The document outlines plans for the third Orphan Café event organized by the Project Team for Rare Diseases. It will feature presentations on the Advice Center for Rare Diseases and the National Plan for Rare Diseases in the Netherlands. Nicole Kien will also speak about patients with rare diseases, reimbursement, trends, and case studies. The event aims to provide information and networking opportunities for participants. Additionally, the document discusses the formation of an Advice Center for Rare Diseases to serve as a central hub for information and support regarding rare disease activities, research, and development in the Netherlands.

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