Mechanism of coordinated access to orphan drugs

Ri De RidderDirector-General of the Health Department

National Institute for Health and Disability Insurance (Belgium)

m mechanism

o of

c coordinated

a access

(to orphan drugs)

24-09-2010

commission vice president

Antonio Tajani

announces

the launch of

a process

on corporate responsibility

in the pharmaceutical sector

3 platforms

ethics and transparency

access to medicines in Africa

access to medicines in Europe

process on Corporate Responsibility

in the field of Pharmaceuticals

platform on access to medicines in Europe

MECHANISM OF COORDINATED ACCESS

TO ORPHAN MEDICINAL PRODUCTS

MoCA

14 member states

patients

doctors

industry

European commission

Europe

COMP, ENTR, MARKET, SANCO

Member States

Austria, Belgium, Estonia, Finland, France, Greece, Hungary, Italy, Malta, Netherlands, Poland, Portugal, Spain, Sweden

Patients

AIM, EPF, ESIP, EurordisHealth Care Providers

CPMEIndustry

EFPIA, EGA, EuropaBio,

GIRP

EMINet

the partners

why

MOCA?

OD Cover Europe 2010

0.00%

10.00%

20.00%

30.00%

40.00%

50.00%

60.00%

70.00%

80.00%

90.00%

100.00%

%OD Cover (#62 OD)

Number OD vs GDPsource Euripid

0

10

20

30

40

50

60

70

PIB/inh

Number OD/62

Fabry’s Disease – OD – price

2,800.00

3,300.00

3,800.00

Sweden Denmark Austria Belgium Cyprus Finland Norway Spain CzechRepublic

Slovakia Hungary

Fabrazyme (p/U)

Moyenne

-

5.000

10.000

15.000

20.000

25.000

30.000

35.000

40.000

45.000

-

50.000

100.000

150.000

200.000

250.000

300.000

350.000

400.000

450.000

500.000

14 Orphan Drugs

Sales 2010

myozyme

-

5.000

10.000

15.000

20.000

25.000

30.000

35.000

40.000

45.000

-

10.000

20.000

30.000

40.000

50.000

60.000

70.000

Pompe disease

nexavar

-

5.000

10.000

15.000

20.000

25.000

30.000

35.000

40.000

45.000

-

10.000

20.000

30.000

40.000

50.000

60.000

70.000

80.000

90.000

100.000

renal cell carcinoma

no expertise in

Belgium for

aldurazym file assesment

found in the Netherlands

huge time loss

in defining a

postmarketing study

for flolan in context

of managed entry agreement

optimize investments

by avoiding duplication

by mapping and coordination

by capacity building

by sharing resources

create guidance

“roadbook”

launch a project

coordination between stakeholders and Member States at

EU level

to provide real access to real solutions (orphan medicinal

products )

for real patients with real unmet medical needs,

for which these solutions would otherwise be out of reach

in an affordable and sustainable way (-> “real life

access”).

the project – key concepts - vision

some key issues

horizon scanning

early dialogue

early access programmes

clinical data collection

identifying added value

through collaboration / coordination

mapping existing coordination mechanisms

propose mechanisms for identified gaps

project report

expected for

early november