Situation of biotechnology – national and international · · 2012-08-06Situation of biotechnology – national and international ... a dynamic market – average annual growth

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Pharmaceutical Innovation: Possibilities and Limits of Personalized Medicine

Situation of biotechnology –national and international

Prof. Dr. Georg-Burkhard KresseVP Biologicals Strategy and Communication, Roche Pharma Research, Penzberg, Germany

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Steam engine

Textile industry

Industrial revolution

SteelRailways

Transport

Electricalengineering

Chemistry

MassConsumption

AutomotivePetrochemistry

IndividualMobility

Informationtechnology

InformationCommunication

BiotechnologyNanotechnology

Healthcare

1stKondratiev

2ndKondratiev

3rdKondratiev

4thKondratiev

5thKondratiev

6thKondratiev

1800 20??1990195019001850

Source: modified from M. Lonsert et al. (2007) pharmind 69, 37-41

Modern biotechnology is one of thekey enabling technologies of the 21st century

Overview of Kondratiev waves

Economics is governed by innovation-driven supercycles –healthcare technologies may well be the 6th

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Healthcare is the most prominent fieldof biotechnology

Biotech industry, status 2004: • 2163 biotech companies in 18 European countries• Over 96,500 employees, thereof 42,500 in R&D• Over 21,5 billion € revenue

Source: EuropaBio, Critical I comparative Study 2006

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Economic impact of biotech andbiotech-focussed pharma companies

Number of companies 2006 Employees 2006Revenue 2006

(Biopharmaceuticals only, in Mio. €)

4.010 366 29.909

Source: Boston Consulting Group/VFAbio, Studie „Wirtschaftliche Situation, Nutzen und Einsatz von Biopharmazeutika in Deutschland“, 2007

Companies withtechnology platforms, but without drug development

23.813

6.096

Companies withproducts in themarket and/or

drug development

2006 vs. 2005

+2

+13 % +4 %

-4

+27 %

-20 %

+11 %

-26 %

-0.5%

3.463

547

281

85

Pharma and Biotech companies active in healthcare biotechnology in Germany:Revenues and number of employees are increasing

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Mammalian and microbial fermentation capacity (in ‘000 l, 2005)

mammalian cellsmicrobialfermentation capacity in liter

(1) „microbial“ including Puerto Rico

USA(1) Germany Japan India

Fermentation capacity -Germany in the 2nd place worldwide

Source: Boston Consulting Group/VFAbio, Studie „Medizinische Biotechnologie in Deutschland“, 2006

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Biopharmaceuticals: High economic significance in Europe

Source: E. Zika et al. (2007) Consequences, Opportunities and Challenges of Modern Biotechnology for Europe (Bio4EU study), EC JRC report EUR 22728 EN

Biopharmaceuticals launched (accumulatednumbers, w/o vaccines)

Share of turnover of biopharmaceuticalsout of all pharmaceuticals

Biopharmaceuticals in Europe: a dynamic market – average annualgrowth rate (23%) twice as high as for pharmaceuticals (11%)

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Biopharmaceuticals: A significant share of new drug approvals

2429 28

12

31

1725

10

149

3

7

5

11

2000 2001 2002 2003 2004 2005 2006

Biopharmaceuticals

Other

29 % 33 % 20 % 18 % 23 %ThereofBiopharmaceuticals

Number of new drug approvals in Germany

24 % 31 %


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Biopharmaceuticals are indispensablein various disease areas

CNS Meta-bolism

0 %

20 %

40 %

60 %

80 %

100 %

Infec-tiousDis.

Onco-logy

Hema-tology

Immuno-logy

Others

10 %

23 % 22 % 21 % 24 %19 %

4 %

Example:Insulins for

diabetes

Example:Pneumo-coccal

vaccines

Example:Interferon-

beta formultiple sclerosis

Example: Epoetins

for anemia

Example:TNF-α

Inhibitorsfor

rheumatoidarthritis

Biopharmaceuticals share of total pharma market (based on sales in Germany 2006)

Example :Trastuzumab

for breastcancer

Example:Anti-IL-2 mAb in trans-

plantation

Total pharmaMarket, € 25.4 Bio.

Biopharmaceuticals(12 %), € 3.1 Bio.

e.g. gastroenterology, dermatology, urology etc.


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Biopharmaceuticals addressunmet medical needs

Examples

• Incurable and/or• Drugs/therapies for

relief or retardation of disease progress exist, or

• No therapy available yet Multiple sclerosis

Rheumatoidarthritis

Breast cancer

Chronic, severediseases

• Prevalence max. 5 patientsper 10.000 people

• No therapy available yet

Rare diseases

Morbus Pompe

Morbus Fabry

Mucopolysaccharidosis Type VI

Mucopolysaccharidosis Type I

• Avoidance of diseases• Mainly in infectious

diseases, but also in cancer

Cervical cancerPneumococcal

infection

Prevention/ Vaccination


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Progress in cancer treatmentthrough innovative drugs based on monoclonal antibodies

rNew medicines create value for the patients -Median survival time in cancer increases

Source: Roche

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Pharma Research is risky and increasingly expensive

Source: Tufts Center for the Study of Drug Development; M.W. Vannier (ed), US Measurement System/NIST Workshop (2006), www.sns.gov/workshops/ian2006/TU2/IAN2006oct_Vannier_FINAL.pdf

Average capitalized development costsper „New Chemical Entity“

1976 1986 1987 1990 1997 2001

$54 Mio.$125 Mio.

