The National Institute for England and Wales has now published final guidance recommending the use of PharmaMar’s Yondelis for the treatment of soft tissue sarcomas on the National Health Service.

Soft tissue sarcomas – which develop in tissues such as fat, muscle and blood vessels – account for just 1% of all cancer diagnoses, affecting around 3,000 people a year in the UK, of which around 500-600 in England and Wales suffer from the advanced form of the condition.

Treatment options are limited, especially for patients with advanced stages of the disease, and Yondelis - a marine-based anticancer drug derived from the sea-squirt, which prevents tumour growth and spread by attacking the DNA within the cancer cells - can extend life by at least three months more than other therapies currently used on the NHS.

The cost watchdog has agreed to reimburse treatment with Yondelis (trabectedin) in patients with advanced soft tissue sarcomas but only if previous therapy with anthracyclines and ifosfamide has failed, or patients are unable to take these drugs because of intolerance or contraindications.

Patient access scheme
The news will be especially sweet for the Spanish drugmaker as the drug’s future in the UK was looking very uncertain for a while. Draft guidance published last year actually rejected Yondelis for the NHS because it was considered too expensive, but a subsequent patient access scheme proposed by PharmaMar helped change the Institute’s mind.

Under the scheme, the company will pick up the tab for any treatment with its drug beyond the fifth cycle to help reduce the burden on NHS resources, which means the full cost per patient to the health service is estimated to be around £19,941, assuming seven cycles of treatment and that PharmaMar pays the bill after the fifth one.