The Role of Noncomparative Evidence in Health Technology Assessment Decisions

Area of Science:

• Health Technology Assessment
• Clinical Evidence Evaluation
• Regulatory Science

Background:

• Health technology assessment (HTA) agencies often prefer randomized controlled trials (RCTs) for evaluating new health technologies.
• However, conducting comparative trials is not always feasible or ethical.

Purpose of the Study:

• To assess the role and acceptance of noncomparative evidence in HTA decision-making processes.
• To analyze the frequency and impact of noncomparative evidence in HTA reports from major agencies.

Main Methods:

• A systematic search of HTA reports from NICE (UK), CADTH (Canada), and IQWiG (Germany) published between 2010 and 2015.
• Double-extraction of product, indication, outcome, and evidence type (comparative/noncomparative).
• Definition of noncomparative studies included those not presenting results against another treatment, including dose-ranging studies.

Main Results:

• Noncomparative evidence was considered in 12-38% of submissions across the three agencies.
• Exclusive reliance on noncomparative evidence occurred in 4-6% of appraisals.
• Positive outcome rates for noncomparative-only submissions varied, being similar to overall rates for CADTH but lower for NICE and IQWiG.

Conclusions:

• Noncomparative studies are acceptable clinical evidence for HTA agencies under specific conditions.
• Justifiable study designs and convincingly demonstrated treatment effects are key for acceptance.
• The current utilization of noncomparative evidence in HTA remains limited.