Decision to fund treatments for rare metabolic disorders

12 April 2021

Medicines Decision

We are pleased to announce a decision to fund six treatments for people with rare metabolic disorders.

What we’re doing

We are pleased to announce a decision to fund six treatments for people with rare metabolic disorders. The following treatments will be listed on the Pharmaceutical Schedule from 1 May 2021:

Carglumic acid for acute inpatient treatment of hyperammonaemia arising from severe organic acidaemia as an alternative to haemofiltration.
Various supplements for inborn errors of metabolism:
- Coenzyme Q10 for treatment of inborn errors of metabolism responsive to coenzyme Q10 supplementation.
- Levocarnitine for treatment of inborn errors of metabolism responsive carnitine supplementation.
- Riboflavin for treatment of inborn errors of metabolism responsive to riboflavin supplementation.
- Arginine for the treatment of inborn errors of metabolism responsive to arginine supplementation
- Taurine for the treatment of mitochondrial disorders responsive to taurine supplementation.

PHARMAC currently funds these treatments for people through the Named Patient Pharmaceutical Assessment (NPPA) funding pathway. This decision will improve access to these treatments and reduce administrative burden for clinicians. We expect around 80 people with rare metabolic disorders will benefit each year.

PHARMAC has no agreement with the suppliers of these products, so the price and subsidy will be Cost Brand Source. This means that the original pack will be fully subsidised at the purchase price (GST exclusive).

Any changes to the original proposal?

This decision was subject to a consultation letter dated 5 February 2021.

Following consideration of the feedback received, we have made some changes to the proposal and funding criteria for these agents to better align with patient requirements and product availability.

Who we think will be most interested

People with rare metabolic disorders and their whānau, family and caregivers
Metabolic specialists and clinicians
Hospital and community pharmacists and DHBs

Detail about this decision

The following changes will occur in Section B and Part II of Section H of the Pharmaceutical Schedule.

Carglumic acid

Carglumic acid 200 mg dispersible tablets will be listed in Part II of Section H of the Pharmaceutical Schedule as any brand from 1 May 2021 subject to the following restriction:

Restricted

INITIATION

Metabolic physician

For the acute in-patient treatment of organic acidaemias as an alternative to haemofiltration.

Changes were made to the hospital restrictions applied to carglumic acid to better reflect the intended use as an acute in-patient treatment.

Coenzyme Q10 Section B changes

Funded access to coenzyme Q10 from 1 May 2021 will be subject to the following Special Authority criteria:

Special Authority for Subsidy

Initial application only from a metabolic physician. Approvals valid for 6 months where patient has a suspected inborn error of metabolism that may respond to coenzyme Q10 supplementation.

Renewal only from a metabolic physician. Approvals valid for 24 months for applications meeting the following criteria:

Both:

The patient has a confirmed diagnosis of an inborn error of metabolism that responds to coenzyme Q10 supplementation; and
The treatment remains appropriate and the patient is benefiting from treatment.

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

Coenzyme Q10 will be listed in in Section B of the Pharmaceutical Schedule as Cost Brand Source (CBS), subject to Special Authority criteria from 1 May 2021:

Chemical	Formulation	Brand	Pack size	Price and subsidy
Coenzyme Q10	Cap 120 mg	Solgar	30	CBS
Coenzyme Q10	Cap 160 mg	Go Healthy	60	CBS

Section H listing

Coenzyme Q10 will be listed in Part II of Section H of the Pharmaceutical Schedule as any brand, subject to restrictions from 1 May 2021:

Chemical	Formulation
Coenzyme Q10	Cap 120 mg
Coenzyme Q10	Cap 160 mg

Levocarnitine

Funded access to levocarnitine from 1 May 2021 will be subject to the following Special Authority criteria:

Special Authority for Subsidy

Initial application only from a metabolic physician. Approvals valid for 6 months where patient has a suspected inborn error of metabolism that may respond to carnitine supplementation.

Renewal only from a metabolic physician. Approvals valid for 24 months for applications meeting the following criteria:

Both:

The patient has a confirmed diagnosis of an inborn error of metabolism that responds to carnitine supplementation; and
The treatment remains appropriate and the patient is benefiting from treatment.

Levocarnitine will be listed in Section B of the Pharmaceutical Schedule as CBS subject to Special Authority criteria from 1 May 2021:

Chemical	Formulation	Brand	Pack size	Price and subsidy
Levocarnitine	Cap 500 mg	Balance	60	CBS
Levocarnitine	Tab 500 mg	Solgar	30	CBS
Levocarnitine	Cap 250 mg	Solgar	30	CBS
Levocarnitine	Oral liq 500 mg per 10 ml	Balance	300 ml	CBS

Section H listing

Currently levocarnitine capsules 500 mg, oral solution 1,000 mg per 10 ml, oral solution 1,100 mg per 15 ml and injection presentations are listed as any brand subject to the following restriction:

Prescribed by, or recommended by a neurologist, metabolic physician or metabolic disorders dietitian, or in accordance with a protocol or guideline that has been endorsed by the DHB Hospital.

Additional presentations of levocarnitine will be listed in Part II of Section H of the Pharmaceutical Schedule as any brand, subject to the same restrictions from 1 May 2021:

Chemical	Formulation
Levocarnitine	Tab 500 mg
Levocarnitine	Cap 250mg
Levocarnitine	Oral liq 500 mg per 10 ml

The two currently listed strengths of levocarnitine oral liquid will be delisted from Section H from 1 November 2021, six months after the listing of the oral liquid 500 mg per 10 ml presentation, to avoid initiation of these products in hospital on a strength that would not be available in the community.

