Califf: Leveraging Real World Evidence is 'Top Programmatic Priority' for FDA

Regulatory NewsRegulatory News | 11 May 2016 |  By 

The "top programmatic priority" for the US Food and Drug Administration (FDA), under Commissioner Robert Califf, is to leverage real world evidence from the healthcare system to inform FDA decision making, he told participants at the Food and Drug Law Institute's annual conference last week.

While Califf said his first priority as commissioner is to strengthen FDA's workforce, that stronger workforce will be critical to achieving FDA's goals in specific program areas such as real world evidence.

Specifically, Califf said he wants to see FDA develop a system for "[real world] evidence generation that can meet the demands of the next few decades."

The groundwork for such a system is already being built by FDA and others through recent developments in electronic health records, patient registries and FDA-led initiatives such as Sentinel and unique device identifier (UDI) adoption, he added.

The problem Califf hopes to solve is what he perceives as a lack of good evidence to support healthcare and regulatory decision making.

"Unfortunately, too many of the decisions made today about health and healthcare are not supported by high quality evidence," he said.

This is a particular problem for FDA, in Califf's opinion, as the agency "lacks vital evidence needed to support the definitive regulatory determinations of safety and efficacy and appropriate indications for marketed medical products."

Part of the issue is that most of the data used to support the approval of new products comes from clinical trials, which in many ways do not reflect the reality of clinical care.

"In the past, so called 'regulatory trials' tended to focus on high quality studies with detailed study procedures, restrictive inclusion and exclusion criteria and have been conducted in special study sites," Califf said.

This is because in the past, "there was no way to collect reliable information in clinical practice, and the healthcare system lacked a structure that was amenable to the discipline required to obtain reliable research answers," he added.

Today, however, "Nearly every American has an electronic health record, national health repositories of curated claims data are available and clinical registries are growing at a rapid rate. The consolidation of healthcare delivery into large integrated health systems means that research clinics are being folded into clinical care systems with increasingly robust governance systems and highly curated enterprise data warehouses," Califf said.

Through the use of sophisticated analytical tools, Califf believes FDA and product sponsors are "at the tipping point for generating the scientific evidence needed to make good decisions across the spectrum" of healthcare products.

Rather than primarily relying on randomized controlled trials, Califf said he sees well-designed pragmatic studies as a way of generating high quality evidence.

"Prospectively designed registries and cohort studies in the context of clinical practice are highly valuable, and randomized trials conducted in the context of clinical practice, often called a pragmatic clinical trial may be the most important source of knowledge in the future," he said.

However, for this to work for FDA, Califf says that both data sources and research methods must be optimized.


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