From: Retrospective case series describing the efficacy, safety and cost-effectiveness of a vial-sharing programme for canakinumab treatment for paediatric patients with cryopyrin-associated periodic syndrome
Well defined patient population
Published diagnostic criteria for CAPS are available, and genetic testing for NLRP3 mutations is available.
The cost of one vial of canakinumab is £11,913.
Risk of drug wastage in paediatric population
Example: A 10 kg baby with CAPS would have a typical starting dose of 20 mg. This means that without vial sharing 130 mg of the vial would be wasted.
Patients attend single centre on same day as dose
This service only applies to NHS England patients; some travel long distances every 8 weeks.
Dosage interval acceptable for regular travel to single centre
Patients receive canakinumab every 8 weeks, minimising impact on families of regular travel to the national CAPS clinic.
Sufficient numbers of patients to make vial sharing feasible
Whilst CAPS as an exceptionally rare disease, centralisation of the national clinic provides sufficient patient numbers to ensure that vial sharing becomes a feasible and cost-effective exercise.
Availability of a pharmacy aseptic unit
Individual doses need to be made under aseptic conditions to allow vial sharing. Centres will require an aseptic unit with the capacity to fulfil the demand to implement this innovative practice.
Nationally commissioned service in place
National funding arrangement agreed from NHS England for the use of canakinumab in patients with CAPS. This was a key aspect facilitating centralisation of care, and thus a viable vial sharing programme.
Acute “real-time” changes in drug dose not critical
Changes in canakinumab dose cannot be made on the day of clinic since this is prescribed 2 weeks in advance. Dosage alteration therefore occurs 8 weeks later at the next clinic visit.