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Regulatory Focus – RWE Pilot Shows Promise as FDA Seeks to Leverage EHR Data

Regulatory Focus – RWE Pilot Shows Promise as FDA Seeks to Leverage EHR Data

A pilot study released Tuesday supports the use of real-world evidence (RWE) to understand mortality data while highlighting the barriers to using such data in regulatory decision-making.

The retrospective observational analysis was the result of a pilot project aimed at determining whether RWE can provide early insights into patient outcomes through the use of electronic health records (EHRs) and claims data. Data sets were provided by six partners, including COTA, Flatiron, IQVIA, Kaiser Permanente, OptumLabs and PCORI.

Findings showed a correlation between real-world endpoints and overall survival, with no significant variation across age groups. The data partners and officials from the US Food and Drug Administration (FDA) discussed the implications during a public meeting held by Friends of Cancer Research on Tuesday, including the potential benefits and challenges of RWE use.

There is growing interest among pharmaceutical companies across the US in using real-world data (RWD) to assess the value and quality of drugs.

“Understanding the clinical context becomes really important as you interpret findings,” including those relating to mortality data, said Amy Abernethy, chief medical/scientific officer and SVP of oncology at Flatiron Health. RWD can also be used to supplement data from traditional trials.

For the pilot, investigators evaluated data from patients with at least two documented clinical visits from 2011 until the study’s cutoff date.

Panelists agreed that the “following the patient” approach can help companies and regulators alike in coming up with new ways to analyze data.

Still, a recent medical visit gave Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research (CDER), a rude awakening with the hospital’s inability to locate her previous health records. The US has yet to achieve “basic interoperability” or “good patient supportability,” Woodcock said during the meeting.

Beyond measuring safety and traditional efficacy endpoints, RWE has the potential for supporting expansions of drug labels, acting associate director Sean Khozin at FDA’s Oncology Center of Excellence argued. But the current national health IT infrastructure does not support interoperability for capturing clinical relevant endpoints, Khozin added.

Nancy Dreyer, chief scientific officer and global chief of scientific affairs for IQVIA, told Focus interoperability is not absolutely necessary if study partners can just agree to a data sharing privacy protocol, as was this case in the project.

Further, the lack of data standardization continues to be a major barrier to interoperability in the US healthcare system.


“At the basic level, we may have data but we don’t really have evidence yet,” Woodcock argued. “We just have all kinds of disaggregated data that doesn’t stand and it varies when we think about how to make sense out of it.”

Yet another challenge for FDA relates to crafting an RWD-specific auditing plan, noted Rajeshwari Sridhara, director at the Division of Biometrics V within CDER’s Office of Biostatistics.

Meanwhile, FDA is looking to enhance the generation of patients’ EHR data with a $100 million proposal set forth under President Donald Trump’s FY 2019 budget request.

The agency’s new “medical data enterprise” would involve building an national EHR system with a limit in the number of the records it would contain of about 10 million US patients, FDA Commissioner Scott Gottlieb said in a Tuesday blog post.

“This system would expand the data enterprise that we already maintain by incorporating new information from electronic health records, and other sources that would allow us to more fully evaluate medical products in the post-market setting,” Gottlieb said.…