Listing of ivacaftor (Kalydeco) in the Pharmaceutical Schedule for people with cystic fibrosis and certain class III gating mutations

Medicines

Decision

From 1 March 2021 ivacaftor (Kalydeco) will be listed in the Community and the Hospital Medicines List of the Pharmaceutical Schedule.

What we’re doing

From 1 March 2021 ivacaftor (Kalydeco) will be listed in the Community and the Hospital Medicines List of the Pharmaceutical Schedule because Medsafe approval has been granted for ivacaftor.

Applying for funded ivacaftor

Since 1 March 2020, clinicians have been able to apply for funded access to ivacaftor(external link).

They have been able to do so via PHARMAC’s Exceptional Circumstances(external link) provisions.

As we indicated in our notification in February 2020(external link), now that Medsafe approval has been granted for ivacaftor, funded access to it will be made available via the standard Special Authority system. This will occur from 1 March 2021.

This change:

  • makes it easier for patients to access ivacaftor
  • saves time for prescribers because they can apply for funding online and get a decision faster

All current approvals for funded access to ivacaftor will be transferred to a Special Authority.  Clinicians do not need to do anything extra for those patients currently accessing funded ivacaftor.

Patients will need to pick up their ivacaftor from the hospital pharmacy as it will not be dispensed through community pharmacies.

Who we think will be most interested

  • People with cystic fibrosis who have the G551D mutation (or the following other class III gating mutations: G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R) 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 the listing of ivacaftor

Ivacaftor (Kalydeco) will be listed in Section B (as PCT only) and Section H of the Pharmaceutical Schedule, from 1 March 2021, at the following price (ex-manufacturer, excluding GST):

Chemical

Formulation

Brand

Pack size

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

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, due to the small number of patients and the high list price of the medicine.

A confidential rebate applies to Kalydeco that will reduce the net price to the Funder.

Ivacaftor will be listed in Section B of the Pharmaceutical Schedule subject to the following eligibility criteria:

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:

1. Patient has been diagnosed with cystic fibrosis; and

2. Either:

2.21 Patient must have G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene on at least 1 allele; or

2.22 Patient must have other gating (class III) mutation (G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R) in the CFTR gene on at least 1 allele; and

3. Patient must have a sweat chloride value of at least 60 mmol/L by quantitative pilocarpine iontophoresis or by Macroduct sweat collection system; and

4. Treatment with ivacaftor must be given concomitantly with standard therapy for this condition; and

5. 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

6. The dose of ivacaftor will not exceed one tablet or one sachet twice daily; and

7. Applicant has experience and expertise in the management of cystic fibrosis.

Similar restrictions will apply in Part II of Section H of the Pharmaceutical Schedule.

Information about 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 and oral granules for reconstitution are 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.

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.