www.eurordis.org
NORD CORPORATE COUNCIL MEETING
15 May 2012
Washington D.C. USA
Yann LE CAM
Chief Executive Officer, EURORDIS
Peter Saltonstall
President & Chief Executive Officer, NORD
www.eurordis.org
NORD & EURORDIS
AN INTERNATIONAL COLLABORATION
A LONG-TERM STRATEGIC PARTNERSHIP
ON THE 4th OF JULY 2009
EURORDIS AND NORD SIGNED
A MEMORANDUM OF UNDERSTANDING
TO SEAL THEIR
LONG TERM PARTNERSHIP
BASED ON THEIR
RESPECTIVE STRATEGIES 2010-2015
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NORD CORPORATE COUNCIL 15 MAY 2012
WHY SHOULD NORD & EURORDIS COLLABORATE?
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NORD CORPORATE COUNCIL 15 MAY 2012
TO PROVIDE A GLOBAL APPROACH TO
RARE DISEASES AND ORPHAN DRUGS THROUGH:
I. Coordination of our strategies and implementation
II. International Rare Disease Day: the overall objective is to raise international
awareness of rare diseases as a public health priority to policy makers, health
care professionals and other stakeholders, and the public at large
III. International policy affairs: Closer collaboration based on frequent
exchange of information and close coordination between the US and EU,
and close cooperation on issues of international dimensions
IV. Expand the movement of rare diseases at the international level
V. International Patient Advocate Outreach & Mentoring: The two organisations
will develop their ability to reach out and support patient groups, in the USA,
Europe and worldwide
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NORD CORPORATE COUNCIL 15 MAY 2012
TO PROVIDE A GLOBAL APPROACH TO
RARE DISEASES AND ORPHAN DRUGS THROUGH:
VI. Joint development of our Web Platform: sharing strategy, investments,
technology and skills so as to offer better and global services using the
potential of Web 2.0 Communications and Social Networking
VII. Relationship with pharmaceutical & biotechnology companies:
Bring together more closely the NORD Corporate Council and the
EURORDIS Round Table of Companies
VIII. Fund raising through international high level events: The two
organisations will jointly support the Annual NORD Gala Tribute
Banquet in Washington and an annual EURORDIS Gala Dinner in
Brussels that started in 2012
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NORD CORPORATE COUNCIL 15 MAY 2012
WHAT IS ALREADY VISIBLE?
•
Rare Disease Day
•
NORD Corporate Council & EURORDIS Round Table of Companies
Conferences on Rare Diseases and Orphan Drugs:
•
• Europe
• USA
• Transatlantic
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•
NORD Gala Tribute Banquet & EURORDIS Gala Dinner
•
Online Social Networks: RareConnect
•
NORD-EURORDIS new collaboration on Patient Registries
•
Coordination in international policy affairs
NORD CORPORATE COUNCIL 15 MAY 2012
RARE DISEASE DAY
Rare Disease Day
 International awareness raising campaign
 Last day of February – the « rare » day
 Created by EURORDIS in 2008 with its
national alliances on rare diseases
 Worldwide Coordination by Eurordis
 Driven by patient advocacy groups
 To strenghten and expand the rare
disease communities
 To reach out to policy makers, media and
the public
 NORD coordinates in the USA
 CORD coordinates in Canada
 Etc
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NORD CORPORATE COUNCIL 15 MAY 2012
Rare Disease Day
Five Years of Experience:
2008 : Rare Diseases as a Public Health Priority
“A rare day for special people”
2009 : Patient-centred care
“Patient Care: A Public Affair!”
2010: Bridging Patients and Research
“Patients & Researchers: Partners for Life!’
2011: Rare Diseases and Health Inequalities
“Rare but Equal”
2012: Solidarity with People Living With Rare Diseases
“Rare but Strong Together”
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Rare Disease Day:Participating Countries
18 in 2008
30 in 2009
NORD joined in 2009
46 in 2010
55 in 2011
63 in 2012
25 official partners (Council of National Alliances)
Belgium, Denmark, France, Germany, Greece, Hungary, Ireland, Italy,
Netherlands, Romania, Spain, Sweden, UK, Bulgaria, Croatia,
Luxembourg, Portugal, Russia, Canada and USA.
