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RAREsummit21 Agenda – Draft

Live programme for 7th October 2021

Cambridge Rare Disease Network - RAREsummit21 Agenda - Draft 1

photo credit, Martin Bond, A Cambridge Diary

Talks, panel discussions, fireside chats, Q&A, workshops, live pitching, networking

TimeTopic & session formatSpeakersSession description
08.30Orientation
Virtual platform opens

Familiarise yourself with the venue, visit exhibition stands, preview the gallery and introduce yourself in the chatrooms

0850

Welcome address

CRDNCircling back and looking forwards
CRDN celebrates its 5th RAREsummit. We'll be exploring what progress has been made and what the vision is for the future of rare diseases
09:00Live panel
Rare and Equitable
Facilitator - Nicola Miller, Rare Revolution Magazine
Kerry Leeson-Beavers, Breaking Down Barriers
Other speakers TBC
Addressing inequalities in rare diseases in regards to equality, diversity and inclusion
9:40Live Talk with Q&A
Rare Disease Health Information Delivered Well
Dr. Tim Ringrose - CEO & Co-founder, The Cognitant Group
Patient advocate speaker  - TBC

Providing clear reliable knowledge enabling informed decisions
Exploring the challenges of developing trustworthy, reliable, accessible health information on rare diseases.  How do patients like their health information delivered?  How can the right information allow them to become actively involved in improving their health, and enabling healthcare professionals to optimise their care? 

10.20Networking, exhibition zone and gallery explorationMeet and mingle live with attendees, exhibitors and poster creators
10.50

Live Talk with Q&A
Can We Successfully Run Clinical Trials Without Placebos?

Femida Gawdry-Sridhar - Founder and CEO, Pulse Infoframe
Daniel Lewi - Head of Business Development and Patient Advocacy, Pulse Infoframe and Co-Founder, Cure & Action for Tay-Sachs (CATS) Foundation

Why is structured data collection vital in developing synthetic control arms for rare disease trials?
Synthetic control arms ensure that all trial participants receive the active treatment. Vital for so many rare disease patients where time is in limited supply. This approach is being increasingly considered by companies developing treatments for the rare disease community. But how does this work and why is patient data so important? 

11.25Workshop with live Q&A
Amplifying the Patient Voice through Patient Voice Publications
Emma Sutcliffe - SVP Patient Insights and Solutions, Prime Global 
Jess Duggan  - Syngap Research Fund 
This session will explore PVPs
PVPs tell the patient story; from real world experience and burden of disease to hopes for the future, bringing the patient voice to pharma for enhanced patient engagement. Vitally, PVPs drive engagement and better outcomes for patients by telling the patient story when pharma is meaningfully listening. Join this session to hear about how PVPs support the triple win, for patients, for pharma, and for society.
12.00Networking, exhibition zone and gallery explorationMeet and mingle live with attendees, exhibitors and poster creators
12.30

Live pitching
Innovation Challenge: Rare Diseases 

Alex Lloyd - Principal Advisor, Commercial Delivery Lead, Eastern Academic Health Science Network 
Dragon's Den Judges
Louise Jopling - Commercial Director Eastern AHSN
Alastair Kent - Co-Chair of UK Rare Disease Policy Board
Sean Richardson - General Manager Alexion Astra Zeneca UK
Sophie Muir - Timothy Syndrome Alliance
Charles Steward - Patient Advocacy and Engagement Lead - Congenica
Dr Gemma Chandratilllake - CRDN Chair and Education and Training Lead NHS East Genomics
Five selected innovators TBC

Five Innovators pitch their rare disease challenge solutions to the RAREsummit21 audience
At RAREfest20 the Eastern AHSN (Academic Health Science Network) asked people living with rare conditions what their biggest everyday challenges were. Our community said, we listened.  The innovation community have responded with tech and digital solutions to help with care coordination, wellbeing and mental fatigue or ensuring health information is done well. Today we'll hear 5 of the best solutions live.  
13.30Networking, exhibition zone and gallery explorationMeet and mingle live with attendees, exhibitors and poster creators
14.00Live Talk with Q&A
UK Rare Disease Framework: Action Plans Update and Engagement
Lauren Watson - Rare Diseases Lead, Genomics Science and Emerging Technologies, Department of Health and Social Care
Kath Bainbridge - Head of Genomics Science and Emerging Technologies, Department of Health and Social Care
The UK Rare Disease Framework action plans - where are we now and how do we get involved?

