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NICE recommends mifamurtide for treating bone cancer in children and young people

A drug for treating bone cancer in children and young people is recommended today (Weds 26 October) by NICE, the healthcare guidance body.

The final NICE guidance recommends the use of mifamurtide (Mepact, Takeda) in combination with postoperative multi-agent chemotherapy as an option for treating high-grade resectable non-metastatic osteosarcoma (bone cancer) in children, adolescents and young adults, when it's made available at a reduced cost to the NHS under a patient access scheme.

Although rare, osteosarcoma is the most common form of bone cancer. The evidence indicates that mifamurtide, when added to the usual treatment with surgery and chemotherapy, increases the number of patients who are effectively cured of their cancer. Although the number of additional patients who would benefit in this way is small and uncertain, the treatment effects are both substantial in restoring health and sustained over a very long period - the patients who are cured continue to benefit for the rest of their lives.

The guidance includes additional recommendations supporting further research into the size of the effect of mifamurtide when combined with the type of chemotherapy typically used in UK clinical practice, as well as further collection of quality of life data from people who are cured.

Sir Andrew Dillon, NICE Chief Executive, said: “We're pleased to issue guidance for the NHS today recommending mifamurtide as a treatment for osteosarcoma in children and young people. The drug is recommended in specific clinical circumstances, when it's made available at a reduced cost to the NHS under a patient access scheme.

“For the small number of patients who benefit from mifamurtide, the health benefits continue over the rest of their lives, effectively being a cure. This situation presented specific methodological issues for the independent appraisal committee regarding the way in which health benefits are valued and calculated over a very long period of time. Following a clarification from the NICE Board on this issue, and in addition to a revised patient access scheme from the manufacturer, the extra cost per unit of health gained that the NHS will pay for mifamurtide is now lower than previously determined, but still above the normal range that is usually accepted. After having looked again very carefully at a number of factors (such the curative potential of the drug for some people and the small patient population) and taking these factors into account, the Committee was able to recommend mifamurtide for osteosarcoma as a cost-effective use of NHS resources.

“Today's recommendation of mifamurtide will help children and young people with this very painful and distressing disease, as well as providing some for hope for them, and the people caring for them.”

This guidance is available from 26 October 2011.

Ends

For more information call Dr Tonya Gillis at the NICE press office on 0845 003 7782 or out of hours on 07775 583 813.

Notes to Editors

About the Appraisal

1. The full recommendation is: “Mifamurtide in combination with postoperative multi-agent chemotherapy is recommended within its licensed indication as an option for the treatment of high-grade resectable non-metastatic osteosarcoma after macroscopically complete surgical resection in children, adolescents and young adults and when mifamurtide is made available at a reduced cost to the NHS under the patient access scheme.”

2. The evidence indicates that use of mifamurtide in the circumstance covered by the recommendation can increase overall survival on average by an extra 7 people for every 100 people treated. It's estimated that around 60 people could be treated under this new guidance.

3. The manufacturer (Takeda) submitted a revised patient access scheme (PAS) to the Department of Health, the details of which are confidential as requested by the manufacturer. The new PAS reduced the incremental cost-effectiveness ratio (ICER, which is the extra cost per unit of health gained that the NHS is asked to pay) from £67,000 to £56,000.

4. Following a clarification from the NICE Board on how discounting is applied (which involves assessing the way in which health benefits are valued and calculated over a very long period of time) the independent Appraisal Committee re-examined the evidence for this appraisal. Noting the Board's clarification to the ‘Guide to the methods of technology appraisal' issued in July 2011, resulted in a manufacturer's best-case probabilistic ICER of £36,000 per quality-adjusted life year (QALY) gained.

5. NICE produced the first final draft guidance document for this appraisal in October 2010 which was unable to provisionally recommend mifamurtide for NHS use in combination with post-operative chemotherapy drugs for the treatment of high-grade non-metastatic, surgically treatable osteosarcoma. At that point, the most plausible probabilistic ICER was £67,000 per QALY gained.

The second final draft guidance document was issued in September 2011.

About osteosarcoma

6. Around 150 new cases of osteosarcoma are diagnosed each year in the UK. 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.

7. At present, treatment for osteosarcoma involves chemotherapy first to shrink the tumour, then surgery to remove the part of the bone or limb affected, followed by further chemotherapy after surgery to destroy any remaining cancer cells. Mifamurtide has a marketing authorisation for use after surgery, in combination with post-operative chemotherapy drugs.

About NICE

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

9. 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, medical technologies (including devices and diagnostics) and procedures within the NHS
  • clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.

10. NICE produces standards for patient care:

  • quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
  • Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients

11. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.

This page was last updated: 25 October 2011

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