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Rare Disease Day Video!
• Over 100,000 views
• Translated into 11 languages
• The video was “liked” on facebook 9,213 times
• Ranked 1st on YouTube in NGO category
• 100% pro bono from leading International PR company
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Video promotion
• Shown on several national and regional TV stations in Italy,
Portugal and Ireland
• International Rare Disease Day Website
• NORD’s RDD website
• EU Health Newsletter (on EC’s website)
• OrphaNews Europe
• National Alliances + European Federations + members and
hundreds of other websites
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Joining hands @ the world
Thousands of people responded to our call to Raise & Hold
hands to show Solidarity with RD patients
• 91 photos on rarediseaseday.org
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Some examples…
France
Event in Croatia
Medical students in Mexico
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Parliament in Ireland
Military in Portugal
Rare Disease Day Media Coverage
• Online press clipping –
over 3000 articles during the month
of February
1388 during the week of Feb 29
• CBS News.com
• Le Point
• The Examiner
• Huffington Post
• The Lancet
• Nature
• Gulf News.com
• AztecaNoticias
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Specific Policy Events
• Rare Disease Day Symposium
in Brussels on
“Rare but Strong Together”
• Launch of the Genetic Testing Registry
by the National Institutes of Health in the USA
• All day event at the National Institute of Health
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NORD Corporate Council
&
EURORDIS Round Table of Companies
EURORDIS Round Table of Companies
 35 US and European companies specialised in orphan drugs
and therapies for rare diseases
 2 workshops/ year; High level well prepared topics; break-out
sessions; several social networking opportunities
 70-90 representatives of industry, drug regulatory agencies,
patient groups, clinicians and academics
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THE EURORDIS ROUND TABLE OF COMPANIES
•
12th Workshop: 18th June 2010, Barcelona
“Retooling the EU Orphan Drug Regulation and US Orphan Drug
Act for Better and Faster Development of Rare Disease Therapies?”
•
13th Workshop: 10th December 2010, Paris
“Patients' Access to OMPs, Innovative Pricing Schemes and
National Measures in a Global Financial & Economic Crisis
Environment”
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THE EURORDIS ROUND TABLE OF COMPANIES
•
14th Workshop: 27 May 2011, Barcelona
“Mechanisms for the Implementation of the Clinical Added-Value of
Orphan Drugs (CAVOD) – Common Assessment of Relative
Efficacy, & Relative Effectiveness and Post-Marketing European
research Plans on Risk-Benefit-Effectiveness ”
•
15th Workshop: 21 November 2011, Paris
“Compassionate Use and Early Access Programmes”
21 NORD CORPORATE COUNCIL 15 MAY 2012
THE EURORDIS ROUND TABLE OF COMPANIES
•
16th Workshop: 1st March 2012, Brussels
"The Value of Partnering in Rare Disease Therapies: Benefits of
working with patients along the treatment lifecycle in the areas of
clinical trials, regulatory affairs and health technology assessment"
•
17th Workshop: 28th September 2012, Barcelona
"Determining the "Value" of Orphan Drugs"
22 NORD CORPORATE COUNCIL 15 MAY 2012
NORD GALA TRIBUTE BANQUET
&
EURORDIS GALA DINNER
EURORDIS GALA DINNER
Solidarity & Hope for Rare Disease Patients Throughout Europe
24 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS GALA DINNER
Solidarity & Hope for Rare Disease Patients Throughout Europe
The magnificent « THEATRE ROOM »
of the Royal Family PLAZA HOTEL
25 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS GALA DINNER
Solidarity & Hope for Rare Disease Patients Throughout Europe
A very elegant evening
Partners & Patrons Reception, Champaign & Oister Bar,
Afters of champaigns & coffees, gastronomic dinner,