At RAREsummit19, Baroness Nicola Blackwood, then Minister for Innovation for the UK Government, invited attendees to join a National Conversation on rare diseases and have their say on the future of UK rare diseases policy.  Guided by these responses, the Government launched the new UK Rare Diseases Framework in 2021, outlining a coherent, national vision on how the UK will improve the lives of those living with rare diseases. Join this session to hear about how the action plans related to the Framework are evolving and how you can get involved in the next stages.
14.20Live Panel
Generation Genome
Dr Richard Scott – Clinical Director, Genomics England                                   
Dr Sarah Bowdin – Medical Director, East Midlands and East of England Genomics Laboratory Hub
Sasha Henriques – Principal Genetics Counsellor, Guy's and St. Thomas' NHS Foundation Trust
Rebecca Middleton  - Vice Chair Genomics England Participant Panel
Circling back and looking forwards
The panel will reflect back on the genomic revolution of the last 5 years and the difference it has made for rare disease patients and lay out their plans and vision for the coming 5 years. 
14.55Networking, exhibition zone and gallery explorationMeet the experts, mingle live with attendees, exhibitors and poster creators
15.25

Fireside Chat with Q&A
Medicines Discovery for Rare Diseases


Steve Rees – VP Discovery Biology, AstraZeneca   
Sharon Barr - Senior Vice President, Head of Rare Disease Research and Product Development, Alexion AstraZeneca            Facilitator  – TBC

Medicines Discovery: The potential of genomic medicines for rare diseases
Exploring Astra Zeneca's recent move into the rare disease therapeutics space with Alexion Pharmaceuticals and the potential opportunity offered by the rapid advance in CRISPR medicines, oligonucleotides and other gene therapies

Details tbc 

15:50

Live Panel
The Potential of the Cambridge Cluster to Innovate for Rare Disease Patients

Dr Tim Guilliams - founder and trustee of CRDN and Co Founder and CEO of Healx
Professor David Rowitch,
Head of Paediatrics, University of Cambridge
Other Speakers TBC
This live panel session will explore what the Cambridge Cluster has to offer in terms of innovating for rare diseases. We'll be talking to representatives from Cambridge University, start-up accelerators, the Biomedical Campus, pharma companies and the innovation community. 
16.50

Closing address

CRDN
17.00NetworkingMingle live with attendees
All Day: On DemandShort film
A Path Well Travelled
Shirlene Badger  - Patient advocacy specialist, Illumina
Allison Watson - CEO, Ring 20 Research and Support UK CIO                       
Jo Balfour  - Managing Director, Cambridge Rare Disease Network

Exploring the narrative of a patient group's growth and development
A short documentary film chronicling the journey and growth of a rare disease patient group, the opportunities they have grasped and the impact this has had on their community


All Day: On Demand

Early Access Programs

Naomi Litchfield – Global Patient Advocacy Lead, Bionical Emas (Moderator)
Alix Hall - Managing Director, The Isaac Foundation, Canada
Christine MacCracken - Head, Patient Strategies & Solutions - Janssen Pharmaceutical Companies of Johnson & Johnson, US

Collaborating, listening and educating effectively with patient groups
Where all options have been exhausted, Early Access Programs can offer ethical, compliant, and controlled mechanisms of access to investigational medicines outside of clinical trials, before the commercial launch of the medicine. How can the pharma industry support patient groups to navigate the complexities? 
All day: On demand

Panel discussion
Patient Group and Industry Partnering 

Dr Ron Jortner – Trustee CRDN,  CEO, Masthead Biosciences
Dr Madhu Madhusudhan - Senior Business Manager, LifeArc
Sophie Muir - Timothy Syndrome UK   
Speaker - Dravet Syndrome UK TBC

Can Systematic Serendipity Catalyse New Treatments?

In developing rare disease treatments, it is often through serendipity that new ways forward are discovered. We hypothesize, that systematically linking stakeholders in the right setting could help catalyse treatment development. To test this hypothesis, we recently ran an inaugural, speed-dating-style event partnering rare disease patient groups and companies. Event participants will describe the concept, their experience and interim outcomes.

All Day: On demandGlobal Collaboration: Looking East

Dr. Ammar Masood - Lead Operations South, DRK Pharma Solutions
Dr. Salman Kirmani
 - Associate Professor, Department of Paediatrics and Child Health, Aga Khan University Hospital

Prof Dr. Huma Cheema - Prof. Emeritus Children's Hospital and Institute of Child Health, Lahore
Asim Rauf - Chief Executive Officer, Drug Regulatory Authority of Pakistan
Abdur Rasheed - Director, Division of Pharmacy Services, Drug Regulatory Authority of Pakistan
Dr Harvey F Lodish - Massachusetts Institute of Technology (MIT)
Amer Haider - Principal, GeneEquity

What are the opportunities for developing Rare Disease therapies in South Asia?
This panel session will explore consanguinity and the rare disease burden in this region, and the resulting potential for rare disease research and innovation. We'll examine the need for international cooperation, aligning regulatory ecosystems for clinical trials to best improve the situation in this flourishing destination for rare disease progress.