Scottish cost regulators have given the green light for the use of Roche's RoActemra on the National Health Service, but have closed the door to Gilead Science's Cayston.
The Scottish Medicines Consortium has OK'd funding for RoActemra (tocilizumab) to treat active systemic juvenile idiopathic arthritis (sJIA) in patients aged two years and above who have failed to respond to treatment with non-steroidal anti-inflammatory drugs (NSAIDs) and systemic corticosteroids.
The group concluded that RoActemra was superior to placebo in reducing disease activity and fever in patients with persistent forms of the illness despite having received previous treatment with NSAIDs and corticosteroids, and acknowledged the significant unmet need for a treatment for the condition.
As well as developing arthritis in the joints, patients with systemic JIA can suffer from a range of additional symptoms such as fever, rash, swollen glands, tiredness and lack of energy, weight loss and muscle pain, and the condition can lead to joint damage and permanent disability.
The SMC's guidance notes that patients taking RoActemra show clinical improvement within six weeks of starting treatment, and stresses that continued therapy should be "carefully reconsidered" if no benefit is seen in this timeframe.
Around 10,000 children in the UK have JIA, of which around 10%-20% have the systemic subtype, and RoActemra is the first medicine to be licensed specifically for this indication, bringing new hope to patients.
Approval of the drug for use on the NHS was contingent upon a patient access scheme submitted by Roche, under which it has offered a discount on the list price of the drug to boost its cost-effectiveness.
Thumbs down for Cayston
On the downside, the cost watchdog turned down Gilead Science's inhaled antibiotic Cayston (aztreonam lysine) as a suppressive therapy of chronic lung infections (due to Pseudomonas aeruginosa) in adult patients with cystic fibrosis.
The SMC notes that Cayston has shown superiority in improving lung function and respiratory symptoms three 28-day studies in patients with CF and chronic Pseudomonas aeruginosa infection, but adds that there limited data to support the sustainability of this benefit over further courses of treatment.
The company's justification of the treatment’s cost in relation to its health benefits and the economic case presented were not sufficient to gain access to NHS Scotland, the SMC said.