England’s Health Service Makes Three Treatments Available for Those with Rare Diseases

England’s National Health Service (NHS) has added three treatments to the options it offers patients with rare diseases, one of which is for pulmonary arterial hypertension (PAH).

The additions will help up to 145 people annually. NHS England will cover the cost of doctors performing them.

NHS approved 19 treatments in July 2016. It also said it would fund an extensive trial of an HIV prevention program.

The three rare-disease treatments are:

• Riociguat for pulmonary arterial hypertension – It will benefit 90-125 patients a year whose pervious treatments failed.
• Eculizumab for the kidney condition C3 glomerulopathy – It will benefit an estimated patients a year who suffer a relapse following a kidney transplant.
• Second allogeneic haematopoietic stem cell transplants – It will cover around 15 patients a year who suffer a relapse following a first transplant.

“NHS England has carefully reviewed the evidence to treat PAH with riociguat,” the agency said. “We have concluded that there is enough evidence to make the treatment available.”

“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,” John Stewart, NHS England’s acting director for specialized commissioning, said in a press release.

Fiona Loud, policy director of the British Kidney Patient Association, added: “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 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.”

Today, Feb. 28, 2017, is the 10^th International Rare Disease Day, which is coordinated by EURORDIS, “the voice of rare disease patients in Europe.”