What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted etranacogene dezaparvovec on an interim basis (interim acceptance) for the treatment of adults with moderately severe and severe haemophilia B. Interim acceptance is an option available to SMC for certain* medicines. This means that etranacogene dezaparvovec is accepted for use subject to ongoing evaluation and reassessment once further evidence is available.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of etranacogene dezaparvovec by looking at the SMC Detailed Advice Document (SMC2649).
What does SMC's decision mean for patients?
The decision to accept etranacogene dezaparvovec on an interim basis means that if your healthcare professional thinks that it is the right medicine for you, you should be able to have the treatment on the NHS in Scotland. Once further evidence is available, SMC will reassess the medicine and provide an updated decision on its routine availability in NHSScotland.
You can find more information about making decisions about your 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 a rare condition.
*https://www.scottishmedicines.org.uk/how-we-decide/interim-acceptance-decision-option/
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 haemophilia and their families. SMC is not responsible for the content of any information provided by external organisations.
Haemophilia Scotland
https://haemophilia.scot
0845 874 4004
You can find out more about etranacogene dezaparvovec (Hemgenix®) 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/