For the first time, NICE has today
(Friday, 20 December) recommended an innovative new treatment to
help stop babies, children and young people who are undergoing
cancer treatment from losing their
hearing.
Cisplatin is a powerful chemotherapy
medicine which is routinely used to treat many cancers in
children. Over time, cisplatin builds up in the inner ear and can
cause inflammation and damage, known as ototoxicity, which is a
cause of hearing loss.
Anhydrous sodium thiosulfate, also
known as Pedmarqsi and made by Norgine, is recommendedin final
draft guidance for preventing hearing loss caused by cisplatin
chemotherapy in childrenfrom the ages of one month to
17-years-old with solid tumours which have not spread to other
parts of the body.
About 60% of children having
cisplatin-based treatment develop irreversible hearing loss, and
283 new cases of ototoxic hearing loss were diagnosed in people
under 18 in England between2022 and 2023.
The medicine, which is given as an
infusion by a nurse or doctor, works by binding to and blocking
the action of cisplatin that has not been taken up by cells and
preventing damage to cells in the ear. The use of anhydrous
sodium thiosulfate does not impact the effectiveness of cisplatin
chemotherapy.
It is estimated that just under 60
children and young people in England will be eligible to receive
this drug in the first year of anhydrous sodium thiosulfate's
recommendation.
Evidence from two clinical trials
showed the treatment almost halves the rate of hearing loss
inchildren receiving cisplatin chemotherapy. In one clinical
trial it was found that hearing loss occurred in 32.7% of
children who had cisplatin then anhydrous sodium thiosulfate,
compared with 63% in children who only had
cisplatin.
In another trial 56.4% of children who
had cisplatin only developed hearing loss, compared with 28.6%
who had cisplatin then anhydrous sodium
thiosulfate.
The trials also showed that, if
children did develop hearing loss, it was less severe overall in
children who had anhydrous sodium
thiosulfate.
Speech and language development,
functioning at school and at home, can all be affected if hearing
is impaired through cisplatin chemotherapy, parents told the NICE
independent committee.
Helen Knight, director of medicines evaluation at NICE,
said: “Hearing loss due to
cancer treatment is devastating for children and their families
so we are pleased to be able torecommend this ground-breaking
treatment.
“This is the first drug shown to
prevent and reduce the impact of hearing loss, and it will have a
life changing effect on the lives of children and young
people.
“Our recommendation for this
innovative treatment demonstrates NICE's commitment to focussing
on what matters most by getting the best care to patients fast
and ensuring value for the taxpayer.”
The treatment would be available on
the NHS in England within three months of NICE's final guidance
being published.
The company has a confidential
commercial arrangement in place which makes anhydrous sodium
thiosulfate available to the NHS with a
discount.
Read the full final draft guidance
for anhydrous sodium thiosulfate for preventing ototoxicity
caused by cisplatin chemotherapy in people aged one month to 17
years with localised solid tumours.
Notes to editors
An embargoed copy of the final draft guidance for anhydrous
sodium thiosulfate (Pedmarqsi) for preventing ototoxicity caused
by cisplatin chemotherapy in people aged 1 month to 17 years with
localised solid tumours is attached below:
ID1001 sodium thiosulfate
FDG for appeal [noCON].docx
