giuseppe recchia, convegno mitocon 2015

Bisogni

players

Why?

RD Priorities

Discovery Clinical Trials

competitivenesspharmaceutical

Technologies

Rare Diseases

Scenario

Bisogni

players

Why?

RD Priorities

Discovery Clinical Trials

competitivenesspharmaceutical

Technologies

Rare Diseases

Scenario

Scenario 2015 - 2018

￭ Science

￭ Precision Medicine

￭ Technology

￭ Demography

￭ Society

￭ Healthcare Costs

￭ Patent Expiration

￭ RD Productivity

2012

35

2013

4

23

4 5

2014

36

Bisogni

players

Why?

RD Priorities

Discovery Clinical Trials

competitivenesspharmaceutical

Technologies

Rare Diseases

Scenario

+ T

hera

peut

ic A

ltern

ativ

es -

- Added Benefit +

Priorities – Therapeutic Innovation

Absolute Needs

Residual Needs

Priorities - Absolute Needs

Priorities - Residual Needs

12.300 compounds

6.500 cancer

Priorities – A Divergen Perspective from WHO

Public Health

- Added Benefit ++ T

hera

peut

ic A

ltern

ativ

es -

Bisogni

players

Implications …

RD Priorities

Discovery Clinical Trials

competitivenesspharmaceutical

Technologies

Rare Diseases

Scenario

Pharmaceutical Technologies - Evolution

- Added Benefit ++ T

hera

peut

ic A

ltern

ativ

es -

Chemicals 1897

Biologicals

1982

Advanced Therapy

Medicinal Products

2012 - 2014 - 2016

Advanced Therapy Medicinal Products - 2012

1^ Gene Therapy Approvedn in Europe

discovered in Netherlands

Advanced Therapy Medicinal Products - 2014

1^ Stem-Cell Therapy Approved in Europe

discovered in Italy

Advanced Therapy Medicinal Products - 2016

1^ Cell-based Gene Therapy Approved in Europe

discovered in Italy

Bisogni

players

Implications …

RD Priorities

Discovery Clinical Trials

competitivenesspharmaceutical

Technologies

Rare Diseases

Scenario

New Players in Drug Discovery - Accademia

Absolute Needs

Residual Needs

New Players in Drug Discovery - Accademia

Discovery Partnerships with Academia

Discovery Fast Track Challenge 2015

New Players in Drug Discovery - Patients

New Players in Drug Discovery - Charities

Bisogni

players

Implications …

RD Priorities

Discovery Clinical Trials

competitivenesspharmaceutical

Technologies

Rare Diseases

Scenario

Global Clinical Trials – For Profit 2014

2009 2010 2011 2012 20130

50

100

150

200

250

300

2009 2010 2011 2012 20130

100

200

300

400

500

600

700

Trend in study numbers and

median actual patients enrolled

per study for studies completing

enrolment 2009-2013, by phase

Studies completing enrolment Actual patients enrolled per study

No .

of

stud

ies

Act

ual p

atie

nts

enr o

ll ed

p er

stud

y

Phase IIPhase Ip Phase III

2009

2013

Global Clinical Trials – For Profit 2014

Patient volume (2011-2013) Change in patient volume 2008-2010 vs. 2011-2013 (%)

USA

Japan

Germany

China

Russian Federation

Poland

UK

Canada

Spain

Hungary

France

Ukraine

South Africa

Czech Republic

India

Romania

Mexico

Netherlands

Brazil

Italy

Bulgaria

Argentina

South Korea

Denmark

Australia

0 5000 10000 15000 20000 25000 30000[91729]

Top 25 countries by patient

volume for countries initiating

patient enrolment, 2011-2013

and change in patient volume,

2008-10 vs 2011-13

-75 -50 -25 0 25 50 75 100

USA

JapanGermany

China

Russian Federation

Poland

UK

Canada

Spain

HungaryFrance

Ukraine

South Africa

Czech Republic

India

Romania

Mexico

Netherlands

Brazil

Italy

Bulgaria

Argentina

South Korea

Denmark

Australia

Clinical Trial Actrativeness

Bisogni

players

How?

RD Priorities

Discovery Clinical Trials

competitivenesspharmaceutical

Technologies

Rare Diseases

Scenario

Big Pharma and Rare Diseases

Big Pharma and Rare Diseases

Big Pharma and Rare Diseases

￭ Specialist RD Units with a large

RD organization

￭ Disease Prioritisation

￭ Corporate R&D Engine

￭ Global Reach

Rare Diseases - Pipeline 2015

3.500 / 12.300

Rare Diseases - Pipeline 2015

- Orphan Drug

- Rare Disease

Orphan Drugs Report 2014 – Phase 3 Development

Orphan Drugs Report 2014 – Phase 3 Development

Still Orphans or Happily Adopted?

Orphan Drug Discovery – Italy 2015

opportunity

Orphan Drugs Clinical Trials – Italy

2013 Phase 2 Phase 3 Phase 4 Total

Italy 57 63 8 128

France 244 220 40 504

Spain 325 333 27 685

UK 58 55 8 121

Germany 249 183 14 446

Total 933 858 97 1888

issue

Orphan Drugs Clinical Trials – No Profit Italy

Clinical Trials Profit % No Profit %

CT – Total 64,3 35,7

Rare Diseases CT 87,9 12,1

issue

Mail giuseppe.g.recchia@gsk.com

Twitter @gr0457 Giuseppe Recchia