Context

Context

Canada's Drug Agency undertook an Environmental Scan to understand current practices for detecting and diagnosing sepsis in remote and rural health care settings across Canada.

The key objectives of this Environmental Scan were:

The number of drug for rare diseases (DRD) in the pipeline and the number of marketed DRDs are expanding. As such, policy-makers and clinicians have struggled on how to make DRDs available to patients while ensuring that only clinical- and cost-effective treatments are reimbursed.

Background

Context

Health Technology Assessment (HTA) informs health care decision-makers about the value — such as the safety, efficacy, and cost-effectiveness — of new and existing health technologies. HTA can inform clinical decision-making, as well as formulary listing and reimbursement decisions within health care systems. Given the utility of HTA to support evidence-based decision-making, it has been widely adopted and customized across different countries and regions.

Key Messages

• For rheumatoid arthritis patients with moderate to severe disease in whom treatment with methotrexate has failed or who are intolerant to methotrexate, conventional synthetic disease-modifying antirheumatic drugs (alone or in combination), biologics (including biosimilars), and targeted synthetic disease-modifying antirheumatic drugs appear to be effective for different outcomes.
• It is unclear how the efficacy and safety of the treatments compare with one another.

For an update on the status of the Canada's Drug Agency Therapeutic Review of Third-Line Drugs for Type 2 Diabetes, please visit our news section.

Background

Drugs for rare diseases (DRDs), also referred to as orphan drugs in some jurisdictions, are typically small-molecule drugs or biopharmaceuticals (referred to collectively herein as “drugs”) used to treat rare diseases. Due to a shift in the focus of the biopharmaceutical industry’s research and development priority from blockbuster to niche drugs, the DRD pipeline and the number of marketed DRDs are expanding.

Although Embase is a recommended key database to be searched when undertaking health technology assessments (HTAs), it has several features that hinder efficient searching. One feature is the large number of Emtree index terms that are added to most Embase records; an average of 3 to 4 major terms and up to 50 minor terms. In comparison, MEDLINE records may contain an average of 10 to 20 index terms (both major and minor).