Decision to fund ivacaftor (Kalydeco) for people with cystic fibrosis with the G551D mutation
We are pleased to announce a decision to fund ivacaftor (Kalydeco) for the treatment of people with cystic fibrosis with the G551D mutation (or other class III gating mutations) through an agreement with Vertex Pharmaceuticals Pty Ltd (Vertex).
Ivacaftor tablets and granules will be funded on the Pharmaceutical Schedule (national funding) at a date yet to be determined, following Medsafe approval.
Exceptional circumstances applications from clinicians for individual patients meeting the funding criteria will be considered from 1 March 2020 until such time that ivacaftor is listed on the Pharmaceutical Schedule.
Any changes to the original proposal?
This decision was subject to a consultation letter dated 24 January 2020
Following consultation feedback we have made some changes to the proposal we consulted on.
The regulatory updates required for ivacaftor tablets will take more time than initially expected. Vertex, the supplier of ivacaftor, will be submitting the necessary changes to Medsafe as soon as possible for ivacaftor tablets and granules, and Medsafe will then need time to assess this information. We want to ensure that ivacaftor meets the requirements of Medsafe to be supplied in New Zealand and we will not be listing ivacaftor tablets or granules on the Pharmaceutical Schedule before Medsafe approval.
In the meantime, from 1 March 2020 PHARMAC will consider applications under our exceptional circumstances framework for people with cystic fibrosis who meet the funding criteria, prior to listing on the Pharmaceutical Schedule. Any prescribing, sale and supply of ivacaftor tablets and granules would need to meet the requirements in Section 25 and 29 of the Medicines Act 1981. PHARMAC will communicate directly with clinicians who treat people with cystic fibrosis, so they are aware of and understand how to apply for funding. Patients and their families will need to discuss treatment options with their clinical team.
Some changes were also made to the funding criteria:
- as a result of consultation feedback, we have included an additional method to measure the sweat chloride value that is used in some DHBs
- we have clarified that the granules will only be funded for patients requiring a dose of less than 150 mg, by including a maximum dose in the criteria of 1 tablet or sachet twice daily.
Who we think will be most interested
- People with cystic fibrosis that have the G551D mutation (or other class III gating mutations) and their whānau
- People interested in the funding of medicines for cystic fibrosis and other rare disorders
- Respiratory physicians, paediatricians, respiratory nurses, and other clinicians and health professionals involved in the management of cystic fibrosis
- Hospital pharmacies and DHBs
- Pharmaceutical suppliers
Details about this decision
Ivacaftor (Kalydeco) will be listed in Section B (as PCT only) and Section H of the Pharmaceutical Schedule, at a date to be determined following Medsafe approval, at the following price (ex-manufacturer, excluding GST):
|
Chemical |
Formulation |
Brand |
Pack size |
Proposed price and subsidy |
|---|---|---|---|---|
|
Ivacaftor |
Tab 150 mg |
Kalydeco |
56 |
$29,386.00 |
|
Ivacaftor |
Grans 50 mg, sachet |
Kalydeco |
56 |
$29,386.00 |
|
Ivacaftor |
Grans 75 mg, sachet |
Kalydeco |
56 |
$29,386.00 |
Kalydeco tablets and granules will be listed as soon as reasonably practicable following Vertex’s notification to PHARMAC of any Medsafe approval.
Ivacaftor will be listed as a PCT only-Specialist pharmaceutical in Section B of the Pharmaceutical Schedule, meaning that only DHB hospitals will be able to make subsidy claims. Ivacaftor will not be dispensed through community pharmacies at this time due to the small number of patients and high list price of the medicine.
A confidential rebate will apply to Kalydeco that will reduce the net price to the Funder. Prior to a listing on the Pharmaceutical Schedule (i.e. until Medsafe approval is granted and the listing has been implemented) the above list price and the confidential rebate will apply to any individual patient funding applications approved via exceptional circumstances.
