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Availability of Orkambi on the NHS: Publication of documents

20 December 2018

The Health and Social Care Committee has now considered the documents supplied to it by NHS England, the National Institute for Health and Care Excellence (NICE) and Vertex Pharmaceuticals in response to the Chair's letter of 7 November.

The documents supplied by each party have been made available to the other parties from whom documents were requested, with an invitation to make any further comments they might wish to make before the Committee considers the matter again. Comments have been requested by Wednesday 16 January.

In the meantime, the Committee has published the documents supplied which do not contain any commercially sensitive information. The Committee reserves the right to publish the remainder of the documents it has received, and any further documents it may request, at a later stage.

The Committee will consider the matter again once any further comments have been received. In the meantime, it urges NHS England and Vertex Pharmaceuticals to continue to explore ways in which Orkambi, and the other treatments for cystic fibrosis being developed by Vertex Pharmaceuticals, can be made available to NHS patients in England at a cost-effective price.

List of documents published:

Documents from NHS England:

  • Letter from John Stewart, National Director, Specialised Commissioning, to the Chair of the Committee, dated 28th November 2018

Documents from the National Institute for Health and Care Excellence (NICE):

  • Letter from Sir Andrew Dillon, Chief Executive, dated 29th November 2018
  • Memorandum, Appendix A and B

Documents from Vertex Pharmaceuticals:

  • Letter from Simon Lem, Vice President, Regional General Manager, Europe North (dated 30th November 2018)
  • Tab 1: Submission memorandum
  • Tab 2: Independent Assessment by Meredith Pickford QC and Stefan Kuppen
  • Tab 3b: Observations on and Limitations of the NICE Single Technology Appraisal for Orkambi and other CF Medicines
  • Tab 3c: Summary of Vertex Engagement
  • Tab 3d: Memorandum regarding Why Pricing Confidentiality is in the Public Interest 

Submission from the parents of a young child with Cystic Fibrosis

Submission from European Association of Bioindustries

Submission from UK BioIndustry Association

Further information

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