Agenda for RAREsummit19
MONDAY 23rd SEPTEMBER
08:30 UNTIL 17:30
followed by drinks and canapes reception 17:30 – 19:00
08:30 – 08:55
Registration and welcome refreshments
08:55 – 09:10
RAREsummit 19 Welcome Address
The power of a network to accelerate change
09:10 – 09:30
Keynote
25 years of rare diseases: Reflecting on the successes, challenges and hopes for the future
Alastair Kent, OBE – Expert in policy development and patient engagement for health and medical research in rare and genetic diseases
Morning Session
Patient centricity in rare diseases – Mastery, opportunities and trends in the drug development process
09:30 – 10:30
Panel Discussion
Patient groups partnering in the drug development process. Something for everyone?
Patient groups share their successes and the barriers which stifle progress
Dr. Anna Mingorance – CDO LouLou Foundation, Director Dravet Syndrome Foundation, Spain (Panel Facilitator)
David Bull – CSO, Duchenne UK
Dr. Paul Wicks – Independent Consultant, Wicks Digital Health Ltd
Tanya Collin-Histed – CEO, International Gaucher Alliance
Allison Watson – Co-founder Ring20 Research and Support UK CIO/EPAG EpiCARE ERN
Carina Thurgood – Co-founder and CEO, Maddi Foundation for SPG15
10:30 – 11:10
Moderated Discussion
Disruptive technologies – Patients at the heart of AI.
A vision of 21st century digital health where technologies empower patients to manage their own health, find treatments and be connected
Dr Tim Guilliams – Founder of CamRARE and CEO and co-founder of Healx, AI-powered drug discovery for rare diseases (Moderator)
Dr. Andy Richards – Digital Health Entrepreneur, healthcare and life sciences chairman and director
Dr. David Brown – Co-Founder, Chief Scientific Officer and Chairman, Healx
Elin Haf Davies – CEO, Aparito
Dr. Pete Chan – Head of Research and Analysis, Raremark
11:10 – 11:30
Refreshment break
11:30 – 12:30
Moderated Discussion
Disruptive Innovation and Transformation – Patients at the heart of the drug development process
Industry & patient’s future vision
Dr. Joanna Segieth – Founder of Rare Drug Development Solutions Biosynetix Ltd (Moderator)
Prof. Chas Bountra – Prof. of Translational Medicine, University of Oxford Steve Rees – Vice-President of the Discovery Biology, AstraZeneca
Dr. Daniel O’Connor – Medical Assessor, Medicines and Healthcare products Regulatory Agency, MHRA
Neil Dugdale – VP & GM, UK and Rol, Sobi
Thomas Ogorka – CEO, Orphan Reach, Specialist Rare Clinical Research Organisation
Dr. Nicolas Sireau – CEO and Chair, AKU Society
12:30 – 13:40
Networking lunch
Browse company and charity exhibits and the patient journey poster gallery
Afternoon Session
Co-creators: Where patients become vital and responsible partners with professional teams
13:40 – 14:05
Short Talk
Co-creating genetic reports that are understood by non-specialists
Dr. Gabriel Recchia – Research Associate, Winton Centre for Risk and Evidence Communication, University of Cambridge
Dr. Gemma Chandratillake – E&T Lead at the East Midlands & East of England Genomic Laboratory Hub
Menna Hawkins -Polyposis Nurse Specialist
14:05 – 14:30
Short Talk
Patients as partners in assistive technology design – Collaboration and customisation is the key to success
Dr. Cecily Morrison – Microsoft Research Cambridge
Dr. Sarah Leiter – CamRARE trustee, junior doctor Addenbrookes CUH and albinism advocate
14:30 – 14:55
Short Talk
“Peer-Led to get ahead!” – Developing an education and self-management programme for and with young people affected by haemophilia
Laurence Woollard – Founder and Director of On the Pulse Consultancy and living with haemophilia
14:55 – 15:05
Short Talk
What matters to you? What matters most? We need a national debate on rare diseases to offer the best possible care
Baroness Nicola Blackwood introduces the hackathon challenge and invites delegates to share their priorities for the UK Rare Disease Strategy post 2020 framework
Baroness Nicola Blackwood – Parliamentary Under Secretary of State at the Department of Health and Social Care
15:05 – 15:25
Break & moving into groups
15:25 – 16:25
Cross sector hackathon activity
Delegates work in teams facilitated by speakers and CamRARE staff to collaboratively agree on their priorities for the UK Rare Disease Strategy framework 2020
16:25 – 16:40
Comfort break
16.40 – 17:30
Team pitches, voting, closing address and thoughts from the day
Using Glisser event tech to vote using devices and Q+A with delegates
