To ask the Minister of Health (i) whether he has any plans to approve the use of Trikafta/Kaftrio for the treatment of cystic fibrosis via the Health Service; (ii) if approved, when the drug will be available to patients; and (iii) under what conditions will it be licenced for use on the Health Service.

I announced on 30 July 2020 that an agreement with Vertex Pharmaceuticals had been reached. Subsequently I welcomed the news that Kaftrio received its licence for use in Europe on 21 August 2020. I can also advise that the contractual process has been completed and that Kaftrio is now approved for use in the HSC.

A number of the most severely ill patients have already commenced treatment with Kaftrio. However, there are over 500 children and adults who suffer from cystic fibrosis in Northern Ireland. Not all will be clinically suitable for treatment with Kaftrio and not everyone can be treated immediately. Clinical teams are working on the prioritisation of those patients most likely to see the greatest benefit.

The current EU license provides that eligible patients – aged 12 or over who have two copies of the F508del mutation gene, or one copy of F508del and a minimal function mutation – may be suitable for treatment with Kaftrio. In addition, the Vertex agreement provides for a number of additional patients with genetic mutations assessed as requiring ‘off label’ (off-license) use of the new regime. A clinically led panel will be established at Trust level to support the assessment and suitability of such patients for treatment.