The next 1 0 years A regulatory view Presented by: Jordi Llinares - - PowerPoint PPT Presentation

▶

Nov 27, 2023 92 likes •211 views

The next 1 0 years A regulatory view Presented by: Jordi Llinares Head of Orphan Medicines An agency of the European Union Some headlines I hope I never see (and I am not playing with fear politics) EU bureaucrats kill patients hope

SLIDE 1

An agency of the European Union

Presented by: Jordi Llinares Head of Orphan Medicines

The next 1 0 years

A regulatory view

SLIDE 2

Presentation title (to edit, click View > Header and Footer) 2

Some headlines… I hope I never see (and I am not playing with fear politics)

EU bureaucrats kill patient’s hope Greedy industry push health system to the limit National budgets insensitive to rarity National budgets insensitive to rarity Patient’s Icarus flight

SLIDE 3

Presentation title (to edit, click View > Header and Footer) 3

The future

It is on us, depends on our actions today
Individual responsibility
Aim shared by different stakeholders

Even though we have different short term objectives Responsibility to build on something that already works Different stakeholders to play different roles

keep pushing, but safely

SLIDE 4

Presentation title (to edit, click View > Header and Footer) 4

Regulator’s responsibilities / opportunities ( I )

a. Keep balance between development chances and

data required (at designation and authorisation)

b. Wise management of challenges
Sub setting policy (consistency and communication)
Biological advances and application for designation
c. Transparency
Better communication on designation and SB

– collaboration with Member states and HTA bodies

SLIDE 5

Presentation title (to edit, click View > Header and Footer) 5

Regulator’s responsibilities / opportunities ( I I )

d. Further assistance / incentives
Pre designation assistance (?)
e. Advice to EC : full implementation of the COMP role

(research and policy)

f. Monitoring orphan drugs:
Development
After marketing: registers, shortages of supply

SLIDE 6

Presentation title (to edit, click View > Header and Footer) 6

The future as I see it (I)

Debate about regulation (not future anymore)
Need for in depth assessment
Legal advice (amendment?; to open all or part of text?)
What needs change
Risk assessment
Increased transparency
Procedural / outcome
Development (clinical trials)

SLIDE 7

Presentation title (to edit, click View > Header and Footer) 7

The future as I see it (II)

Increased international collaboration
Development
Post marketing issues
Consolidation of EU structure on rare diseases
Will strengthen rare diseases position
Opportunity to fight back misconceptions

SLIDE 8

Presentation title (to edit, click View > Header and Footer) 8

The people / resources

We will need dedicated people (like you) Ideas and challenges Continuous support to Agency and fee reduction policy Collaboration, discussion and more collaboration

SLIDE 9

Presentation title (to edit, click View > Header and Footer) 9

And a big thanks to all those at the Agency that made this possible… .

Patrick Melanie Francesco Spiros Agnes Bo Channa Teresa Marisa Monica Johanna Sandra Eric Frida Paolo Thomas Marco Sarah Cathrin Driss and more … Federica Juan Florence Cinzia Agnieszka Stiina Stelios