Proposal for Pulmonary Arterial Hypertension (PAH) treatments

Medicines Consultation Closed

What we’re proposing

We are seeking feedback on a proposal to change the way that pulmonary arterial hypertension (PAH) treatments are funded. In summary, from 1 August 2023:

  • Applications to the PAH Panel for ambrisentan (Ambrisentan Viatris, Ambrisentan Mylan), iloprost (Vebulis) and epoprostenol (Veletri), would no longer be required and all funded PAH treatments would be accessed by a standard Special Authority application in the same way as many other funded medicines.
  • The funding criteria for all PAH treatments would be amended to align with current international diagnostic criteria and New Zealand funded clinical practice.
  • The PAH treatments that currently have a renewal period of one year would be amended to two years. 

The currently funded PAH treatments which are assessed via the PAH panel process are : ambrisentan (Ambrisentan Viatris, Ambrisentan Mylan), iloprost (Vebulis) and epoprostenol (Veletri). Sildenafil (Vedafil) and bosentan (Bosentan Dr Reddy’s) are already funded via standard Special Authority applications. We anticipate that consolidating all treatment access via Special Authority would provide an easier, more streamlined way for clinicians to apply, and people to access all the funded PAH treatments. 

We welcome feedback on this proposal. Consultation closes at 5 pm on Wednesday, 17 May 2023, and feedback can be emailed to consult@pharmac.govt.nz

Further details on this proposal can be found below. 

What would the effect be?

People with a current Panel Special Authority approval for any of the PAH treatments would continue to receive funded treatment. The currently funded PAH treatments are:

  • ambrisentan (Ambrisentan Viatris/ Ambrisentan Mylan) – Assessed via PAH panel
  • iloprost (Vebulis) – Assessed via PAH panel
  • epoprostenol (Veletri) – Assessed via PAH panel
  • sildenafil (Vendafil) – Funded via Special Authority application
  • bosentan (Bosentan Dr Reddy’s)- Funded via Special Authority application

Funding for all PAH treatments would be via Special Authority Application

Funding for sildenafil and bosentan can currently be accessed via standard Special Authority applications. However, funded access to ambrisentan, iloprost and epoprostenol is presently managed by the PAH Panel. The PAH Panel consists of expert clinicians in the field of PAH treatment who assess funding applications for the PAH treatments ambrisentan, iloprost and epoprostenol against a set of eligibility criteria(external link).  We are proposing that access to these treatments change from access via Panel application, to access via Special Authority, in line with current access to sildenafil and bosentan. 

From 1 August 2023, funded access to ambrisentan (Ambrisentan Viatris/ Ambrisentan Mylan), iloprost (Vebulis) and epoprostenol (Veletri) would be managed through standard Special Authority applications without the requirement for review by the PAH Panel. Applications could be made either electronically, via the electronic Special Authority system, or manually on the Special Authority forms(external link) available on the Pharmac website. 

People with a current approval for ambrisentan (Ambrisentan Viatris /Ambrisentan Mylan) iloprost (Vebulis) and/or epoprostenol (Veletri) would be automatically issued a new Special Authority approval from 1 August 2023 to align with the new Special Authority number. At a minimum, the new Special Authority numbers would be valid for 6 months (please note however, it is proposed that some new Special Authority numbers would be valid for longer periods as detailed further below). This would allow treating clinicians to then apply for a renewal approval for their current patients, without the requirement for assessment by the PAH Panel. 

PAH treatments that currently have a renewal period of one year would be amended to two years.

The renewal period for ambrisentan, iloprost and epoprostonol, is currently one year; we are proposing to amend this to two years. This would mean clinicians would only need to apply for a funding renewal every two years, as is the current renewal period for bosentan. There would be no change to the sildenafil renewal period, which is currently indefinite. 

Funding criteria for all PAH treatments would be aligned with current funded clinical practice in New Zealand

From 1 August 2023, the Special Authority criteria for sildenafil (Vedafil) and bosentan (Bosentan Dr Reddy’s) would be amended to align key diagnostic criteria for PAH with those of the latest international guidelines, reflect current funded clinical practice in New Zealand and ensure alignment with the Special Authority criteria for ambrisentan, iloprost and epoprostenol. Each individual PAH treatment would have a separate Special Authority form and number.

