New price deal paves way for NICE approval of treatment for rare genetic condition
A new treatment for Duchenne muscular dystrophy (DMD) is set to be available to around 1,700 people in England following positive final draft guidance from NICE published today.
Vamorolone (also called Agamree and made by Santhera), is an alternative to currently available corticosteroids used to reduce muscle inflammation and maintain muscle strength and function in people with DMD.
NICE’s previous draft guidance concluded that uncertainties in the clinical evidence and economic model meant that more evidence was needed to establish the cost-effectiveness of vamorolone.
New analysis presented by the company, together with an improved discount to the price of vamorolone, mean that NICE can now recommend it as a cost-effective use of NHS resources for people 4 years and over.
Side effects of currently available corticosteroids can include osteoporosis, reduced bone strength, increased risk of spinal fractures, weight gain, negative behaviour changes, growth restriction, reduced bone density and delayed puberty.
Vamorolone could offer important benefits because of its potential to reduce adverse events associated with corticosteroids.
Vamorolone becomes one of several non-cancer treatments recommended by NICE following the introduction of the new severity modifier. This allows NICE’s independent committees to give greater weight to severe conditions, including conditions such as DMD, that would not have qualified for additional weighting under the previous end of life criteria.
The committee agreed that a severity weight of 1.7 was appropriate to reflect the severity of DMD.
The committee felt vamorolone could offer important benefits to patients and their families because of its potential to reduce adverse events associated with current steroid treatments.
Helen continued: “Given the significant burden this disease places on children with the condition and on their parents and carers, as well as the negative impact this can have on their quality of life, a treatment with the potential to reduce side effects associated with current treatments is to be welcomed.
“We’re therefore delighted the company has been able to work with us to enable vamorolone to be made available to people with DMD.”
One of a group of muscular dystrophies, DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin, a protein important for muscle cells. Present from birth and with symptoms that usually appear at around age 3, DMD leads to gradual decline in muscle function with children often needing to use a wheelchair by early adolescence and eventually requiring artificial ventilation to breathe.