Post-market Review of Pulmonary Arterial Hypertension (PAH) Medicines

Page last updated: 25 May 2020

Disclaimer

Every effort has been made to ensure web accessibility to the Post-market Review of Pulmonary Arterial Hypertension medicines, however complete web accessibility cannot be guaranteed.  If you have trouble accessing information within the report and you would like a copy to be provided to you, please direct all queries via email to PBS postmarket.

Background

Pulmonary Arterial Hypertension (PAH) is a type of high blood pressure that arises when the arteries on the right side of the heart that supply blood to the lungs (pulmonary arteries) thicken and become rigid.  When the pulmonary arteries thicken, the heart needs to work harder to provide adequate blood flow to the lungs, meaning that the body may not get all of the oxygen it needs to function well. Patients with PAH can experience shortness of breath, dizziness, chest pain and fatigue.

In February 2015, the Drug Utilisation Sub-Committee (DUSC) of the Pharmaceutical Benefits Advisory Committee (PBAC) reviewed the Pharmaceutical Benefits Scheme (PBS) utilisation of PAH medicines.  The DUSC considered that the PBS restrictions for PAH medicines are not consistent with current treatment guidelines in that they:

  • Require failure to respond to six or more weeks of appropriate vasodilator treatment for WHO class III patients with a mean right atrial pressure of 8 mmHg or less.
  • Do not allow treatment of functional class II patients.
  • Do not allow combination therapy.

In July 2015, the PBAC considered a submission from the sponsor of bosentan, epoprostenol and macitentan to remove the requirement for patients to be assessed as having achieved a response to treatment for continuing access to these medicines. The submission claimed that the PBS restriction requiring ongoing evidence of improvement or stability in disease status was inconsistent with best clinical practice and international guidelines. The submission further argued that in the case of an inadequate response to initial therapy, sequential combination therapy, rather than cessation and switching therapies, was recommended in the guidelines. Noting the submission’s claim that the current PBS continuing restriction for PAH medicines is inconsistent with best clinical practice, the PBAC considered that there were a number of issues around PAH medicines that could not be assessed within the scope of the submission.

The PBAC recommended a post-market review of the efficacy and cost‑effectiveness of PAH medicines, including the existing listing for class III and class IV patients, and the additional clinical place of these therapies as recommended in international guidelines.

Further information is available in the PBAC Public Summary Documents for July 2015 for these medicines.

The PBAC recommended a Post-market Review of the efficacy and cost-effectiveness of PAH medicines in December 2015.  The purpose of the Post-market Review of PAH Medicines is to review the safety, efficacy and cost-effectiveness of these medicines, in the context of Quality Use of Medicines and patient access to optimal treatment.  The Review was approved by the Minister for Health on 7 April 2016.

The Review is being carried out under the Australian Government’s post-market monitoring program, which aims to ensure the continued safe, cost-effective and quality use of medicines listed on the PBS.

Medicines to be included in the PAH Review are:

  • Macitentan
  • Ambrisentan
  • Bosentan
  • Iloprost
  • Epoprostenol
  • Sildenafil
  • Tadalafil
  • Riociguat

