NICE has recommended for the first time an innovative treatment that could help babies, children and young people undergoing cancer treatment avoid hearing loss.
       Cisplatin is a powerful chemotherapy drug widely used to treat many types of childhood cancer. Over time, cisplatin can build up in the inner ear and cause inflammation and damage known as ototoxicity, which is one cause of hearing loss.
       The final draft recommendations recommend the use of anhydrous sodium thiosulfate, also known as Pedmarqsi and manufactured by Norgine, to prevent hearing loss caused by cisplatin chemotherapy in children aged 1 month to 17 years with solid tumors that have not spread to other parts of the body.
       Around 60% of children treated with cisplatin will develop permanent hearing loss, with 283 new cases of ototoxic hearing loss diagnosed in children under 18 years of age in England between 2022 and 2023.
       The drug, which is given as an infusion by a nurse or doctor, works by binding to cisplatin that has not been taken up by cells and blocking its action, thereby preventing damage to ear cells. The use of sodium thiosulfate anhydrous does not affect the effectiveness of cisplatin chemotherapy.
       It is estimated that in the first year of recommendation for the use of anhydrous sodium thiosulfate, around 60 million children and young people in England will be eligible to receive the drug.
       Hearing loss due to cancer treatment can have a devastating impact on children and their families, so we are pleased to be able to recommend this innovative treatment option.
       This is the first drug proven to prevent and reduce the effects of hearing loss and will have a dramatic impact on the lives of children and young people.
       Helen continued: “Our recommendation of this innovative treatment demonstrates NICE’s commitment to focusing on what matters most: delivering the best care to patients quickly and ensuring good value for money for the taxpayer.”
       Data from two clinical trials showed that the treatment nearly halved the rate of hearing loss in children who received cisplatin chemotherapy. One clinical trial found that children who received cisplatin chemotherapy followed by anhydrous sodium thiosulfate had a 32.7% rate of hearing loss, compared with a 63% rate of hearing loss in children who received cisplatin chemotherapy alone.
       In another study, 56.4% of children receiving cisplatin alone experienced hearing loss, compared with 28.6% of children receiving cisplatin followed by anhydrous sodium thiosulfate.
       The trials also showed that if children did develop hearing loss, it was generally less severe in those who used anhydrous sodium thiosulfate.
       Parents have told an independent NICE committee that if hearing loss occurs as a result of cisplatin chemotherapy, it could impact on speech and language development, as well as functioning at school and at home.
       We are pleased to announce that this groundbreaking drug will be used in young patients undergoing cancer treatment to prevent hearing loss as a side effect of cisplatin chemotherapy.
       Ralph continued: “We look forward to seeing this drug in hospitals across the country and hope that all children who could benefit from it will soon have access to this life-saving treatment. We are grateful to our supporters for their contribution, which has enabled RNID to provide NICE with important ideas and evidence to help make this drug widely available across the UK. This is the first time that a drug has been developed specifically to prevent hearing loss and is recommended for use on the NHS. This is an important milestone that will give those investing in and developing treatments for hearing loss confidence that they can successfully bring a drug to market.”
       The treatment will be available on the NHS in England within three months of the publication of the final NICE guidance.
       The company has entered into a confidential commercial agreement to supply anhydrous sodium thiosulfate to the National Health Service at a reduced price.