

• Spreading of excellence (training)

• Common management

To structure the EU research potential by integrating existing research capacities

Governing Council Funding Bodies Representatives

Partner Organisation Representative

Scientific Council


Integrated projects: Objectives

Training

Ethical aspects, science-society dialogueEthical aspects, science-society dialogue

Technology transfer, exploitationTechnology transfer, exploitation

strong managementstructure

ImplementationPlan

RTD 4

RTD 1

RTD 2

RTD 3

RTD 5

Demonstration

ManagementManagement

addressing major societal needs increasing EU competitiveness

predefined S/Tresults and

clear deliverables

To integrate the critical mass of activities/resources needed for :


Overview of the Selection : 2nd CallOverview of the Selection : 2nd Call

Instrument Response Priority Main Priority Reserve No-Go &(Nr of prop.) List (1) List Ineligible

IP 48 11 8 3 37NoE 14 7 6 1 7STREP 28 14 14 - 14SSA 14 5 5 - 9CA 2 0 0 -

Total 109(*) 37 33 4 69

(*) 3 ineligible(1) Proposals having passed the thresholds


Evaluation criteriaEvaluation criteria

Relevance

Potential impact

Excellence of the participants

Degree of integration and the JPA

Organisation and management

S&T excellence

Quality of the consortium

Quality of the management

Mobilisation of the resources

Network of Excellence

X

X

X

X

X

Integrated projects

X

X

X

X

X

X


Integrated projects Integrated projects Reasons for failing evaluation criteriaReasons for failing evaluation criteria

1. S&T excellence

2. Overall

3. Relevance

4. Potential impact


Evaluation criteria : S&T Excellence (IP)Evaluation criteria : S&T Excellence (IP)

COMMENT• “…clear ambition…clear

and relevant objectives.”• “…S&T very good …

objectives clearly defined…”

• “….limited applications…objectives too narrow…over ambitious”

• “…unfocussed work plan…”

MARK

• 4 - 5

• 2.5 - 3.5


Evaluation criteria : Relevance (IP)Evaluation criteria : Relevance (IP)

COMMENT• “…excellent relevance• “….exactly matches

the objectives …”• “…. addresses the

objectives• “…partially addresses

scope of the Call…”• “…does not fit the

objectives of the Call…”

MARK

• 4 - 5

• 3 – 3.5

• 2.5

• 0 - 1


Evaluation criteria : Overall (IP)Evaluation criteria : Overall (IP)

COMMENT• “…(some issues)

should have been dealt with in greater detail…”

• “…some partners not involved…

• over ambitious goals…

• management insufficient”

MARK

JUST BELOW THE THRESHOLD


Networks of ExcellenceNetworks of Excellence Reasons for failing evaluation criteria Reasons for failing evaluation criteria

1. Degree of Integration, JPA

2. Potential impact

3. Overall


Networks of ExcellenceNetworks of Excellence Evaluation criteria : Degree of Integration Evaluation criteria : Degree of Integration

& JPA& JPA

COMMENT• “…missing

information…partners (industrial)...”

• “…lower than expected integration…”

• “…no lasting integration…”

MARK• 3.5

• 2


STREPsSTREPs Reasons for failing evaluation criteria Reasons for failing evaluation criteria

1. S&T excellence

2. Relevance


Evaluation criteria : S&T Excellence Evaluation criteria : S&T Excellence (STREP)(STREP)

COMMENT• “…objectives clearly

stated...”• “….[adds] no real value

to the state of the art”• “ …fails to develop

(x,y,z…)• “…unfocussed work

plan…”

MARK

• 4 - 5

• 3


Evaluation criteria : Overall remarksEvaluation criteria : Overall remarks

• Incomplete/Absent Work Packages, absent elements in the research & development programme,

– Necessary partners absent : regulators, societal, industrial etc.– SMEs underrepresented/absent/de facto subcontractors

• Financial plans non-transparent– budget overinflated– distribution of funds inadequately justified

• Partial or no relevance to Work Programme• Project management inadequately described

– no clear management structure– fragmented / overlapping activities – no role definition for partner(s)

• Inappropriately qualified personnel


Number of proposals: 8 IP6 NoE14 STREP5 SSA33 Total (32 topics)

All topics where proposals passed the threshold are covered

Budget: 161,31 Mio Euro (Indicative budget 165 Mio Euro)

SME participation: 23,2% (budget) and 16,9% (Nr of participants)

Budget requested by SME (in %): 27.3 IP22.6 NoE14.5 STREP

Industrial participation: 14%

Ratio NI / TI (budget): 79.8 / 20.2

Overview of the SelectionOverview of the Selection



SMEs are defined as enterprises which:

have fewer than 250 employees, and have either an annual turnover not exceeding € 40

million, or an annual balance-sheet total not exceeding in € 27

million

(Ref. 96/280/EC: Commission Recommendation of 3 April 1996 concerning the definition of small and medium-sized enterprises (Text with EEA relevance), Official Journal L 107 , 30/04/1996 P. 0004 - 0009)

SMEs



SMEs

Academia + others

83,7%

LARGE IND

1,7% SME 14,6 %


Warsaw, POLAND 22.6.2004

EU contribution to SME contractors in IPs and STREPs was 14.6 % (22.7 M€). Adding large industries (1.7 %) will increase EU contribution to 16.3 % (25.3 M€).


SMEs


63 SME contractors out of 335 partners (18.8 %).

SME19%

Academia + others

77%

IND4%


SMEs

A large majority (95%) of the SME contractors is industrial SMEs.

SME-other5%

IND-SME95%



SME Dimension of the Selection


0

5

10

15

20

25

30

Response Priority List Main Priority List2nd Call

Main Priority List1st Call

IP

NoE

STREP

Average


IP New approaches for accelerated development of new, safe and more effective medicines

IP Pharmacogenomics for individualised medicinesIP Exploring the potential of stem cells and/or primary cells,

for the understanding of monogenic rare diseases and the development of new drugs for their treatment

STREP Standardised assays for determining immunogenicity of biopharmaceuticals STREP 'Microdosing' studies to gain enhanced absorption, distribution, metabolism, excretion (ADME) - parameters for biopharmaceuticals (especially orientated towards involvement of SMEs)STREP Targeted delivery of protein-based drugs (especially orientated towards

involvement of SMEs) SSA Interdisciplinary educational approaches on the biological responses to

new biopharmaceuticals ( e.g. training events, workshops)

IP New approaches for accelerated development of new, safe and more effective medicines

IP Pharmacogenomics for individualised medicinesIP Exploring the potential of stem cells and/or primary cells,

for the understanding of monogenic rare diseases and the development of new drugs for their treatment

STREP Standardised assays for determining immunogenicity of biopharmaceuticals STREP 'Microdosing' studies to gain enhanced absorption, distribution, metabolism, excretion (ADME) - parameters for biopharmaceuticals (especially orientated towards involvement of SMEs)STREP Targeted delivery of protein-based drugs (especially orientated towards

involvement of SMEs) SSA Interdisciplinary educational approaches on the biological responses to

new biopharmaceuticals ( e.g. training events, workshops)

Rational and accelerated development of new, safer and more effective drugs (topics in the third call 2004)

Lif

e S

cien

ces,

Gen

omic

s &

biot

echn

olog

y fo

r he

alth


IP Screening techniques and strategies in the early diagnosis of solid tumors

STREP Highly sensitive tests for the assessment of haematological malignancies (especially orientated towards involvement of SMEs)

STREP Genetic and molecular diagnostic markers of alcoholic liver diseases (especially orientated towards involvement of SMEs)

STREP New diagnostic tools for human and animal prion associated diseases (especially orientated towards involvement of SMEs)

SSA Workshop on the monitoring of safety of human blood origin products

SSA Workshop on ethical issues raised by new diagnostic methods

SSA Workshop on trends and perspective in new diagnostics

IP Screening techniques and strategies in the early diagnosis of solid tumors

STREP Highly sensitive tests for the assessment of haematological malignancies (especially orientated towards involvement of SMEs)

STREP Genetic and molecular diagnostic markers of alcoholic liver diseases (especially orientated towards involvement of SMEs)

STREP New diagnostic tools for human and animal prion associated diseases (especially orientated towards involvement of SMEs)

SSA Workshop on the monitoring of safety of human blood origin products

SSA Workshop on ethical issues raised by new diagnostic methods

SSA Workshop on trends and perspective in new diagnostics

Development of new diagnostics(topics in the third call 2004)

Lif

e S

cien

ces,

Gen

omic

s &

biot

echn

olog

y fo

r he

alth


IP Cell systems as a means to enhance toxicity testing

STREP Optimization and pre-validation of in-vitro models for the study of drug absorption, modification and detoxification in the liver and in the intestinal

epithelium (especially orientated towards involvement of SMEs)

SSA Socio-economic impact on the "Three Rs" of the portfolio of the 5 research contracts in the "Cell Factory" 5th Framework Programme,

area 3.1.4

SSA Workshop: how to integrate and make optimal use of animal data, non-animal data and predictions from computer-based modelling in

assessing the risk of chemical compounds

SSA Mini-pigs as models for toxicity testing of new drugs and chemicals: impact assessment

IP Cell systems as a means to enhance toxicity testing

STREP Optimization and pre-validation of in-vitro models for the study of drug absorption, modification and detoxification in the liver and in the intestinal

epithelium (especially orientated towards involvement of SMEs)

SSA Socio-economic impact on the "Three Rs" of the portfolio of the 5 research contracts in the "Cell Factory" 5th Framework Programme,

area 3.1.4

SSA Workshop: how to integrate and make optimal use of animal data, non-animal data and predictions from computer-based modelling in

assessing the risk of chemical compounds

SSA Mini-pigs as models for toxicity testing of new drugs and chemicals: impact assessment

Development of new in vitro tests to replace animal experimentation(topics in the third call 2004)

Lif

e S

cien

ces,

Gen

omic

s &

biot

echn

olog

y fo

r he

alth


IP Overcoming the limitations of stem cell therapy for solid tumours

IP Chemokines: A new therapeutic approach for chronic inflammation and autoimmune diseases

NoE Quality and safety standards for clinical gene transfer

STREP Development of new non-viral gene delivery technologies for ex vivo gene-based therapies (especially orientated towards involvement of

SMEs)STREP Human stem cells for spinal cord injury repairSTREP Exploring the potential of human umbilical cord blood stem cells for application other than allogeneic haematopoietic transplantationsSTREP Oncolytic virus vectors for cancer virotherapy

(especially orientated towards involvement of SMEs) SSA Workshop on use of stem cells as a tool for gene therapy

IP Overcoming the limitations of stem cell therapy for solid tumours

IP Chemokines: A new therapeutic approach for chronic inflammation and autoimmune diseases

NoE Quality and safety standards for clinical gene transfer

STREP Development of new non-viral gene delivery technologies for ex vivo gene-based therapies (especially orientated towards involvement of

SMEs)STREP Human stem cells for spinal cord injury repairSTREP Exploring the potential of human umbilical cord blood stem cells for application other than allogeneic haematopoietic transplantationsSTREP Oncolytic virus vectors for cancer virotherapy

(especially orientated towards involvement of SMEs) SSA Workshop on use of stem cells as a tool for gene therapy

Development and testing of new preventative and therapeutic tools such as somatic gene and cell therapies and immunotherapies

(topics in the third call 2004)

Lif

e S

cien

ces,

Gen

omic

s &

biot

echn

olog

y fo

r he

alth


IP Post genomic approaches to understand the molecular basis of asthma aiming at a preventive or therapeutic control

IP Post genomic approaches for inflammatory rheumatic disease, leading to the development of improved therapy

IP Post-genomic approaches to a human pandemic influenza vaccine

STREP Post genomic approaches for tackling complex multifactorial diseases (except Cancer) (especially orientated towards involvement of SMEs)

IP Post genomic approaches to understand the molecular basis of asthma aiming at a preventive or therapeutic control

IP Post genomic approaches for inflammatory rheumatic disease, leading to the development of improved therapy

IP Post-genomic approaches to a human pandemic influenza vaccine

STREP Post genomic approaches for tackling complex multifactorial diseases (except Cancer) (especially orientated towards involvement of SMEs)

Innovative research in post genomics(topics in the third call 2004)

Lif

e S

cien

ces,

Gen

omic

s &

biot

echn

olog

y fo

r he

alth


Technology Platform


Technological platforms have the primary objective of defining a coherent and unified approach to tackling a major economic, technological or societal challenge of vital importance for Europe’s future competitiveness and economic growth.


