giuseppe recchia, convegno mitocon 2015
TRANSCRIPT
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 [email protected]
Twitter @gr0457 Giuseppe Recchia