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NICE unable to recommend cancer drug in draft guidance owing to lack of robust data

In preliminary draft guidance published today (24 June), a new treatment for patients with a form of leukaemia has not been recommended for use in the NHS because evidence does not yet show how well the drug works compared with current NHS treatment, for the price the NHS is being asked to pay.

This draft guidance has now been issued for consultation: NICE has not yet published final guidance to the NHS.

Ofatumumab (Arzerra, GlaxoSmithKline) is being appraised by the National Institute for Health and Care Excellence (NICE) for the treatment of chronic lymphocytic leukaemia that is refractory (in other words, does not respond) to the drugs fludarabine and alemtuzumab.

An independent appraisal committee reviewed research submitted by the manufacturer, GlaxoSmithKline, but concluded that the data, which centred on interim results from a small subgroup of patients in an ongoing clinical trial, was not robust enough to demonstrate how well the drug improves overall survival rates compared to currently available best supportive care. These uncertainties combined with the additional cost of ofatumumab compared to current NHS treatment, led the committee to conclude that the drug would not be a good use of NHS resources.

Chronic lymphocytic leukaemia is the most common form of leukaemia in the UK with nearly 2,000 cases diagnosed in England in 2004[1]. It affects the body's immune system by encouraging a type of white blood cell called B lymphocytes to grow abnormally, causing anaemia and an increased susceptibility to infection. The condition is termed ‘refractory' if it does not respond to treatment. This can happen either at the beginning of, or during, treatment.

Sir Andrew Dillon, NICE Chief Executive, said: “We know that patients will want to try any new treatment when their disease does not respond to currently available drugs. However, we can only recommend drugs where the evidence shows them to work better than current NHS treatments, and when they represent good value for money for the NHS. In this case, the manufacturer's evidence included interim results from a clinical trial where all patients received the ofatumumab and which is not scheduled to report its full findings until next year. This meant no direct comparison could be made between ofatumumab and best supportive care and the committee that is appraising ofatumumab was unable to judge how well the drug works compared to best supportive care.

“This uncertainty, combined with the additional cost of ofatumumab compared to currently available treatment, meant the committee could not recommend the drug as an efficient use NHS resources.

“The lack of hard evidence on disease-free and overall survival rates has also been acknowledged by the UK's Medicines and Healthcare products Regulatory Agency and also the Food and Drug Administration in the USA. They have both granted a conditional licence for the drug on the proviso that more research must be carried out to prove its clinical benefit.”

NICE's preliminary guidance is now available for public consultation until 19 July 2010. Comments can be made via the NICE website; www.nice.org.uk. Any feedback received during this consultation will be considered by the committee and, following this meeting, the next version of draft guidance will be issued.

Final guidance for ofatumumab for chronic lymphocytic leukaemia that is refractory to fludarabine and alemtuzumab is expected to be published later this year. Until then, NHS bodies should make decisions locally on the funding of specific treatments.

Ends

Notes to Editors

· The guidance is available to view at: http://guidance.nice.org.uk/TA/Wave22/7 (from Thursday 24 June 2010).

· According to the draft guidance, ofatumumab is not recommended for the treatment of chronic lymphocytic leukaemia that is refractory to fludarabine and alemtuzumab.

· In forming the guidance recommendation, the committee reviewed evidence from the manufacturer of ofatumumab, clinical experts and patients.

· The manufacturer's evidence compared ofatumumab to best supportive care only. However, the data only compared the cost effectiveness of the two and did not look at how much extra benefit ofatumumab may offer patients compared to best supportive care.

· Best supportive care means regular outpatient appointments and blood transfusions and bone marrow transplants as necessary.

· Cost-effectiveness is the additional cost of one year of healthy life (expressed as the cost per quality adjusted life year, or QALY, gained). The committee considered the best available estimate for the base case ICER to be greater than £81,500 per QALY gained.

· According to the manufacturer, ofatumumab costs £182.00 per 100 mg vial, excluding VAT. The manufacturer of ofatumumab, GlaxoSmithKline, did agree a patient access scheme (PAS) with the Department of Health, in which ofatumumab will be made available to the NHS at a discounted price. However, even with this in place, ofatumumab was judged to be too costly for the uncertain benefit it may offer patients.

· Because of the uncertainties around how well ofatumumab may improve overall survival rates, as well as the cost of treatment once the disease has progressed, the committee concluded that the drug was not a cost effective use of NHS resources.

· Ofatumumab works by targeting a protein called CD-20 that is found on the surface of B lymphocytes (a type of white blood cell) and cancer cells. Ofatumumab kills these cells allowing normal lymphocytes to grow and replace those destroyed.

· Ofatumumab is administered directly into the bloodstream via an infusion. It is given once a week for eight weeks and then once a month for a total of four months. Patients receive a first dose of 300mg with subsequent doses of 2,000mg. Before treatment, patients are given analgesics, anti-histamine and IV corticosteroid therapy to reduce the likelihood of any infusion-related adverse effects.

· NICE has previously recommended the following treatments for chronic lymphocytic leukaemia

o Fludarabine for the treatment of B-cell chronic lymphocytic leukaemia: http://guidance.nice.org.uk/TA29

o Rituximab as a first-line treatment for patients with the disease: http://guidance.nice.org.uk/TA174

o Rituximab for relapsed or refractory chronic lymphocytic leukaemia: http://guidance.nice.org.uk/TA/Wave18/23 (currently pending an appeal)

About NICE

1. The National Institute for Health and Care Excellence (NICE) is the independent organisation responsible for providing national guidance on the promotion of good health and the prevention and treatment of ill health.

2. NICE produces guidance in three areas of health:

  • public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
  • health technologies - guidance on the use of new and existing medicines, treatments and procedures within the NHS
  • clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.

[1] Office of National Statistics, Cancer Statistics series MB1 no. 35. http://www.statistics.gov.uk

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This page was last updated: 23 June 2010

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Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.