Importance Although randomized clinical trials are considered to be the criterion standard
for generating clinical evidence, the use of real-world evidence to evaluate the efficacy and …

Physicians and insurers need to weigh the effectiveness of new drugs against existing
therapeutics in routine care to make decisions about treatment and formularies. Because …

High development cost, low development success, cost‐disciplined health‐care policies,
and intense competition demand an efficient drug development process. New compounds …

Real‐world data/evidence (RWD/RWE) may provide insightful information on medicines'
clinical effects to guide regulatory decisions. While its contribution has been recognized for …

Objectives To document reporting of study characteristics of randomized controlled trials
(RCTs) in the four leading general medical journals and to appraise the generalizability of …

A central question in the assessment of benefit/harm of new treatments is: how does the
average outcome on the new treatment (the factual) compare to the average outcome had …

Comparative‐effectiveness research (CER) aims to produce actionable evidence regarding
the effectiveness and safety of medical products and interventions as they are used outside …

Background Classically, randomised controlled trials (RCTs) are considered the gold
standard for demonstrating product efficacy for the regulatory approval of medicines …

Background: In this era of evidence-based medicine, clinicians require a comprehensive
range of well-designed studies to support prescribing decisions and patient management. In …

Objectives: Health insurers, physicians, and patients worldwide need information on the
comparative effectiveness and safety of prescription drugs in routine care. Nonrandomized …