In January 2025, Pedmarqsi became the first drug approved in the UK to help prevent hearing loss in children caused by cisplatin-based chemotherapy – a major milestone for hearing therapeutics and paediatric cancer care.
Our Hearing Therapeutics Initiative (HTI) played a key role in this achievement by contributing lived experience evidence to both NICE and the Scottish Medicines Consortium (SMC), helping ensure the treatment was approved for use in NHS England and NHS Scotland.
Understanding the challenge: hearing loss in childhood cancer treatment
Cisplatin is a chemotherapy drug routinely used to treat many types of cancer in children. While effective, it can cause permanent hearing loss in around 60% of children who receive this treatment. It does this by causing oxidative damage to the cells of the inner ear.
Hearing loss can have lifelong consequences, affecting communication, education, and emotional wellbeing.
What is Pedmarqsi?
Pedmarqsi is a medicine that helps prevent this hearing loss. It is an infusion of sodium thiosulphate, and works by binding to free cisplatin molecules, preventing oxidative damage. Clinical trials showed that Pedmarqsi treatment nearly halves the incidence of hearing loss in children who undergo cisplatin chemotherapy.
RNID’s Hearing Therapeutics Initiative helped to get Pedmarqsi approved and recommended by the NHS.
Helen Knight, Director of Medicines Evaluation at NICE, described the approval as a life-changing moment for children and young people:
“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.”
Contributing lived experience to drive change
In the UK, new medicines must be recommended by national bodies such as NICE and the SMC before they can be made available through the NHS. These assessments are based on clinical evidence, cost-effectiveness, and the lived experiences of patients and carers.
Working in collaboration with the National Deaf Children’s Society (NDCS), we provided essential insight and evidence to both NICE and the SMC. This included written submissions and verbal contributions during committee and engagement meetings, ensuring the real-world impact of hearing loss was fully understood by decision-makers.
By representing the voices of children, families and clinicians affected by hearing loss, we helped shape the final recommendations for Pedmarqsi.
Pedmarqsi is now approved for use in NHS England and NHS Scotland. This marks a landmark moment for hearing therapeutics and for children undergoing cancer treatment.
Ralph Holme, our Director of Research, emphasised the significance of this moment for the wider hearing therapeutics field:
“Pedmarqsi is the first drug specifically developed to help prevent hearing loss 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 be successfully brought to market.”
Continuing our commitment to hearing therapeutics
Our work in helping innovative hearing loss treatments reach healthcare providers isn’t finished yet. We continue to:
- collaborate with other organisations in the hearing loss sector to ensure lived experience remains central to future medicine assessments
- raise awareness of Pedmarqsi and the importance of hearing protection in cancer treatment
- horizon scan for emerging therapeutics under clinical evaluation, engaging with communities to understand their perspectives early in the development process
We will continue to advocate for access to innovative treatments and ensure that lived experience remains a driving force in health technology assessments.
