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    Has all of the relevant evidence been taken into account?

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    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

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1 Recommendations

1.1

Leniolisib is not recommended, within its marketing authorisation, for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in people 12 years and over.

1.2

This recommendation is not intended to affect treatment with leniolisib that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers.

Why the committee made these recommendations

APDS is an ultra-rare genetic condition that can severely affect the quality of life of people with the condition, and their families and carers, and can significantly shorten life. It can cause organs and lymph nodes to swell and the body's immune system to attack healthy tissue. People with the condition are also at high risk of serious infections, and it can cause cancer. There are no licensed treatments for APDS. Standard care includes antimicrobial treatment, surgery, immunosuppressants, immunoglobulin (antibody) replacement therapy, and stem cell transplants.

Clinical trial evidence shows that leniolisib, compared with placebo plus selected standard care treatments, reduces the size of people's lymph nodes and increases levels of immune cells called B cells.

There are uncertainties in the economic modelling around how best to model the effect of stopping treatment.

Because of the uncertainties in the economic model, it is not possible to determine the most likely cost-effectiveness estimates for leniolisib. So it is not recommended.