KOL and Payers' seminar

KEY OPINION LEADERS' SEMINAR

08:00 Registration and networking breakfast

08:55

Opening remarks

Jennifer Kwon
09:00

Improving and standardizing the care of children with rare diseases that were identified through newborn screening

Adeline Vanderver
09:20

Creating standards of care for patients and spearheading pre-clinical and clinical research projects to improve diagnosis for leukodystrophy

Diana Bharucha
09:40

The role of natural history studies in clinical trial readiness for rare diseases – the Giant Axonal Neuropathy case

James Wilson
10:00

The age of gene therapy; the impact on cystic fibrosis and other rare disorders

10:20 Networking coffee break

Florian Eichler
11:00

Creating an accelerated research environment for rare neurological diseases

Samuel Seward
11:20

Driving research and accelerating treatments for patients with Hermansky- Pudlak syndrome (HPS)

Jerry R. Mendell
11:40

Gene therapy and other novel treatment strategies for Inherited muscle and nerve disorders

12:00 NETWORKING LUNCH

PAYERS’ SEMINAR

13:00

Opening remarks

Peter Dehnel
13:05

Alternative funding methods, that will spur innovation and not rely solely on payers for the reimbursement of orphan drugs

Jeff Myers
13:25

Medicaid state overview and the impact on orphan drug reimbursement in the post-election era

Invited: John Coster
13:45

Current landscape for tackling increased drug costs through more reasonable reimbursement

Edmund Jessop
14:05

Ultra-orphan drugs: the NHS model for managing extremely rare diseases

2:25 Networking coffee break

Diane Kleinermans
15:00

Belgium, Netherlands Luxemburg and Austria together in orphan drug assessment

Anna Bucsics
15:20

Pricing and reimbursement challenges to access European markets and ways to present value and evidence to payers and regulators

Einar Andreassen
15:40

Creating true value for patients- severity as a measurement to prioritize reimbursement of orphan drugs in Norway

Detlev Parow
16:00

Proposed new approaches for the affordability of orphan drugs and the sustainability of the healthcare system in Germany

David Elvira Martinez
16:20

Improving access to rare disease therapies: lessons learned from the Catalan experience

David Elvira Martinez, Director, CatSalut, Catalunya Health Service

4:40 Welcome drinks reception

17:20 End of Pre-conference day

last published: 24/Mar/17 14:35 GMT