25-10-2012

1

Societeit “De Vereeniging” 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

25-10-2012

2

Advise Center Orphan Diseases

The Netherlands

Johan Hanstede,

Chairman Project Team Orphan Diseases

Strategic Advisor BioFarmind

Chairman Dutch Vaccines 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

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)

Foundation Advice Center Orphan Diseases (2013)

Project Team Orphan Diseases

Orphan Drug Project Team Eric van der Aa (Pantarhei Bioscience);

Roger Legtenberg (PSR-group);

Hans Schikan (Prosensa);

Paul Flapper (Shire Human Genetic Therapies);

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.

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

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

• Accelerating Time to Market

• Positive image OD

• Identify problems

• Potential threats

• Financial support

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

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