Cancer groups challenge NICE ruling on Vidaza drug

Some MDS patients develop an aggressive form of leukaemia

Cancer groups are to appeal against a ruling which would deny NHS patients in England and Wales a drug for treating life-threatening bone marrow diseases.

The drug, Vidaza, gives patients with myelodysplastic syndromes (MDS) an extra nine months of life on average.

But the National Institute for Health and Clinical Excellence (NICE) ruled that Vidaza is too costly, relative to the benefits it brings.

The drug costs £45,000 a year per patient.

MDS means that the bone marrow does not produce enough of one or more types of blood cells. Most patients need regular blood transfusions.

The average survival of patients with MDS is about 20 months. Nearly a third of patients progress to acute myeloid leukaemia, an aggressive form of leukaemia.

How can it be in the patient's best interest to deny them an average of an extra nine months of life? David Hall, MDS UK Patient Support Group

Research has shown that the average survival for higher-risk MDS patients receiving Vidaza (also known as azacitidine) was 24.5 months, compared with 15 months for patients receiving conventional treatments such as supportive care or chemotherapy.

The appeal against the NICE ruling is being lodged by three cancer patient groups - the MDS UK Patient Support Group, the Leukaemia Society and the Rarer Cancers Forum. The manufacturer of Vidaza, Celgene, is also appealing against the decision.

'Significant advance'

David Hall, an MDS patient who is also chairman of the MDS UK Patient Support Group, said Vidaza was the first significant advance in medical treatment for this condition.

Mr Hall, whose group is supported by pharmaceutical companies, including Celgene, said: "The negative recommendation is a return to hopelessness for UK MDS patients," he said.

"The recommendations also create an ethical dilemma for doctors treating this condition. How can it be in the patient's best interest to deny them an average of an extra nine months of life?

"Some patients have their lives extended by much longer than the average nine months," he added.

The charity argues that the cost for the NHS is unlikely to be significant because there are only around 700 people in the UK with higher risk MDS.

'Not cost effective'

NICE published its draft guidance on Vidaza earlier this month and will now consider the appeal.

Their spokeswoman, Dr Carole Longson, said: "We are disappointed not to be able to recommend this drug."

She said that Vidaza is not a cure for MDS but could potentially prolong the lives of people with these conditions by around nine months longer than standard treatment.

However, she said: "The Appraisal Committee concluded that, relative to the benefits, the price the NHS is being asked to pay for azacitidine is still too high for it to be recommended as a cost-effective use of NHS resources."