World Orphan Drug Congress 2018 - Agenda Day 2

Day 2, Nov 8

OPENING KEYNOTE PLENARY

Karen Facey
08:35

A pan European study: The realities of orphan drug access

  • How do European countries differ in their value assessment to new orphan drugs?
  • What next steps are needed for more harmonized, comparable and rational evidence based decision-making in Europe?
Followed by live feedback from HTAs:Dr Karen Facey, Member, Scottish Health Technologies Group & Impact HTA - Co-Lead Investigator for WP10 - Appraisal of Orphan Medicinal ProductsEdmund Jessop, Medical Adviser, NHS EnglandSophia Brodin, Deputy Head of Department, Department of Value Based Pricing, TLV, The Dental and Pharmaceutical Benefits AgencySenior representative, H-AS, France
Ad Schuurman
09:25

Speaking from experience, expedited regulatory pathways in gaining EMA PRIME examples – Where do we go from there?

  • Paving the way for biotechs to gain approval and access
  • Recent changes and future amendments to regulatory issues surrounding orphan drugs approval
  • How are companies interacting the payers and how to close the gap to allow treatments to reach patients
Discussion with payer and PRIME designated companies
Nathalie Moll
10:10

Recommendations on sustainable access to innovative therapies

Presentation:
  • Ensuring the sustainability of health spendingas well ascontinued innovation that meets patient needs
  • How does the current pricing system need to change to compliment this?
  • Are novel pricing & reimbursement mechanisms for gene therapy products necessary?
Followed by feedback from global stakeholders on value vs pricing and affordability
  • Are we paving the way to a fair, inclusive and on-going multi-stakeholder approach with the potential to generate sustainable, affordable and actionable improvements in patient access to rare disease therapies?
  • Balancing between recouping costs for R&D and incentives to support investment in future treatments that ultimately access to patients
10:55

Morning Networking Break

Cell & Gene Therapy
11:55

Chair’s opening remarks

Science & Strategy
11:55

Chair’s opening remarks

Manufacture
11:55

Chair’s opening remarks

Clinical Development

Product Development

Approval & Access

Global Market Access

Cell & Gene Therapy

Pricing & Reimbursement

Science & Strategy

The Business of Orphan Drugs

Manufacture

Manufacturing & Commercialization of Cell & Gene Therapies

13:00

Networking Lunch & Patient Poster Session

15:30

Networking refreshment break

CLOSING PLENARY SESSION

Diego Ardigo
16:00

IRDiRC: Enabling all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention. How do evolve to achieve target?

Presentation:
  • The IRDiRC goals, a community effort for a better future in rare disease research and clinical management and a call for international collaboration to an unprecedented scale
  • How are we going to achieve quicker diagnosis for 7000+ diseases and to solve unsolved diagnostic cases?
  • How are we going to deliver the best possible care and give access to all available therapies to rare disease patients?
  • What change in pace do we need to develop new treatments for thousands of rare diseases?
  • Summary of learnings from the congress and targets for next year
Followed by panel discussion on; how far we are from the IRDiRC vision and how much ambitious its goals are& what we need to change in the system to achieve these goals and vision

Chair’s Closing Remarks & Close of congress

last published: 17/Oct/18 12:05 GMT

 

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