Cancer drug not good value for the NHS, according to latest draft guidance
The drug ofatumumab (Arzerra, GlaxoSmithKline) is not recommended for NHS use in patients with chronic lymphocytic leukaemia that is refractory (does not respond) to the drugs, fludarabine and alemtuzumab, in draft guidance published today (16 September). The National Institute for Health and Care Excellence (NICE) is unable to recommend the treatment for this group of patients because the benefits that ofatumumab may provide patients compared with current NHS treatment does not justify the extra cost the Health Service is being asked to pay.
This draft guidance is now with consultees who have the opportunity to appeal against the proposed recommendations. NICE has not yet issued final guidance to the NHS.
As part of this appraisal, the manufacturer was asked to supply evidence of ofatumumab's effectiveness compared with best supportive care but instead submitted data on a small subgroup of patients in an ongoing clinical trial where all participants received ofatumumab. Because of this, a direct comparison with best supportive care was not possible. The Appraisal Committee concluded that this evidence was not robust enough to demonstrate how much extra time the drug may give patients' compared with best supportive care.
In a second meeting to discuss ofatumumab, the manufacturer responded to comments made by clinical experts who highlighted other studies that might contain data about patients in whom fludarabine and alemtuzumab no longer work, which could be used to help estimate the relative effectiveness of ofatumumab from the main trial that was originally submitted as evidence. This could, in effect, compare ofatumumab with best supportive care. The Appraisal Committee thought this may provide a more robust estimate of survival for best supportive care, but concluded that, because this new preliminary data indicated survival figures similar to the ones originally estimated by the manufacturer, the evidence would not change the Committee conclusions and would still not make ofatumumab an efficient use of NHS resources.
Sir Andrew Dillon, NICE Chief Executive, said: "Chronic lymphocytic leukaemia is the most common form of leukaemia in the UK and treatment can depend on a range of factors, which is why it's important to have a variety of therapies available for this disease. However, NICE can only recommend a treatment when it works better than current NHS treatments and represents good value for money for the NHS for the amount of benefit it offers patients.
"The Appraisal Committee formed its recommendations after analysing all of the evidence presented to them by the manufacturer and clinical and patient representatives at two separate meetings. In this case, they concluded that the amount of benefit that ofatumumab might provide patients over and above currently available treatments does not justify the extra cost that the NHS would have to pay for the drug."
Consultees now have until Thursday 30 September to appeal against the recommendations via the NICE website. 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.
Notes to Editors
- View the Ofatumumab draft guidance (from Thursday 16 September 2010).
- According to the draft guidance, ofatumumab is not recommended for the treatment of chronic lymphocytic leukaemia that is refractory to fludarabine and alemtuzumab.
- The draft guidance also states that people currently receiving ofatumumab for the treatment of chronic lymphocytic leukaemia that is refractory to fludarabine and alemtuzumab should have the option to continue treatment until they and their doctor consider it appropriate to stop.
- Nearly 2,000 people were diagnosed with chronic lymphocytic leukaemia in England in 2004. 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.
- In forming the guidance recommendation, the committee reviewed evidence from the manufacturer of ofatumumab, clinical experts and patients. Initial draft guidance was developed and a consultation period followed to allow consultees to provide feedback. Following this, the Appraisal Committee met for a second time and produced this latest draft guidance.
- 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 is between more than £60,500 and more 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.
- 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
- Fludarabine for the treatment of B-cell chronic lymphocytic leukaemia: http://guidance.nice.org.uk/TA29
- Rituximab as a first-line treatment for patients with the disease: http://guidance.nice.org.uk/TA174
- Rituximab for relapsed or refractory chronic lymphocytic leukaemia: http://guidance.nice.org.uk/TA193
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.
 Best supportive care is defined as regular outpatient appointments and blood transfusions and bone marrow transplants as necessary.
This page was last updated: 15 September 2010