The National Institute for Health and Clinical Excellence (NICE) has issued new draft guidance, which recommends against the use of ipilimumab (Yervoy, Bristol-Myers Squibb) for advanced malignant melanoma in people who have received prior chemotherapy.
Leading healthcare experts have challenged the decision, saying it represents a major setback for skin cancer patients.
“The news comes as little surprise to IHS Global Insight, who had long questioned whether a drug with such a high initial price point could feasibly reach the cost-effectiveness threshold which guides NICE’s decision,” said Gustav Ando, Director of Healthcare at IHS Global Insight.
“Still, it is a major setback for cancer patients, who had been hoping that the highly innovative therapy could give some respite to the lack of treatment options.”
Sir Andrew Dillon, Chief Executive of NICE, defended the decision, saying: “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.
“Unfortunately, no patient characteristics or biomarkers have yet been identified to help identify this small group of people most likely to gain long-term benefit from receiving ipilimumab.
“The drug is also associated with a number of adverse reactions including diarrhoea, rash, fatigue, nausea, vomiting, decreased appetite, and abdominal pain which can significantly affect a patient’s quality of life.
“The Committee considered all these factors and concluded that, on the basis of the evidence provided so far, ipilimumab could not be considered a cost effective use of NHS resources.
“However, consultees, including the manufacturer, healthcare professionals and members of the public are now able to comment on the preliminary recommendations which are available for public consultation.
“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 Ipilimumab currently costs around £80k per patient whether the treatment is effective for them or not.”