About this event

  • Date and time Tue 15 Feb 2022 from 6:30pm to 8:00pm
  • Location Online
  • Organised by Medicine and Me, Action for Rare Disease Empowerment (ARDEnt)

Jointly organised with ARDEnt, this webinar will explore the impact of the pandemic response on UK clinical trials and drug development for rare diseases.

This episode refers to theme 3 in the Making The Unseen Seen: Rare disease and lessons learned from the pandemic report and will discuss how safety considerations, travel restrictions, shielding, trial-sites being repurposed to COVID-19 wards, research staff either being called to the front- line or called to replace others who were, have compounded the already fragile world of clinical development for rare diseases.

Read Making The Unseen Seen: Rare disease and lessons learned from the pandemic report to find out more on this episode, the series, and the ARDEnt Team's research.

CPD learning applied.

This webinar is part of the 'Lessons learned from the COVID-19 pandemic' series, a series that will cover all themes outlined in the Making The Unseen Seen report, from diagnostic delay in episode 1 to health and social care in episode 2, clinical trials and drug development in episode 3, and concluding with the UK Rare Diseases Framework and action plans in episode 4. 

This webinar is available for on-demand viewing. The webinar recording will be available for registered delegates up to 30 days after the live webinar broadcast via Zoom. The link will be sent 24 hours after the webinar takes place. 

Join in the conversation online #RSMLive, #UnseenSeen
Follow us on Twitter: @RoySocMed 

About ARDEnt

Action for Rare Disease Empowerment is an open group of stakeholders from across different sectors who all have a passion for bettering the lives of those living with rare conditions. The three founding organisations of ARDEnt are
Medics4RareDiseases, Cambridge Rare Disease Network and Rare Revolution Magazine, however over 30 individuals and groups were involved in the creation of the ‘Making The Unseen Seen’ report, and the group welcomes new participants and contributors at any time. Although currently, the focus of ARDEnt has been around the pandemic the group hope that this open collaboration sets the template for future activities. Rare is stronger together. If you would like to be involved please do not hesitate to contact any of the Theme Leads from the Cambridge Rare Disease Network.

About Medicine and Me

Medicine and Me meetings aim to provide an outline of current best practices, future updates of important conditions, and give a direct voice to patients and their carers and enable them to discuss their concerns and reflections on the impact of diagnosis, investigation and management. Physicians, surgeons and indeed all healthcare professionals, continue to learn from and be inspired to greater efforts to improve care by hearing directly from patients.

We would like to thank our anonymous donor for their generous support enabling this platform for patient focused charities and free access to the Medicine and Me programme.

Key speakers

Dr Melita Irving

Dr Melita Irving

Consultant in Clinical Genetics, Guy's and St Thomas' NHS Trust

Speaker's biography

Dr Melita Irving trained in paediatrics before joining the clinical genetics department at Guy's and St Thomas'. She undertook a fellowship in skeletal dysplasia at the Royal Children's Hospital, Melbourne and has since established multidisciplinary paediatric clinics in skeletal dysplasia and achondroplasia at the Evelina London Children's Hospital. She is chief investigator for a number of clinical trials in achondroplasia and a proponent of embedding clinical research synergistically within clinical service.    

Chris McDermott

Professor Christopher McDermott

Professor of Translational Neurology, University of Sheffield

Speaker's biography

Professor McDermott studied for his medical degree at the University in Leeds graduating in 1994. He then continued is general medical and specialist neurology training in Leeds before taking up a clinical research training fellowship at the University of Newcastle upon Tyne. 

He moved to the University of Sheffield with Professor Dame Pamela Shaw in 2000 to undertake his Wellcome Trust Research Training PhD Fellowship and to complete his Specialist Training in Neurology to become a Consultant Neurologist in 2006. Professor McDermott is now the Professor of Translational Neurology at SITraN, a NIHR Research Professor and a Consultant Neurologist at the Sheffield Teaching Hospitals Foundation NHS Trust regularly undertaking specialist MND and neuromuscular clinics in Sheffield. 

The main drive of Professor McDermott’s research programme is developing the evidence base for effective treatments and delivering supportive and symptomatic care for patients living with motor neuron disease. 