Currently cardiologists, respiratory specialists and medical practitioners on the recommendation of a cardiologist or respiratory specialist can apply for funding for PAH treatments. We are proposing to include rheumatologists and amend ‘medical practitioners’ to ‘any relevant practitioner’ to ensure all relevant health care practitioners working within their scope of practice, who care for people with PAH, can apply for funded treatment. 

We consider that the proposed changes to access to funded PAH treatments would provide a more streamlined way for clinicians to apply for, and renew, funding for people who need PAH treatments. The proposal aims to reduce the burden placed on applicants and people who need PAH treatments, by enabling prescribers to complete assessments and gain approvals for funding faster and more easily.   

Who we think will be interested

  • People and their families/whānau currently receiving treatment for pulmonary arterial hypertension (PAH)
  • Patient support organisations
  • Health care professionals involved in the management of PAH
  • Pharmacists, Te Whatu Ora - Health New Zealand and suppliers of PAH treatments 

About PAH

Pulmonary arterial hypertension (PAH) is a chronic, progressive and life threatening cardiovascular and respiratory disease. PAH causes right heart failure (from increased resistance of blood vessels in the lungs), and the key symptom is worsening shortness of breath. PAH affects males and females of all ethnicities and ages. 

PAH most commonly presents in people aged between 20 and 40 years, however it can affect people of any age. Females, of any ethnicity, are reported to be disproportionally affected. PAH does not appear to disproportionately affect Māori or Pacific peoples.    

Background to the proposal

There are currently five treatments for pulmonary arterial hypertension listed in Section B and Part II of Section H of the Pharmaceutical Schedule: sildenafil, bosentan, ambrisentan, iloprost and epoprostenol. Sildenafil, bosentan and iloprost have been funded since 2009, ambrisentan has been funded since 2010, and epoprostenol has been funded since 2018. Funding for sildenafil and bosentan is currently accessed via a standard Special Authority application. However, to access funding for ambrisentan, iloprost and epoprostenol, an application to a special access panel (the PAH Panel) is required. 

Around 75 people in New Zealand are currently accessing funded treatments for PAH via the PAH Panel. 

Diagnosing PAH is complex, and a PAH Panel was established in 2009 to assess Special Authority applications to ensure funding was targeted to those most likely to benefit from treatment. The PAH Panel is made up of clinicians who review applications to ensure the person’s clinical circumstances meet the specialised detailed Special Authority eligibility criteria(external link). 

Why we’re proposing this

We sought advice from members of the PAH panel to develop appropriate Special Authority criteria to enable a transition from a Panel assessment to standard Special Authority process. The clinical advisors recommended transferring funded access for ambrisentan, iloprost and epoprostenol to a standard Special Authority that would contain revised, but similar, criteria. Considerations in relation to the transition from Panel to Special Authority included: 

  • generating Special Authority criteria for all currently funded PAH treatments
  • aligning diagnostic criteria for PAH with international guidelines, and to reflect current funded clinical practice.
  • ensuring that clinicians working within their scope of practice are able to apply for funding for PAH treatments.
  • amending the renewal periods of the PAH treatments that currently have a renewal period of one year to two years. 

This proposal to transition to a standard Special Authority and amend funding criteria is in line with the recommendations from our clinical advisors and would provide a simpler, more streamlined way for clinicians to apply, and for people to access, funded PAH treatments.

Details about our proposal 

Ambrisentan (Ambrisentan Viatris / Ambrisentan Mylan) iloprost (Vebulis) and epoprostenol (Veletri)

From 1 August 2023 applications for ambrisentan (Ambrisentan Viatris / Ambrisentan Mylan), iloprost (Vebulis) and epoprostenol (Veletri) would be made by standard Special Authority applications. The current criteria(external link) would be replaced with the following: 

ambrisentan (Ambrisentan Viatris / Ambrisentan Mylan)

