The Scottish Medicines Consortium (SMC) has advised that ruxolitinib should be made available to myelofibrosis patients in Scotland on the NHS.
They had initially decided not to make the drug available, but reversed their decision after hearing from clinicians and charities, including Leukaemia & Lymphoma Research.
Myelofibrosis is a blood disorder caused by an abnormality in cells that make platelets. As the disease develops, tissue in the bone marrow scars over and becomes useless. Blood cell production is taken over by the liver and spleen, resulting in low blood counts. Symptoms can vary but include anaemia, persistent infections, headaches and skin irritation.
Ruxolitinib is the only treatment shown to be able to lessen debilitating symptoms, reduce spleen size and improve quality of life for patients.
Leukaemia & Lymphoma Research was one of the charities that participated in the Patient and Clinical Engagement (PACE) meeting. We were able to provide personal testimony from patients who had been treated with ruxolitinib, who were all extremely supportive of the drug. We also included the views of two clinical experts from our Medical Advisory Panel.
The SMC’s decision to recommend ruxolitinib additionally took into account a Patient Access Scheme offered by the drug’s manufacturer, Novartis, which improved its cost effectiveness.
Yvonne Dickson, Regional Head of Scotland at Leukaemia & Lymphoma Research, said: “This decision is great news for Scottish patients. Myelofibrosis can be horribly debilitating and many patients have reported that ruxolitinib has had a hugely positive impact on their quality of life. Thanks to the response of patients and clinicians, we were able to present a really strong case that the drug should be made available on the NHS.”