Proposal to widen funded access to pulmonary arterial hypertension treatments

Medicines Consultation Closed

PHARMAC is seeking feedback on a proposal to make several changes to the funding of medicines for pulmonary arterial hypertension (PAH).

In summary, we propose to make the following changes from 1 February 2018:

  • New listing: IV epoprostenol would be listed in Section B (community) of the Pharmaceutical Schedule, and available via Special Authority upon approval by the PAH Panel.
  • Access widening: The PAH severity requirements in the Special Authority criteria would be widened to include all patients with NYHA/WHO Functional Class II.
  • Treatment selection: Dual therapy would be available to patients who had trialled monotherapy and had no response or deteriorated, instead of requiring two trials of different monotherapies. Triple therapy would be available to patients meeting certain severity criteria.
    • Funded access mechanism: Funding for sildenafil and bosentan would be through a standard Special Authority application (electronic or manual) process instead of via the PAH Panel. Funding for iloprost, ambrisentan and epoprostenol injection would remain available via Special Authority upon approval by the PAH Panel.

Feedback sought

PHARMAC welcomes feedback on this proposal. To provide feedback, please submit it in writing by 29 November 2017 to:

Alexander Rodgers
Therapeutic Group Manager
PHARMAC

Email:  alexander.rodgers@pharmac.govt.nz

Fax:     04 460 4995
Post:   PO Box 10 254, Wellington 6143

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.

Background

Pulmonary arterial hypertension (PAH) is a rare, 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.

There are currently four treatments for pulmonary arterial hypertension listed in Section B of the Pharmaceutical Schedule, subject to Special Authority criteria: sildenafil, bosentan, iloprost, and ambrisentan. The first three were funded from 2009, while ambrisentan was funded from 2010. Funding is available for these four products via application to a special access panel (the PAH Panel). Epoprostenol injection is listed in Part II of Section H of the Pharmaceutical Schedule for patients with PAH who are on the active waiting list for lung transplantation, who have been approved by the PAH Panel.

Around 200 people are currently accessing funded treatments for PAH in New Zealand.

Diagnosing PAH is complex, and PAH treatments were all very high cost when first funded, so the PAH Panel was established to assess Special Authority applications for funding. The PAH Panel is made up of clinicians who are tasked with examining applications to ensure the patient’s clinical circumstances meet the Special Authority criteria set out in the application form. Since then, generic forms of sildenafil and bosentan have become available, which has reduced the cost of those treatments.

PHARMAC has received applications from clinicians proposing widenings of access to the five PAH treatments listed in Section B and Section H. These applications were reviewed by the Pharmacology and Therapeutics Advisory Committee (PTAC) and by PHARMAC. More information, including links to advisory committee minutes and previous consultation and notification letters, can be found in the Application Tracker records for PAH treatments at:

www.pharmac.govt.nz/patients/ApplicationTracker?ProposalId=1406 [link no longer available]

www.pharmac.govt.nz/patients/ApplicationTracker?ProposalId=1407 [link no longer available]

Details of the proposal

New listing – IV epoprostenol

Intravenous epoprostenol (Veletri) would be listed in Section B of the Pharmaceutical Schedule from 1 February 2018 at the following price and subsidy (ex-manufacturer, excluding GST):

Chemical Presentation Brand Pack size Proposed subsidy and price
Epoprostenol Inj 500 mcg vial Veletri 1 $36.61
Epoprostenol Inj 1.5 mg vial Veletri 1 $73.21

Access to funding for epoprostenol would be through the PAH Panel. In addition to general limitations on the number of agents a patient can receive at any time, epoprostenol would be restricted to:

Any of the following:

  1. Patient is presenting acutely with idiopathic pulmonary arterial hypertension (IPAH) in New York Heart Association/World Health Organization (NYHA/WHO) Functional Class IV; or
  2. Patient is deteriorating rapidly to NYHA/WHO Functional Class IV who may be lung transplant recipients in the future, if their disease is stabilised; or
  3. Patient is with PAH associated with the scleroderma spectrum of diseases (APAHSSD) who have no major morbidities and are deteriorating despite combination therapy, or
  4. For use as a bridge to transplant for patients with pulmonary arterial hypertension who are on the active waiting list for lung transplantation.

Intravenous epoprostenol is already listed in Part II of Section H. Its restrictions would be widened to match those proposed for Section B.

Access widening - PAH severity requirements

Severity of PAH is measured using the New York Heart Association Functional Classification (NYHA/WHO FC), which ranks severity of PAH into one of four categories, FC I being the lowest severity and FC IV being the highest. Further information on NYHA/WHO classification is available at http://www.pah-info.com/Assessing-the-severity-of-PAH. Currently, the initiation criteria for any funded PAH treatment limits funding to patients with NYHA/WHO FC IV, FC III or patients in FC II where there is clear evidence of disease progression despite optimal therapy.

As recommended by PTAC, we propose widening the initiation criteria to enable patients with NYHA/WHO Functional Class II or higher, regardless of disease progression, to access funded treatment (subject to the other criteria). We received advice is that this would allow patients to start treatment earlier, improving quality of life and delaying disease progression.

Treatment selection

Dual therapy

We propose that patients would be only required to try one PAH treatment as monotherapy before moving to dual therapy. This would change the current restriction that requires trying two monotherapies. Patients would be expected to initially try sildenafil as monotherapy, except under certain circumstances described in the proposed Special Authorities.