➽SAQQQQ Special Authority for Subsidy

Initial application only from a respiratory specialist or cardiologist, rheumatologist, or  any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has pulmonary arterial hypertension (PAH); and
  2. PAH is in Group 1, 4 or 5 of the WHO (Venice 2003) clinical classifications; and
  3. PAH is in New York Heart Association/World Health Organization (NYHA/WHO) functional class II, III or IV; and
  4. Any of the following:
    1. All of the following:
      1. PAH has been confirmed by right heart catheterisation; and
      2. A mean pulmonary artery pressure (PAPm) > 20 mmHg (unless peri Fontan repair); and
      3. A pulmonary capillary wedge pressure (PCWP) less than or equal to 15 mmHg; and
      4. Pulmonary vascular resistance > 2 Wood Units or > 160 International Units (dyn s cm-5); and
      5. Either
        1. PAH has been demonstrated to be non-responsive in vasoreactivity assessment using iloprost or nitric oxide, as defined in the 2022 ECS/ERS Guidelines for PAH (5.1.12.2)(external link); or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
    2. Patient is a child with idiopathic PAH or PAH secondary to congenital heart disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures; and
  5. Any of the following:
    1. Both:
      1. Ambrisentan is to be used as PAH monotherapy; and
      2. Any of the following:
        1. Patient has experienced intolerable side effects with both sildenafil and bosentan; or
        2. Patient has an absolute contraindication to sildenafil and an absolute or relative contraindication to bosentan (eg due to current use of a combined oral contraceptive or liver disease); or
        3. Patient is a child with idiopathic PAH or PAH secondary to congenital heart disease; or
    2. All of the following:
      1. Ambrisentan is to be used as PAH dual therapy; and
      2. Either:
        1. Patient has tried a PAH monotherapy (sildenafil or bosentan) for at least three months and has not experienced an acceptable response to treatment according to a validated risk stratification tool**; or
        2. Patient has tried PAH dual therapy including bosentan and has experienced intolerable side effects on bosentan; and
      3. Both:
        1. Patient is presenting in NYHA/WHO functional class III or IV, and in the opinion of the treating clinician would benefit from initial dual therapy; and
        2. Patient has an absolute or relative contraindication to bosentan (eg due to current use of a combined oral contraceptive or liver disease); or
    3. Both:
      1. Ambrisentan is to be used as PAH triple therapy; and
      2. Any of the following:
        1. Patient is on the lung transplant list; or
        2. Both
          1. Patient is presenting in NYHA/WHO functional class IV; and
          2. Patient has an absolute or relative contraindication to bosentan (e.g. due to current use of a combined oral contraceptive or liver disease); or
        3. Both:
          1. Patient has tried PAH dual therapy for at least three months and remains in an unacceptable risk category according to a validated risk stratification tool**; and
          2. Patient does not have major life-threatening comorbidities and triple therapy is not being used in a palliative scenario.

Renewal from a respiratory specialist, cardiologist, rheumatologist, or any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist. Approvals valid for 2 years where the patient is continuing to derive benefit from ambrisentan treatment according to a validated PAH risk stratification tool**. 

Note

** the requirement to use a validated risk stratification tool to determine insufficient response applies to adults. Determining insufficient response in children does not require use of a validated PAH risk stratification tool, where currently no such validated tools exist for PAH risk stratification in children.

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

iloprost (Vebulis)

➽SAQQQQ Special Authority for Subsidy

Initial application only from a respiratory specialist or cardiologist, rheumatologist, or any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has pulmonary arterial hypertension (PAH); and
  2. PAH is in Group 1, 4 or 5 of the WHO (Venice 2003) clinical classifications; and
  3. PAH is in New York Heart Association/World Health Organization (NYHA/WHO) functional class II, III or IV; and
  4. Any of the following:
    1. All of the following:
      1. PAH has been confirmed by right heart catheterisation; and
      2. A mean pulmonary artery pressure (PAPm) > 20 mmHg (unless peri Fontan repair); and
      3. A pulmonary capillary wedge pressure (PCWP) less than or equal to 15 mmHg; and
      4. A pulmonary vascular resistance > 2 Wood Units or > 160 International Units (dyn s cm-5); and
      5. Either:
        1. PAH has been demonstrated to be non-responsive in vasoreactivity assessment using iloprost or nitric oxide, as defined in the 2022 ECS/ERS Guidelines for PAH (5.1.12.2)(external link); or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
    2. Patient is a child with idiopathic PAH or PAH secondary to congenital heart disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures; and
  5. Any of the following:
    1. Both
      1. Iloprost is to be used as PAH monotherapy; and
      2. Either:
        1. Patient has experienced intolerable side effects on sildenafil and both the funded endothelin receptor antagonists (ie. Both bosentan and ambrisentan) or
        2. Patient has an absolute contraindication to sildenafil and an absolute or relative contraindication to endothelin receptor antagonists; or
    2. All of the following:
      1. Iloprost is to be used as PAH dual therapy with either sildenafil or an endothelin receptor antagonist; and
      2. Either:
        1. Patient has an absolute contraindication to or has experienced intolerable side effects on sildenafil or
        2. Patient has an absolute or relative contraindication to or experienced intolerable side effects with a funded endothelin receptor antagonist; and
      3. Either:
        1. Patient has tried a PAH monotherapy for at least three months and remains in an unacceptable risk category according to a validated risk stratification tool**, or
        2. Patient is presenting in NYHA/WHO functional class III or IV, and in the opinion of the treating clinician would benefit from initial dual therapy; or
    3. Both:
      1. Iloprost is to be used as PAH triple therapy; and
      2. Any of the following:
        1. Patient is on the lung transplant list; or
        2. Patient is presenting in NYHA/WHO functional class IV; or
        3. Both:
          1. Patient has tried PAH dual therapy for at least three months and has not experienced an acceptable response to treatment according to a validated risk stratification tool**; and
          2. Patient does not have major life-threatening comorbidities and triple therapy is not being used in a palliative scenario.

