World Orphan Drug Congress 2018 - Agenda Day 2

Day 2, Nov 8


Karen Facey

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

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

Recommendations on sustainable access to innovative therapies

  • 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

Morning Networking Break

Cell & Gene Therapy

Chair’s opening remarks

Science & Strategy

Chair’s opening remarks


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


Manufacturing & Commercialization of Cell & Gene Therapies


Networking Lunch & Patient Poster Session


Networking refreshment break


Diego Ardigo

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?

  • 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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