Real-World Data & RWE

Data relating to patient health and/or delivery of care routinely collected from sources such as electronic health records, claims, registries, or wearables (FDA RWE Framework).

Clinical evidence about use and benefits/risks of a medicine derived from analysis of RWD (FDA). Used to support regulatory decisions including post-market safety.

Use of real-world data (e.g., claims, EHR, registries) to evaluate or validate safety signals, estimate rates, or assess confounders.

Assessment of whether a given RWD source is adequate for the intended PV or safety question (completeness, accuracy, relevance, timeliness).

Prospective, organized collection of data on pregnancy outcomes following exposure to a drug; used for risk characterization and RMP (GVP Module V, XVI).

Systematic follow-up of reported pregnancies in which the suspect drug was used, to collect outcome data (live birth, miscarriage, congenital anomaly, etc.).

Analysis of healthcare claims data (e.g., insurance) to assess drug use, outcomes, and safety in real-world populations.

Study carried out after approval to collect safety (or efficacy) data; may be imposed by authority or voluntary.

Study to assess efficacy in real-world use when condition of approval; distinct from PASS but may include safety.

Classification of drug use in pregnancy (e.g., FDA categories, SmPC). Relevant for pregnancy reporting and PPP.

Predefined protocol for RWD study (objectives, data source, analysis plan) to ensure scientific rigor.

Digital record of patient health information; source of RWD for safety studies.

Bias in RWD where another factor is associated with both exposure and outcome; must be addressed in analysis.

FDA's national system for monitoring medical product safety using electronic health data.

EU register of post-authorization studies (PASS, PAES); transparency and protocol registration.

Study of drug use and effects in populations; methods used in signal evaluation and RWE.

Study comparing cases (with outcome) to controls (without) for exposure; used in signal evaluation.

Study following exposed (and sometimes unexposed) cohort for outcomes.

Non-interventional study (e.g., cohort, case-control) using RWD; protocol and ethics required.

Bias where drug is prescribed to different patient types; confounds RWD analyses.

Registry of patients on a drug or with a condition; used for safety and effectiveness.