Renewal from a respiratory specialist, cardiologist, rheumatologist, or any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist. Approvals valid for 2 years where patient is continuing to derive benefit from iloprost treatment according to a validated PAH risk stratification tool**. 

Note

** the requirement to use a validated risk stratification tool to determine insufficient response applies to adults. Determining insufficient response in children does not require use of a validated PAH risk stratification tool, where currently no such validated tools exist for PAH risk stratification in children.

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

epoprostenol (Veletri)

➽SAQQQQ Special Authority for Subsidy

Initial application only from a respiratory specialist, cardiologist, rheumatologist, or  any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has pulmonary arterial hypertension (PAH); and
  2. PAH is in Group 1, 4 or 5 of the WHO (Venice 2003) clinical classifications; and
  3. PAH is in New York Heart Association/World Health Organization (NYHA/WHO) functional class III or IV; and
  4. Any of the following:
    1. All of the following:
      1. PAH has been confirmed by right heart catheterisation; and
      2. A mean pulmonary artery pressure (PAPm) > 20 mmHg (unless peri Fontan repair); and
      3. A pulmonary capillary wedge pressure (PCWP) less than or equal to 15 mmHg; and
      4. A pulmonary vascular resistance > 2 Wood Units or > 160 International Units (dyn s cm-5); and
      5. Either:
        1. PAH has been demonstrated to be non-responsive in vasoreactivity assessment using iloprost or nitric oxide, as defined in the 2022 ECS/ERS Guidelines for PAH (5.1.12.2)(external link); or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
    2. Patient is a child with idiopathic PAH or PAH secondary to congenital heart disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures; and
  5. Either:
    1. All of the following:
      1. Epoprostenol is to be used as part of PAH dual therapy with either sildenafil or an endothelin receptor antagonist; and
      2. Patient is presenting in NYHA/WHO functional class IV; and
      3. Patient has tried a PAH monotherapy for at least three months and remains in an unacceptable risk category according to a validated risk stratification tool; or
    2. Both:
      1. Epoprostenol is to be used as PAH triple therapy; and
      2. Either:
        1. Patient is on the lung transplant list; or
        2. Patient is presenting in NYHA/WHO functional class IV; or
        3. Both:
          1. Patient has tried PAH dual therapy for at least three months and has not experienced an acceptable response to treatment according to a validated risk stratification tool; and
          2. Patient does not have major life-threatening comorbidities and triple therapy is not being used in a palliative scenario.

Renewal from a respiratory specialist, cardiologist, rheumatologist, or any relevant practitioner on the recommendation of a respiratory specialist or cardiologist. Approvals valid for 2 years where patient is continuing to derive benefit from epoprostenol treatment according to a validated PAH risk stratification tool**. 

Note

** the requirement to use a validated risk stratification tool to determine insufficient response applies to adults. Determining insufficient response in children does not require use of a validated PAH risk stratification tool, where currently no such validated tools exist for PAH risk stratification in children.

