10 Sources of evidence considered by the Committee

10 Sources of evidence considered by the Committee

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

  • Tappenden P, Bessey A, Pandor A, et al. Eculizumab for treating atypical haemolytic uraemic syndrome. Final report to the National Institute for Health and Care Excellence, November 2013

B. The following organisations accepted the invitation to participate in this evaluation as consultees and commentators. They were invited to comment on the draft scope, and the evaluation consultation document. Organisations listed in I, II and III 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 evaluation determination.

I. Company/sponsor:

  • Alexion Pharma UK

II. Professional/specialist and patient/carer groups:

  • aHUS Action

  • aHUS UK

  • British Society for Haematology

  • Genetic Alliance UK

  • Kidney Research UK

  • Royal College of Nursing

  • Royal College of Paediatric and Child Health

  • Royal College of Pathologists

  • Royal College of Physicians

III. Other consultees:

  • NHS England

  • Department of Health

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

  • Healthcare Improvement Scotland

  • National Institute for Health Research Health Technology Assessment

  • Welsh Kidney Patients Association

  • Welsh Government

C. The following individuals were selected from clinical and patient expert nominations from the consultees and commentators. They gave their expert personal view on eculizumab by providing oral and written evidence to the Committee.

  • Dr Rodney Gilbert, Consultant Paediatric Nephrologist, nominated by Alexion Pharma UK – clinical expert

  • Professor Tim Goodship, Professor of Renal Medicine, nominated by aHUS Action and aHUS UK – clinical expert

  • Dr Marie Scully, Consultant Haematologist, nominated by the British Society for Haematology and the Royal College of Pathologists – clinical expert

  • Elena Lilley, nominated by aHUS UK – patient expert

  • Len Woodward, Treasurer, nominated by aHUS UK – patient expert

D. The following individuals were nominated as NHS Commissioning experts by NHS England. They gave their expert/NHS commissioning personal view on eculizumab by providing oral and written evidence to the Committee.

  • Barbara Howe, National Programme of Care Director – Highly Specialised, selected by NHS England – NHS Commissioning expert

  • Edmund Jessop, Public Health Adviser, selected by NHS England – NHS Commissioning Expert

  • Fiona Marley, Assistant Head of Specialised Services, selected by NHS England – NHS Commissioning Expert

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

  • Alexion Pharma UK