"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 guidance will help innovators and researchers do just that.”
The FDA has provided new guidance for researchers developing prevention and treatment options for coronavirus disease 2019 (COVID-19) in order to accelerate the process of developing new drugs. Guidance documents should make the submission process for the initiation of studies for new drugs and biological products more efficient, and recommendations outline ways to design trials to evaluate the safety and effectiveness of new treatments, according to a press release from the FDA.
The guidance builds upon the steps the FDA has already taken to streamline the review process of potential new treatments for COVID-19 as quickly and efficiently as possible.
“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, MD, in a statement “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 guidance will help innovators and researchers do just that.”
The first guidance from the FDA outlines the processor for developers receiving agency feedback on their data with the primary goal of initiating clinical trials as quickly as possible. The FDA strives to help sponsors move potentially effective products into clinical trials quickly while maintaining safety in patients. The guidance describes types of data and information that should be provided to address clinical, nonclinical, and quality considerations prior to application submission for the initiation of new studies.
The purpose of the second guidance it to provide the FDA’s current recommendations on later-stage studies that are meant to establish safety and effectiveness, outlining critical sponsor considerations such as appropriate patient selection, which includes the evaluation of therapy in high-risk patients. With this guidance, sponsors should understand how to design their trials, including factors such as study duration, assessment of efficacy, and safety monitoring. This guidance should help sponsors design studies efficiently that could lead to the review of and potential approval of safe, effective agents.
The guidance documents form the FDA are supplemented by a variety of ongoing activities and broad agency guidance in support of innovative study designs to test multiple agents and patient populations simultaneously, with the end result being understanding the safety and efficacy as soon as possible.
The FDA continues to expedite preventative and therapeutic options and is engaged in a public-private partnership with the National Institutes of Health and others to accelerate the development of novel vaccines and treatments aimed at COVID-19.
The Coronavirus Treatment Acceleration Program was launched by the FDA in an effort to move new medical products to patients with COVID-19 quickly while simultaneously determining the effectiveness and the benefits. The FDA has been working closely with federal health partners, academia, and industry to advance developments for COVID-19.
Over 130 trials are underway to potentially offer treatment for COVID-19, and the FDA is overseeing the programs for other agents still in the planning stages. Drugs under investigation or in development include antiviral drugs to prevent viruses from multiplying and immunomodulatory therapies aimed at correcting the immune system to react to the virus, particularly in cases where the body starts attacking the patient’s own organs.
Among these clinical trials, the randomized phase III PRE-VENT study is evaluating pacritinib, an agent primarily explored for the treatment of myelofibrosis, as treatment of patients with severe COVID-19 is underway in both the United States and Europe. Ruxolitinib (Jakafi), another agent from the oncology space, is being investigated as treatment of cytokine storm associated with COVID-19 in the global phase III RUXCOVID clinical trial. The BTK inhibitor acalabrutinib (Calquence) is under investigation in the CALAVI study for the treatment of cytokine storm in patients who are severely ill with COIVD-19.
The toxicity of these agents and many others is under evaluation in clinical trials to determine their effectiveness against COVID-19. Data has already begun to be shared with the FDA from some clinical trials, such as leronlimab, which had data recently published in a pre-print manuscript online. The FDA intends to engage with pharmaceutical sponsors and other government agencies to facilitate patient access to these potential treatments as soon as favorable results are seen.
The Accelerating COVID-19 Therapeutic Interventions and Vaccines partnership between the United States and European government partners, as well as more than 12 biopharmaceutical companies, aims to develop a collaborative framework to prioritize vaccine and drug candidates, as well as streamline clinical trials, coordinate regulatory processes, and leverage assets among all partners.
Coronavirus (COVID-19) Update: FDA Takes New Actions to Accelerate Development of Novel Prevention, Treatment Options for COVID-19 [news release]. FDA; May 11, 2020. https://bit.ly/2LpUpRM. Accessed May 13, 2020.