Patients with the bleeding disorder chronic immune (idiopathic) thrombocytopenic purpura are a giant step closer to being able to access GlaxoSmithKline's Revolade (eltrombopag) on the National Health Service.
The National Institute for Health and Care Excellence has this week published draft final guidance endorsing the use of the drug in adult patients who have had their spleens removed (splenectomy) and are not responding to other treatments.
Revolade will also be routinely funded as a second-line treatment in those unable to have a splenectomy.
In both cases, GSK's drug should only be used when patients have severe disease and a high risk of bleeding needing frequent courses of rescue therapies, the Institute said.
Around 3,000–3,500 people are affected at any one time in England and Wales by ITP.
Revolade helps to reduce bleeding by works by boosting platelet production, but with an average daily price tag of £56.43 (as determined by data from the EXTEND study), the drug does not come cheap.
Revolade's use in this setting was actually originally rejected back in 2010, after NICE estimated its cost to be £104,100 per QALY for splenectomised patients and £116,750 per QALY for those without, and thereby far above the ceiling limit for cost-effectiveness.
However, a subsequent offer from GSK of a discount via a Patient Access Scheme prompted a new review of the drug and NICE's current positive recommendation.
Final guidance is expected in July.