NICE is not recommending Novartis’s Jakavi after it found problems with the firm’s data and costing model for its drug.
The Institute has published final draft guidance not recommending Novartis’ Jakavi (ruxolitinib) for the treatment of disease-related splenomegaly (enlarged spleen) or its symptoms in adults with primary myelofibrosis, or post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosisi.
Novartis believes that its drug is worth £74,000 per QALY gained, but NICE argues that it is actually double this – NICE does not usually recommend a drug that has a QALY over £30,000.
Jakavi costs £3,600 for a 60-tablet pack of 15 mg or 20 mg tablets, or £1,800 for a 60-tablet pack of 5 mg, meaning an annual cost of around £43,200 per patient. The firm has not offered a patient access scheme to help lower the cost of the medicine.
NICE concluded that Jakavi was clinically effective and a step change in innovation for treating the condition, but could not be considered a cost-effective use of NHS resources compared with best available therapy for treating disease-related splenomegaly or symptoms in adults with myelofibrosis.
It also said that although it was plausible the drug could offer an overall survival benefit, the reason for this “remained unclear” because of uncertainties in the evidence.
Commenting on the final draft guidance, Sir Andrew Dillon, chief executive at NICE, said: “Myelofibrosis and splenomegaly can be extremely debilitating, with symptoms such as severe itching and fatigue. It is disappointing not to be able to recommend this new treatment in our final draft guidance, but in order to do this we have to be sure that the treatment is both clinically and cost effective, because money has to be diverted from elsewhere in the health service to pay for it.”
NICE also said there were “concerns” around the limitations of the Novartis’ economic model when cost assessing its medicines. NICE’s committee concluded that there were “fundamental issues” with the structure of the Novartis’ model, and that the associated assumptions increased the uncertainty of its cost effectiveness.
Final guidance is expected to be published in June 2013, but it looks likely that the medicine will not be approved for NHS funding unless Novartis offers a discount, or can come up with a better economic model.
Myelofibrosis is a type of haematological cancer and is a rare condition, with around 0.4 cases per 100,000 each year in the UK. The bone marrow becomes scarred (fibrosed) making it is less able to make blood cells.
To compensate for this, other organs in the body, including the liver and the spleen, begin to produce them. As the spleen begins to produce blood cells, it grows in size causing splenomegaly (enlarged spleen).