Osteosarcoma - mifamurtide

Mifamurtide for the treatment of osteosarcoma

Status: History
Expected date of issue: October 2011
Referral date: June 2008
Process: STA
Topic area:
  • Cancer

NICE project team

Executive Lead: Gillian Leng
Technical Lead: Pall Jonsson
Communications manager: Tonya Gillis
Project manager: Bijal Joshi


Provisional schedule

Closing date for invited submissions / evidence submission: 23 October 2008
1st appraisal committee meeting: 18 February 2009
2nd appraisal committee meeting 18 March 2010
3rd appraisal committee meeting: 18 August 2010
4th appraisal committee meeting: 18 January 2011
5th appraisal committee meeting: 18 May 2011


Consultees and commentators

Consultees Commentators (no right to submit or appeal)
Manufacturers / Sponsors General
  • Takeda (mifamurtide)

Patient / Carer Groups

  • Action for Sick Children
  • Adam Dealey Foundation for Ewing Sarcoma
  • Afiya Trust
  • Age Concern England
  • Anthony Pilcher Bone Cancer Trust
  • Association for Children with Life Threatening or Terminal Conditions
  • Black Health Agency
  • Bone Cancer Research Trust
  • British Ethnic Health Awareness Foundation
  • CANCERactive
  • Cancer Black Care
  • Cancer Equality
  • Cancer Voices
  • Cancerbackup
  • Children's Society
  • Chinese National Healthy Living Centre
  • CLIC Sargent
  • Confederation of Indian Organisations
  • Counsel and Care
  • Equalities National Council
  • Help Adolescents with Cancer
  • Help the Aged
  • Leonard Cheshire Disability
  • Limbless Association
  • Limb Loss Information Centre
  • Macmillan Cancer Support
  • Maggie's Centres
  • Marie Curie Cancer Care
  • Muslim Council of Great Britain
  • Muslim Health Network
  • National Alliance of Childhood Cancer Patient Organisations
  • National Association for the Relief of Paget's Disease
  • National Cancer Alliance
  • National Children's Bureau
  • National Council for Palliative Care
  • NCH – The Children's Charity
  • RADAR - Royal Association for Disability & Rehabilitation
  • Rarer Cancers Forum
  • Sarcoma UK
  • Skill: National Bureau for Students with Disabilities
  • South Asian Health Foundation
  • Specialised Healthcare Alliance
  • Steps
  • Sue Ryder Care
  • Teenage Cancer Trust
  • Tenovus Cancer Information Centre
  • WellChild

Professional Groups

  • Association of Cancer Physicians
  • Association of Surgeons of Great Britain and Ireland
  • British Association for Services to the Elderly
  • British Association of Surgical Oncology
  • British Institute of Musculoskeletal Medicine
  • British Oncological Association
  • British Orthopaedic Association
  • British Orthopaedic Oncology Society
  • British Psychosocial Oncology Society
  • British Sarcoma Group
  • British Society of Rehabilitation Medicine
  • Cancer Networks Pharmacists Forum
  • Cancer Research UK
  • Children's Cancer and Leukaemia Group
  • Royal College of Anaesthetists
  • Royal College of General Practitioners
  • Royal College of Nursing
  • Royal College of Paediatric and Child Health
  • Royal College of Pathologists
  • Royal College of Physicians, Medical Oncology Joint Special Committee
  • Royal College of Radiologists
  • Royal College of Surgeons
  • Royal Pharmaceutical Society
  • Royal Society of Medicine – Intellectual Disabilities Forum
  • United Kingdom Clinical Pharmacy Association
  • United Kingdom Oncology Nursing Society
  • Vascular Society


  • Department of Health
  • Sheffield PCT
  • Welsh Assembly Government
  • West Sussex Teaching PCT
  • Age Concern Cymru
  • Board of Community Health Councils in Wales
  • British National Formulary
  • Cancer Care Cymru
  • Department of Health, Social Services and Public Safety for Northern Ireland
  • Medicines and Healthcare products Regulatory Agency
  • National Association of Primary Care
  • National Public Health Service for Wales
  • NHS Alliance
  • NHS Confederation
  • NHS Purchasing and Supply Agency
  • NHS Quality Improvement Scotland
  • Scottish Medicines Consortium

Comparator manufacturer(s)

