What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted voretigene neparvovec, for treating adults and children with vision loss due to inherited retinal dystrophy (a rare genetic condition that causes damage to the retinal cells at the back of the eye).
Voretigene neparvovec is for use in patients who still have enough working cells left in the retina and when the disease is caused by mutations (genetic changes) in the RPE65 gene (a gene that is necessary to make a protein needed for vision) which are biallelic (passed down from both parents).
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of voretigene neparvovec by looking at the SMC Detailed Advice Document (SMC2641).
What does SMC's decision mean for patients?
Voretigene neparvovec for use as described above is available for prescribing on the NHS in Scotland. Your healthcare professional can discuss with you if it is the right treatment for you or your child.
You can find more information about making decisions about your or your child’s treatment in this booklet called: Medicines in Scotland: What’s the right treatment for me?
More about SMC's decision
SMC’s decision takes into account a confidential discount offered by the pharmaceutical company. SMC was also able to be more flexible* in its decision-making because the medicine is for, an extremely rare condition.
How does SMC make its decision?
SMC carefully considers every new medicine to make sure it benefits patients and is likely to be a good use of NHS resources.
To do this SMC studies the following:
- Evidence from the company about how well the medicine works compared with current treatments available in Scotland, in relation to how much they will cost to buy and use to treat patients.
- Information from patient groups about the potential impact of the medicine on patients and carers.
- Advice from healthcare professionals about any benefits of the new medicine compared to current treatment, along with how the new medicine is likely to be used.
When SMC assesses a medicine it takes account of the needs of all patients in NHSScotland, not only those who may be treated with the medicine.
You can find out more about how SMC decides here: htps://www.scottishmedicines.org.uk/how-we-decide/
More information and support
The organisation below can provide more information and support for people with vision loss and their families. SMC is not responsible for the content of any information provided by external organisations.
Retina UK
https://retinauk.org.uk
0300 111 4000
You can find out more about voretigene neparvovec (Luxturna®) in the Patient Information Leaflet (PIL) by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.
https://products.mhra.gov.uk/