Decision for Pulmonary Arterial Hypertension (PAH) treatments

Medicines Decision

What we’re doing

We are pleased to announce a decision to change the funding process for access to pulmonary arterial hypertension (PAH) treatments. From 1 August 2023 the PAH Panel application process will change to a standard Special Authority process. We anticipate that this change will provide a simpler way for clinicians to apply for funded access to PAH treatments.

In summary:

  • Applications to the PAH Panel for ambrisentan (Ambrisentan Viatris, Ambrisentan Mylan), Iloprost (Vebulis) and epoprostentol (Vetetri), will no longer be required and all funded PAH treatments will be accessed by a standard Special Authority application in the same way as many other funded medicines.
  • Special Authority criteria for all PAH treatments have been aligned to reflect diagnostic criteria used in clinical practice in New Zealand.
  • PAH treatments that currently have a renewal period of one year will be amended to two years.

What does this mean for people?

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

From 1 August 2023, funded access to ambrisentan (Ambrisentan Viatris and Ambrisentan Mylan), nebulised iloprost (Vebulis) and epoprostenol (Veletri) will be managed through standard Special Authority(external link) applications. Respiratory specialists, cardiologists, rheumatologists, or any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist will be able to submit a Special Authority application for new patients without a requirement for review by the PAH Panel. Applications can be made either electronically, via the electronic Special authority system, or manually on the Special Authority forms, which will be available on the Pharmac website.

People with a current PAH Panel Special Authority approval for ambrisentan, iloprost and/or epoprostenol will be issued a new Special Authority approval from 1 August 2023 to align with the new Special Authority form number. At a minimum, the new Special Authority numbers will be valid for 6 months. This will allow treating clinicians to then apply for a renewal approval for their current patients, without the requirement for assessment by the PAH Panel.

From 1 August 2023, the renewal period for ambrisentan, iloprost and epoprostenol will be changed from one year to two years.

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

From 1 August 2023, the Special Authority criteria for sildenafil (Vedafil) and bosentan (Bosentan Dr Reddy’s) will 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 will have a separate Special Authority form(external link) and approval number; these forms and approvals are not interchangeable across the treatments.

Any changes to the proposal

This decision was subject to a consultation letter dated 2 May 2023. We’re really grateful for the time people took to respond to this consultation. We have made some changes to the Special Authority criteria following consideration of the consultation feedback, as follows:

  • We have removed the requirement to perform vasoreactivity testing at the time of Right Heart Catheterisation (RHC) for people with chronic thromboembolic pulmonary hypertension (CTEPH). Vasoreactivity testing will now only be required for people with idiopathic / heritable or drug-associated PAH type.
  • We have removed the requirement for children to undergo Right Heart Catheter (RHC) diagnostic studies to initiate PAH treatment.

Who we think will be interested

  • People and their families/whānau currently receiving treatment for pulmonary arterial hypertension (PAH)
  • Consumer support organisations
  • Health care professionals involved in the care of people with PAH
  • Te Whatu Ora - Health New Zealand hospitals and other organisations who deliver services and support for people with PAH
  • Pharmaceutical suppliers and wholesalers

Details about the decision

Ambrisentan (Ambrisentan Viatris /Ambrisentan Mylan)

From 1 August 2023, applications for ambrisentan (Ambrisentan Viatris and Ambrisentan Mylan), will be made by the standard Special Authority application process.

Ambrisentan access criteria will be amended in Section B of the Pharmaceutical Schedule and replaced with the following: 

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) greater than 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 greater than 2 Wood Units or greater than 160 International Units (dyn s cm-5); and
      5. Any of the following:
        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 (see note below for link to these guidelines)†; or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
        3. Patient has PAH other than idiopathic / heritable or drug-associated type; or
    2. Patient is a child with PAH secondary to congenital heart disease or PAH due to idiopathic, congenital or developmental lung disorders including chronic neonatal lung disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures or a major complication of the Fontan circulation requiring the minimising of pulmonary/venous filling 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 (e.g. 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 (e.g. 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 only 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 European Respiratory Journal Guidelines can be found here: 2022 ECS/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension(external link)

** 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 will apply in Part II of Section H of the Pharmaceutical Schedule.

Iloprost (Vebulis)

From 1 August 2023, applications for nebulised iloprost (Vebulis), will be made by the standard Special Authority application process.

Iloprost access criteria will be amended in Section B of the Pharmaceutical Schedule and replaced with the following:

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) greater than 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 greater than 2 Wood Units or greater than 160 International Units (dyn s cm-5); and
      5. Any of the following:
        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 (see note below for link to these guidelines)†; or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
        3. Patient has PAH other than idiopathic / heritable or drug-associated type; or
    2. Patient is a child with PAH secondary to congenital heart disease or PAH due to idiopathic, congenital or developmental lung disorders including severe chronic neonatal lung disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures or a major complication of the Fontan circulation requiring the minimising of pulmonary/venous filling 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 (i.e. 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 only 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 European Respiratory Journal Guidelines can be found here:  2022 ECS/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension(external link)

** 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 will apply in Part II of Section H of the Pharmaceutical Schedule.