International class Live Music Performance,
Photo & Video Exhibition, EURORDIS Awards
26 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS
GALA
DINNER
EURORDIS
GALA
DINNER
CONCEPT
Solidarity & Hope for Rare Disease Patients Throughout Europe
CO-CHAIRS GALA COMMITTEE
Eric Emmanuel Schmitt (Belgium & France)
Milan Macek (Czeck Republic & ESHG)
VICE-CHAIRS
Axel van der Mersch (Luxembourg)
Reem Boustany (Belgium & Lebanon)
MEMBERS
Peter Salstonstall (USA)
Franck Sasinowski (USA)
Elis Kakkis (USA)
Pat Mahaffy (USA)
Mark Kruger (USA)
Bo Jesper Hansen (Sweden)
Fabio Cavalli (Switzerland)
Riccardo Braglia (Switzerland)
27 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS GALA DINNER
• 250 attendees on-site
• High level politically
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Strong political message by President of the European Union
Council, Herman Van Rompuy
Honorary Patrons included 3 First Ladies + 2 Commissioners + 3
MEPs + 2 Ambassadors
In the presence of 1 First Lady, 2 Commissioners, 3 Ambassadors
+ other attendees of public offices
President of the Law Bar of Belgium
Owners and top executives of corporate sponsors
Leaders of scientific community
28 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS GALA DINNER
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EURORDIS GALA DINNER
30 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS GALA DINNER FUNDING
42 Organisations contributed, including 39 business, 1 foundation,
1 trade association and 1 patient organisation, from Europe (29),
USA (12) and Canada (1)
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50 000: Bayer, Shire
35 000: CSL Behring, GSK, EveryLife Foundation for Rare Diseases
26 000: Sigma Tau
20 000: Alexion, Celgene
15 000 – 16 000: Idis, Medunik Canada
10 000 – 11 000: Genzyme, Biogen Idec, BioMarin, CLOVIS Oncology, Ernst & Young,
Helsinn, Intermune, LFB Biomédiament, Orphan Europe
7 500: Amgen, Krueger & Assoc., Lysogene, SOBI
3 000 – 5 000: Amicus, Ariad, Enobia, Laboratoire CTRS, Merck Serono, Orfagen,
Pfizer, PTC Therapeutics, Synageva
500 – 2 700: Antigone, Cystinosis Foundation, Dompé, EuropaBio, Lucane, Medtronic,
Novartis, Prosensa, Raptor, Voisin Consulting
EURORDIS GALA DINNER
Conferences on Rare Diseases
and
Orphan Products
The Conferences on Rare Diseases
and Orphan Products
• An annual conference:

in the USA in October « US Conference on Rare Diseases and
Orphan products »

in EU in May « European Conference on Rare Diseases and
Orphan Products »
• A partnership EURORDIS-NORD-DIA
• A common brand, a common format, a coordinated content
• EURORDIS and NORD bring the focus and the transatlantic effect
• The DIA brings know-how, marketing, new audiences
• A format with a balanced mix of policy advocacy, information &
experience sharing, capacity building and networking
• Plenary sessions, parallel themes of several sessions, tutorials
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NORD CORPORATE COUNCIL 15 MAY 2012
6th European Conference on Rare Diseases
and Orphan Products 2012 Brussels
23-24-25 May 2012 in Brussels
Plenary + 600 people
Translation 6 languages in plenary room
(English, French, German, Dutch, Spanish, Russian)
36 additional Sessions
Spacious foyer/lunch/coffee break/poster area
34 NORD CORPORATE COUNCIL 15 MAY 2012
6th European Conference on Rare Diseases
and Orphan Products 2012
• Organised in collaboration with DIA Europe
• Partnership include all key stakeholders: European Commission (EC),
EU Committee of Experts on Rare Diseases (EUCERD), European
Medicines Agency (EMA) and its scientific committees (COMP, PDCO,
CAT, CHMP, PhVg), Industry (European Emerging Biotech of EFPIA and
EuropaBio), ORPHANET, NORD
• Themes: “Orphan Products”, “Research Policy”, “Health Policy”, “Social
Policy” of 2 to 6 sessions each, covering a broad range of 30 topics :
designation, marketing authorisation, clinical added value and
