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

Page last updated: 26 July 2017

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

Consultation on Draft Terms of Reference (closed)

Public consultation on the draft Review Terms of Reference was open between 02 May and 16 May 2016.  To view submissions please go to the Public Consultation page.

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

Review Process

This Review will follow the Post-market Review Framework available on the Reviews website. This process includes public consultation on the draft Terms of Reference (completed), an opportunity to provide submissions to the Review (complete), and an opportunity to comment on the draft Review Report. The draft Review Report is expected to be released for consultation in early 2018.

Further Information

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

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