Epoprostenol (Veletri)

From 1 August 2023, applications for epoprostenol (Veletri), will be made by the standard Special Authority approval process. Initial applications and renewals for this treatment will be limited to Respiratory specialists, cardiologists, rheumatologists, or any relevant practitioner on the recommendation of a respiratory specialist, cardiologist or rheumatologist.

Iloprost access criteria will be added to Section B of the Pharmaceutical Schedule and replaced with the following:

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) greater than 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 greater than 2 Wood Units or greater than 160 International Units (dyn s cm-5); and
      5. Any of the following:
        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 (see note below for link to these guidelines)†; or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
        3. Patient has PAH other than idiopathic / heritable or drug-associated type; or
    2. Patient is a child with PAH secondary to congenital heart disease or PAH due to idiopathic, congenital or developmental lung disorders including severe chronic neonatal lung disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures or a major complication of the Fontan circulation requiring the minimising of pulmonary/venous filling 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. 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 only 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 European Respiratory Journal Guidelines can be found here: 2022 ECS/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension PAH(external link)

** 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 will apply in Part II of Section H of the Pharmaceutical Schedule.

Sildenafil (Vedafil)

From 1 August 2023 sildenafil (Vedafil) access criteria for PAH criteria will replaced in Section B of the Pharmaceutical Schedule with the following:

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) greater than 2 Wood Units or greater than 160 International Units (dyn s cm-5); and
      5. Any of the following:
        1. PAH is non-responsive in vasoreactivity assessment using iloprost or nitric oxide, as defined in the 2022 ECS/ERS Guidelines for PAH (see note below for link to these guidelines)†; or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
        3. Patient has PAH other than idiopathic / heritable or drug-associated type; or
    2. Patient is a child with PAH secondary to congenital heart disease or PAH due to idiopathic, congenital or developmental lung disorders including severe chronic neonatal lung disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures or a major complication of the Fontan circulation requiring the minimising of pulmonary/venous filling pressures.

Note: Indications marked with * are Unapproved Indications.

† The European Respiratory Journal Guidelines can be found here: 2022 ECS/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension PAH(external link)

** 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.

Sildenafil special authority criteria for non-PAH indications (Raynaud’s Phenomenon / Erectile dysfunction due to spinal cord injury) remain unchanged.

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

Bosentan (Dr Reddy’s)

From 1 August 2023, bosentan (Dr Reddy’s) access criteria will be replaced in Section B of the Pharmaceutical Schedule with the following:

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) greater than 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 greater than 2 Wood Units or greater than 160 International Units (dyn s cm-5); and
      5. Any of the following:
        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 (see note below for link to these guidelines)†; or
        2. Patient has not experienced an acceptable response to calcium antagonist treatment, according to a validated risk stratification tool**; or
        3. Patient has PAH other than idiopathic / heritable or drug-associated type; or
    2. Patient is a child with PAH secondary to congenital heart disease or PAH due to idiopathic, congenital or developmental lung disorders including severe chronic neonatal lung disease; or
    3. Patient has palliated single ventricle congenital heart disease and elevated pulmonary pressures or a major complication of the Fontan circulation requiring the minimising of pulmonary/venous filling 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 only 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 European Respiratory Journal Guidelines can be found here: 2022 ECS/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension(external link)

** 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 will apply in Part II of Section H of the Pharmaceutical Schedule.

Our response to what you told us

We’re really grateful for the time people took to respond to 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 set out below. 

Theme

Pharmac Comment

Support for the proposal

General support for the proposal to move the assessment of PAH treatments from panel assessment to standard Special Authority process and reducing the burden of the requirement for serial Right Heart Catheter diagnostic studies

We are pleased to be making changes to the PAH funding process and criteria to make these treatments easier for people with PAH and their clinicians to access. We hope these changes will reduce issues of access equity and will reduce the administrative and resource burden on the health sector.

Amendments to eligibility criteria

Feedback questioning the necessity to perform vasoreactivity testing for patients with CTEPH as detailed in the proposed Special Authority criteria.

Based on the feedback and additional clinical advice from the PAH Panel, the Special Authority criteria has been revised so that there is no longer a requirement to perform vasoreactivity testing for patients with CTEPH. This now aligns with the recommendations contained in the 2022 ESC/ERS Guidelines(external link).

