Use of Real-World Data in Health Technology Assessment? Read the comparative Study on policies of Six HTA Agencies.

In light of rising health care costs and the introduction of innovative, yet expensive, pharmaceutical products, health tech- nology assessment (HTA) agencies are seeking robust methods for relative effectiveness assessments (REAs) of drugs in routine clinical practice. The relative effectiveness of an intervention is defined as “[t]he extent to which an intervention does more good than harm, when compared to one or more intervention alter- natives for achieving the desired results and when provided under the routine setting of health care practice (i.e. real-world setting)” [1].

Conventionally, data on treatment effects for drugs are collected in the context of randomized controlled trials (RCTs), whereby a selected, homogeneous group of patients is ran- domly assigned to either the experimental drug or a comparator (e.g., placebo or active comparator) under highly controlled conditions. This study design is ideal to demonstrate the efficacy of a drug, because of its ability to minimize problems with confounding, information bias, and selection bias.