8 Sources of evidence considered by the Committee

A. The Evidence Review Group (ERG) report for this appraisal was prepared by Kleijnen Systematic Review Ltd (UK):

  • Wolff R, Joore MA, Ramaekers B et al. Tolvaptan for treating autosomal dominant polycystic kidney disease: a single technology appraisal, March, 2015.

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 make written submissions. Organisations listed in I, II and III also have the opportunity to appeal against the final appraisal determination.

I. Company:

  • Otsuka Pharmaceuticals

II. Professional/expert and patient/carer groups:

  • British Kidney Patient Association

  • British Renal Society

  • British Society for Histocompatibility and Immunogenetics

  • Kidney Research UK

  • PKD Charity

  • Renal Association

  • Royal College of Pathologists

  • Royal College of Physicians

III. Other consultees:

  • Department of Health

  • NHS England

  • 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

  • Healthcare Improvement Scotland

C. The following individuals were selected from clinical expert and patient expert nominations from the consultees and commentators. They gave their expert personal view on tolvaptan by attending the initial Committee discussion and providing a written statement to the Committee. They were also invited to comment on the ACD.

  • Professor Albert Ong, Professor of Renal Medicine, University of Sheffield, nominated by Otsuka Pharmaceuticals, the PKD Charity and the Renal Association – clinical expert

  • Professor Bruce Hendry, Professor of Renal Medicine, King's College London, nominated by Otsuka Pharmaceuticals – clinical expert

  • Dr John Sayer, Senior Clinical Lecturer in Nephrology, nominated by PKD Charity – clinical expert

  • Simone Goren, nominated by the PKD Charity – patient expert

  • Theresa Williams, nominated by the PKD Charity – patient expert

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

  • Otsuka Pharmaceuticals

  • National Institute for Health and Care Excellence (NICE)