Riboflavin

Funded access to riboflavin from 1 May 2021 will be subject to the following Special Authority criteria:

Special Authority for Subsidy

Initial application only from a metabolic physician or neurologist. Approvals valid for 6 months where patient has a suspected inborn error of metabolism that may respond to riboflavin supplementation.

Renewal only from a metabolic physician or neurologist. Approvals valid for 24 months for applications meeting the following criteria:

Both:

The patient has a confirmed diagnosis of an inborn error of metabolism that responds to riboflavin supplementation; and
The treatment remains appropriate and the patient is benefiting from treatment.

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

Riboflavin will be listed in in Section B of the Pharmaceutical Schedule as CBS, subject to Special Authority criteria from 1 May 2021:

Chemical	Formulation	Brand	Pack size	Price and subsidy
Riboflavin	Tab 100 mg	Solgar	100	CBS
Riboflavin	Tab 100 mg	Country Life	100	CBS

Section H listing

Riboflavin will be listed in Part II of Section H of the Pharmaceutical Schedule, subject to restrictions from 1 May 2021 as any brand:

Chemical	Formulation
Riboflavin	Tab 100 mg

Arginine

Funded access to arginine from 1 May 2021 will be subject to the following Special Authority criteria:

Special Authority for Subsidy

Initial application only from a metabolic physician. Approvals valid for 6 months where patient has a suspected inborn error of metabolism that may respond to arginine supplementation.

Renewal only from a metabolic physician. Approvals valid for 24 months for applications meeting the following criteria:

Both:

The patient has a confirmed diagnosis of an inborn error of metabolism that responds to arginine supplementation; and
The treatment remains appropriate and the patient is benefiting from treatment.

Arginine will be listed in in Section B of the Pharmaceutical Schedule as CBS, subject to Special Authority criteria from 1 May 2021:

Chemical	Formulation	Brand	Pack size	Price and subsidy
Arginine	Tab 1,000 mg	Clinicans	90	CBS
Arginine	Cap 500 mg	Solgar	50	CBS
Arginine	Powder	Biomed	400 g	CBS

Section H listing

Currently arginine powder and injection presentations are listed as any brand without restriction.

Additional presentations of arginine will be listed in Part II of Section H of the Pharmaceutical Schedule from 1 May 2021 as any brand:

Chemical	Formulation
Arginine	Tab 1,000 mg
Arginine	Cap 500 mg

Taurine

Funded access to taurine from 1 May 2021 will be subject to the following Special Authority criteria:

Special Authority for Subsidy

Initial application only from a metabolic physician. Approvals valid for 6 months where patient has a suspected specific mitochondrial disorder that may respond taurine supplementation.

Renewal only from a metabolic physician. Approvals valid for 24 months for applications meeting the following criteria:

Both:

The patient has a confirmed diagnosis of a specific mitochondrial disorder which responds to taurine supplementation; and
The treatment remains appropriate and the patient is benefiting from treatment.

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

Taurine will be listed in in Section B of the Pharmaceutical Schedule as CBS, subject to Special Authority criteria from 1 May 2021:

Chemical	Formulation	Brand	Pack size	Price and subsidy
Taurine	Cap 1,000 mg	Life Extension	90	CBS
Taurine	Powder	Life Extension	300 g	CBS

Section H listing

Taurine will be listed in Part II of Section H of the Pharmaceutical Schedule, subject to restrictions from 1 May 2021 as any brand:

Chemical	Formulation
Taurine	Cap 1,000 mg
Taurine	Powder

Our response to what you told us

We’re really grateful for the time people took to respond to this consultation. A summary of the main themes raised in feedback, our responses to the feedback received, and changes we have made after listening to you are listed below:

Theme	PHARMAC Comment
Responders were generally supportive of the proposal.	We are really grateful to everyone who took the time to provide feedback. We believe this decision will result in positive outcomes for patients.
Requests to change the Special Authority criteria and hospital restrictions to better align with patient requirements and product availability.	PHARMAC staff have worked closely with the Metabolic Service team on amendments to the Special Authority and hospital restrictions consulted on to better align with patient requirements and clinical practice. This included changes to the listed products. In general, this included extending initial approvals to 6 months to allow for accurate diagnosis of metabolic conditions and aligning terminology between the individual Special Authority and hospital restrictions as well as changes to the presentations listed. Changes were made to the hospital restrictions applied to carglumic acid to better reflect the intended use as an acute in-patient treatment.
Request to also consider inclusion of biotinidase deficiency, riboflavin-5-phosphate tablets which is used in the treatment of BVVL, pyridoxal-5-Phosphate tablets are used for pyridoxal-5-phosphate dependent epilepsy.	Consideration of these agents is beyond the scope of this proposal. PHARMAC staff will consider these as separate requests.
Request that a tablet or capsule formulation of arginine be added to the Schedule for individuals that may have difficulty measuring out a powder.	Following consultation and additional clinical advice we have included tablet and capsule formulations with different strengths of arginine, including a 1000mg tablet.
A responder noted that some of the products are not Medsafe approved and will require additional resource to provide product information because datasheets are not readily available.	With the exception of carglumic acid, the proposed agents are not Medsafe approved and are considered dietary supplements in New Zealand. This means that Medsafe approved products are unlikely to become available. These supplements are considered to provide significant benefit to patients with rare disorders, and their prescribing will be under the care of metabolic physicians. These products are readily available over the counter in many different doses and formulations in New Zealand. A wide range of formulations and brands have been included in the Schedule to provide choice and security regarding product availability.
A responder requested contracted products to assure product availability.	Given the relatively low volumes, these products have not been contracted for.

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 660 050.