Concerns regarding the requirement for paediatric patients to undergo Right Heart Catheter study as part of diagnostic requirement, due to clinical risk.

Based on the feedback and additional clinical advice from the PAH Panel, the Special Authority criteria have been revised so that children no longer require cardiac catheterisation as part of the diagnostic workup for funding of PAH treatments.

A request that vasodilator testing at the time of right heart catheterisation (RHC) be restricted to patients with idiopathic, heritable and drug-related PAH (NYHA I to III) as per the 2022 ESC/ERS Guidelines(external link)

Based on the feedback and clinical advice from the PAH Panel, the Special Authority criteria have been revised to restrict the right heart catheterisation vasodilator requirement to only those patients with idiopathic, heritable, and drug-related PAH (NYHA I to III).

A respondent suggested that iloprost should no longer be used as a therapy in clinical practice except for those already on therapy and where needed in pregnancy.

The 2022 ESC/ERS guidelines(external link) suggest that the addition of inhaled prostacyclin analogues (e.g. iloprost) to PDE5i therapy is recommended to reduce the risk of morbidity/mortality events (Class I recommendation). Clinical advice received from the PAH Panel confirmed that inhaled iloprost remains an effective treatment option.

Request to limit the prescription of PAH-specific treatments to PAH specialist centres.

We appreciate that where possible people with PAH are assessed and treated in centres with PAH specialist teams. To facilitate equitable access to PAH treatment across the country we consider it is appropriate that the listed relevant specialists (cardiologists/rheumatologists/respiratory specialists) have the ability to apply for funded treatment and recommend treatment to any relevant practitioner to access funding where appropriate.

Concern that the term PAH usually refers only to patients with Group 1 disease, whereas in the proposal it is used instead of PH (point 2 in the special authority requirements). 

The term PAH has been used throughout the special authority criteria for consistency and simplicity. Clinical advice from the PAH Panel suggests that this is a reasonable approach.

Pharmacy and Related Concerns

 

Concerns with regards to pharmacy dispensing/wastage

No decision has been made to change the way PAH treatments are dispensed/accessed from pharmacies.

Concerns about provision of iloprost nebulisers for initiation of treatment in hospitals.

Nebuliser devices for Iloprost are a device and are outside of the scope of the Hospital Medicines List. Te Whatu Ora hospitals can buy the nebulisers and give them to patients in accordance with rule 8.2.6(external link). of the Pharmaceutical Schedule.

Requests for wider access

Concern that bosentan is positioned as a first line endothelin receptor antagonist (ERA) in the special authority criteria rather than ambrisentan, which is not supported by the 2022 ESC/ERS Guidelines.(external link)

The 2022 ESC/ERS Guidelines(external link) for the diagnosis and treatment of pulmonary hypertension have several recommendations with regard to different treatment strategies for patients with PAH. The clinical advice received from the PAH Panel was that ambrisentan is the clinically preferred first line ERA, and that bosentan is the currently funded first line treatment option in NZ for those people without contraindications. We are aware that there is a clinical desire to use ambrisentan first line and we plan to work with treating clinicians to obtain the information we need to assess this further.

Alternative PAH medicines

A respondent noted that the drugs Tadalafil, selexipag, macitentan and riociguat, are available internationally with some of these treatments having the benefit of once per day dosing, which may result in greater adherence.

The primary purpose of this decision is to change the funding process for access to PAH treatments from the PAH Panel assessment to the standard Special Authority application process.

We have assessed  funding applications for  both selexipag(external link) and macitentan(external link) and these have both been ranked on our Options for Investment list, which means these are treatments that we would like to fund subject to funding availability.

We have not received funding applications for either tadalafil or riociguat to date but would welcome interested parties to submit these applications via our Funding Application portal.

A respondent highlighted the need for Pharmac to engage in horizon scanning, including consideration of funding for inhaled treprostinol in PH-ILD and PAH, and sotatercept

Horizon scanning is part of our day-to-day work. We also regularly meet with pharmaceutical companies to discuss their future medicine development pipelines. Pharmac welcomes funding applications for either new medicines or expanded indications for existing funded medicines from interested parties.

Concerns noted about a lack of new funded treatments for PAH. The Pharmac review specifically recommended that for rare disorders a patient voice be considered in decision-making and that the disproportionate effect of the diagnosis of rare disorders on Māori and Pacific people be recognised.

We are aware that there is a desire in the clinical community to access selexipag and macitentan/tadalafil as these may have a positive impact on suitability. The impact to Māori and Pacific people, and suitability are all part of our decision-making framework, the Factors for Consideration. More details about our decision making framework is available here(external link).

We are working towards having more consumer involvement in our process and have already started by ensuring we have consumer representation on PTAC and some of our specialist advisory committees, including the most recent Rare Disorders Advisory Committee.

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.