Skin cancer drug rejected

14^th October 2011 - The skin cancermedication ipilimumab is not suitable for use in the NHS, according to the National Institute for Health and Clinical Excellence (NICE), which highlighted doubts about clinical evidence submitted by the manufacturer and concern over side effects.

NICE also raised concerns over the expense of ipilimumab, which is marketed as Yervoy by maker, Bristol-Myers Squibb.

Ipilimumab is used to treat advanced malignant melanoma in people who have already undergone chemotherapy. It is estimated that there are between 400 and 500 people in the UK each year in whom melanoma has become advanced.

Cancer deaths

Malignant melanoma is a rare type of skin cancer which can spreads to other parts of the body in a process known as metastasis.

Although melanoma only makes up 10% of all skin cancer cases, it is responsible for most skin cancer deaths. Around 2,070 people die from melanoma each year in England and Wales.

One trial of ipilimumab, which involved tests on 676 patients, found that ipilimumab improved survival rates. Participants, who had previously been treated for advanced melanoma, were either given ipilimumab, an experimental medicine called gp100, or both ipilimumab and gp100.

The study found that patients survived for around 10 months on treatment with ipilimumab or the combination treatment, compared with six months on gp100 alone.

Clinical data

Sir Andrew Dillon, chief executive of NICE, said in a statement: "We need to be sure that new treatments provide sufficient benefits to patients to justify the significant cost the NHS is being asked to pay.

"In the case of ipilimumab, the data submitted by the manufacturer primarily came from a trial called the MDX010 20 trial. This did not compare ipilimumab with the drugs currently used to treat people with advanced or metastatic melanoma, but the results did show the drug could potentially be very effective for a small percentage of patients. However, the follow up from the trial was too short to determine how long this effect would last.

"Clinical specialists also told the independent appraisal committee that only around 30% of people treated with ipilimumab would have improved survival, with only 10% potentially experiencing long-term benefits."

In making its judgement, NICE also took into account the side-effects of ipilimumab, which include diarrhoea, rash, fatigue, nausea, vomiting, decreased appetite and abdominal pain.

Cost

Sir Andrew said there was also concern about the cost of the medication, particularly as it was difficult to identify the small number of patients most likely to gain long-term benefit from receiving it. "The manufacturer can also consider whether it wishes to reduce the acquisition cost to the NHS of the drug by proposing a patient access scheme," he said.  Patient access schemes involve a drug company offering treatments to the NHS at a special discount or deal. "Ipilimumab currently costs around £80,000 per patient whether the treatment is effective for them or not."

The draft guidance issued by NICE will now go out for consultation, with a final decision due in February 2012. Until then, any decision on whether to prescribe ipilimumab for patients must be taken locally.

Professor Peter Johnson, chief clinician at Cancer Research UK, said in an emailed statement: "It is disappointing that the high cost of Ipilimumab makes it impossible for NICE to recommend its routine use in the NHS.  

"This is a novel and potentially effective treatment for some people with melanoma, but we still do not have a good way to identify those most likely to benefit and those at risk of the occasionally severe side effects."