Designing a robust Real-World Evidence (RWE) study is crucial for generating reliable and valid insights that are acceptable to regulators. Here are some scientific best practices to consider according to regional drug regulatory authorities:
 
EUROPEAN UNION – According to the European Medicines Agency (EMA), relevant scientific guidance should be considered by marketing authorisation holders and investigators for the development of study protocols, the conduct of studies and the writing of study reports…These scientific guidelines include the:
1. ENCePP Guide on Methodological Standards in Pharmacoepidemiology
2. ENCePP Checklist for Study Protocols, and
3. Guidelines for Good Pharmacoepidemiology Practices of the International Society of Pharmacoepidemiology (ISPE GPP)
Ref: Section VIII.B.1. of GVP Module VIII
 
GERMANY – The observation plan is to be drawn up according to recognized recommendations of scientific or regulatory guidelines…for example:
1.  Guidelines for Good Pharmacoepidemiology Practices of the International Society for Pharmacoepidemiology (ISPE GPP),
2. Recommendations of the ” ENCePP Guide on Methodological Standards in Pharmacoepidemiology”, and
3. Guidelines for Good Epidemiological Practice (GEP) of the German Society for
Epidemiology (DGEpi)
Ref:  Section 2.7.1 of the BfArM/PEI Recommendations – December 2022
 
CANADA – All research protocols should be well-designed and include a comprehensive assessment of good research principles. There are fifteen key elements that should be considered for each protocol, and are reflective of the:
1. European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) Protocol Checklist, and
2. The Guidelines for Good Pharmacoepidemiology Practices (GPP).
Both prospective and retrospective designs should attempt to address each element, or provide justification why it may not be applicable to the specific study.
Ref: Health Canada – Elements of Real World Data/Evidence Quality throughout the Prescription Drug Product Life Cycle, March 2019
 
USA – According to the FDA’s “Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data” of May 2013, the following provide general guidance applicable to all pharmacoepidemiologic safety studies:
1. ISPE guidelines (ISPE GPP),
2. STROBE reporting framework, and
3. The ENCePP methods checklist
Ref: FDA Guidance – Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data
 
These scientific best practices will help ensure that the RWE study is robust, credible, and provides meaningful information to patients, healthcare providers, and policy makers.