Methods Applied to Assess Real-World Effectiveness of Drugs: A Scoping Review

Area of Science:

• Pharmacoepidemiology
• Real-world data analysis
• Drug effectiveness research

Background:

• Bridging the gap between clinical trials and practice requires robust drug effectiveness evaluations.
• Current methods for real-world effectiveness studies are not well-defined.
• Real-world data (RWD) offers valuable insights into drug performance in routine clinical settings.

Purpose of the Study:

• To identify and categorize pharmacoepidemiological studies utilizing RWD.
• To analyze the drugs, outcomes, and statistical methods employed in these studies.
• To highlight common limitations in current RWD-based effectiveness research.

Main Methods:

• Systematic literature search of PubMed and Embase (July-December 2019).
• Inclusion criteria: RWD-based, evaluated drug exposures, and included effectiveness outcomes.
• Categorization based on methods, drugs, outcomes, and patient population size.

Main Results:

• 87 studies met the inclusion criteria from 4820 identified records.
• Chemotherapy was the most studied drug class, and all-cause mortality the most frequent outcome.
• Survival models were dominant statistical methods; 13 studies used only descriptive statistics.

Conclusions:

• Established pharmacoepidemiological methods were widely applied in RWD effectiveness studies.
• Many studies featured large patient cohorts, enhancing generalizability.
• Limitations included reliance on descriptive statistics and infrequent use of active comparators.