Triple therapy

We propose that patients on the active lung transplant list could apply to the Panel for funded access to triple therapy. Triple therapy would be required to include sildenafil as one component, unless a patient cannot tolerate it.

We also propose that patients who meet the criteria for epoprostenol could apply to the Panel for triple therapy, on the condition that sildenafil and bosentan are two of the three treatments used.

Funded access mechanism – sildenafil and bosentan

We propose that, instead of requiring applications to the PAH Panel, sildenafil and bosentan be listed in Section B subject to the following Special Authority criteria:

Sildenafil

SAQQQQ Special Authority for Subsidy

Initial application — (pulmonary arterial hypertension*) only from a relevant specialist or medical practitioner on the recommendation of a relevant specialist. 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. Any of the following:
    1. PAH is in Group 1 of the WHO (Venice) clinical classifications; or
    2. PAH is in Group 4 of the WHO (Venice) clinical classifications, or
    3. PAH is in Group 5 of the WHO (Venice) clinical classifications; and
  3. Any of the following:
    1. PAH is in NYHA/WHO functional class II; or
    2. PAH is in NYHA/WHO functional class III; or
    3. PAH is in NYHA/WHO functional class IV; and
  4. Patient has a pulmonary capillary wedge pressure (PCWP) less than or equal to 18 mmHg; and
  5. Either:
    1. Patient has a mean pulmonary artery pressure (PAPm) > 25 mmHg; or
    2. Patient is peri Fontan repair; and
  6. The patient must have a pulmonary vascular resistance (PVR) of > 3 Wood Units or > 240 International Units (dyn s cm-5).

* indicates an Unapproved Indication

Note: Definitions of WHO (Venice) clinical classifications and NYHA/WHO functional classes can be found at www.pharmac.govt.nz/xxxxx

Note that the current Special Authority criteria for sildenafil for Raynaud’s Phenomenon would be unchanged by this proposal and would be incorporated into this new Special Authority.

Bosentan

SAQQQQ Special Authority for Subsidy

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

All of the following:

  1. Patient has pulmonary arterial hypertension (PAH); and
  2. Any of the following:
    1. PAH is in Group 1 of the WHO (Venice) clinical classifications; or
    2. PAH is in Group 4 of the WHO (Venice) clinical classifications, or
    3. PAH is in Group 5 of the WHO (Venice) clinical classifications; and
  3. Any of the following:
    1. PAH is at NYHA/WHO functional class II; or
    2. PAH is at NYHA/WHO functional class III; or
    3. PAH is at NYHA/WHO functional class IV; and
  4. Any one of the following:
    1. Both:
      1. Bosentan is to be used as PAH monotherapy; and
      2. Either:
        1. Patient is intolerant or contraindicated to sildenafil; or
        2. Patient is a child with idiopathic PAH or PAH secondary to congenital heart disease; or
    2. Both:
      1. Bosentan is to be used as PAH dual therapy; and
      2. Either:  
        1. Patient has tried a PAH monotherapy for at least three months and failed to respond, or
        2. Patient deteriorated while on a PAH monotherapy; or
    3. Both:
      1. Bosentan 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 acutely with idiopathic pulmonary arterial hypertension (IPAH) in New York Heart Association/World Health Organization (NYHA/WHO) Functional Class IV; or
        3. Patients deteriorating rapidly to NYHA/WHO Functional Class IV who may be lung transplant recipients in the future, if their disease is stabilised; or
        4. Patients with PAH associated with the scleroderma spectrum of diseases (APAHSSD) who have no major morbidities and are deteriorating despite combination therapy, or
        5. For use as a bridge to transplant for patients with pulmonary arterial hypertension who are on the active waiting list for lung transplantation.

Note: Definitions of WHO (Venice) clinical classifications and NYHA/WHO functional classes can be found at www.pharmac.govt.nz/xxxxx

Renewal only from a relevant specialist or medical practitioner on the recommendation of a relevant specialist. Approvals valid for 2 years for applications meeting the following criteria:

  1. Any one of the following:
    1. Both:
      1. Bosentan is to be used as PAH monotherapy; and
      2. Patient is stable or has improved while on bosentan; or
    2. Both:
      1. Bosentan is to be used as PAH dual therapy; and
      2. Patient has tried a PAH monotherapy for at least three months and either failed to respond or later deteriorated; or
    3. Both:
      1. Bosentan is to be used as PAH triple therapy; and
      2. Any of the following:
        1. Patient is on the lung transplant list; or
        2. Patients presenting acutely with idiopathic pulmonary arterial hypertension (IPAH) in New York Heart Association/World Health Organization (NYHA/WHO) Functional Class IV; or
        3. Patients deteriorating rapidly to NYHA/WHO Functional Class IV who may be lung transplant recipients in the future, if their disease is stabilised; or
        4. Patients with PAH associated with the scleroderma spectrum of diseases (APAHSSD) who have no major morbidities and are deteriorating despite combination therapy, or
        5. For use as a bridge to transplant for patients with pulmonary arterial hypertension who are on the active waiting list for lung transplantation.

The proposed Special Authorities above are intended to be cover all existing rules around funding for sildenafil and bosentan, as well as the changes proposed in this consultation.

Ambrisentan, iloprost, and epoprostenol

Access to funded ambrisentan and iloprost would still require application to the PAH Panel. Funding for epoprostenol would also be done through the Panel.

The PAH Panel’s guiding document would be changed to account for the proposed changes as well as some changes to clarify wording.

Proposed changes to the PAH panel's guiding document [PDF, 143 KB]