Ivacaftor will be listed in Section B of the Pharmaceutical Schedule subject to the following Special Authority criteria (changes made following consultation are indicated in bold):
Special Authority for Subsidy - PCT only - Specialist
Initial application only from a respiratory specialist or paediatrician. Approvals valid without renewal unless notified for applications meeting the following criteria:
All of the following:
- Patient has been diagnosed with cystic fibrosis; and
- Either:
2.1. Patient must have G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene on at least 1 allele; or
2.2. Patient must have other gating (class III) mutation in the CFTR gene on at least 1 allele; and
- Patients must have a sweat chloride value of at least 60 mmol/L by quantitative pilocarpine iontophoresis or by Macroduct sweat collection system; and
- Treatment with ivacaftor must be given concomitantly with standard therapy for this condition; and
- Patient must not have an acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease in the last 4 weeks prior to commencing treatment with ivacaftor; and
- The dose of ivacaftor will not exceed one tablet or one sachet twice daily; and
- Applicant has experience and expertise in the management of cystic fibrosis.
Similar restrictions would apply in Part II of Section H of the Pharmaceutical Schedules.
Funding for individual patients prior to listing on the Pharmaceutical Schedule
From 1 March 2020, exceptional circumstances applications will be considered for individuals who meet the approved funding criteria prior to listing on the Pharmaceutical Schedule. A mechanism has been set up to facilitate this process through exceptional circumstances, and to ensure that clinicians are aware of and seek informed consent from their patients regarding the regulatory status of both formulations.
The exceptional circumstances application form for ivacaftor will be available on the PHARMAC website before 1 March 2020.
Our response to what you told us
We appreciate the time people took to respond to this consultation. Overall, responders were strongly supportive of the proposal to fund ivacaftor. A summary of the main themes raised in feedback and our responses to the feedback and changes we have made after listening to you are set out below:
|
Theme |
Comment |
|---|---|
|
Many responders were strongly supportive of the proposal and noted the high health need of people cystic fibrosis with the G551D mutation and the expected benefit from treatment with ivacaftor. |
PHARMAC acknowledges that there is a high degree of public interest in the funding of ivacaftor and other rare disorder treatments. |
|
Responders raised concerns about the potential use of ivacaftor granules and tablets prior to Medsafe regulatory approval, given that regulatory approval is important for establishing the safety, efficacy and quality of the medicine. |
PHARMAC recognises the importance of ensuring that funded medicines are approved by Medsafe where possible. Ivacaftor will only be listed on the Pharmaceutical Schedule use following Medsafe approval. PHARMAC will consider applications for individual patients to receive funded ivacaftor under our exceptional circumstances framework, where it will be made clear to applicants that any prescribing, sale and supply of ivacaftor tablets and granules would need to meet the requirements in Section 25 and 29 of the Medicines Act 1981. PHARMAC will communicate this to clinicians treating these patients. |
|
Some responders suggested PHARMAC should consider funding ivacaftor for a further 23 mutations where ivacaftor may be effective. |
PHARMAC received clinical advice recommending funding for the G551D and other class III gating mutations based on the evidence reviewed. The other class III gating mutations considered to meet the intent of the criteria include G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R. We would welcome a funding application for widening access to other mutations, including the residual 23 mutations. |
|
Some responders suggested PHARMAC consider funding more treatments for cystic fibrosis that would benefit a larger proportion of people with cystic fibrosis, such as Symdeko and Trikafta. |
PHARMAC has not received any funding applications for these other treatments for cystic fibrosis to date. We would welcome funding applications for further cystic fibrosis treatments. |
|
Some responders requested that the eligibility criteria be amended to allow measurement of sweat chloride values by the Macroduct sweat collection system as this is a method currently used by some treatment centres. |
We have amended the funding criteria to allow for measurement of sweat chloride values by either pilocarpine iontophoresis or Macroduct sweat collection system. |
|
Some responders noted that there would likely be increased work for hospital pharmacists supporting the use of ivacaftor as it interacts with several liver enzyme systems as well as several medicines and foods. There would also be administrative costs from the claiming process. |
While there may be some increased resource impact on DHBs associated with monitoring ivacaftor and the claiming mechanism, the treatment provides significant health benefits for a small, well defined group of patients. |
If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 66 00 50.