The FDA Needs to Get Back to Basics

People want access to potentially life-saving drugs as soon as possible, especially during a pandemic. That’s why the FDA has the power to issue emergency use authorizations to provide patients with access to drugs that might otherwise be inaccessible during public health emergencies. Since authorizing the use of two investigational drugs to treat Covid-19 in recent months, however, the FDA has not properly carried out its duty to assure the safety and efficacy of those drugs. After issuing emergency use authorizations, the FDA needs to actively oversee the generation of high-quality evidence on the effects of promising but largely-unproven drugs on patient health.

Since the passage of the Food, Drug, and Cosmetic Act in 1938, the FDA has been responsible for evaluating new therapies before those products become widely available to patients. Federal lawmakers charged the FDA with this mission after an unproven new product killed dozens of people, including many children. The FDA was born out of the need to prevent similar tragedies in the future.

More than 80 years later, the agency’s origins as a safeguard against dangerous drugs still serve as a valuable guide for future action. Following its legacy of protecting patients from unsafe drugs, the FDA should quickly revoke emergency use authorizations for drugs when evidence emerges that shows products to be unsafe or ineffective. Regulators did just that in mid-June, revoking a previously-issued emergency use authorization for hydroxychloroquine and chloroquine.

The decision to revoke that emergency use authorization came too late, though. To make more timely decisions about emergency authorization revocations, regulators need to have better access to high-quality data on drug-related patient outcomes. Passively monitoring drugs under emergency authorization does not produce the kind of robust data that regulators need to make decisions in times of crisis.

According to federal law, the FDA may approve emergency use authorizations on the condition that data on the safety and effectiveness of authorized medications will be collected and analyzed. To protect patients from unsafe drugs and ensure the effectiveness of treatments, the FDA needs to routinely exercise that power. The ramifications of allowing the widespread use of investigational drugs without sufficient regulatory oversight could be dire.

As Ameet Sarpatwari, Anna Kaltenboeck, and Aaron Kesselheim note in a recent article in JAMA on the FDA’s emergency use authorization for remdesivir, the agency could use patient registries to conduct more active surveillance. Using patient registries, the FDA could rapidly develop a thorough profile of how authorized drugs are being used and the effects of treatment. The authors recommend that these registries, which are frequently used in post-approval safety monitoring programs, should be run by the FDA or an independent third party to assure unbiased and high-quality data collection. Insisting on high standards of evidence, particularly when evaluating the safety of investigational drugs, should remain a top priority for the agency in times of crisis.

The havoc of this pandemic has brought a palpable sense of urgency to the FDA’s work. To get us through this emergency and the next, the agency needs to more aggressively pursue its most basic mission: ensuring that drugs are safe and effective.

Research for this piece was supported by the Laura and John Arnold Foundation.


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