The shortage of tests for diagnosing COVID-19, like other aspects of the public health crisis, has drawn attention to the U.S. government’s emergency powers.
In recent weeks, the Food and Drug Administration in particular has taken a series of actions to address COVID-19, the disease caused by the new coronavirus.
The Trump administration’s declaration of a public health emergency Jan. 31 triggered provisions in law that permit the FDA to exercise greater discretionary authority in meeting its responsibilities to approve and regulate medical products.
The purpose of those emergency provisions is to ensure that the statutory standards and requirements that the Food and Drug Administration is tasked with administering do not become impediments to quickly getting patients and health care providers the medical products they need in an emergency.
When it comes to regulating medical products such as drugs, devices, and tests, the FDA’s congressionally mandated responsibilities can be summarized as ensuring that the product works as intended and that the product information provided by the manufacturer to doctors and patients is complete, accurate, and based on sound science.
Congress has given the Food and Drug Administration authority to determine whether medical products may be sold in the U.S. That authority extends to every new product, even if it is just a copy of one already on the market (such as a new version of a generic drug), or is a modification to an existing product (such as adding remote monitoring functionality to a ventilator).
The FDA also regulates the content and wording of the information that manufacturers provide, both to consumers (such as medication guides or advertisements) and to physicians and other medical professionals.
The latter is called the product’s “label,” though it actually is a lengthy document containing extensive, scientific information about the product, including how it works, the conditions it has been proven to treat, recommended dosing, and any safety issues such as side effects, interactions with other medicines, or considerations when prescribing it for patients with other medical conditions.
Normally, the Food and Drug Administration requires that a product’s approval and associated information be supported by data from controlled clinical trials designed to prove the product’s effectiveness in treating one or more conditions and to identify any safety issues associated with using the product as intended.
Also important is what the FDA does not do. Although the agency regulates what manufacturers say about their products, it does not regulate how medical providers use medical products or what they say or publish about their use of those products.
Doctors can, and do, prescribe drugs for uses other than those included on the FDA-approved label. They can, and do, publish articles reporting their results.
Indeed, the practice of “off label” use is fairly common in some specialties such as oncology, where a physician may try treating patients with one type of cancer using a drug approved to treat a different type of cancer.
In response to the COVID-19 pandemic, the Food and Drug Administration has issued “emergency use authorizations” under provisions of the law that allow the agency, during a public health emergency, to make exceptions to the standards it normally is tasked with applying.
Emergency use authorizations are not a different regulatory process, but rather reflect temporary adjustments to the FDA’s standard practices. Under both normal and emergency conditions, the FDA is tasked with answering the same basic question: Does the possible benefit outweigh the possible harm?
The difference is that during a public health emergency many people could suffer harm, including death, before the Food and Drug Administration gets results from the controlled clinical trials needed to reach definitive conclusions about a product’s effectiveness and safe use.
Consequently, during an emergency, the FDA instead must rely on information that it can gather from other sources, such as physicians who report the effects of using a drug off-label to treat patients with a particular condition.
In an emergency situation, the FDA instead looks at the “totality of scientific evidence available” to make determinations, with the caveat that the agency subsequently may change its authorizations or recommendations as more data becomes available.
To date, the Food and Drug Administration has issued emergency use authorizations to increase physician and patient access to needed drugs, tests, and equipment and to enable manufacturers to develop and bring to market innovative products more quickly in response to the emergency. For example:
—Off-label drug use. On March 28, the FDA issued an emergency use authorization for two generic drugs (chloroquine phosphate and hydroxychloroquine sulfate), which long have been approved for treating malaria and several other conditions. Some doctors have reported success in using the drugs to treat patients with COVID-19.
Although doctors already could use those drugs off-label to treat COVID-19, this emergency use authorization allows federal, state, and local public health authorities to obtain those drugs for that purpose from the Department of Health and Human Services’ Strategic National Stockpile, a reserve supply of essential drugs and medical products set aside for use during public health emergencies.
This emergency use authorization also permits the stockpile to receive and distribute versions of those drugs that have not been approved for marketing in the U.S. and are not accompanied by FDA-approved packaging and labeling. That is to address instances where a quantity of the drug may have been produced for use in another country with different requirements.
For each of those drugs, the FDA also created an accompanying fact sheet for health care providers, giving guidance on the administration and dosing of the drug to treat COVID-19 (based on currently available information) and details on the drug’s known safety issues.
—Tests. To address the shortage of tests for COVID-19, the Food and Drug Administration so far has issued 34 emergency use authorizations for tests (or testing components) developed by laboratories and manufacturers.
These range from specialized tests at single-site laboratories to mass-produced test kits developed by large, well-known companies such as Abbott, Roche, and Thermo Fisher Scientific.
Again, these authorizations are accompanied by FDA fact sheets for health professionals on how to properly administer the tests.
—Respirators. The Food and Drug Administration has issued emergency use authorizations for protective respirators used by workers in other industries but not previously approved for use in health care settings, for some disposable and reusable respirator filters, and for a system that decontaminates reusable filters.
—Ventilators. Finally, the FDA issued an emergency use authorization to address the shortage of ventilators. This authorization covers ventilators, their components, attachments, and related equipment.
During the emergency, it authorizes the marketing of ventilators and components that meet certain specifications but have not yet received normal marketing approval.
It also authorizes modification of anesthesia gas machines so they may be used as ventilators, and provides guidance to companies (such as Medtronic) that are working to develop hardware and software solutions to enable a ventilator to support more than one patient.
That guidance is part of the FDA’s effort to work with manufactures to approve and quickly bring to market any innovations that can help address the emergency.
Although the Food and Drug Administration’s usual practice is to seek more complete and definitive answers based on controlled clinical trials, a health emergency alters the risk/benefit calculus by dramatically increasing the degree and extent of potential harm.
The greater the potential harm, the more important it becomes to pursue solutions—and quickly—even when the potential benefits are not fully understood or quantified.
That is why Congress previously gave the Food and Drug Administration flexibility in an emergency to deviate from the normal statutory standards for approving medical products, and why the agency now exercises that authority to help meet the crisis.