Haemophilia treatments funding proposal
PHARMAC is seeking feedback on a proposal involving the funding of haemophilia treatments.
The treatments include recombinant factor VIII (rFVIII), recombinant factor IX (rFIX), recombinant factor VIIa (rFVIIa) and factor VIII inhibitor bypassing fraction. We have reached provisional agreements for the listing and supply of these treatments.
We are seeking feedback on our proposal, with a closing date for submissions of 4 pm Friday, 19 June 2015 (extended from Monday, 15 June 2015). Details of how to provide feedback are provided on the final page of this document.
The purpose of this consultation is to understand whether there are any practical or clinical issues we need to be aware of, and to seek views and any information that we may not be aware of, prior to making a decision.
This consultation document contains the following sections:
- What PHARMAC is proposing to do;
- Background to proposal;
- Details of the proposal; and
- How to provide feedback.
Specific questions are set out below; however, we encourage you to provide comment to us on any aspects of this proposal.
Our proposal is to continue funding of four haemophilia treatments, and to make changes to the funding rules. The four treatments are rFVIII, rFIX, rFVIIa and factor VIII inhibitor bypassing fraction.
The changes proposed for rFVIII would result in funding for a Preferred Brand, a Second Brand and a Rare Clinical Circumstances Brand. An expert panel would be established (the Haemophilia Treatments Panel) which would consider applications for funded access to the Second Brand or Rare Clinical Circumstances Brand of rFVIII for specific patients, taking into account a patient’s specific clinical circumstances and the suitability of the relevant brand sought.
If implemented, the changes to rFVIII funding would mean that some patients would need to change the brand of rFVIII they currently use.
Changes would occur from 1 September 2015, and would be phased-in over a six-month period, giving patients time to make any adjustments necessary to move to the Preferred Brand or clinicians time to make funding applications for the appropriate funded Second Brand or Rare Clinical Circumstances Brand of rFVIII.
This proposal would involve only minor changes to the listing of the other haemophilia treatments – rFIX, rFVIIa and factor VIII inhibitor bypassing fraction.
Recombinant Factor VIII
Preferred Brand Status, Second and Rare Clinical Circumstances Brand Statuses
- Moroctocog alfa (Xyntha, Pfizer) would be awarded Preferred Brand Status and would be the nationally preferred or first treatment choice of rFVIII. An application to the Haemophilia Treatments Panel would not be required for patients prescribed Xyntha.
- Octocog alfa (Kogenate FS, Bayer) would be awarded Second Brand Status and would be the second treatment choice of rFVIII for patients. Funded access would be by application to the Haemophilia Treatments Panel.
- Octocog alfa (Advate, Baxalta) would be awarded Rare Clinical Circumstances Brand Status and would only be funded for patients where treatment with Xyntha and Kogenate FS are clinically inappropriate. Funded access would be by application to the Haemophilia Treatments Panel.
- A Haemophilia Treatments Panel would be established, and managed by PHARMAC. The Haemophilia Treatments Panel would be largely comprised of haematologists who treat haemophilia. Clinicians would need to make an application to the Haemophilia Treatments Panel for funded access to Kogenate FS or Advate if they considered a switch to Xyntha would compromise appropriate clinical care for their current patients.
- PHARMAC proposes that patients would, where possible, be treated with Xyntha in order to obtain the best value for money from the more favourable price for this product. Clinicians would be able to seek access to funded treatment with Kogenate FS as the second treatment choice in cases where it is considered that the use of Xyntha would be likely to compromise appropriate treatment and care. Funded access to Advate would only be provided in cases where the use of Xyntha and Kogenate FS would be considered likely to compromise treatment and care.
- PHARMAC has received clinical advice that a brand switch may not be appropriate for some patients including those who:
- previously had high titre inhibitor levels;
- are undergoing active or have undergone immune tolerance therapy;
- have a known product allergy; and
- have recently commenced therapy (Previously Untreated Patients or PUPs).
- All new patients commencing rFVIII treatment from 1 March 2016 would be commenced on Xyntha as the nationally preferred brand where possible.
- This proposed arrangement would apply for three years (1 March 2016 to 28 February 2019).
- PHARMAC is aware of the impending availability of new longer-acting rFVIII products. This proposal would not prevent the funding of these products. However, because they are new treatments, they would need to be assessed by PHARMAC through its normal funding processes before a funding decision is made.
What is your view on the appropriateness of having brand preferences for funded rFVIII treatments?
What is your view on PHARMAC establishing a Haemophilia Treatments Panel to assess applications for funded access to the brands which do not have Preferred Brand Status?
