The coronavirus pandemic has infected more than 2.5 million people worldwide, with the United States accounting for nearly 1 million of those cases. The U.S. Department of Health and Human Services (HHS) declared a national public health emergency on March 13, giving federal agencies, including the Food and Drug Administration (FDA), emergency authorities to expedite investigations into the cause of COVID-19 and development of treatments or methods for prevention. FDA has not yet approved any drugs to treat, cure, or prevent the disease, but patients can potentially access investigational therapies, for example through clinical trials, the expanded access program, Right to Try law, and emergency use authorizations.
Clinical trials—controlled investigations of drug or device safety and efficacy—are essential for understanding both novel products and new uses for approved therapies. Such trials adhere to a central protocol that outlines the study’s design, method, and outcome measures, among other details. Public health and medical experts agree that rigorously designed clinical trials are the best method for understanding what products might prevent or treat COVID-19. Currently, there are several such trials underway, and many more will begin enrolling patients over the coming months.
Accessing an investigational therapy through a clinical trial has several advantages. FDA and investigational review boards (IRBs) require that patients are fully informed of the known benefits and risks of an intervention and that they receive close monitoring and follow-up to ensure that any adverse reactions or side effects are treated quickly. Trials allow product developers to collect robust data that can demonstrate whether the benefits of an intervention outweigh its risks, and they can use that data to submit a formal application for FDA approval. The findings from clinical trials are also essential for clinicians making treatment decisions, even in cases in which the developer decides not to pursue an approval. If a treatment does not work for a disease or causes significant harm, physicians can rule it out.
If a patient cannot participate in a clinical trial—for example, because they do not meet the eligibility criteria or cannot travel to a trial site—the patient might be able to access the experimental therapy through FDA’s expanded access program (EAP).
EAP applications can be granted to individuals or groups of patients, which can number in the hundreds or thousands. Typically, FDA approves applications if:
FDA typically responds to such applications in less than a week. In emergency cases, treatment can begin immediately upon verbal authorization from the agency, usually over the phone. Manufacturers must submit a summary to FDA of the outcomes of that expanded access, including any adverse events, which can inform the premarket approval process.
Several hundred patients received remdesivir—an antiviral drug under investigation as a potential COVID-19 treatment—through the EAP; the final results of the clinical trial are pending.
Since 2018, patients have also been able to access experimental treatments through the Right to Try (RTT) law. Much like the EAP, RTT allows certain patients to request access to investigational treatments if they are ineligible for clinical trials and generally have no other FDA-approved treatment options available to them.
But RTT differs in several ways from the EAP. Patients must be terminally ill to qualify, and the program is available only for drugs—not medical devices. RTT also removes FDA and IRB oversight from the approval process, leaving only the treating physician and the manufacturer to authorize the use of a drug on a patient. Additionally, manufacturers are required to report only known serious adverse events to FDA, and they are protected from liability arising from use of the product. The purpose of removing these steps, according to the law’s supporters, is to reduce the administrative burden on physicians and incentivize manufacturers to make their experimental products available to patients. However, just like with the EAP, manufacturers are not obligated to participate. To date, the number of patients who have accessed treatment under the RTT law can be measured in single digits.
If HHS has declared a public health emergency related to chemical, biological, radiological, or nuclear agents, FDA can authorize the emergency use of an unapproved medical product, or the unapproved use of an approved medical product. That therapy can then be legally used as long as the emergency declaration remains active. In response to the coronavirus pandemic, FDA has issued dozens of emergency use authorizations for treatments, diagnostic tests, and medical devices such as facial respirators and ventilators.
During a public health emergency, especially one that is caused by a widespread infectious disease such as COVID-19, FDA can use its existing authorities to play a critical role in ensuring that safe and effective treatments and prevention tools reach patients as quickly as possible. FDA has made broad use of its authorities to allow access to investigational medical products and should continue to do so to combat the pandemic. The agency must also ensure that allowing access to these products outside of the clinical trial pathway will not undermine attempts to develop rigorous evidence on their safety and effectiveness.
Liz Richardson directs The Pew Charitable Trusts’ health care products project.