For the first time, NICE has 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Anhydrous sodium thiosulfate, also known as Pedmarqsi and made by Norgine, is recommended in final draft guidance for preventing hearing loss caused by cisplatin chemotherapy in children from the ages of one month to 17-years-old with solid tumours which have not spread to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 between 2022 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.
"Hearing loss due to cancer treatment is devastating for children and their families so we are pleased to be able to recommend 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, " commented Helen Knight, director of medicines evaluation at NICE.
She added: "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."
Evidence from two clinical trials showed the treatment almost halves the rate of hearing loss in children 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.
Ralph Holme, Director of Research at the Royal National Institute for Deaf People (RNID), commented: "We are thrilled by the announcement that this groundbreaking drug will be made available to young patients undergoing cancer treatment to prevent hearing loss as a side effect of their cisplatin chemotherapy.
"We look forward to seeing it being rolled out in hospitals across the country, and we are hopeful that all children who may benefit will soon have access to this vital treatment.
"Our sincere thanks go to our supporters whose contributions have enabled RNID to provide essential insight and evidence to NICE helping to make this treatment widely accessible in England. It is the first drug specifically developed to prevent hearing loss to be recommended for use in the NHS. This is an important milestone that will give confidence to those investing in and developing treatments for hearing loss that they can by successfully brought to market.”
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.
