Specialised treatments for rare diseases to be made available in England

NHS England has confirmed that three new specialised treatments are to be made available for patients suffering from certain rare diseases.

For the first time, patients undergoing allogeneic haematopoietic stem cell transplants who suffer a relapse following a first transplant will be allowed to have a second one. This is expected to benefit around 15 patients a year.

Meanwhile, eculizumab therapy for the kidney condition C3 glomerulopathy – which includes C3 glomerulonephritis and dense deposit disease – will be offered to the approximately five sufferers a year who suffer a relapse following a kidney transplant.

Finally, around 90 to 125 patients a year will be able to receive treatment with riociguat, subject to confirmation of a discounted price offered by the manufacturer, for pulmonary arterial hypertension in cases where other treatment options have failed.

John Stewart, NHS England's acting director for specialised commissioning, said: "This is really good news for the relatively small number of patients for whom these new treatments could prove beneficial."

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