RAREsummit21 Agenda – Draft

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

Welcome address

Nicola Miller, Rare Revolution Magazine
Kerry Leeson-Beavers, Breaking Down Barriers
Kevin Mott - Member, Board of Directors, NephCure Kidney International    
Jennifer Wilson, Director of Patient Advocacy in Europe, Travere 
Tammy Boyd, Director of Health Policy and Legislative Affairs, Black Women’s Imperative                             
Farah Akhtar, Rare mum and member of the xeroderma pigmentosum community 

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? 

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

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? 

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 - CamRARE Chair and Education and Training Lead NHS East Genomics

Innovators pitching:

(in alphabetical order)  

• Ascelpius Digital – A paediatric rare disease diagnostic portal
• Medwise.ai – Providing health care professionals with vetted and contextualized knowledge about the treatment and management of rare diseases
• nOink - Real world data capture and information for rare disease patients and their families to improve care.  A tool for rare disease families to diarise their condition(s) for the benefit of themselves and their clinicians
• Thriving AI Limited – A tool that integrates health and social care, formal and informal care around a Thriver, the person being cared for and is underpinned by machine learning and AI capabilities
• Vitaly Collaborative decision making - a state-of-the-art tool for healthcare professionals embedded in an interoperable platform on top of video-conferencing, image sharing and other HIE-relevant functionalities

Fireside Chat with Q&A
Medicines Discovery for Rare Diseases

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

Live Panel
Patient Group and Industry Partnering: What are the right ingredients to help catalyse new treatments? 

Dr Ron Jortner – Trustee CamRARE,  CEO, Masthead Biosciences
Dr Madhu Madhusudhan - Senior Business Manager, LifeArc
Sophie Muir - Timothy Syndrome Alliance
Julia Hawkins - General Partner at LocalGlobe, Board Trustee of Shwachman Diamond Syndrome UK
Dr Bruce Bloom - Chief collaboration Officer, Healx 

In developing rare disease treatments, it is often through serendipity that new ways forward are discovered. Can systematically linking stakeholders in the right setting help to catalyse treatment development, what are the right ingredients and how can patient groups and industry best prepare for collaborations? This panel will discuss the concept and share their thoughts and experiences.

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. Ring20 took to the stage at RAREsummit 2019 and shared their challenges and unmet needs with the audience. Who could help solve their diagnostic odyssey? Who is actively listening?

Closing address

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

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.