Vague FDA policies on adverse event data are keeping patients from accessing investigational drugs

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Drug manufacturers are unsure how the FDA is using adverse event data collected through the expanded access program.

Ambiguous adverse event data policies from the Food and Drug Administration are preventing drug manufacturers from making investigational drugs accessible to patients with life-threatening illnesses.

The FDA has made strides in recent years to provide greater access to drugs that are still in the clinical trial phase by issuing expanded access exemptions to drug manufacturers, allowing them to provide drugs that are still being tested to patients with diseases for which there are limited medical options. Some states have also passed “Right-to-Try” laws in an attempt to provide access to investigational drugs.

But manufacturers are constrained by a lack of clarity from the FDA regarding how the agency uses adverse event data, according to a report released by the Government Accountability Office. Drugmakers told the watchdog agency they were concerned adverse event data collected through the expanded access program would prompt the FDA to put a “clinical hold” on their drug, delaying approval. That, in turn, influences their decision to offer access to certain drugs that are still being tested.

Historically, a clinical hold almost never occurs. The GAO found just two instances in which the FDA used adverse event data to put a hold on a drug, but the agency recommended that the FDA provide more clarity about how it uses data reported by manufacturers, noting that existing documents offer little guidance on the issue.

The FDA agreed with the recommendations.

RELATED: FDA plans to use computer modeling to speed up drug, device evaluations

The use of data—for both drugs and devices—has emerged as a key focal point for the FDA to speed innovative medical products. Earlier this week, FDA Commissioner Scott Gottlieb explained how the agency plans to use computer modeling to speed up drug and device evaluations.

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