Nobody understands your community better than you do.
About the workshop
This three-part, online workshop is designed to give rare disease patient group leaders the knowledge and confidence to engage with clinical research and make their voices heard.
Across three two-hour interactive sessions, we’ll explore the unique challenges of rare disease trials, share practical solutions, and highlight where patient groups can add value.
We will cover
Session 1 – Clinical trials in the rare disease context
18th November, 10:00 – 12:00 GMT
Learn more:
This session will provide an accessible overview of what clinical trials are and why they are important. It will cover key concepts such as trial phases, protocols, and the role of regulators, as well as the unique challenges and opportunities that arise when conducting research in rare diseases. Attendees will gain a clear understanding of how trials work and the factors that make rare disease research distinct.
Session 2 – The patient voice in trial design
19th November, 10:00 – 12:00 GMT
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We will hear from The Lily Foundation on the important work they are doing in this area!
Session 3 – The patient voice in trial delivery and beyond
20th November, 10:00 – 12:00 GMT
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Why join?
- Build your understanding of how clinical trials work
- Learn practical ways to influence and support research
- Hear real-world examples of patient involvement in rare disease trials
- Connect with other patient group leaders facing similar challenges
Register below to secure your FREE place!
Important Notice: Our ticketing provider, Eventbrite, now allows you to confirm tickets before completing the order form on the next page. To finalise your booking, please ensure you fill out the form in full after paying your deposit.
Meet the speakers
Christa Van Kan
Christa van Kan is a clinical trials expert with 25+ years’ experience across multiple diseases, including rare and paediatric indications. A co-founder of PSR Orphan Experts and former Director of Clinical Operations, she now consults for small pharma and biotech, specialising in vendor selection, oversight, and stakeholder management ensuring the successful execution of clients’ clinical trials.
Melita Irving
Dr Melita Irving is a clinical geneticist at Guy’s and St Thomas’ NHS Foundation Trust, specialising in genetic conditions across all ages with a focus on skeletal disorders. She leads multidisciplinary paediatric clinics in skeletal dysplasia and achondroplasia, serves as chief investigator on related clinical trials, and authored the Haynes Manual book Human DNA – Understanding Your Genetic Code.
Katie Waller
Katie Waller is Head of Patient Programmes at The Lily Foundation and a former paediatric and research nurse in early-phase trials. She now works across patient support, research, policy and advocacy, serving as PPIE lead for the LifeArc Centre for Rare Mitochondrial Disorders, and leading IMPACT, the Lily Foundation’s patient involvement group.
Maria O’Hanlon
Dr Maria O’Hanlon is Research Manager at The Lily Foundation. Before joining Lily, Maria completed a PhD in biology focusing on the role of mitochondria in Parkinson’s disease. Inspired by a master’s project in mitochondrial disease, she joined the Lily Foundation in January ‘24 and hasn’t looked back. As a Research Manager, she looks after all things Lily research (portfolio, international scientific efforts, research comms), leading ‘The Research Zone’ hub.
Lee Aiyegbusi
Prof Lee Aiyegbusi is Professor of Patient-Centred Research and Deputy Director at the Centre for Patient-Reported Outcomes Research (CPROR), University of Birmingham, UK. He leads the patient-reported outcome (PRO) work package of the LifeArc Centre for Acceleration of Rare Disease Trials, co-leads the PRO theme of the Birmingham NIHR BRC, and serves as PPIE lead for projects including the NIHR Blood and Transplant Research Unit in Precision Cellular Therapeutics (BTRU-PCT).
Pan Pantziarka
Dr Pan Pantziarka is Director of Drug Repurposing at the Anticancer Fund, working on drug repurposing in oncology – from drug candidate identification to clinical trials and health policy work.
Pan is also a member of the Executive Committee of FOSTER (Fight Osteosarcoma Through European Research). He also Chairs and is the Co-founder of George Pantziarka TP53 Trust – the UK organisation for people with Li Fraumeni Syndrome.
Deposit system
Registering for this workshop will require paying a refundable deposit of £15 upon registration. This deposit will be kept safe in Eventbrite until after the training has been completed. Following the event, if you have attended and signed in to 2 out of 3 of the sessions, you will get your full deposit back.
We understand that circumstances can change, especially in the rare disease community. Should you register for the event and realise you can no longer attend please let us know as soon as possible. We will endeavour to refund your deposit in full provided reasonable notice is given. Reasonable notice for this event is no later than 3 working days before.
Find out more about our deposit scheme here. If you have any questions, please contact projects@rarebeacon.org.
