Comment on Framework for FDA's Real-World Evidence Program - part 2 (ENG)

There’s a current trend toward using Real World Evidence (RWE) for effectiveness studies. However, utilizing RWE requires comprehensive evaluation of whether the research design is suitable for real-world data (RWD), if the study is scientifically sound, and if the data is reliable. RWD must also be selected according to specific regulatory requirements.

RWD has clear limitations. Being not primarily designed for clinical trials, it may not fully answer researchers’ questions. For example, understanding how ICD-10 codes and EHR variables are applied in actual clinical settings is important. Integration can be challenging due to differences between EHR systems.

RWD is used to create external control arms for measuring comparative effectiveness. While propensity score matching improves comparability, potential bias exists due to different timeframes and populations. Like clinical trials, pre-sharing research design such as clinical protocol and analysis plans is important for credibility. While clinical trials routinely pre-register protocols on clinicaltrials.gov, observational studies have historically done this less frequently. Recent trends encourage pre-registration of RWE studies on platforms like ClinicalTrials.gov, EU-RWD catalogue, and ISPOR-ISPE.

RWE study protocol registers ClinicalTrial.gov: https://clinicaltrials.gov/

EU-RWD catalogue : https://catalogues.ema.europa.eu/search?f%5B0%5D=content_type%3Adarwin_study

ISPOR-ISPE : https://osf.io/registries/rwe/discover

Regarding RWE’s future, the FDA framework discusses stakeholder engagement, including internal FDA discussions and external input from patient advocacy groups, pharmaceutical companies, and academia. The direction of RWE utilization will likely depend on stakeholders’ perspectives. I think RWE usage will increase because of necessity.

Article written by Presenter, InYoung Jun

Source: Original Korean article | English translation by Claude 3.5 Sonnet (New)