  • Baxter Healthcare (ifosfamide)
  • Cephalon UK (doxorubicin)
  • Goldshield Pharmaceuticals  (methotrexate)
  • Hospira UK (cisplatin, doxorubicin, methotrexate)
  • Medac (doxorubicin)
  • Pfizer (cisplatin, doxorubicin)
  • Teva UK (cisplatin, methotrexate)
  • Wockhardt UK (cisplatin, doxorubicin, methotrexate)
  • Wyeth Pharmaceuticals (methotrexateGilead Sciences (adefovir dipivoxil)

Relevant research groups

  • Bone Research Society
  • Christopher Hardman Osteosarcoma Research Fund
  • Institute of Cancer Research
  • MRC Clinical Trials Unit
  • National Cancer Research Institute
  • National Cancer Research Network
  • Policy Research Institute on Ageing and Ethnicity
  • Research Institute for the Care of Older People

Evidence Review Group

  • National Institute for Health Research Health Technology Assessment Programme
  • School of Health & Related Research Sheffield (ScHARR)

Associated Guideline Groups

  • National Collaborating Centre for Cancer
  • National Collaborating Centre for Women and Children's Health

Associated Public Health Groups

  • None



Project history

Date Update
20 July 2011

The Board was invited to consider whether it would be helpful to the appraisal committees to clarify paragraph 5.6.2 in the Guide to the Methods of Technology Appraisal which refers to the use of discounting. The Board considered the matter and has issued a clarification to the Guide, in the following way:


‘5.6.2 The need to discount to a present value is widely accepted in economic evaluation, although the specific rate is variable across jurisdictions and over time. The Institute considers it appropriate to normally discount costs and health effects at the same rate. The annual rate of 3.5%, based on recommendations of the UK Treasury for the discounting of costs, should be applied to both costs and health effects. Where the Appraisal Committee has considered it appropriate to undertake sensitivity analysis on the effects of discounting because treatment effects are both substantial in restoring health and sustained over a very long period (normally at least 30 years), the Committee should apply a rate of 1.5% for health effects and 3.5% for costs.’


The amendment has immediate effect and will be published in due course on the Institute’s website.  Given the application this amendment has to the appraisal of mifamurtide, the Appraisal Committee will be asked to take it into account in preparing the Final Appraisal Determination (FAD). It will do so at its meeting on 17th August 2011.

6 April 2011 Mifamurtide for the treatment of osteosarcoma will be discussed at the next committee meeting on 18 May 2011.

Guidance Executive have reviewed this topic and recommended that further discussion should take place on the differential impact of discounting on costs and benefits in the context of the mifamurtide technology appraisal.
18 December 2009 The Appraisal Committee met on 18 February 2009 to discuss the above mentioned appraisal. At that time the manufacturer of the drug, IDM Pharma, informed us that the date for the launch of this drug in the UK had not been finalised, and consequently the final price had not been formally announced. We therefore could not release the decision agreed by the Committee and the evidence documentation related to the appraisal at that time.

In June 2009 IDM Pharma Inc was acquired by Takeda Pharmaceuticals Company Group. In agreement with the NICE appraisal team, Takeda will be submitting an addendum with a new cost effectiveness analysis, including a proposed patient access scheme. Furthermore, the Evidence Review Group (School of Health & Related Research Sheffield) will critique the addendum submitted by Takeda.

In light of the above, the next Committee Meeting will be scheduled for 18 March 2010, to discuss this appraisal. The appraisal has been scheduled to produce guidance as close as possible to the anticipated launch of the product in the UK.

We will not release the decision agreed by the Committee in February 2009 because the evidence on which this decision has changed.  All evidence documentation related to this appraisal will be released after the March 2010 meeting.
6 May 2009 Mifamurtide has now received a centralised marketing authorisation from the European Commission for the treatment of osteosarcoma. 

Please note that the date this drug will be launched in the UK has not been finalised and the final price of the drug has not been formally announced by the manufacturer. Therefore, we cannot release the decision agreed by the Committee and the evidence documentation related to the appraisal at this time.

This appraisal continues to be temporarily on hold, and the website will be updated once further information is available.
5 March 2009 Mifamurtidehas not received a centralised marketing authorisation from the European Commission and therefore we are unable to circulate the Appraisal Consultation Document (ACD) for consultation. As a result we have also cancelled the Appraisal Committee Meeting scheduled for 22 April 2009. This appraisal is temporarily on hold, and the website will be updated once further information is available.


Key documents

This page was last updated: 26 October 2011

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