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SMC green light for rare blood cancer treatment

People in Scotland with the rare blood cancer polycythaemia vera (PV) will soon gain access to a new treatment option on the NHS after cost regulators approved funding for Novartis’ Jakavi (ruxolitinib).

Specifically, the drug can be used on the NHS to treat adults with polycythaemia vera (PV) who are resistant to, or intolerant of, hydroxyurea (HU), thought to represent around 15% of the PV population.

Until now, patients unable to take HU have had to rely on regular venesection (the removal of blood) to reduce their red blood cell count and manage their condition.

The SMC’s backing of Jakavi marks a major milestone for Scotland, which will be the first UK country to offer this treatment option to patients with PV who cannot tolerate the current standard of care.

Three other medicines were also backed for NHS use in Scotland:

Takeda’s Takhzyro (lanadelumab) was accepted for the routine prevention of frequent severe attacks of hereditary angioedema. Current preventative treatment is given intravenously which can be difficult for some patients and may not always be feasible. Takhzyro can help maintain an acceptable level of attack control and is well tolerated, which can help reduce anxiety around the threat of further attacks, the SMC noted. Also, as the treatment is given by subcutaneous injection, with the opportunity for patients to self-administer, fewer hospital visits are required;

The SMC also approved funding for AstraZeneca’s Lynparza (olaparib) for ovarian cancer. Lynparza maintenance treatment, after initial chemotherapy, increases the time that patients are free of recurrence, delaying the time to next chemotherapy and potentially offering increased survival benefits; and

Shionogi’s Mulpleo (lusutrombopag) was accepted for use to prevent excessive bleeding in adults with thrombocytopenia caused by long-standing liver disease. The drug is an oral medicine taken to reduce the need for platelet transfusions in thrombocytopenic patients with chronic liver disease undergoing invasive procedures.

On the downside, the Committee was unable to accept Immedica’s Yondelis (trabectedin) for the treatment of advanced soft-tissue sarcoma. The SMC said it was unable to accept the medicine because the company’s evidence around the clinical and cost effectiveness of treatment “was not clear”.