Canada’s Drug Agency publishes first-ever methods guide for health technology assessment

Canada’s Drug Agency (CDA-AMC) has released a ‘Methods Guide for Health Technology Assessment’ (HTA), offering a detailed, transparent framework for evaluating clinical evidence submitted for drug reimbursement. The guide outlines key appraisal methods and evidence expectations, supporting consistent decision-making across Canada’s health system.

The comprehensive document, the first of its kind from the agency, outlines the methods used to evaluate clinical evidence submitted by sponsors through the Drug Reimbursement Review program. The guide was developed to enhance transparency and improve understanding of how CDA-AMC conducts assessments to inform drug reimbursement decisions.

The guide focuses on how clinical evidence is assessed to determine the comparative effectiveness and potential harms of drug products relative to treatments used in Canadian clinical practice. “This guidance is intended for use by those who generate and submit evidence, and those who conduct the evidence appraisal,” CDA-AMC explains, noting that it will be updated periodically as scientific methods evolve.

The document was shaped by extensive input from stakeholders, including patient groups, health professionals, HTA organizations, regulators, and industry representatives. CDA-AMC conducted two rounds of consultation, receiving “overwhelmingly positive” feedback that acknowledged the importance of the initiative.

Based on national and international best practices, the guide outlines key methodologies used to evaluate different types of clinical evidence. It defines expectations of what the evidence sponsors must provide, including randomized controlled trials, systematic reviews, real-world evidence (RWE), and indirect treatment comparisons, depending on the drug and disease context.

“The main HTA question of interest is as follows: What are the effectiveness and harms of the drug product under review relative to relevant comparators used in clinical practice in Canada?” the guide notes.

In line with this, the guide emphasizes transparency in study design and outcome reporting, and encourages use of established frameworks such as PICO(T)(S) and the Grading of Recommendations Assessment, Development and Evaluation (GRADE).

The guide also addresses the appraisal of surrogate outcomes, real-world data (RWD), and long-term extension studies, while stressing the importance of methodological rigor, particularly when nonrandomized evidence is used. It includes standards for critical appraisal, risk of bias assessment, and the use of appropriate statistical methods to interpret effect estimates.

CDA-AMC highlights that while clinical evidence is a key input into expert committee deliberations, other dimensions, such as patient experiences, ethical considerations, and system-level impacts, also contribute to reimbursement recommendations. As the agency states, HTA is a “multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its life cycle.”