Appendix B: Sources of evidence considered by the Committee

A. The Evidence Review Group (ERG) report for this appraisal was prepared by the West Midlands Health Technology Assessment Collaboration:

  • Edlin R, Connock M, Round J et al. Azacitidine for the treatment of myelodysplastic syndrome, chronic myelomonocytic leukaemia, and acute myeloid leukaemia, April 2009

    The Decision Support Unit (DSU) prepared three reports for this appraisal:

  • Miners A. DSU report for NICE – Azacitidine STA economic model 09/12/09, December 2009

  • Davis S, Wailoo A and Carroll C. Myelodysplastic syndromes – Azacitidine: A critical appraisal of additional evidence submitted by Celgene and the MDS Foundation, September 2010

  • Davis S. Myelodysplastic syndromes – Azacitidine: Review of Celgene's response to the post-appeal ACD, December 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:

  • Celgene

II) Professional/specialist and patient/carer groups:

  • British Committee for Standardisation in Haematology

  • British Society for Haematology

  • Cancer Research UK

  • Leukaemia CARE

  • Leukaemia Research Fund

  • Leukaemia Society (UK)

  • Macmillan Cancer Support

  • MDS Patient Support Group

  • Rarer Cancers Forum

  • Royal College

  • Royal of Pathologists

  • Royal College, Medical Oncology Joint Special Committee

  • United Kingdom Oncology Nursing Society

III) Other consultees:

  • Department of Health

  • Harrow PCT

  • Stockton-On-Tees PCT

  • Welsh Assembly 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

  • NHS Quality Improvement Scotland

  • Pharmacia

  • West Midlands Health Technology Assessment Collaboration

  • Winthrop Pharmaceuticals UK

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 azacitidine by attending the initial Committee discussion and providing written evidence to the Committee. They were also invited to comment on the ACD.

  • Dr David T Bowen, Consultant Haematologist, nominated by the NCRI Haematological Oncology Clinical Studies Group/RCP/RCR/ACP/JCCO – clinical specialist

  • Dr Dominic J Culligan, Consultant Haematologist, nominated by the Royal College of Pathologists and British Committee for Standardisation in Haematology – clinical specialist

  • Mr Paul Harford, nominated by MDS UK – patient expert

  • Ms Stella Pendleton, nominated by the Rarer Cancers Forum – patient expert

  • Professor Rodney Taylor, nominated by the MDS UK Patient Support Group – patient expert

  • Ms Sophie Wintrich, nominated by the MDS UK Patient Support Group – 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.

  • Celgene

  • National Institute for Health and Care Excellence (NICE)