Real World Evidence An Important—and Controversial—Piece of the Cures Act

By a 392 to 26 vote, the House of Representatives passed the 21st Century Cures Act, which would speed FDA approval for drugs and medical devices and allow the agency to include real world evidence in the approval process. Drug companies will be able to submit drug data summaries, instead of the clinical trials that are a much longer process, according to STAT. In addition, the legislation gives states $1 billion to fight the opioid crisis, and allocates $4.8 billion for medical research.

The Act, passed in the House on Tuesday, now heads to the Senate next week, where approval is expected. Patient advocacy groups celebrated. Opponents argue that the Act lowers safety and efficacy standards and contend that the FDA already moves faster than similar agencies in other countries.

Representative Fred Upton led the charge for the Act in the House. According to STAT, he said, “We are on the cusp of something special—a once-in-a-generation opportunity to transform how we treat disease. With today’s vote, we are taking a giant leap on the path to cures.”

Still, there are doubts, some of which were expressed in an opinion(link is external) piece in STAT by Reshma Ramachandran, MD, of Johns Hopkins Bloomberg School of Public Health, and Zackary Berger, MD, an assistant professor of medicine at the Johns Hopkins School of Medicine. They argue that the Act weakens the idea of “FDA approved,” and take issue with the role real world evidence would play. They worry that new drugs would be approved for unmet medical needs without having been tested in specific patient populations.

In addition, new medical devices could be “approved based on anecdotal case studies or reports in medical journals, rather than having to be held to the gold standard of testing in clinical trials. On top of that, manufacturers would be able to make changes to these high-risk devices without any FDA oversight.”

Janet Woodcock, director of the Center for Drug Evaluation and Research, also takes issue with the place of real world evidence in the Act, telling the Regulatory Affairs Professional Society(link is external) (RAPS), that “data gathered from health care has always had one characteristic, it's not very good.”

According to RAPS, the FDA’s definition of real world evidence includes:

  • “generation of hypotheses to be tested in a prospective clinical study
  • as a historical control, a prior in a Bayesian trial, or as one source of data in a hierarchical model or a hybrid data synthesis
  • in a setting where a registry or some other systematic data collection mechanism exists and the data can potentially be used as a concurrent control group or as a mechanism for collecting data related to a clinical study to support device approval or clearance
  • in some circumstances where real-world use of a device is in a broader patient population or wider set of circumstances than described in the device labeling, it may be possible to use existing systematically collected real-world data to expand the labeling
  • for public health surveillance efforts and to understand the evolution of the benefits and risks of devices after they have been approved or cleared in the U.S.”