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
Further details of this proposal, including how to provide feedback, proposed eligibility criteria and background information, can be found below.
Consultation closes at
What would the effect be
From
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
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
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
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
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
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
The application was further considered by
Ivacaftor had previously been considered by
We have been in negotiations with
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.
Contact:
Tel: 021 863 342
Email: media@pharmac.govt.nz
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