Before the case you have been asked to read the following:
https://www.scientificamerican.com/article/four-success-stories-in-gene-therapy/
Baby Arthur was born in January 2021, to non-consanguineous parents. He was well at birth and discharged without incident, Apgar score was 7 at 1 minute and 9 at 5 minutes. At 4 months old he visited his paediatric GP as he was unable to control his head. He looked alert, showed social smiling and babbling, and could contact his eyes with her mother’s eyes, but his posture was severely hypotonic. His upper extremity power was grade 3 and lower extremity power was grade 2 symmetrically. There were no deep tendon reflexes.
After admission to hospital, he was diagnosed with mutation in SMN1 gene causing type 1 Spinal Muscular
Atrophy (SMA). Infants born with type 1 SMA rarely live past 2 years of age. Approximately 1 in 50 people are carriers of this mutation.
In June 2021 was prescribed a novel gene therapy called Zolgensma, becoming the first NHS patient to be prescribed this drug. The drug carries a list price of US$2.125 million per treatment, making it the most expensive medication in the world as of 2019.
Whilst potentially lifesaving, the cost of the gene therapy is high leading to questions over whether the cost outweighs the benefits and whether the money would be better spent elsewhere. There are also further ethical considerations with gene therapy which need to be considered.
Within the UK the decision as to whether drugs should be made available via the NHS is made by NICE, which in November 2021 recommended the use of an alternative treatment called Risdiplam for SMA.
Discuss whether gene therapy treatments such as Zolgensma should be made available on the NHS?
Essay topic: Should gene therapy be made available on the NHS? You should write a 1000- word argument and support your position with critically evaluated evidence.
Case adapted from the BBC article “Gene therapy is a game changer for our son”, which can be found here.
https://www.bbc.co.uk/news/uk-57309613
