NICE agrees cost-reducing scheme for new cancer drug trabectedin
Patients with a rare form of cancer called advanced soft tissue sarcoma could now benefit from a new drug called trabectedin, after the National Institute for Health and Clinical Excellence (NICE) approved the drug for NHS use.
The disease develops in the body's soft supportive tissue - such as fat, muscle and blood vessels - and affects around 2,000 people a year in the UK.
Trabectedin works by damaging the DNA in cancer cells, preventing them from growing and spreading to other parts of the body.
Research suggests that the drug may extend life by at least three months more than other NHS treatments and that it may therefore be beneficial for some of the 500 to 600 people in England and Wales with advanced soft tissue sarcoma.
In its preliminary draft guidance, NICE had argued that trabectedin would not be a cost-effective use of NHS resources, but it has since changed its mind thanks to a cost-sharing scheme agreed with manufacturer PharmaMar.
The drug has now been recommended in the institute's final draft guidance, with the NHS paying for each patient's first five treatment cycles and PharmaMar funding any cycles beyond this.
Under the latest guidance, the drug is recommended as a treatment for people with advanced soft tissue sarcoma who have previously failed to respond to treatment with anthracyclines and ifosfamide, or who are unable to tolerate those treatments.
Dr Carole Longson, director at the NICE Health Technology Evaluation Centre, said: "We are delighted the Independent Appraisal Committee has been able to recommend trabectedin in its draft guidance. It has certainly not been an easy decision to make; soft tissue sarcoma is a rare cancer and the evidence was limited.
"However, 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. 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."
The decision has been welcomed by Sarcoma UK, an organisation that disseminates information about the disease and develops support services for patients.
Director Roger Wilson commented: "I am delighted that trabectedin has been approved. This drug benefits a large proportion of the small number of patients who receive it."
Hilary Tovey, Cancer Research UK's policy manager, said that the decision is "great news for people with advanced soft tissue sarcoma".
"We are delighted with this decision and pleased to see manufacturers responding to opportunities to make their drugs available on the NHS," she added.
- NICE technology appraisals - Soft tissue sarcoma - trabectedin: final appraisal determination (FAD)