LEUKAEMIA patients in Scotland will be the first to receive a new drug on the NHS which has been shown to achieve complete remission in some cases after other treatments have failed.
Campaigners and medics said access venetoclax offered patients with chronic lymphocytic leukaemia (CLL) - the most common form of the disease in adults - the chance for long-term survival without symptoms and a better quality of life. In clinical trials, patients prescribed venetoclax lived around 27 months on average without any progression in the disease and 77 per cent of all patients responded to the treatment. This compares to survival of as little as three months for half of patients failing on existing treatments.
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Around 168 new cases of CLL are diagnosed in Scotland each year. However, until now, only seven patients in Scotland with certain types of CLL have been able to benefit from the drug through an early access scheme, with the manufacturer AbbVie providing it free of charge on the expectation that it will be reimbursed by the health service later.
However, it is now expected to be offered to 322 patients in year one, rising to 409 within five years, following a ruling by the Scottish Medicines Consortium. It costs £1,108 per patient for the first cycle and £4,789 per patient for the second cycle onwards - up to £4000 cheaper than current drugs.
It will be routinely available to adult CLL patients with particular chromosome abnormalities - the 17p deletion or a TP53 mutation - who are either unsuitable for or have failed to respond to treatment with a type of drug known as a B-cell receptor (BCR) inhibitor, or to patients without these chromosome traits who have failed both chemo-immunotherapy and a BCR treatment.
Dr Mike Leach, consultant haematologist at the Beatson West of Scotland Cancer Centre, said: “The SMC approval of venetoclax is an important decision for patients with difficult-to-treat forms of CLL, particularly in cases where existing treatments such as BCR inhibitors have failed and patients have limited options left.
"The data and our clinical experience show that patients respond well to treatment with a number achieving complete remission justifying not only today’s acceptance by the SMC but also its inclusion in the latest treatment guidelines from [the European Society for Medical Oncology]. This positive decision has the potential to make a difference to the lives of this patient population.”
CLL, a cancer of the white blood cells, typically develops very slowly and many patients will not require treatment for months or years. It is caused when patients' bone marrow makes too many abnormal white blood cells, called lymphocytes, which do not work properly and over time build up in the lymphatic system where they can cause large, swollen lymph nodes to develop. Sometimes the lymphocytes also accumulate in the bone marrow, squeezing out normal white and red blood cells.
For CLL patients who develop or harbour certain gene mutations treatment is particularly challenging. One of the worst findings is a 17p deletion - meaning a tiny portion of their 17th chromosome is missing - as this means they need treatment much quicker after diagnosis, respond less well to standard chemotherapy and have shorter overall survival rates. There is also a very poor prognosis for patients with a mutation in the gene known as TP53. Mutations of this gene are believed to occur in up to 37 per cent of CLL patients.
David Innes, Chair of the CLL Support Association, said: “The positive recommendation by the SMC is very welcome news for CLL patients in Scotland. There is a clear unmet need for patients with this type of cancer, and the evidence clearly demonstrates the potential impact of venetoclax as a new therapy option that may increase survival and improve the quality of life for someone with CLL, providing an important new option for both patients and their families.”
It was among three medicines approved by the SMC yesterday, including Carfilzomib to extend survival for patients with multiple myeloma - an incurable and complex cancer of the blood cells - and Desmopressin for a urine condition.
Dr Alan MacDonald, chairman of the SMC, said: “I am pleased we have been able to accept these three new medicines for routine use by NHS Scotland.
“We heard how venetoclax can substantially reduce symptoms in CLL patients and give them a better quality of life, so we know this decision will be welcomed."
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