Daniel Lewi

Daniel Lewi

Co-Founder and Chief Executive Officer, Cure and Action for Tay-Sachs (CAT’s) Foundation UK

Speaker's biography

Dan's eldest daughter Amelie was diagnosed with Tay-Sachs in 2011 at 15 months of age and upon finding that there was no dedicated charity providing support for families affected by this disease in the UK he set set-up the Cure & Action for Tay-Sachs (CATS) Foundation with his wife Patricia. Since the charity was launched, they have been able to grow The CATS Foundation so that it now offers a variety of services to its family members and has actively involved in the research investigating therapeutic treatments for Tay-Sachs and Sandhoff disease. In addition to this, Dan is the one of the founding members and Chairman of the European Tay-Sachs and Sandhoff Charity Consortium which brings together all the European patient groups leading the fight against the diseases. In 2017, Amelie sadly lost her battle against Tay-Sachs at the age of 8, but that has not stopped Dan or Patricia and they continue to work towards the charity's goal of finding a treatment so that there is hope for those families affected by Tay-Sachs and Sandhoff in the future.

Tom Kenny

Dr Thomas Kenny

Chief Executive Officer, Rare Disease Research Partners

Speaker's biography

Tom started his professional career in General Practice using improvement science to transform his own practice. From there he moved into NHS management and then into Public Health Medicine. Tom has worked at every level of commissioning in the NHS as clinical director for practice-based commissioning, director of commissioning for a primary care trust, medical advisor for national highly specialised commissioning. During this time Tom worked with NSCAG, NCG, and AGNSS in the programme evaluating drugs for use in rare diseases that became the NICE process for evaluating drugs for Ultra-Orphan diseases. A key task of this role was turning real-world data into useable clinical evidence. Tom was director of external relations and then director of research delivery and impact for the NIHR Evaluation, Trials and Studies Co-ordinating Centre. During this time Tom oversaw the delivery or a portfolio of >800 active research projects across 5 of the NIHR programmes. Tom has worked with several pharmaceutical companies supporting the design of research, interpretations of finding and articulation of evidence to facilitate better payer decision-making. Tom now works for Rare Disease Research Partners, designing, developing and delivering bespoke research programmes ad overseeing the clinical content of projects. Tom has a Masters in Business Administration and a Masters in public health and has written books and published a range of papers particularly on services for rare diseases.

Agenda

View the programme

Welcome and introduction

Jo Balfour, Founding Member and Managing Director, Cambridge Rare Disease Network

The challenges of delivering research and collecting data in rare diseases pre, during and post the COVID-19 pandemic, case studies and multi-stakeholder

Chair: Jo Balfour, 

 

Dr Melita Irving, Consultant in Clinical Genetics, Guy's and St Thomas' NHS Trust, Professor Christopher McDermott, Professor of Translational Neurology, University of Sheffield, Daniel Lewi, Co-Founder and Chief Executive Officer, Cure and Action for Tay-Sachs (CAT's) Foundation UK, Dr Thomas Kenny, Chief Executive Officer, Rare Disease Research Partners and Alexandra Griffiths Rayson, Patient

Overcoming the challenges in rare disease research and data collection - lessons learned, adaptations and solutions, looking to the future

Chair: Gavin Jones, Global Advisor Rare Diseases, OPEN Health Group

Dr Melita Irving, Professor Christopher McDermott, Daniel Lewi, Dr Thomas Kenny and Alexandra Griffiths Rayson 

Questions and answers
Closing remarks and close of meeting

Location

Online

Disclaimer: All views expressed in this webinar are of the speakers themselves and not of the RSM nor the speaker's organisations. 

Special rates for difficult times
The RSM wishes to offer healthcare professionals continued learning opportunities during the coronavirus pandemic. The RSM’s weekly COVID-19 Series webinars remain free of charge, while there will be small charges to register for other online education. These fees will enable the RSM to continue its programme of activities and will apply during the course of the pandemic.

Registration for this webinar will close 2 hours prior to the start time. You will receive the webinar link 2 hours before the meeting. Late registrations will not be accepted.

This webinar will be available for registered delegates 30 days after on Zoom. The link will be sent 24 hours after the webinar takes place. 

This webinar will be recorded and stored by the Royal Society of Medicine and may be distributed in future on various internet channels. 

Upcoming episodes in this series

The Lessons learned from the COVID-19 pandemic series will cover all themes outlined in the Making The Unseen Seen report, from diagnostic delay in episode 1, to health and social care in episode 2, clinical trials and drug development in episode 3, and concluding with the UK Rare Diseases Framework and action plans in episode 4. 

All webinars in this series are available for on-demand viewing. The recording of each episode will be available for registered delegates up to 30 days after the live webinar broadcast via Zoom. The link will be sent 24 hours after the webinar takes place. 

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Free Public Webinar CPD

Lessons learned from the COVID-19 pandemic: Looking to the future with The UK Rare Diseases Framework

Jointly organised with ARDEnt, this webinar will discuss the recommendations the ARDEnt team is using to help inform how the UK is going to deliver its objectives set out in the UK Rare Diseases Framework, published in January 2021.

The webinar panel includes members of ARDEnt and those responsible for overseeing the impact of the Framework in the UK.

  • Price Free