How Kaftrio changed the world of Cystic Fibrosis (a LinkAGE webinar)

This LinkAGE webinar provides an look at CFTR modulators and how they have changed the world of cystic fibrosis (CF). It is presented by paediatric respiratory medicine consultant Dr Ian Balfour-Lynn. We have provided timestamps, video chapters and transcriptions (coming soon) to help you find what you need. The webinar and Q&A was broadcast live on 12 June 2023.

By the end of this sixth LinkAGE webinar you will be able to:
- describe the natural history of CF before the era of CFTR modulators;
- discuss the clinical trials that led to the licensing of these drugs; and
- explore the real world experience of using Kaftrio.

What is the LinkAGE expert webinar series?
The Genomics Education Programme's LinkAGE (Linking Academia and Genomics Education) series will aim to provide expert seminars on exciting ‘bench-to-bedside’ projects that are likely to impact on clinical practice in the not-too-distant future.

Who is the LinkAGE expert webinar series aimed at?
This webinar series is aimed at researchers, clinicians and other healthcare professionals who already have a strong understanding of fundamental genomics concepts. If you are just starting your learning journey, or need a quick refresher, we encourage you to visit our website: www.genomicseducation.hee.nhs.uk

Meet the speaker
Dr Ian Balfour-Lynn is a consultant in paediatric respiratory medicine at the Royal Brompton Hospital, London. Until his retirement from NHS clinical practice in March 2023, Dr Balfour-Lynn specialised in all aspects of paediatric respiratory medicine, including both tertiary and secondary care. He was the director of the Royal Brompton’s paediatric cystic fibrosis unit for 14 years and has multiple publications on clinical aspects of cystic fibrosis.

His other work has included being the chairman of the Cystic Fibrosis group (Paediatric assembly) in the European Respiratory Society, president of the British Paediatric Respiratory Society and sitting on NHSE’s Clinical Reference Group where he represented paediatric cystic fibrosis for 10 years.

TIMESTAMPS
00:00 Welcome and introductions
02:05 Discovering the molecular background to Cystic Fibrosis
05:14 What is the genetics of CF?
07:49 Phenotypes seen in CF and patient prognosis
12:46 CFTR types overview
13:45 CFTR modulators and triple therapy
16:27 Ivacaftor - clinical trials, real-life data, patient experiences and costs
24:02 Double therapy - Orkambi and Symkevi outcome data, patient experiences and costs
28:15 Triple therapy - What did the Kaftrio clinical trials show?
31:53 Kaftrio is commissioned by NHS England in 2020
35:16 What are patients on Kaftrio experiencing?
38:00 The estimated Katrio cost to the NHS
40:36 Ethical and access considerations
43:13 Type-1 CFTR drug efforts - CRISPR and gene editing
45:34 Talk conclusions
46:30 Q&A and close

About us
Health Education England's Genomics Education Programme is developing a substantial education programme to inform healthcare professionals about the impact of genomics on clinical practice. This video is the one of the many educational resources from the programme.

For more information visit https://www.genomicseducation.hee.nhs.uk