effectiveness, early access programmes, market access...to national
plans on rare diseases, population screening, diagnostic, registries,
health provision services, healthcare pathways, e-health....to
rehabilitation services, quality of life, training of healthcare
professionals
35 NORD CORPORATE COUNCIL 15 MAY 2012
6th European Conference on Rare Diseases
and Orphan Products 2012
• Tutorials for industry and for academia
• Poster sessions – 160 Selected
• Social Networking such as “Meet the EU regulators, National
Competent Authorities and HTA representatives”, “Meet the
EUCERD, the EU & national health authorities” etc
• Meeting Points by specific therapeutic area of common interest
or international networking etc
• On Day 1: Parallel workshops of European networks (Council of
National Alliances, Orphanet, Help Lines, Coordinators of
National Plans, etc) + EURORDIS Membership Meeting with
sessions of capacity building for patient advocates
• Broad dissemination: website, webcast, e-newsletters, report
36 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS
GALA
DINNER
TRANSATLANTIC
FORUM
EURORDIS
GALA
DINNER
Solidarity & Hope for Rare Disease Patients Throughout Europe
on Rare Diseases & Orphan Products 2013
• Croatia: Dubrovnik
• 30 – 31 May: Transatlantic Forum on Rare Diseases &
Orphan products
• 1 June : EURORDIS Membership Meeting
• Organised by: EURORDIS – NORD – CORD
• Co-organised by: DIA
• Partners (tbc):
EUCERD, EMA, NIH, FDA, Orphanet, EFPIA, EuropaBio,
Pharmaceutical Research Manufacturers of America (PhRMA),
Biopharmaceutical Industry Organization (BIO)
• Associates (tbc):
ICORD (?), World Health Organization (WHO), Council of Europe and the
Organisation for Economic Cooperation and Development (OECD)
37 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS
GALA
DINNER
TRANSATLANTIC
FORUM
EURORDIS
GALA
DINNER
Solidarity & Hope for Rare Disease Patients Throughout Europe
on Rare Diseases & Orphan Products 2013
• 200 – 300 participants
• 1/3 policy-makers, health professionals and academia, 1/3
patients and patients representatives and 1/3 industry
representatives
• Content: Forum of exchange to sustain a strategic collaboration
between both sides of the Atlantic (the EU and Canada/US) as
well as to position Rare Diseases on the international scene. A
policy / technical working meeting focused on concrete
objectives.
38 NORD CORPORATE COUNCIL 15 MAY 2012
EURORDIS
GALA
DINNER
TRANSATLANTIC
FORUM
2013
EURORDIS
GALA
DINNER
Solidarity & Hope for Rare Disease Patients Throughout Europe
on Rare Diseases & Orphan Products
• Format: 1,5 days comprised of one Plenary session + 10 parallel
sessions + an international Video Conference with participants
from Washington DC, Ottawa, London and Brussels. Specialised
satellite meetings for networks
• English only
• No tutorials
• No posters
• No exhibition
39 NORD CORPORATE COUNCIL 15 MAY 2012
RareConnect
Rare Disease Communities:
Rebranding by mid 2012
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• New Name, New Logo, New Graphic Identity
• www.rareconnect.org
• “Join the Global Conversation”
• Presence on eurordis.org & NORD website
• 21 Communities live
• 1544 registered Participants
• 191 partner Patient Organisations from 32 countries
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RareConnect: 21 Communities live
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RareConnect: Strategic Plan
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EURORDIS and NORD joint strategic plan developed in
partnership by staff of both organisations
Includes situation analysis and project components:

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Marketing strategy
Project implementation strategy
Technology plan
Fundraising
Budget
Governance
Project planning & narrative report completed
Design for rebranding completed
Budget being revised (ongo)
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NORD-EURORDIS
New collaboration on
Patient Registries