Proposal to fund ivacaftor (Kalydeco) for the treatment of patients with cystic fibrosis with the G551D mutation
We are seeking feedback on a proposal to fund ivacaftor (Kalydeco) for the treatment of cystic fibrosis:
with the G551D mutation (or other class III gating mutations) from 1 March 2020 through a provisional agreement with Vertex Pharmaceuticals Pty Ltd (Vertex).
Further details of this proposal, including how to provide feedback, proposed eligibility criteria and background information, can be found below.
Consultation closes at 10am on Monday, 10 February 2020 and feedback can be emailed to consult@pharmac.govt.nz.
A decision has been made
Read the notification of PHARMAC's decision – published 26 February 2020
What would the effect be?
From 1 March 2020 people with cystic fibrosis who have the G551D mutation (or other class III gating mutations) and meet certain clinical criteria would have funded access to ivacaftor. Funding is proposed for ivacaftor oral tablets, as well as the granule formulations for use in children under the age of 6.
Our clinical advice suggests that treatment with ivacaftor significantly slows the progression of cystic fibrosis and leads to reductions in pulmonary exacerbations and hospital admissions for this patient group.
We estimate that there are 30 patients in New Zealand and would be eligible for treatment under the proposed criteria.
Patients would be able to access ivacaftor through their hospital specialist. Prescriptions would be processed through DHB hospital pharmacies.
For prescribers, hospital pharmacies and DHBs
From 1 March 2020 ivacaftor would be funded for eligible people with cystic fibrosis and it would be available through hospital pharmacies and claimed via a PCT only restriction (refer to the “Details about this proposal” section for further information). This would mean that only DHB hospitals would be able to make subsidy claims. DHBs would need to determine how best to manage prescriptions for ivacaftor for patients being managed in the community.
We are not proposing that ivacaftor be dispensed through community pharmacies at this time due to the small number of patients and high list price of the medicine.
Who we think will be interested
- People with cystic fibrosis that have the G551D mutation (or other class III gating mutations) and their whanau
- 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
About ivacaftor and cystic fibrosis
Cystic fibrosis is a genetic condition that affects the lungs, the digestive system and other organs. It is usually diagnosed soon after birth. There are over 500 children and adults living with cystic fibrosis in New Zealand.
Cystic fibrosis is caused by changes in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, leading to thick and sticky mucus that blocks organ function. Symptoms and severity can vary – some people with cystic fibrosis remain well for a long period of time with minimal symptoms or hospital admissions, while others require more intensive medical care. Cystic fibrosis can lead to severe lung damage that is life threatening and eventually leads to death. There is no cure for cystic fibrosis but there are treatments that help manage the symptoms. There are currently no disease-modifying therapies funded for cystic fibrosis in New Zealand.
In patients with cystic fibrosis with the G551D mutation (or other class III gating mutation), the CFTR protein reaches the cell surface, but the ability of the protein channel to open is impaired.
Ivacaftor
Ivacaftor is an oral treatment for patients with cystic fibrosis who have the G551D mutation (or other class IIII gating mutations) in the CFTR gene. Ivacaftor improves the transport of chloride ions by helping the CFTR protein channel stay open longer. The movement of chloride ions may help keep a balance of salt and water in the lungs. Ivacaftor is effective in only a small number of patients with cystic fibrosis.
Ivacaftor is available as a 150 mg film-coated tablet. Granules for oral reconstitution is available in sachets of 50 mg and 75 mg for children under the age of 6 years. Ivacaftor is taken orally every 12 hours and treatment is lifelong.
Ivacaftor is Medsafe approved for people aged 6 years and older in New Zealand. The use of ivacaftor tablets in children under the age of 6 would be an unapproved indication. The granule formulations for children under the age of 6 are currently not approved by Medsafe. The supplier is in the process of submitting changes to Medsafe to update the regulatory status of ivacaftor granules. However, the supply, sale and prescribing of ivacaftor granules would need to meet the requirements in Section 29 of the Medicines Act 1981 until the changes are approved.
Why we’re proposing this
A funding application for ivacaftor for the treatment of patients with cystic fibrosis with the G551D mutation was considered by the Rare Disorders Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) in November 2018 and was recommended for funding with a medium priority. This recommendation was based on high health need, a lack of disease-modifying treatment options, and moderate quality evidence of a health benefit noting the limited availability of long-term data.
The application was further considered by PTAC in February 2019 and was recommended for funding with a low priority. This recommendation was based on high health need, a lack of disease-modifying treatment options, moderate quality evidence of a health benefit noting the limited availability of long-term data, and concerns regarding markers of surrogacy and high cost.
Ivacaftor had previously been considered by PTAC and the Respiratory Subcommittee of PTAC in 2014 and 2015; however, PTAC did not recommend funding at the time due to uncertainty of the evidence, the high cost and poor cost-effectiveness.
We have been in negotiations with Vertex following the positive recommendations from our clinical advisory committees and have been able to reach a commercial agreement that is satisfactory to PHARMAC.
Details about our proposal
Ivacaftor (Kalydeco) would be listed in Section B (as PCT only) and Section H of the Pharmaceutical Schedule from 1 March 2020 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 |
A confidential rebate would apply to Kalydeco that would reduce the net price to the Funder.
Ivacaftor would be listed as a PCT only-Specialist pharmaceutical in Section B of the Pharmaceutical Schedule, meaning that only DHB hospitals would be able to make subsidy claims. We are not proposing that ivacaftor be dispensed through community pharmacies at this time due to the small number of patients and high list price of the medicine.
Ivacaftor granules for oral formulation would initially be listed in the Pharmaceutical Schedule as a Section 29 product until Medsafe approval and restricted to children under the age of 6 years.
Ivacaftor would be listed in Section B of the Pharmaceutical Schedule subject to the following Special Authority criteria:
Special Authority for Subsidy
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; 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
- 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.
To provide feedback
Send us an email: consult@pharmac.govt.nz by 10am on Monday, 10 February 2020.
All feedback received before the closing date will be considered by PHARMAC’s Board (or its delegate) 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.