Appendix B: Sources of evidence considered by the Committee

A The Evidence Review Group (ERG) report for this appraisal was prepared by the School of Health and Related Research (ScHARR), University of Sheffield:

  • Pandor A et al. Mifamurtide for osteosarcoma, January 2009

  • Stevenson M, Mifamurtide for osteosarcoma: addendum critiquing the revised submitted economic model incorporating a patient access scheme, February 2010

B The following organisations accepted the invitation to participate in this appraisal as consultees and commentators. They were invited to comment on the draft scope, the ERG report and the appraisal consultation document (ACD). Organisations listed in I were also invited to make written submissions. Organisations listed in II and III had the opportunity to give their expert views. Organisations listed in I, II and III also have the opportunity to appeal against the final appraisal determination.

I Manufacturer/sponsor:

  • Takeda UK (mifamurtide)

II Professional/specialist and patient/carer groups:

  • Adam Dealey Foundation for Ewing Sarcoma

  • Bone Cancer Research Trust

  • Rarer Cancers Forum

  • 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

  • Sarcoma UK

III Other consultees:

  • Department of Health

  • Welsh Government

IV Commentator organisations (did not provide written evidence and without the right of appeal):

  • Department of Health, Social Services and Public Safety for Northern Ireland

  • National Collaborating Centre for Cancer

  • National Institute for Health Research (NIHR) Health Technology Assessment Programme (HTA Programme)

  • Healthcare Improvement Scotland

  • School of Health and Related Research (ScHARR), The University of Sheffield

C The following individuals were selected from clinical specialist and patient expert nominations from the non-manufacturer/sponsor consultees and commentators. They gave their expert personal view on mifamurtide by attending the Committee discussions and providing written evidence to the Committee. They were also invited to comment on the ACD.

  • Professor Tim Eden, Professor of Teenage and Young Adult Cancer, nominated by the Bone Cancer Research Trust – clinical specialist

  • Professor Anthony Freemont, Professor of Bone and Joint Pathology, nominated by the Royal College of Pathologists – clinical specialist

  • Dr Maria Michelagnoli, Consultant paediatric and adolescent oncologist, nominated by the Bone Cancer Research Trust – clinical specialist

  • Professor Andrew Bassim Hassan, Professor of Medical Oncology and Consultant Medical Oncologist, nominated by Bone Cancer Research Trust– clinical specialist

  • Dr Bruce Morland, Consultant Paediatric Oncologist, nominated by Royal College of Paediatrics and Child Health. Supported by Children's Cancer and Leukaemia Group– clinical specialist

  • Ms Sally Hurst, nominated by the Bone Cancer Research Trust – patient expert

  • Mr Michael Francis, nominated by the Bone Cancer Research Trust – patient expert

  • Ms Hannah Millington, nominated by the Bone Cancer Research Trust – patient expert

  • Master Callum Flynn, nominated by the Bone Cancer Research Trust – patient expert

D Representatives from the following manufacturer/sponsor attended Committee meetings. They contributed only when asked by the Committee chair to clarify specific issues and comment on factual accuracy.

  • Takeda UK

  • National Institute for Health and Care Excellence (NICE)