Patient numbers and cost utility analysis for ivacaftor (Kalydeco)

OIA response

Information on the cost-utility analysis performed on ivacaftor and the number of Exceptional Circumstances applications approved.

24 September 2020

OIA response: Patient numbers and cost utility analysis for ivacaftor (Kalydeco)

Dear [name and contact details redacted]


Thank you for your request dated 27 August 2020 under the Official Information Act 1982 (OIA) for information relating to ivacaftor, brand name Kalydeco.  You asked for:

  • All information on the cost-utility (IUCR) analysis performed on Kalydeco (Ivacaftor) including information on the QALY indicators and the number of patients currently being prescribed Kalydeco in New Zealand.

PHARMAC does not hold details of the number of patients currently being prescribed ivacaftor in New Zealand, however, we are able to provide the number of Exceptional Circumstances (EC) applications approved for the use of ivacaftor in the treatment of cystic fibrosis with the G551D mutation (or other class III gating mutations).

The total number of EC applications approved for ivacaftor since 1 March 2020 (which is the effective date for considering EC applications for the funding of ivacaftor) is 34.

Please note that this may not be the same as the total number of unique patients being prescribed ivacaftor in New Zealand. PHARMAC’s budget impact analysis (BIA) of ivacaftor assumes a prevalent pool of 30 patients being eligible for ivacaftor.

Please find PHARMAC’s Technology Assessment Report (TAR) for ivacaftor included with this response. A supplementary appendix (Appendix 1) to the TAR is also provided by way of background and for completeness. This supplementary appendix sets out relevant parts of the earlier economic documentation referred to in the first paragraph of the TAR which relates to the analysis for ivacaftor.

Technology Assessment Report (TAR) [PDF, 1.3 MB]

Appendix 1 supplementary material to TAR [PDF, 447 KB]

TARs are detailed analyses of new pharmaceuticals. They are used to determine the cost-effectiveness of pharmaceuticals that we are being asked to fund.

Please note that PHARMAC approaches its assessment of requests for information under the OIA on the basis that, once released, the information becomes publicly available - in other words once we release the information to you it becomes available to any other party in that exact form (whether by you distributing it to others or by virtue of us receiving the same request from a different third party).

We don't normally publish our TARs because of commercial sensitivity; however, we have released this TAR under the OIA with a small amount of information redacted as we consider this is necessary to:

  • protect information where the making available of the information would be likely to unreasonably prejudice the commercial position of the person who supplied or who is the subject of the information (section 9(2)(b)(ii));
  • protect information which is subject to an obligation of confidence or which any person has been or could be compelled to provide under the authority of any enactment, where the making available of the information would be likely to prejudice the supply of similar information, or information from the same source, and it is in the public interest that such information should continue to be supplied (section 9(2)(ba)(i));
  • enable PHARMAC to carry on, without prejudice or disadvantage, negotiations, including commercial negotiations (section 9(2)(j)).

As required under the OIA, we also considered whether, in the circumstances, the withholding of this information was outweighed by other considerations which render it desirable, in the public interest, to make this information available.  In this case we did not consider that the public interest outweighed the reasons for withholding the information.

Please note you have the right, by way of complaint under section 28(3) of the OIA to an Ombudsman, to seek an investigation and review of our decision.

We trust that this information answers your queries.  We are making our information more freely available, so we will now publish selected OIA responses (excluding personal details) on our website.  Please get in touch with us if you have any questions about this.

Yours sincerely

Rachel Read
Manager, Policy and Government Services