Day 1 Day 2
DAY ONE – Wednesday 1 April 2020

08:15

09:00

Registration, breakfast, coffee and exhibition

09:00

09:05

Welcome address

Laura Dormer, Editorial Director, Future Science Group

09:05

09:20

Opening remarks

Conference Chair: Charles Makin, Global Head Real-World Evidence Strategy, Biogen

09:20

09:50

Opening keynote conversation

Understanding the human angle – the impact of rare diseases: my family’s story

Jean-Christophe Novelli

09:50

11:05

Panel conversation: co-ordination and collaboration: rare diseases across Europe

  • Supporting national plans for rare diseases
  • Implementing the UK Strategy for Rare Diseases: are we meeting the 2020 deadline?
  • Harnessing real-world evidence to facilitate earlier diagnosis and intervention
  • Understanding the impact of the 100,000 Genome Project

Speakers:

Alastair Kent, Independent

Joanne Hackett, CCO, Genomics England

Eric Low, Eric Low Consulting

11:05

11:35

Morning coffee and exhibition

LISTEN & LEARN ZONE

CONVERSATION & COLLABORATION ZONE

Regulatory tools and pathways

11:35

12:05

Handling real-world evidence to support conditional access to treatments: the NICE experience

Speaker: Brad Groves, Associate Director, Managed Access, The National Institute for Health and Care Excellence (NICE)

Roundtable conversations – a choice of concurrent conversations, designed to enable collaboration

12:05

12:35

Why real-world evidence matters to payers: managing uncertainty and facilitating novel payment models

Speaker: Lotte Steuten, Vice President & Head of Consulting, Office of Health Economics

12:35

13:05

Responding to regulatory requests with data generation

Speaker: Chris Knight, Senior Director, Health Economics, RTI Health Solutions

13:05

14:10

Lunch and exhibition

 LISTEN & LEARN ZONE

CONVERSATION & COLLABORATION ZONE

Understanding reimbursement

14:10

14:30

Handling real-world evidence to support reimbursement

  • Overcoming the challenges of reimbursement decisions in rare diseases
Roundtable conversations – a choice of concurrent conversations, designed to enable collaboration

14:30

15:00

Gaining clarity on real-world evidence and payer models across Europe

  • What is the process for rare diseases and orphan drugs?

Speaker: Paolo Morgese, EU Director of Market Access and Member Relations, Alliance for Regenerative Medicine

15:00

15:40

Afternoon tea and exhibition

15:40

16:10

Post-launch data collection for the purpose of ATMP reimbursement

Speaker: Panos Kefalas, Head of Health Economics and Market Access, Cell and Gene Therapy Catapult

Roundtable conversations – a choice of concurrent conversations, designed to enable collaboration

16:10

16:40

Policy and cost–effectiveness in rare diseases: how should we assess value?

Speaker: Christian Hill, CEO, MAP BioPharma

16:40

16:55

Closing questions

17:00

18:30

Drinks reception

 

DAY TWO – Thursday 2 April 2020

08:15

09:00

 Registration, breakfast, coffee and exhibition

09:00

09:20

Welcome address

Laura Dormer, Editorial Director, Future Science Group

09:20

09:50

Big data, genomics and trust – making the UK the centre for health data science

09:50

10:35

Panel conversation: regulation and access to treatments for rare diseases: an industry perspective

Speaker:

Leslie Galloway, Chairman, Ethical Medicines Industry Group

10:35

11:05

Morning coffee and exhibition

LISTEN & LEARN ZONE

CONVERSATION & COLLABORATION ZONE

Patients and transparency

11:05

11:35

Effective strategies for locating patients using real-world evidence

Speaker: Maya Zlatanova, Co-Founder & CEO, FindMeCure

Roundtable conversations – a choice of concurrent conversations, designed to enable collaboration

11:35

12:05

Achieving transparency in the generation and use of real-world evidence

Speaker: Richard White, Chief Operating Officer, Oxford PharmaGenesis Ltd.

12:05

12:35

The use of RWE across the medicine lifecycle: benefits, challenges and solutions

Speaker:

Amanda Cole, Principal Economist, Office of Health Economics

12:35

13:05

Transforming health care through healthcare data

13:05

14:10

Lunch and exhibition

LISTEN & LEARN ZONE CONVERSATION & COLLABORATION ZONE
Session title TBA

14:10

14:40

Collaboration and transparency: creating a platform for developing data sharing across Europe for orphan drugs

Speaker: Karen Facey, Senior Research Fellow, IMPACT HTA, Co-Lead Investigator, WP10 Appraisal of Orphan Medicinal Products

Roundtable conversations – a choice of concurrent conversations, designed to enable collaboration

14:40

15:10

Next-generation health technology assessment: supporting patient-centred, real-time decision making

Speaker: Dalia Dawoud, Scientific Adviser, Science, Policy and Research Programme, NICE

15:10

15:40

Afternoon tea and exhibition

15:40

16:10

Drug repurposing for rare diseases

Speaker: Rick Thompson, CEO, Findacure

Roundtable conversations – a choice of concurrent conversations, designed to enable collaboration

16:10

16:30

Fireside chat: topic TBA

Charles Makin, Global Head Real-World Evidence Strategy, Biogen

16:30

17:00

Closing keynote: Next steps in real-world evidence and rare diseases

Speaker: Charles Makin, Global Head Real-World Evidence Strategy, Biogen