Reference Group Membership

Name

Nominating Body

Type of Membership

Area of Expertise

Professor Terence Campbell

PBAC

Chair

Pharmaceutical benefits policy, cardiology

Professor Anna Kemp-Casey

DUSC

Individual - Technical Expert

Drug utilisation

Professor Susanna Proudman

Individual - Technical Expert

Pulmonary disease

Professor Trevor Williams

Individual - Technical Expert

Pulmonary disease

Ms Michelle Tew

Australian Health Economics Society

Technical Expert

Health economics

Dr Eugene Kotlyar

Cardiac Society of Australia and New Zealand

Organisational representative

Cardiac disease

Professor Garry Jennings

Heart Foundation

Organisational representative

Cardiology

Ms Elizabeth De Somer

Dr Vicki Gardiner

Medicines Australia

Organisational representative

Pharmaceutical Industry

Professor Geoff Strange

Pulmonary Hypertension Society of Australia and New Zealand

Organisational representative

Pulmonary disease

Dr Charlotte Hespe

Royal Australian College of General Practitioners

Organisational representative

General practice

Mr Garth Birdsey

Society of Hospital Pharmacists

Organisational representative

Hospital pharmacy

Dr Jane Cook

Dr Grant Pegg

Therapeutic Goods Administration

Organisational representative

Regulation and safety of medicines

Dr Jeremy Wrobel

Thoracic Society of Australia and New Zealand

Organisational representative

Pulmonary disease

Dr Christine Walker

Consumers’ Health Forum

Organisational representative

Health consumer advocacy

Final Terms of Reference

The PBAC considered the draft Review Terms of Reference and comments from stakeholders at the August 2016 PBAC special meeting.  Following the August 2016 PBAC special meeting the Minister for Health approved the final Terms of Reference for the Review:

  1. Review recent clinical guidelines for the management of PAH and compare this to the PBS restrictions and Therapeutic Goods Administration (TGA) indications for the use of PAH medicines.
  2. Review the utilisation of PAH medicines in Australia, including sources of data that can provide additional information on clinical use that is not available from PBS data.
  3. Review the clinical outcomes that are most important or clinically relevant to patients with PAH, and the extent to which these outcomes are included in the evidence previously considered by PBAC.
  4. Collate and evaluate evidence on the comparative effectiveness of PAH medicines, including combination use and use in the WHO functional class II patient populations.
  5. Following ToR 1-4 consider reviewing the cost-effectiveness of existing PBS listings for PAH medicines, and in treatment of WHO functional class II and combination treatment in class III and class IV patients.

Public Submissions addressing the final Terms of Reference (closed)

The public submission process for the Pulmonary Arterial Hypertension (PAH) Medicines Review was open between 13 February 2017 and 27 March 2017. To view submissions please go to the Public Consultation page

Consumer Session

A consumer session was held in Sydney on 14 October 2017 with members of the Pulmonary Hypertension Association Australia (PHAA).  To view the Consumer Outcome Statement please go to the Public Consultation page.

Public Consultation on the Draft Review Report (closed)

Public Consultation on the draft Review Report was open between 21 May 2018 and 10 June 2018.

PBAC Consideration of the Final Report

The PBAC considered the Final Report in November 2018, March 2019 and November 2019, including PBAC sub-committee advice and stakeholder comments. The PBAC Minutes and Final Report for the PAH Review are now available. ‘Commercial-in-confidence’ information in the Final Report and the PBAC Minutes has been redacted.

PBAC Minutes for the Post-market Review of PAH Medicines

Post-market Review of PAH Medicines – Final Report to the PBAC

The Final Report is provided in ten parts in DOCX and PDF format:

  1. Executive Summary (PDF  1797 KB) - (Word 290 KB)
  2. Background and Term of Reference 1 (PDF  3193 KB) - (Word 2021 KB)
  3. Term of Reference 2 (PDF 1473 KB) - (Word 392 KB)
  4. Appendix 2A (PDF 1473 KB) - (Word 1466 KB)
  5. Appendix 2B (PDF 605 KB) - (Word 392 KB)
  6. Appendix 2C (PDF 1225 KB) - (Word 213 KB)
  7. Term of Reference 3 (PDF 1025 KB) - (Word  61 KB)
  8. Appendix 3A (PDF 475 KB) - (Word 45 KB)
  9. Term of Reference 4 (PDF 4731 KB) - (Word 1315 KB)
  10. Term of Reference 5 (PDF 864 KB) - (Word 67 KB)

Review Process

This Review followed the Post-market Review Framework available on the Reviews website. This process included public consultation on the draft Terms of Reference, an opportunity to provide submissions to the Review, and an opportunity to comment on the draft Review Report.

Further Information

Further information on post-market reviews is available on the Reviews website.

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