What has SMC said?
Leniolisib was assessed by the Scottish Medicines Consortium (SMC) and meets the definition of an ultra-orphan medicine, which is a medicine to treat an extremely rare condition.
This document summarises the initial SMC assessment of leniolisib for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in adult and paediatric patients who are 12 years of age and older. APDS is a rare genetic condition that affects the immune system.
You can find more detailed information by looking at the SMC ultra-orphan initial assessment report (SMC2836).
What does SMC’s assessment mean for patients?
The Scottish Government will confirm when leniolisib is available for prescribing on the NHS in Scotland as part of the ultra-orphan pathway.
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?
What has SMC said in this assessment?
- There are no other medicines licensed for the treatment of APDS, there is an unmet need for targeted therapies.
- In the clinical study there was no difference in quality-of-life measurements in patients treated with leniolisib compared with patients treated with placebo (a dummy treatment) over 12 weeks. However, there appeared to be an improvement in general health over the longer-term extension study and an increase in work or school days.
- A health economic model suggested that leniolisib was associated with increased costs compared with current treatment. However, treatment with leniolisib was also predicted to improve health outcomes, as measured by quality adjusted life years (QALYs; a way of measuring how well a treatment lengthens/and or improves patients’ lives) compared with current best available treatment. Although there were some areas of uncertainty which may have affected the economic model.
How does SMC assess ultra-orphan medicines?
SMC uses a broad assessment framework for ultra-orphan medicines. This is part of the ultra-orphan pathway in NHSScotland.
You can find out more about how SMC assesses medicines in the ultra-orphan pathway here: https://www.scottishmedicines.org.uk/how-we-decide/ultra-orphan-medicines-for-extremely-rare-conditions
More information and support
The organisation below can provide more information and support for people with APDS and their families. SMC is not responsible for the content of any information provided by external organisations.
Immunodeficiency UK
https://www.immunodeficiencyuk.org
0800 987 8986
You can find out more about leniolisib (Joenja®) in the Patient Leaflet by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website: https://products.mhra.gov.uk/