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

Sildenafil (Vedafil) and bosentan (Bosentan Dr Reddy’s)

From 1 August 2023 sildenafil(external link) (Vedafil) and bosentan(external link) (Bosentan Dr Reddy’s) access criteria would be replaced in Section B of the Pharmaceutical Schedule with the following:

Sildenafil (Vedafil) – PAH criteria only shown

➽SAQQQQ Special Authority for Subsidy

Initial application – (Pulmonary arterial hypertension*) only from a respiratory specialist, cardiologist, rheumatologist, or any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist. Approvals valid without further renewal unless notified for applications meeting the following criteria:

All of the following:

  1. Patient has pulmonary arterial hypertension (PAH)*; and
  2. PAH is in Group 1, 4 or 5 of the WHO (Venice 2003) clinical classifications; and
  3. PAH is in New York Heart Association/World Health Organization (NYHA/WHO) functional class II, III or IV; and
  4. Any of the following:
    1. All of the following:
      1. PAH is confirmed by right heart catheterisation; and
      2. A mean pulmonary artery pressure (PAPm) of greater than 20 mmHg; and
      3. A pulmonary capillary wedge pressure (PCWP) that is less than or equal to 15 mmHg; and
      4. Pulmonary vascular resistance (PVR) of at least 2 Wood Units or at least 160 International Units (dyn s cm-5); and
      5. Either:
        1. PAH is non-responsive in vasoreactivity assessment using iloprost or nitric oxide, as defined in the 2022 ECS/ERS Guidelines for PAH (5.1.12.2)(external link); or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
    2. Patient is a child with idiopathic PAH or PAH secondary to congenital heart disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures. 

Note: Indications marked with * are Unapproved Indications.

** the requirement to use a validated risk stratification tool to determine insufficient response applies to adults. Determining insufficient response in children does not require use of a validated PAH risk stratification tool, where currently no such validated tools exist for PAH risk stratification in children.

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

Bosentan (Bosentan Dr Reddy’s)

➽SAQQQQ Special Authority for Subsidy

Initial application only from a respiratory specialist, cardiologist, rheumatologist, or any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has pulmonary arterial hypertension (PAH)*; and
  2. PAH is in Group 1, 4 or 5 of the WHO (Venice 2003) clinical classifications; and
  3. PAH is in New York Heart Association/World Health Organization (NYHA/WHO) functional class II, III or IV; and
  4. Any of the following:
    1. All of the following:
      1. PAH has been confirmed by right heart catheterisation; and
      2. A mean pulmonary artery pressure (PAPm) > 20 mmHg (unless peri Fontan repair); and
      3. A pulmonary capillary wedge pressure (PCWP) less than or equal to 15 mmHg; and
      4. Pulmonary vascular resistance > 2 Wood Units or > 160 International Units (dyn s cm-5); and
      5. Either
        1. PAH has been demonstrated to be non-responsive in vasoreactivity assessment using iloprost or nitric oxide, as defined in the 2022 ECS/ERS Guidelines for PAH (5.1.12.2)(external link); or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
    2. Patient is a child with idiopathic PAH or PAH secondary to congenital heart disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures; and
  5. Any of the following:
    1. Both:
      1. Bosentan is to be used as PAH monotherapy; and
      2. Any of the following:
        1. Patient has experienced intolerable side effects on sildenafil; or
        2. Patient has an absolute contraindication to sildenafil; or
        3. Patient is a child with idiopathic PAH or PAH secondary to congenital heart disease; or
    2. Both:
      1. Bosentan is to be used as part of PAH dual therapy; and
      2. Either:
        1. Patient has tried a PAH monotherapy (sildenafil) for at least three months and has experienced an inadequate therapeutic response to treatment according to a validated risk stratification tool**; or
        2. Patient is presenting in NYHA/WHO functional class III or IV, and in the opinion of the treating clinician would likely benefit from initial dual therapy; or
    3. Both:
      1. Bosentan is to be used as part of PAH triple therapy; and
      2. Any of the following:
        1. Patient is on the lung transplant list; or
        2. Patient is presenting in NYHA/WHO functional class IV; or
        3. Both:
          1. Patient has tried PAH dual therapy for at least three months and has not experienced an acceptable response to treatment according to a validated risk stratification tool**; and
          2. Patient does not have major life-threatening comorbidities and triple therapy is not being used in a palliative scenario 

Renewal from a respiratory specialist, cardiologist, rheumatologist, or any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist. Approvals valid for 2 years where patient is continuing to derive benefit from bosentan treatment according to a validated PAH risk stratification tool**. 

Note

** the requirement to use a validated risk stratification tool to determine insufficient response applies to adults. Determining insufficient response in children does not require use of a validated PAH risk stratification tool, where currently no such validated tools exist for PAH risk stratification in children.

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

To provide feedback

Send us an email: consult@pharmac.govt.nz by 17 May 2023. 

All feedback received before the closing date will be considered by Pharmac Board (or its delegate) prior to making a decision on this proposal. 

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