NICE makes first-of-a-kind recommendation for hearing loss drug

The National Institute for Health and Care Excellence (NICE) has recommended the first drug for NHS use to prevent cisplatin-induced hearing loss (ototoxicity) in children and young people between the ages of one month and 17 years old. Pedmarqsi ^® (anhydrous sodium thiosulfate) is indicated for these children who have localised, non-metastatic, solid tumours.

NICE and Norgine Pharmaceuticals Ltd highlighted that ototoxicity impacts around 60 percent of children given cisplatin chemotherapy cancer treatment.

“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,” Ralph Holme, Director of Research at the Royal National Institute for Deaf People (RNID) remarked.

“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,” Helen Knight, Director of medicines evaluation at NICE noted.

Evidence of efficacy in preventing and reducing hearing loss

Two clinical trials have demonstrated that the medicine almost halved the rate of ototoxicity in children given cisplatin chemotherapy. One clinical trial found that 32.7 percent participants experience hearing loss when given cisplatin then anhydrous sodium thiosulfate. This was compared to 63 percent in children only given cisplatin, NICE shared.

Another trial showed that 56.4 percent of children who were just administered with cisplatin developed hearing loss. Comparatively, 28.6 percent of participants developed ototoxicity when given cisplatin then anhydrous sodium thiosulfate.

However, these trials revealed that of the participants who developed hearing loss, it was overall less severe when they were given anhydrous sodium thiosulfate, according to NICE.

“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,” Holme explained.

The hearing loss treatment is set to be available for NHS patients within three months following publication of NICE’s final draft guidance. It is expected that just under 60 children and young people in England will be eligible for Pedmarqsi in the first year following recommendation of the drug.