AGENDA DAY TWO

 

Day 2 Orphan 15th Nov

Panel discussion
Emily Crossley
09:30

Case study: Moving Mountains: how one parent led charity raised and spent millions, accelerating research and impacting the landscape

  • Developing innovative and holistic funding model to accelerate trials in rare disease
  • Leveraging investment model to bridge the “valley of death”
  • How we did it
Frederic Revah
09:55

Introducing a new industrial player emerging from a patient organisation, for the industrialisation of cell & gene therapies to be commercialised at a fair price

 
  • Materializing research outcomes into products
  • The role of a patient group, AFM-Telethon from innovation to production
  • Ability to provide production to pharma and biotech
  • Using new models to give a fair opportunity to all stakeholders
10:20

Morning Networking Break

11:20

INTERACTIVE ROUNDTABLES

Covering a number of hot topics and delivered by patient groups, industry and leading solution providers, choose which ONE roundtable discussion you would like to join, for ‘off the beaten track’ learning and information you can’t find online. The session will last 45:


REGIONAL PROVISIONS FOR ORPHAN DRUGS roundtables

Developments in Eastern Europe: Unique challenges and opportunities
Pawel Wozniak, Managing Director, Komtur Polska

A landscape analysis of Rare Diseases and Orphan Drugs availability in South Eastern Europe

Anja Kladar, Rare Diseases Croatia, Executive Director

The future of a fully integrated multi-stake holder model – what can we learn from USA advocate perspective?

Debbie Drell, Director of Membership, NORD

Differences in OD development and registration in the Nordic markets

Einar Andreassen, Senior Adviser, Norwegian Medicines Agency
Dr Douglas Lundin, Chief Economist, TLV, Dental and Pharmaceutical Benefits Agency, Sweden


Linking registries and clinical trials through ERNs:  The paradigm shift in meeting Marketing Authorization requirements
George Reynolds, COO, Vitro Software Int

PATIENT ENGAGEMENT roundtables

ADCY5-related Dyskinesia case study: how research and clinical advancements occur through engagement of the rare disease patient community

Christina Waters, CEO and Founder, RARE Science

How we can ensure rapid development of gene therapies for rare diseases by working with patients and patient organisations?

Emily Culme-Seymour, External Strategy Manager, Gene Therapy, GSK

GOSgene: enabling innovation through gene discovery. Application of precision medicine within rare disease development

Dr Hywel Williams, Senior Research Associate, The Centre for Translational Omics – GOSgene, Genomics and Systems Medicine, UCL, GOS Institute of Child Health
 

A guide to effective patient submissions for HTA processes
Sandra Nestler-Parr, Director of Rare Access and as Trustee of Alpha-1 UK Support Group
Sheela Upadhyaya, Associate Director Highly Specialised Technologies, NICE

How to develop a platform to unite clinical research on paediatric RDs
Beygonya Nafria Escalera, Patient Advocacy Manager, Sant Joan de Deu Children's Hospital

First-hand experience as a patient representative: Making a difference from trial design to access negotiations in orphan drugs
Dr Jasmin Barman, Scientific Advisor, Swiss Society for Porphyria
 
12:05

Networking Lunch & Patient Poster Session

How Collaborative Research is Translated into Products

Pitch to Potential Partners & Funders

Tackling Commercialisation Hurdles

Precision Medicine and Clinical Advancements

SCIENCE & STRATEGY
14:35

The potential of gene editing therapies for orphan drugs: Harnessing CRISPR technology for rare disease drug development

  • The current state of CRISPR gene editing technology  
  • Employing revolutionary gene editing technology to treat monogenic rare diseases
  • The opportunities and the challenges 
GENE THERAPY
15:05

Advances in genetics for rare diseases: Is gene therapy the best way forward?

SCIENCE & STRATEGY
15:05

Making the drug development process better and more efficient for patients through biomarker development and precision medicine

15:35

Networking refreshment break

Gene Therapy and the Patient

Paediatric Advancements

PITCH & PARTNER
16:05

Pitch & Partner 5

PITCH & PARTNER
16:20

Pitch & Partner 6

PRODUCT DEVELOPMENT
16:35

Paediatric drug development: The interaction between industry, physicians and patient groups to get the drug into the market and subsequent P&R approval


Senior representative, Novartis
PITCH & PARTNER
16:35

Pitch & Partner 7

PITCH & PARTNER
16:55

Pitch & Partner 8

17:05

Chair closing remarks

17:10

End of Congress

last published: 10/Nov/17 16:05 GMT