NICE Says no to Life Saving Treatment for Childhood Bone Cancer(1,2)

July 8, 2010
    HIGH WYCOMBE, England, July 9, 2010 /PRNewswire/ --

    - In a draft appraisal the National Institute for Health and
      Clinical Excellence (NICE) has refused to recommend the first treatment
      shown to improve survival in childhood bone cancer (osteosarcoma) in
      more than 20 years(1,2)

    - Data shows Mepact(R) (mifamurtide) reduces the risk of death
      by almost one third when added to standard chemotherapy treatment,
      compared with chemotherapy alone(3)

    - Using Mepact(R) with standard chemotherapy treatment after
      surgery has the potential to save an additional eight lives each

    - NICE has refused to fund lifesaving treatment for children
      that could only cost GBP2.5 million,(6) when the NHS spends over GBP700
      million on treating preventable lifestyle diseases, such as obesity,
      smoking and alcoholism(7-10)

    - Between half and one-third of patients receiving standard
      chemotherapy treatment will not survive beyond five years(5)

Takeda UK announces that in its draft appraisal the National Institute
for Health and Clinical Excellence (NICE) does not recommend the use of
Mepact(R) for the treatment of bone cancer (osteosarcoma) in children,
adolescents and young adults.(1) This comes following NICE’s inability to
recommend Mepact(R) due to its strict criteria for cost-effectiveness – which
does not currently accommodate the assessment of rare, ultra orphan diseases
- and despite the fact that Mepact(R) meets many of the criteria for
‘Deviating from the Threshold’ by the NICE Citizen’s Council reviewed by the
NICE Board on 20 May 2009.(11) This stated that if the treatment in question
is life saving, the patients are children, the intervention will have a major
impact on patient’s family and the illness is extremely severe and/or rare,
then the medicine is of additional value to society and shouldn’t have to
meet NICE’s current cost-effectiveness criteria.(11) In these conditions,
Mepact(R) meets all of these criteria.

Chairman of the Bone Cancer Research Trust, Michael Francis, said: “The
Trust is astonished by the decision taken by NICE. We really thought that
NICE would recognise that Mepact(R) offers the first real opportunity to help
these young people. Money seems to be available for treatments that will
extend the life of a patient with another type of cancer for a matter of a
few months but not when a young life could be extended into a working life,
paying taxes, raising a family and contributing to society. Of course we do
not begrudge funding for treatment for other cancers, but it seems perverse
that we are prepared to let those young people die for the sake of GBP2.5
– a sum which would dramatically allow more young people to survive.
We hope that some means can now be found within the NHS to review how
Mepact(R) can help at least some of those with osteosarcoma.”

Sarcoma UK is also disappointed by the recommendation from NICE that
Mepact(R) should not be funded for the treatment of osteosarcoma in the NHS.
Sarcoma UK’s director, Roger Wilson, has been an open critic of NICE and its
approach to assessing new drugs for rare cancers. He comments: “If we take
the decision down to its essentials, this means that NICE is prepared to
sacrifice children on its self-justified altar of cost-effectiveness,” he
said. “I believe that our society has higher moral principles and will not
allow an academic process, which has already been shown to be flawed, to take
this unprincipled route.”

Osteosarcoma is a rare and often fatal form of bone cancer, with
approximately 100-150 new patients diagnosed in the UK each year.(4) It is a
cancer that is mainly found in children, adolescents and young adults with
the average age of patients being 15 years, although children as young as two
years have been diagnosed with it.(12) It is usually a highly aggressive
disease that can spread to other parts of the body, usually the lungs in
about one-fifth of patients.(13) For up to one-third of newly diagnosed
patients the cancer will return.(14)

Although Takeda are disheartened with NICE’s decision, they want to
ensure that the suitable young patients that are diagnosed with osteosarcoma
each year are provided with a fighting chance, and will therefore work with
NICE through to the end of its appraisal.

There have not been any advances in the treatment of osteosarcoma for
more than 20 years,(1) with patients currently being treated with
chemotherapy given before and after the tumour has been surgically removed
from the bone (resection).(15) Between half and one-third of patients
receiving this treatment will not survive beyond five years.(5)

The new bone cancer drug, Mepact(R) can reduce the risk of death by
almost one-third(3), which means that it has the potential to save an
additional eight lives each year.(3-5)

Leading osteosarcoma expert Professor Ian Lewis, Professor of Cancer
Studies at St James University Hospital, Leeds, said of the announcement,
“It’s a great disappointment and cause for much frustration that we now have
another hurdle to get over and I would urge those involved to make Mepact(R)
a priority so that we can start saving more young lives. We hope that another
avenue be sought to ensure that Mepact(R) can be made available to patients
as soon as possible.”


1. National Institute for Health and Clinical Excellence. Appraisal
consultation document – Mifamurtide for the treatment of osteosarcoma. July

2. IDM Pharma’s Mepact (Mifamurtide, L-MTP-PE) Receives Approval in
Europe for Treatment of Patients with Non-metastatic, Resectable

3. Meyers PA, et al. Osteosarcoma: The Addition of Muramyl Tripeptide to
Chemotherapy Improves Overall Survival – A Report From The Chrildren’s
Oncology Group. J Clin Oncol 2008;26:633-638

4. MattsonJack Epi Data Feb 2009 (Based in the assumption that 56% of
sarcoma is osteosarcoma (NCI))

5. Lewis I, et al. Improvement in Histologic Response But Not Survival in
Osteosarcoma Patients Treated With Intensified Chemotherapy: A Randomized
Phase III Trial of the European Osteosarcoma Intergroup. J Natl Cancer Inst

6. Takeda UK new submission of evidence to NICE: Mifamurtide for the
treatment of Osteosarcoma: 10th December 2009

7. The Health and Social Care Information Centre. Statistics on alcohol:
England, 2009. Available at
cohol/statistics-on-alcohol-england-2009-[ns] (Last accessed: 30/04/2010)

8. The Health and Social Care Information Centre. Statistics on obesity,
physical activity and diet: England, February 2010. Available at
cal_Activity_and_Diet_England_2010.pdf (Last accessed: 30/04/2010)

9. The Health and Social Care Information Centre. Prescribing for
diabetes in England: Supplement – January 2002 to March 2009. Available at
9 (Last accessed: 30/04/2010)

10. The Health and Social Care Information Centre. Statistics on smoking:
England, 2009. Available at
oking/statistics-on-smoking-england-2009 (Last accessed:30/04/2010)

11. NICE Citizen’s Council Report on Departing from the Cost
Effectiveness Threshold document; Nov 27-29, 2008.

12. Mascarenhas L, Siegel S, Spector L, et al. Malignant Bone Tumours.
SEER AYA Monograph, 2002;8:97-109

13. Meyers PA, Gorlick R. Osteosarcoma. Pediatr Clin North Am

14. Grimer RJ. Surgical options for children with osteosarcoma. Lancet
Oncol 2005;6:85-92

15. Bielack S, Carrle D, Casali PG, et al. Osteosarcoma: ESMO Clinical
Recommendations for diagnosis, treatment and follow-up. Annals of Oncology,
2009;20(supplement 4);iv 137-iv 139, 2009

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Source: newswire

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