17.30 – 19:00
Drinks and canapes networking reception
08:30 – 08.55
Registration and welcome refreshments
08:55 – 09:10
RAREsummit19 Welcome Address:
The power of a network to accelerate change
09:10 – 09:30
Keynote
25 years of rare diseases: Reflecting on the successes, challenges and hopes for the future
Alastair Kent, OBE – Expert in policy development and patient engagement for health and medical research in rare and genetic diseases
Morning Session
Patient centricity in rare diseases – Mastery, opportunities and trends in the drug development process
09:30 – 10:30
Panel Discussion
Patient groups partnering in the drug development process. Something for everyone? Patient groups share their successes and the barriers which stifle progress
Dr. Anna Mingorance – CDO LouLou Foundation, Director Dravet Syndrome Foundation, Spain (Panel Facilitator)
David Bull – CSO, Duchenne UK
Dr. Paul Wicks– Independent Consultant, Wicks Digital Health Ltd
Tanya Collin-Histed – CEO, International Gaucher Alliance
Allison Watson – Co-founder, Ring20 Research and Support UK CIO/EPAG EpiCARE ERN
Carina Thurgood – Co-founder and CEO, Maddi Foundation for SPG15
10:30 – 11:10
Moderated Discussion
Disruptive Technologies – Patients at the heart of AI. A vision of 21st century digital health where technologies empower patients to manage their own health, find treatments and be connected
,Dr. Tim Guilliams – Founder of CamRARE and CEO and co-founder of Healx, AI-powered drug discovery for rare diseases (Moderator)
Dr. Andy Richards – Digital Health Entrepreneur, healthcare and life sciences chairman and director
Elin Haf Davies – CEO, Aparito
Dr. Pete Chan – Head of Research and Analysis, Raremark
Dr. David Brown – Co-Founder, Chief Scientific Officer and Chairman, Healx
11:10 – 11:30
Refreshment break
11:30 – 12:30
Moderated Discussion
Disruptive Innovation and Transformation – Patients at the heart of the drug development process
Industry & patient’s future vision
Dr Joanna Segieth – Founder of Rare Drug Development Solutions Biosynetix Ltd (Moderator)
Prof. Chas Bountra – Prof of Translational Medicine, University of Oxford
Dr Daniel O’Connor – Medical Assessor, Medicines and Healthcare products Regulatory Agency, MHRA
Neil Dugdale – VP & GM, UK and Rol, Sobi Steve Rees – Vice-President of the Discovery Biology, AstraZeneca Thomas Ogorka – CEO, Orphan Reach, Specialist Rare Clinical Research Organisation
Dr. Nicolas Sireau – CEO and Chair, AKU Society
12:30 – 13:40
Networking lunch
Browse company, and charity exhibits and patient journey poster gallery
Afteroon Session
Co-creators: Where patients become vital and responsible partners with professional teams
13:40 – 14:05
Short Talk
Co-creating genetic reports that are understood by non-specialists
Dr. Gabriel Recchia – Research Associate, Winton Centre for Risk and Evidence Communication, University of Cambridge
Dr. Gemma Chandratillake – E&T Lead at the East Midlands & East of England Genomic Laboratory Hub
Menna Hawkins -Polyposis Nurse Specialist
14:05 – 14:30
Short Talk
Patients as partners in assistive technology design – Collaboration and customisation is the key to success
Dr Cecily Morrison – Microsoft Research Cambridge
Dr. Sarah Leiter – CamRARE trustee, junior doctor Addenbrookes CUH and albinism advocate
14:30 – 14:55
Short Talk
“Peer Led to Get Ahead” – Developing an education and self-management programme for and with young people affected by haemophilia
Laurence Woollard – Founder and Director of On the Pulse Consultancy and living with haemophilia
14:55 – 15:05
Short Talk:
What matters to you? What matters most? We need a national debate on rare diseases to offer the best possible care.
Baroness Nicola Blackwood introduces the hackathon challenge and invites delegates to share their priorities for the UK Rare Disease Strategy post 2020 framework
Baroness Nicola Blackwood – Parliamentary Under Secretary of State at the Department of Health and Social Care
15:05 – 15:25
Break & moving into groups
15:25 – 16:25
Cross sector hackathon activity
Delegates work in teams facilitated by speakers and CamRARE staff to collaboratively agree on their priorities for the UK Rare Disease Strategy framework 2020
16:25 – 16:40
Comfort break
16.40 – 17:30
Team pitches, voting, closing address and thoughts from the day
Using Glisser event tech to vote using devices and Q+A with delegates
17.30 – 19:o0
Drinks and canapes networking reception