Are there any other points you wish to raise about the proposed funding arrangements for rFVIII?
Transition process and implementation
- There would be a six-month transition period from 1 September 2015 to 29 February 2016 to enable current patients to be safely transitioned to Xyntha, where possible, during their next routine clinic visit.
- This six-month transition period would also allow time for clinicians to make applications to the Haemophilia Treatments Panel for continued funding of Kogenate FS or Advate in situations where they consider a change to Xyntha would be clinically inappropriate for any of their patients.
- From 1 March 2016, only patients with access approved from the Haemophilia Treatments Panel would be eligible for funded treatment with Kogenate FS and Advate. For existing patients, applications to the Haemophilia Treatments Panel should be made prior to 1 March 2016 to avoid interruption to clinical care.
- A Haemophilia Treatments Panel application is not required for patients who remain on, or are switched to, Xyntha.
- PHARMAC would continue to work closely with the National Haemophilia Management Group, the Haemophilia Treaters Group and the Haemophilia Foundation to support clinicians and patients through this proposed change process.
What is your view on a six-month transition period to allow people to transition to a new brand of rFVIII?
What might be the main concerns for a person with haemophilia (or their family) if they need to change their brand of rFVIII?
What can doctors or nurses do to help a person with haemophilia (or their family) if a change in brand of rFVIII was required?
Would a video demonstration on how to use a different brand of rFVIII be helpful?
What other resources might help people with the proposed change?
Is there anything else we should consider with regard to the proposed rFVIII transition as part of this consultation?
Recombinant Factor IX
- Nonacog alfa (BeneFIX, Pfizer) would continue to be funded and there would be no substantive changes to its current funded access arrangements.
- A 3000 iu presentation of BeneFIX would be listed on the Pharmaceutical Schedule once it has been approved by Medsafe.
- An alternative brand of rFIX, nonacog gamma (Rixubis, Baxalta) could be listed in the future, subject to Medsafe registration. It would be listed subject to restriction criteria similar to those currently in place for BeneFIX.
- There would be no brand funding restrictions for rFIX so choice of brand for treatment would be based on clinician and patient preference.
Recombinant Factor VIIa
- Eptacog alfa (NovoSeven RT, Novo Nordisk) would continue to be funded on the Pharmaceutical Schedule and there would be no substantive changes to its current funded access arrangements.
- Eptacog alfa would continue to be able to be used in non-haemophilia indications in hospitals.
Factor Eight Inhibitor Bypassing Fraction
- Baxalta’s brand of factor eight inhibitor bypassing fraction (FEIBA) would continue to be funded on the Pharmaceutical Schedule and there would be no substantive changes to its current funded access arrangements.
Haemophilia is a condition that affects the blood’s clotting ability due to a deficiency in blood clotting factors. Haemophilia A is associated with factor VIII deficiency and haemophilia B is associated with factor IX deficiency. There is an estimated 400-600 people with haemophilia in New Zealand but not all patients require treatment with factor VIII or IX replacement, depending on the severity of their condition. Every year, approximately 200 patients require factor VIII replacement therapy and 40 patients require factor IX replacement.
A complication of haemophilia treatment is the development of an inhibitor, which usually occurs shortly after replacement therapy has been initiated. The inhibitors are antibodies directed against the specific deficient factor. Recombinant factor VIIa and factor eight inhibitor bypassing agent (FEIBA) are treatments used in the treatment and management of patients with inhibitors.
2015 competitive process
The National Haemophilia Management Group (NHMG) was established in 2006 and is responsible, on behalf of 20 DHBs, for management oversight of a national haemophilia service in New Zealand. The Haemophilia Treaters Group is made up of clinicians who are involved in the clinical management of patients with haemophilia in New Zealand, and it collaborates closely with the NHMG. Since 2007, PHARMAC has managed haemophilia treatments procurement activities for DHBs in close collaboration with the NHMG and Haemophilia Treaters Group.
In July 2013, Government decided that expenditure on most haemophilia treatments, including rFVIII, rFIX, rFVIIa and factor eight inhibitor bypassing fraction, should be part of the Combined Pharmaceutical Budget managed by PHARMAC on behalf of DHBs. In performing its statutory function of making decisions about which haemophilia treatments will be funded, PHARMAC works closely with the NHMG and the Haemophilia Treaters Group.
In February 2015, PHARMAC issued a request for proposals (RFP) for the funding of the haemophilia treatments (rFVIII, rFIX, rFVIIa and factor eight inhibitor bypassing fraction). Bids received were evaluated f