$231 Mio.

$359 Mio.

$500 Mio.

$802 Mio.

100

80

60

40

20

0

Idea Lead Dev.Candidate Phase 2a Products

Nu

mb

er

of

pro

ject

s

40%75% 75%75%

Estimate 2006 for biotech projects:

1,2 Bio. US-$due to longer development time

and higher cost of capitalShare of terminated projects

//

12-15 years

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The complexity of proteins

Size

Structure

Modification

Stability

Epoetin

Aspirin

Denaturation, Aggregation, Degradation, Oxidation, ...

Glycosylation, Acylation, etc.

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Biopharmaceuticals -„The process is the product“

DNA Vector

Large-scale fermentation

e.g. bacterial or mammalian cellFormulation

...ATG STOP... Human Gene Sequence

Cloning into a DNA Vector

Transfection into the host cell, expression, screening/selection Product purification

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Can two biologics be “the same“, or just “similar“?

“Sameness” cannot be determined for biologics because of the complexity ofboth the products and their manufacturing processes.

Therefore, “biogenerics” cannot exist – just independently developed original products, or “biosimilars”.

According to the EMEA guidelines, appropriate preclinical and clinical data are required for their approval.

Pictures taken from: http://savingsandclone.com/news/press_room.html

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The challenge of personalized healthcare

Source: JAMA 296, 1453-1454 (2006)

What the public came to expect of PHC are truly individualized therapies –something science and industry may not be able to deliver for some time

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More efficiency in healthcarethrough stratified medicines

Non-Responder

Responder

DiagnosticAssay

A

B

CUnacceptableAdverse Drug Events

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Trastuzumab -An antibody used in breast cancer treatment

Normal Cell HER2 overexpressingtumor cell

HER2 overexpressingTumor cell plus Trastuzumab

HER2 Receptors arepresent, in low density, on the surface of healthybreast cells. These cellsdivide only as needed.

In 20-30% of breast cancer patients, HER2 receptor density is increased. Thus, the cell receives proliferationsignals and divides again and again. A tumor growing in an uncontrolled manner is formed.

The monoclonal antibodyTrastuzumab blocks the HER2receptor and inhibitstransduction of proliferationsignals to the cells. This will slow down tumor growth.

r

Source: Roche

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Getting Trastuzumab to the right patients Patient stratification improves treatment efficiency

FISH+ FISH–

Eligible for Trastuzumab treatment Not eligible

IHC3+ IHC < 3+

Formalin fixed, paraffin-embedded tumor tissue slide

HER2testing

r

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Drug innovation continues withbiotechnology as a major driver

Penicillin SulfonamidesAspirin

Psycho-active drugs

NSAIDS

H2 antagonistsbeta-blockers

Proteintherapeutics

Chronicage-related

degenerative diseases, inflammation, cancer;

regenerative medicine, stem cells

Naturalproducts and derivatives

RandomchemicalResearch

Receptorantagonists

Enzyme inhibitors

Gene technology

Cell and molecular

biology

1900 1950 1960 1970 1980 1990 2000 2010 2020

Source: modified from Roland Berger & Partner, Consors Capital

Monoclonalantibodies

Emerging newtherapies

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39.000

122.000

83.000 27.000

10.0004.000 42.000

Not all patients get access to therapy yet

MS Patients Eligible forimmune

modulatortherapy

Treated withinterferons/

glatiramer acetate

Treated withimmune

suppressantdrugs

Therapyterminated(1)

UntreatedPatients

(1) e.g. due to compliance pissues, contraindication, adverse effects, wish to have children, etc.

33 %

12 % 55 %

One third of MS patients

without therapy

12% of patientsterminate therapy

Not eligible forimmune modulator

therapy

Share of MS patients in Germany according to treatment status in 2006


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Innovation creates value - outlook

• Biopharmaceuticals are economically important, and this field will continueto grow

• Biopharmaceuticals are indispensable for the prevention and treatment of diseases and offer medical benefit for the patients

• Access of patients to biopharmaceuticals is not yet optimal and has to beimproved (e.g., through cost-benefit analyses performed according to international standards, clear and reliable rules for reimbursement, and „compassionate use“programs for orphan drugs)

• A predictable and stable regulatory framework has to be provided• EU should promote innovation and ensure adequate financing of research• A strong industrial base in Europe needs to be preserved by means of

modern industrial policy to stimulate growth potential

Modified from: VFA-bio; EFPIA; G. Verheugen, speeach at Lyon, April 14, 2005

Innovation

Value

Documents

Situation of biotechnology – national and international · · 2012-08-06Situation of biotechnology – national and international ... a dynamic market – average annual growth