NICE recommends first treatment for rare blood disorder
People with a rare blood disorder have access to a new treatment following draft guidance from NICE.
In draft guidance published today (19 August 2021), NICE has recommended midostaurin (Rydapt, Novartis) as a treatment for advanced systemic mastocytosis in adults.
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 diverse subtypes of the condition: aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasms, and mast cell leukaemia. Symptoms are varied and can range from frequent and unexpected diarrhoea and vomiting to anaphylaxis.
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The new draft guidance means that around 170 patients will now be eligible for treatment with midostaurin, the first targeted therapy for advanced systemic mastocytosis to be available on the NHS. The treatment is taken orally twice a day and works by blocking multiple enzymes involved in the condition.
Clinical trial evidence 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 evidence around its effectiveness is uncertain.
Therefore, midostaurin is considered a cost-effective use of NHS resources and is recommended for routine use on the NHS.
We are pleased to be able to recommend midostaurin as a treatment option for people with advanced systemic mastocytosis, despite the limitations in the clinical and comparative effectiveness evidence.
The symptoms experienced by patients with this rare disease can be devastating and limiting. By recognising 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.
“As the first targeted therapy for this form of blood disorder available on the NHS, patients with advanced systemic mastocytosis will now have their chances of survival improved while experiencing a better quality of life.
“This deal is another example of how the NHS is securing access to treatments for rare conditions, that are value for money for taxpayers.”