Experts Ask FDA to Rethink Trial Exclusion Criteria

August 27, 2018

Ethical concerns about testing on vulnerable groups may be unnecessary and may be depriving trials of key critical data, a panel of experts says, urging the FDA to reconsider rigid rules excluding certain groups from clinical trials—or at least make decisions on a case-by-case basis.

Debate has swirled around this issue for years, with critics questioning whether the exclusions have led to trial pools that don’t accurately reflect potential treatment targets. So the FDA in the spring convened a roundtable of experts to address barriers preventing some populations, including children, seniors, pregnant women and those with multiple health conditions, from enrolling in trials.

In a summary report released last week, the panel—which consisted of sponsors, researchers and academics—said it’s time to revisit and update those rules.

“Excluding these patients limits the ability of a trial to generate data that are relevant to the actual users of the drug and limits the ability to describe how investigational therapies affect the pathophysiology of common chronic conditions and interact with other therapies,” the report cautions, calling for regulators to explore erasing or at least considering exclusions on a per case basis.

The report notes that the 21st Century Cures Act requires the NIH to take another look at issues blocking older adults from participating in clinical trials—and urges regulators to move quickly to broaden access for what is now the nation’s largest single demographic group.

The panel also advises sponsors, researchers and regulators to be more open with patients about how eligibility criteria are determined and even to solicit patients’ views on how to structure trials.

“More frequent and routine patient involvement in trial design could lead to more trials that explicitly address outcomes important to patients and achieve greater patient enrollment,” the summary stresses.

It adds that expanded access not only can inform a drug’s risk-benefit profile but may also offer hope to patients out of other options.

“It is critical to consider the need for humanitarian access to an investigational therapy while not undermining the overall clinical trial process,” the summary states.

The bottom line, concludes the report: “Enhancing inclusion and encouraging greater diversity in clinical trial populations is a priority for regulators, sponsors, investigators, and patient advocates.”

Read the roundtable summary here.