Technology Platform


Key features:

• A shared vision and the commitment to put it into reality

• Strategic research agenda• Very large scale, highly

ambitious and complex• Industry-driven• Very substantial public and

private investment• Very high economic potential


ETP: Innovative Medicines for Europe


Objective:

To enhance and accelerate the development process of medicines to ensure a rapid translation of scientific breakthroughs to approved medicines. In particular through reinforced public-private partnerships early access to new, more targeted medicines shall be provided to the European population, while strengthening the European Science base and fostering economic growth in the pharma/biotech industry




Stakeholders:

Industry (Pharmaceutical & Biopharmaceutical)

Public authorities (e.g. policymakers, regulatory agencies)

Academic community (Universities, Research centres, Ethical experts)

Financial community (e.g. venture capital, EIB, EIF)

Users, consumers and civil society (e.g. Hospitals, Clinicians, Patients associations)




Expected benefits:

Increased effectiveness of public and private R&D investment

Fostering of innovation and competitiveness

More efficacious medicines and Reduced treatment periods for

patients

Less medicines withdrawals from market

Shorter drug development times

Overall reduced healthcare cost

Improvement of public perception of pharmaceutical industry

Increase Europe’s attractiveness for researchers and industrial

investment




Implementation

Short term (FP6)

Definition of strategic research agenda Mobilisation of public and private resources Establishment of communication/dialogue process

and supporting structure/secretariat Develop education/training opportunities




Strategic research agenda

• Is a key deliverable• Setting out R&D priorities for medium/long term• Should address obstacles for future market

penetration




Implementation :

• Medium and long term (FP7):

• Through existing instruments (IP, NoE, CA, SSA)

• Via Joint European Technology Initiatives (as defined in Art. 171 of EU treaty)



Joint European Technology Initiatives

Criteria:• Highly innovative Science and Technology• Multiple industrial users• Research agenda building upon existing

programmes, seeking for integration of efforts• Significant economic impact• Feasible only at European level• Key stakeholders with full commitment• Technical/operational framework defined



Evaluators in FP6


Evaluators in FP6


Evaluators in FP6


Evaluators in FP6



Total

10%6%

84%

EU 15

10 New MS

3 Cand.Ctries

Evaluators in FP6



Nr. of experts by country

349

874 951

65

1119

3873

3309

846

14111304

85

543

4664

1198887 1004

1462

554335 204

254 128 159

631

3384

84204132

4187

0

500

1000

1500

2000

2500

3000

3500

4000

4500

5000

BG RO TR CR PL EE LV LT HU CZ SK SI CY MT IRL I UK F E PT B NL L DK D S FI A EL

Evaluators in FP6



Evaluators in FP6

% of total experts by country

BG1%

RO3%

TR3%

CR0%

PL3%

F11%

E10%

PT2%

B4%

NL4%

L0%

DK2%

D14%

S4%

FI3%

A3%

EL4%

HU2% CZ

1%SK1%

SI1% CY

0% MT0%IRL2%

UK10%

LV0%

LT1%

EE0%

I12%


Success will depend on mobilisation of all actors

Industrial

Platforms

Academia

Private Sector

Civil Society

European

Investment Bank

RESEARCH DEVELOPMENT REGULATIONDELIVERABLES

FOR SOCIETY

ParliamentCommissionMember States

Ethical Bodies

SMEs

Clinicians


Documents

Science, research and developmentEuropean Commission Dr Fergal DONNELLY Biotechnology and Applied Genomics Unit Research Directorate General European Commission