Proposal to list sapropterin for use in pregnancy by women with phenylketonuria
What we’re proposing
PHARMAC is seeking feedback on a proposal to fund sapropterin tablets, a treatment for the rare condition phenylketonuria (PKU), for women who are pregnant or actively planning a pregnancy and require pharmacological support to manage PKU in pregnancy.
Sapropterin (Kuvan) would be funded from 1 November 2018, through a provisional agreement with BioMarin Pharmaceutical Australia Pty Ltd.
Consultation closes at 12 noon on Monday, 17 September 2018 and feedback can be emailed to Sarita Von Afehlt, Therapeutic Group Manager: sarita.vonafehlt@pharmac.govt.nz.
What would the effect be?
For patients
From 1 November 2018, women with PKU who are pregnant or actively planning a pregnancy and require pharmacological support to manage their PKU in pregnancy would be eligible for funded treatment with sapropterin, subject to Special Authority criteria.
Only some patients with PKU respond to sapropterin. Patients who meet the criteria below would initially get a one-month supply of sapropterin to see if it works for them. If it does, those women could access funded sapropterin for up to 12 months if they are actively planning a pregnancy and also for the duration of their pregnancy.
We estimate that about six women each year may be eligible for treatment with sapropterin under this proposal.
For clinicians and dietitians
Management of patients’ phenylalanine levels would be improved, enabling less restrictive dietary advice and monitoring of these patients.
Who we think will be interested
- Women with PKU who are pregnant or planning a pregnancy
- PKU consumer interest groups
- Health practitioners involved in the management of patients with PKU
- Hospital and community pharmacists, DHBs, suppliers and wholesalers.
About sapropterin and PKU
Sapropterin is the dihydrochloride salt of synthetic tetrahydrobiopterin (BH4), a co-factor required for the metabolism of phenylalanine. It is indicated for the treatment of hyperphenylalaninemia in sapropterin-responsive patients with PKU or BH4 deficiency. Only 20% to 30% of patients respond to sapropterin treatment. You can find more information about the brand of sapropterin proposed for funding, Kuvan, in the Medsafe datasheet.(external link)
PKU is a rare metabolic genetic condition in which the body is unable to process phenylalanine, an amino acid found in high-protein foods. In New Zealand, there are about 160 people with PKU. This condition is usually managed by severe dietary restrictions including not eating high protein foods, strict planning of meals, taking special food supplements and closely monitoring phenylalanine blood levels.
Uncontrolled maternal phenylalanine levels can cause serious harm to the unborn child, including birth and developmental defects, cognitive impairment and intellectual disability, as well as prematurity and low birth weight. Dietary restriction and use of supplements may not be appropriate or sufficient to adequately control phenylalanine levels in pregnancy.
Why we’re proposing this
PHARMAC received a funding application for sapropterin in February 2016 and subsequently sought advice from the Pharmacology and Therapeutics Advisory Committee (PTAC), which recommended that sapropterin be funded for the treatment of hyperphenylalaninaemia due to PKU in women who are planning to become pregnant, or are pregnant, with a high priority. PTAC advised that in this patient group, sapropterin would help support successful pregnancy and reduce the risk of PKU-related birth defects.
More information on this funding proposal is available here(external link).
PTAC recommended that PHARMAC seek advice from the National Metabolic Service regarding possible Special Authority criteria for this patient group. PHARMAC has developed the proposed criteria in consultation with a clinician from this service.
We are aware that there are other groups of patients with PKU who may benefit from sapropterin. In May 2016, PTAC also considered a funding application for sapropterin for all other patients with PKU (i.e. non-pregnant patients), and recommended that the funding application be declined. We have since received a further sapropterin funding application for patients with PKU who are at risk of cognitive impairment and this wider group was considered by PTAC in August 2018. Minutes from this discussion will be available on the PHARMAC website once finalised.
More information on these other funding proposals is available here(external link).
Details about our proposal
Sapropterin (Kuvan) would be listed in Section B and in Part II of Section H of the Pharmaceutical Schedule from 1 November 2018 at the following price and subsidy (ex-manufacturer, excluding GST):
|
Chemical |
Presentation |
Brand |
Pack size |
Proposed price and subsidy |
|---|---|---|---|---|
|
Sapropterin dihydrochloride |
Tab soluble 100 mg |
Kuvan |
30 OP |
$1,452.70 |
A confidential rebate would apply to Kuvan that would reduce its net price to the Funder.
Kuvan 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 metabolic physician. Approvals valid for 1 month for applications meeting the following criteria:
All of the following:
- Patient has phenylketonuria (PKU) and is pregnant or actively planning to become pregnant; and
- Treatment with sapropterin is required to support management of PKU during pregnancy; and
- Sapropterin to be administered at doses no greater than a total daily dose of 20 mg/kg; and
- Sapropterin to be used alone or in combination with PKU dietary management; and
- Total treatment duration with sapropterin will not exceed 22 months for each pregnancy (includes time for planning and becoming pregnant) and treatment will be stopped after delivery.
Renewal only from a metabolic physician or relevant practitioner on the recommendation of a metabolic physician. Approvals valid for 12 months for applications meeting the following criteria:
All of the following:
- Either:
- Following the initial one-month approval, the patient has demonstrated an adequate response to a 2 to 4 week trial of sapropterin with a clinically appropriate reduction in phenylalanine levels to support management of PKU during pregnancy; or
- On subsequent renewal applications, the patient has previously demonstrated response to treatment with sapropterin and maintained adequate phenylalanine levels to support management of PKU during pregnancy; and
- Any of the following:
- Patient continues to be pregnant and treatment with sapropterin will not continue after delivery; or
- Patient is actively planning a pregnancy and this is the first renewal for treatment with sapropterin; or
- Treatment with sapropterin is required for a second or subsequent pregnancy to support management of their PKU during pregnancy; and
- Sapropterin to be administered at doses no greater than a total daily dose of 20 mg/kg; and
- Sapropterin to be used alone or in combination with PKU dietary management; and
- Total treatment duration with sapropterin will not exceed 22 months for each pregnancy (includes time for planning and becoming pregnant) and treatment will be stopped after delivery.
Similar restrictions would apply in Part II of Section H of the Pharmaceutical Schedule (the Hospital Medicines List; HML).
Kuvan would have protection from delisting and subsidy reduction until 1 November 2019.
Original (whole) pack (OP) dispensing is proposed because Kuvan is supplied in bottles of 30 tablets, which should be used within two months after first opening. Also, the weight-based dosing requirements mean that eligible patients would need to take large numbers of tablets daily and the dose of sapropterin changes over the course of pregnancy so there would be variation in the number of tablets required. OP dispensing would reduce the risk of pharmacies being left with part-packs that are not able to be dispensed or used due to being open for more than two months.
To provide feedback
Send us an email: sarita.vonafehlt@pharmac.govt.nz by 12 noon, Monday 17 September 2018.
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.