Decision to fund treatments for rare metabolic disorders

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:

  1. The patient has a confirmed diagnosis of an inborn error of metabolism that responds to coenzyme Q10 supplementation; and
  2. 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:

  1. The patient has a confirmed diagnosis of an inborn error of metabolism that responds to carnitine supplementation; and
  2. 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:

  1. The patient has a confirmed diagnosis of an inborn error of metabolism that responds to riboflavin supplementation; and
  2. 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:

  1. The patient has a confirmed diagnosis of an inborn error of metabolism that responds to arginine supplementation; and
  2. 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