It was good news for Spanish drugmaker PharmaMar this week after cost regulators changed track and gave a green light to its soft tissue sarcoma drug Yondelis for use on the National Health Service.

The National Institute for Health and Clinical Excellence has now published draft guidance recommending Yondelis (trabectedin) as an option for a restricted number of patients with advanced soft tissue sarcoma, and says it expects final guidance to be published next year.

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

Yondelis is an alkylating agent designed to prevent tumour growth and spread by attacking the DNA within the cancer cells, but the Institute had originally voted that, at around £3,500 per cycle, the drug was too expensive despite accepting that it can extend life by at least three months more than other therapies on the NHS.

However, a proposed patient access programme - under which PharmaMar has agreed to foot the bill for any treatment beyond five cycles, thus reducing the cost - helped the drug win favour with the appraisal committee and thereby its entry into the NHS.

Assessing the drug in accordance with new guidelines on so-called end-of-life therapies and alongside the patient access programme, NICE has now concluded that Yondelis is indeed a cost-effective use of resources in patients with advanced soft tissue sarcomas but only when treatment with anthracyclines and ifosfamide has failed, or in patients who cannot take these drugs because of intolerance or contraindications.

“We are delighted the Independent Appraisal Committee has been able to recommend trabectedin in its draft guidance,” commented Dr Carole Longson, Health Technology Evaluation Centre Director at NICE. “Treatment options for this type of cancer are limited and in the last 20 years there have been no major developments to treat the advanced stages of this disease,” she said, adding: “Being able to recommend trabectedin for use on the NHS represents a step forward in the care of this group of patients who may have very few treatment options left”.

Roger Wilson, Director of Sarcoma UK, also expressed his delight at the decision. “This drug benefits a large proportion of the small number of patients who receive it,” he said, and praised PharmaMar “for its determination to get this drug approved and…its constructive approach to making the treatment accessible to patients in the UK".