Orphan Café, 18 oktober 2012 Presentation by Johan Hanstede (DutchCC)

1. 25-10-2012
1
Societeit “DeVereeniging” Utrecht
Het Orphan Café wordt voor de derde maal georganiseerd
door het Projectteam Zeldzame Ziekten en is
mogelijk gemaakt door de steun van:
Programma
 15.00 inloop
 15.30 Programma
 Johan Hanstede
update over het Adviespunt Zeldzame Ziekten.
 Frits Lekkerkerker
Toelichting op het Nationaal Plan Zeldzame Ziekten
 Nicole Kien
spreekt over patiënten met zeldzame ziekten,
vergoedingen,trends en casuïstiek.
17.00 uur Netwerkborrel

2. 25-10-2012
2
Advise Center Orphan Diseases
The Netherlands
Johan Hanstede,
Chairman ProjectTeam Orphan Diseases
Strategic Advisor BioFarmind
Chairman DutchVaccines Group
Director DutchCC
Overview of the current situation
• Pharmaceutical imago
• Price Transparency and CE Orphan Drugs
• Cost of Care reduction
• Pharmacy Manufacturing
• Public Development Orphan Drugs
• Public commitment towards orphan diseases’
• Particular treatment. How long particular?
• Transfer of orphan drugs to hospital budget
Identification of key ongoing issues in our market

3. 25-10-2012
3
Ongoing developments and activities
 National Plan Orphan Diseases Netherlands (2013)
Sounding board group Orphan Diseases
 Expert Centers (2013)
Dutch Federation of University Medical Centers (NFU)
 FoundationAdvice Center Orphan Diseases (2013)
ProjectTeam Orphan Diseases
Orphan Drug Project Team
 Eric van der Aa (Pantarhei Bioscience);
 Roger Legtenberg (PSR-group);
 Hans Schikan (Prosensa);
 Paul Flapper (Shire Human GeneticTherapies);
 Bert de Jong (Genzyme);
 Peter Bertens (Nefarma);
 Annemiek Verkamman(BioFarmind);
 Marcel Gerritsen (BioFarmind);
 Margaret van der Meijden (BPRA)
 Jolanda Huizer (NPZZ/ ZonMw);
 Johan Hanstede Chairman.

4. 25-10-2012
4
“ Advice Center Orphan Diseases”
 Central advice center For Business,Research
Centers, Patiënt organizations and others
 Monitoring OD activities (Clinical and R&D)
 Questions & Answers
 Portal as spin in a virtual web
 Finding the Experts
 Financial opportunities
 Advice Center will be part of the National Plan
OD Netherlands
Advice Center Orphan Diseases
R&D
TRM
Universities
Institutes
Companies
Media
Patients
Society
Authorities
Politics
Portal site
OD Network
OD Registry
OD Monitor
Training & Education
Signaling Function
Communication

5. 25-10-2012
5
Critical Success Factors
 Independent organization
 Broad support
 Strong Infrastructure
 Continuity
 Committee of Recommendation
 Professional support OD Portal
 Social image Orphan Drug development
 Joining forces
Future potential outcomes
• Start with Advice Center will lead to
• AcceleratingTime to Market
• Positive image OD
• Identify problems
• Potential threats
• Financial support

6. 25-10-2012
6
Planned activities and next steps
2012/2013 Oct Nov Dec Jan Febr Mrt Apr May June July Aug Sept Oct Nov Dec
General
Orphan Café X X X X
Launch Symposium X
Sentiments monitor X
Orphan Disease Registry
ACZZ Business Plan X
Media & Com. Plan X
Training & Educational Plan X
E-learning Plan X
Organization
Organization structure X
Governance & Board X
Consortium & OD Network X
Funding
ZonMw Subsidary X
Innovation Contract LS&H
Crowd & Charaty
Letters of Intent (LOI)
Letters of Support (LOS) X
Portal site
Website Phase I
Website Phase 2
Website Phase 3 X

7. 25-10-2012
7
Projectteam Orphan Diseases
Information / support
J.G.Hanstede,chairman
E-mail info@hanstede.nl
Mobile 06 5152 6780
Programma
 15.00 inloop
 15.30 Programma
 Johan Hanstede
update over het Adviespunt Zeldzame Ziekten.
 Frits Lekkerkerker
Toelichting op het Nationaal Plan Zeldzame Ziekten
 Nicole Kien
spreekt over patiënten met zeldzame ziekten,
vergoedingen,trends en casuïstiek.
17.00 uur Netwerkborrel