NICE Concerned Over Bone Cancer Drug's Level Of Efficacy And High Cost In Draft Guidance

Main Category: Cancer / Oncology
Also Included In: Bones / Orthopedics
Article Date: 09 Jul 2010 - 4:00 PDT

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In evidence given to the National Institute for Health and Clinical Excellence (NICE), the drug, mifamurtide (Mepact, Takeda) has not been shown to significantly increase the overall survival of patients with a form of bone cancer more than currently available treatments.

These uncertainties over the drug's effectiveness, combined with the high cost that the NHS is being asked to pay for the treatment, means NICE is unable to recommend mifamurtide, in combination with post-operative chemotherapy drugs, for the treatment of non-metastatic, surgically treatable osteosarcoma in draft guidance published today (9 July).

Sir Andrew Dillon, NICE Chief Executive, said: "Our independent advisory committee considered that although mifamurtide could prove to be a valuable new treatment for osteosarcoma, when used in combination with postoperative chemotherapy. However, they had to base their opinion on the evidence currently available, which suggests that the drug has limited and still relatively uncertain clinical benefit."

According to the manufacturer, Takeda, mifamurtide costs £114,000 per patient for a full treatment course of 48 doses. Takeda has proposed to make the drug free of charge for a patient's first seven doses. However, the independent advisory committee did not consider this to be sufficient to overcome its concerns about the value to patients on the drug, given its very high cost.

This draft guidance has now been issued for consultation: NICE has not yet published final guidance to the NHS.

Although rare, osteosarcoma is the most common form of bone cancer. Around 150 new cases are diagnosed each year in the UK with most cases being seen in children, teenagers and young adults. Current treatment involves chemotherapy before and after surgery, first to shrink the tumour and then to destroy any remaining cancer cells, and surgery to remove the part of the bone or limb affected. In most cases limb-saving surgery can be performed although some patients will need an amputation.

Sir Andrew Dillon added: "We understand a diagnosis of cancer is very distressing, and especially so when children and young adults are affected. With this in mind, we are disappointed that the evidence for mifamurtide is not stronger. It is important to remember, though, that other, effective treatments are available in the NHS for treating this condition."

NICE's preliminary guidance is now available for public consultation until 2 August 2010. Comments can be made via the NICE website. 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. Until final guidance is published, NHS bodies should make decisions locally on the funding of specific treatments.

Notes

- View the Osteosarcoma - mifamurtide guidance for further information.

- Osteosarcoma is the most common form of primary bone cancer. Tumours can grow anywhere in the skeleton, but the most common places are in the legs or upper arm. Common symptoms include pain, redness and swelling in the affected area.

- The exact causes of osteosarcoma are unknown. It is thought genetic factors, previous exposure to radiotherapy, past bone damage and certain bone diseases like Paget's disease may increase the risk of developing osteosarcoma. However, the reasons for this are unclear.

- Mifamurtide works by stimulating macrophages, a type of white blood cell that helps the body fight infections by absorbing the disease-causing organism. These cells can normally be found in the bloodstream and connective tissue.

- Mifamurtide is a powder, which is mixed with saline and administered via an intravenous drip. It is given after surgery with the recommended dose being 2 mg/m² body surface area and is used in combination with postoperative multi-agent chemotherapy. Patients should receive mifamurtide twice a week at least 3 days apart for 12 weeks, followed by once-weekly treatments for another 24 weeks.

- According to the manufacturer's submission, mifamurtide costs £2,375 for one dose and £114,000 for a full treatment course of 48 doses. A Patient Access Scheme has been proposed by the manufacturer to reduce the cost of the drug to the NHS. Under the scheme, mifamurtide would be available at no charge to the NHS for the first 7 doses.

- The independent Appraisal Committee agreed the incremental cost-effectiveness ratio (ICER) based on the evidence available would be at least £50,000 per QALY gained, and possibly even above £100,000 per QALY gained.

- The research presented to the committee for consideration was a clinical trial where the primary focus was to compare mifamurtide in combination with postoperative multi-agent adjuvant chemotherapy (three or four agent using high-dose methotrexate, doxorubicin and cisplatin with or without ifosfamide) with postoperative multi-agent adjuvant chemotherapy (three or four agent) alone. Patients recruited to the trial all had non-metastatic, surgically treatable, malignant osteosarcoma.

- The analyses highlighted no significant increase in overall survival and, in most cases, no significant improvement in disease-free survival (the amount of time a patient can remain free of the disease).

Source:
NICE

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