The US Food and Drug Administration has taken important actions to help accelerate the development of prevention and treatment options for COVID-19 by providing new guidance with recommendations for innovators and researchers conducting work in this area. These guidance documents aim to make the process for submitting applications to initiate studies for new drugs and biological products more efficient and outline recommendations for ways to design clinical trials to evaluate safety and effectiveness of these medical products for COVID-19.

"Our staff continues to work around the clock with the world’s best innovators and researchers to help expedite the development of prevention and treatment options for COVID-19," said FDA Commissioner Stephen M. Hahn, M.D. "Accelerating the investigation of safe and effective therapies that could benefit people affected by the COVID-19 pandemic is one of the FDA’s highest priorities. We are committed to maximizing our regulatory flexibility and using every tool at our disposal to speed the development and availability of these medical products and believe these new guidances will help innovators and researchers do just that."

Quickly after the emergence of this virus, the FDA began working directly with federal health partners, academia, and industry to advance medical countermeasures against COVID-19. Given the urgent nature of the pandemic, the FDA launched a new program called the Coronavirus Treatment Acceleration Program to help move new medical products to patients as soon as possible, while at the same time determine whether they are effective and if the benefits outweigh the risks.

So far, more than 130 clinical trials of potential COVID-19 related drugs and biological products are underway with FDA oversight and additional development programs for other agents are in the planning stages. These include studies on therapies such as antiviral drugs to keep viruses from multiplying, as well as therapies called immunomodulators aimed at tamping down the body’s own immune reaction to the virus, in cases where the body starts attacking the patient’s own organs. Studies like these, which are conducted by the pharmaceutical industry and academic researchers, assess the products’ toxicity and help to find out if they work against COVID-19. The FDA has started to receive data from clinical studies and expects to receive more data regarding investigational products soon. The agency intends to engage with pharmaceutical sponsors and other government partners to facilitate patient access as soon as possible when favorable results are observed.

The guidances issued build on steps the agency has taken to streamline its review and advice process to get studies started as quickly as possible, and they incorporate information provided to individual innovators or researchers over the past several months.

The first guidance, "COVID-19 Public Health Emergency: General Considerations for Pre-IND (Investigational New Drug application) Meeting Requests for COVID-19 Related Drugs and Biological Products," outlines a more efficient process for developers to receive agency feedback on their supporting data with the goal of starting clinical trials as soon as possible. The FDA is committed to helping sponsors get potentially effective products into study quickly, while protecting the safety of patients. To that end, the guidance provides sponsors clarity on the types of data and information they should provide to address clinical, nonclinical and quality considerations before submitting an application to initiate studies.

The second guidance, "COVID-19: Developing Drugs and Biological Products for Treatment or Prevention," provides the FDA’s current recommendations on later stage clinical trials intended to establish safety and effectiveness for COVID-19 products. The guidance outlines critical sponsor considerations such as appropriate patient selection, including the evaluation of therapies in patients at high risk of complications from COVID-19 (e.g., the elderly). In addition, the guidance helps sponsors to understand how to design their trials, including considerations of study duration, assessment of efficacy and monitoring for safety. The FDA anticipates this guidance will help sponsors to efficiently design studies that may lead to the review and potential approval of safe and effective drugs and biological products to address the COVID-19 pandemic.

These guidance documents are supplemented by a variety of ongoing activities and broad agency guidance to support innovative study designs, including master protocols, to test multiple products and multiple populations simultaneously and get answers on safety and efficacy as quickly as possible.

In addition to the FDA's work to expedite preventative and therapeutic options, the agency is engaged in a public-private partnership with the National Institutes of Health and others to speed the development of COVID-19 vaccine and treatment options. The Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) partnership, which also includes other US and European government partners and more than a dozen biopharmaceutical companies, will develop a collaborative framework for prioritizing vaccine and drug candidates, streamlining clinical trials, coordinating regulatory processes and/or leveraging assets among all partners to rapidly respond to COVID-19 and future pandemics.