RARE Daily

NICE Recommends First Treatment for Rare Blood Disorder

August 19, 2021

The National Institute for Health and Care Excellence, the United Kingdom’s watchdog on the value of drugs, issued draft guidance recommending Novartis’ midostaurin to treat advanced systemic mastocytosis in adults. 

Photo: Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE

Mastocytosis is a rare condition caused by an excess number of specific blood cells called mast cells, which play a key role in the immune system by triggering inflammation in the body. Advanced systemic mastocytosis is a severe form of the disease where mast cells gather in body tissues, such as the skin, internal organs, and bones. There are three subtypes of the condition and symptoms can vary widely.

Because of the individual nature of the disease and the diversity of the subtypes, there is no current standard treatment for advanced systemic mastocytosis. Instead, treatment is based on symptoms, and can include a variety of therapies.

The new draft guidance means that about 170 patients will now be eligible for treatment with midostaurin, the first targeted therapy for advanced systemic mastocytosis to be available within the United Kingdom’s National Health Service.

The treatment, which is administered orally twice a day, works by blocking multiple enzymes involved in the condition.

Clinical studies suggests that midostaurin improves the overall survival of people with advanced systemic mastocytosis compared to several comparator treatments. There is also a substantial improvement in the quality of life for patients using midostaurin, however NICE said evidence around its effectiveness is uncertain.

NICE said that midostaurin is considered a cost-effective use of NHS resources and is recommended for routine use on the NHS.

“The symptoms experienced by patients with this rare disease can be devastating and limiting,” said Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE. “By recognizing that patients with advanced systemic mastocytosis have a limited life expectancy without midostaurin, and by working closely with the company, we can support access to this innovative treatment.”

Author: Rare Daily Staff

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