CADTH will undertake a systematic review to compare the efficacy and safety of new and existing drug therapies for pulmonary arterial hypertension (PAH), and will examine their cost-effectiveness. The review will include drug therapies that are available in Canada (epoprostenol, treprostinil, bosentan, ambrisentan, macitentan, sildenafil, tadalafil) and one for which the clinical development program is advanced but is not yet approved for the treatment of PAH in Canada (riociguat).

The clinical and cost-effectiveness evidence will be reviewed by the Canadian Drug Expert Committee (CDEC) for the purpose of making recommendations. Recommendations and advice delivered by CDEC are provided to CADTH participating jurisdictional drug programs to inform their policy decisions.

Contents

• ABBREVIATIONS
• 1. INTRODUCTION AND RATIONALE
• 2. DELIVERABLES
• 3. POLICY QUESTIONS
• 4. RESEARCH QUESTIONS
• 5. METHODS
  • 5.1. Literature Search Strategy
  • 5.2. Selection Criteria
  • 5.3. Data Extraction and Critical Appraisal of Clinical Studies
  • 5.4. Data Analysis and Synthesis
  • 5.5. Data Availability
• REFERENCES

This report is prepared by the Canadian Agency for Drugs and Technologies in Health (CADTH). This report contains a comprehensive review of existing public literature, studies, materials, and other information and documentation (collectively the “source documentation”) available to CADTH at the time it was prepared, and it was guided by expert input and advice throughout its preparation.

The information in this report is intended to help health care decision-makers, patients, health care professionals, health systems leaders, and policy-makers make well-informed decisions and thereby improve the quality of health care services. The information in this report should not be used as a substitute for the application of clinical judgment in respect to the care of a particular patient or other professional judgment in any decision-making process, nor is it intended to replace professional medical advice. While CADTH has taken care in the preparation of this report to ensure that its contents are accurate, complete, and up-to-date, CADTH does not make any guarantee to that effect. CADTH is not responsible for any errors or omissions or injury, loss, or damage arising from or as a result of the use (or misuse) of any information contained in or implied by the information in this report. CADTH takes sole responsibility for the final form and content of this report. The statements, conclusions, and views expressed herein do not necessarily represent the view of Health Canada or any provincial or territorial government.

Production of this report is made possible through a financial contribution from Health Canada.