Proposal to decline a funding application for eculizumab

Medicines Consultation Closed

PHARMAC is seeking feedback on a proposal to decline the application from Alexion Pharmaceuticals for funding eculizumab (Soliris) for the treatment of paroxysmal nocturnal haemoglobinuria.

Background information relevant to the proposal can be found in the documents linked below.

Why is PHARMAC proposing to decline this funding application?

This proposal to decline the funding application is consistent with the clinical advice we have received, which recommended that the application be declined because although it is an effective treatment, it is extremely expensive.

PHARMAC’s cost-utility analysis of the use of eculizumab in patients who have PNH shows that eculizumab is not very cost-effective compared with other funding options. The reason PHARMAC is proposing to decline funding is because the price requested by the supplier is extreme and, given the available budget, appears to be out of reach.

There are always more medicine funding applications than the available budget will allow. Even if DHBs had much more money available, at the current price, eculizumab is not cost-effective and would be likely to be at the back of the queue of medicines that could be funded. 

PHARMAC’s current view is that we cannot justify progressing eculizumab for funding in light of other funding options for DHBs.  By way of example, in the 2011/12 financial year, we estimate that $5.5 million was spent on new community medicine investments which benefitted approximately 19,000 patients. A decision to fund eculizumab for 12-20 patients from 2013/14 onwards would potentially mean that 40,000 other patients would be missing out on health gains (from other treatments that could be funded instead). 

We recognise that a decline proposal is not what patients with PNH are hoping for, but we are proposing to decline this funding application because we understand that certainty is something that patients with PNH and their families would prefer.

What does a proposal to ‘decline’ this funding application mean?

PHARMAC has not made a decision about the funding of eculizumab.

Before we do, we are making information available that we have based this proposal on for people to consider and comment on in detail. We want to hear from the community on its views about whether it would be appropriate to decline the funding of eculizumab. We are also interested to hear views as to whether any decision criteria (other than the eight we regularly consider) should be taken into account when deciding this application and, if so, what they should be.

All consultation responses received for this proposal will be provided to the PHARMAC Board for consideration when it makes a decision on the funding of eculizumab for PNH.

If the PHARMAC Board makes a decision to decline this funding application, it would mean that PHARMAC would not progress eculizumab for funding. However, if such a decision was made, it would not prevent PHARMAC from reconsidering funding for this treatment in the future if (for instance) material new evidence became available or if the price reduced substantially.

Feedback sought

PHARMAC welcomes feedback on this proposal. Your response may include any material which is relevant to the proposal.

Written feedback

To provide feedback, please submit it in writing by Wednesday, 31 July 2013 to:

Sue Anne Yee
Therapeutic Group Manager
Fax: 04 460 4995
Post: PO Box 10 254, Wellington 6143

All feedback received before the closing date will be considered by PHARMAC’s Board prior to making a decision on this proposal. 

Feedback we receive is subject to the Official Information Act 1982 (OIA) and we will consider any request to have information withheld in accordance with our obligations under the OIA. Anyone providing feedback, whether on their own account or on behalf of an organisation, and whether in a personal or professional capacity, should be aware that the content of their feedback and their identity may need to be disclosed in response to an OIA request.

We are not able to treat any part of your feedback as confidential unless you specifically request that we do, and then only to the extent permissible under the OIA and other relevant laws and requirements. If you would like us to withhold any commercially sensitive, confidential proprietary, or personal information included in your submission, please clearly state this in your submission and identify the relevant sections of your submission that you would like it withheld. PHARMAC will give due consideration to any such request.

Consultation meetings

In addition to providing feedback in writing, PHARMAC would welcome requests to discuss this proposal face-to-face with any interested parties. Please forward meeting requests to the email address above.

Associated documents: