FDA has raised the bar on real-world evidence (RWE) studies with its new draft guidance. FDA’s expectations that RWE studies be good quality, rigorous, and conducted under a protocol and with IRB approval are not new. But the depth of data assessment, documentation, and requirements for submission outlined in this guidance have been augmented beyond what may be reasonable. A greater desire by regulators for clinical data to support medical device safety and effectiveness is here to stay, not just at FDA, but globally. While the motivations are genuinely for the benefit of public health, the reality is that clinical and observational research is expensive, complicated, time consuming, and burdensome. These costs will ultimately be passed down to payers and patients. I am not sure the incremental confidence FDA has in its regulatory decisions is worth the cost.
Read MoreThe desire for RWD/RWE by global regulators to demonstrate medical device safety and efficacy is growing. We exist in an era of unprecedented technological capabilities for data linking, tokenization to protect privacy, and big data analytics. However, many challenges remain with the use of these data for medicines and device evaluation. Data quality, common data models, alignment on statistical methodologies, data linkage technology, data access and privacy, stakeholder agreements for data sharing, patient input and consent, and ethical concerns persist. Regulators must collaborate such that industry can leverage these capabilities in a least burdensome way and ensure RWE is used to effectively and efficiently get innovative medicines and technologies to patients around the world.
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