Patients with rare conditions to benefit from new treatments
NHS England has recently confirmed that three new specialised treatments will be made available for patients in England.
It is estimated that up to 145 people a year will meet the clinical criteria to benefit from the following treatment options being made routinely available to their clinicians with funding from NHS England:
- Second allogeneic haematopoietic stem cell transplants – estimated to cover around 15 patients a year who suffer a relapse following a first transplant.
- Eculizumab for the treatment of the kidney condition C3 glomerulopathy – which includes C3 glomerulonephritis and dense deposit disease – estimated to benefit up to 5 patients a year who suffer a relapse following a kidney transplant.
- Riociguat for pulmonary arterial hypertension – estimated to benefit around 90-125 patients a year for whom other treatments have failed, subject to confirmation of a commercially in confidence discounted price offered by the manufacturer.
NHS England’s Acting Director for Specialised Commissioning, John Stewart, said: “This is really good news for the relatively small number of patients for whom these new treatments could prove beneficial. Last year with the extra funding available we were able to give the go ahead to those new treatments ranked as most important by medical experts. And with additional investment now coming on stream from April we are able to finish the job by funding all 22 of those new treatment options.”
Fiona Loud, Policy Director at the British Kidney Patient Association, recently said: “The British Kidney Patient Association welcomes today’s announcement from NHS England that funding is now being granted for the use of eculizumab in the rare condition known as C3 glomerulopathy following a kidney transplant.
“It is an important decision for people with this rare disease, and key to their being able to receive a transplant. It is vital that we maximize the success of every single kidney transplant as there are still not enough donated kidneys to allow everyone who needs a transplant to have one. Tragically, some people die whilst they are on the waiting list. This decision has taken a long time to reach, nevertheless, today there is considerable relief amongst patients who are affected.”
These three treatments are in addition to the 19 which were approved for routine use earlier in the current financial year, along with funding for an extensive trial of a prevention programme for those at risk of contracting HIV.
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