3. Clinical effectiveness assessment in HTA

At the heart of HTA lies the evaluation of the effectiveness of a new medicine for patients in real-world clinical settings.

Evidence from multinational randomised controlled trials (RCTs) is typically used as a starting point. However, this evidence must be interpreted in the local context and supplemented with additional information. It cannot be assumed that the available data fully meets the needs of an ideal HTA.

HTA bodies and decision-makers often rely on a combination of sources to build a complete picture. These may include:

• 📚 Scientific publications
• 🏛️ Regulatory assessment reports for marketing authorisation of medicines
• 🏢 Data provided by the company developing or marketing the medicine

While some HTA bodies conduct or commission their own research, most rely on existing evidence generated by others

Suggested steps

Clinical effectiveness assessments go beyond evaluating efficacy and safety (benefit-risk) as done by regulatory authorities. They compare the effect of a new technology on the health of patients in standard care settings against current treatment options, aiming to reflect what is likely to happen in real-world healthcare systems.

High-quality clinical effectiveness assessments require input from various expert disciplines. The methodology used is largely drawn from epidemiology and medicine.

Four key steps are often followed in clinical effectiveness assessment: