Responding to a community outbreak through expanded access
On a Friday in late July, Clinical Trials Research Project Manager Hema Buddha, MBBS, received an urgent call from a physician at the Kaiser Permanente South Bay Medical Center.
The physician had a patient with symptoms of mpox, then commonly referred to as monkeypox, and needed help accessing an investigational new drug called tecovirimat.
While the physician waited on blood tests to confirm the diagnosis, Buddha began to navigate the process of obtaining the drug through an expanded access protocol managed by the U.S. Centers for Disease Control & Prevention. A representative of the County of Los Angeles Department of Public Health, which served as a local intermediary for the CDC, provided guidance.
“We were coming up on the weekend, and everybody wanted to get treatment for the patient as quickly as possible,” said Buddha, whose primary focus is on helping physicians procure drugs available through expanded access (sometimes called compassionate use). “There were a lot of steps in getting the medication and approvals needed before treatment could be initiated.”
Buddha reached out to several resources in Kaiser Permanente Southern California, including the Institutional Review Board, infectious disease specialists, pharmacy staff, and colleagues from the clinical trials team. She traded calls and emails with the Department of Public Health. After a flurry of activity, the process moved forward. Within days, the treatment was started.
Infectious disease physicians, pharmacists jump in to help
Regional Chief of Infectious Disease Elizabeth Hudson, DO, MPH, had been preparing for a local outbreak since late spring, when mpox cases first began appearing in Europe.
“It became imperative for us as infectious disease specialists to understand the disease so we could help our fellow clinicians identify it, especially since the cases we were seeing didn’t look like textbook examples,” she said. “By late summer, we started seeing an awful lot of cases, particularly in Los Angeles and San Diego counties.”
Dr. Hudson and her colleagues worked closely with the clinical trials team to make sure all 51 infectious disease specialists within Kaiser Permanente Southern California had the training and documentation to be able to initiate treatment with tecovirimat. To make sure each specialist could quickly initiate the informed consent process, the clinical trials teams created electronic forms that were individualized to each physician, which could be sent out via patient messages from Kaiser Permanente HealthConnect®.
The clinical trials team worked closely with the infectious disease specialists, pharmacists, and others, meeting at least weekly to develop and refine the workflows that supported access to tecovirimat. Over time, Dr. Hudson noted a reduction in the administrative burden for the infectious disease specialists. Most important, physicians were able to move swiftly to help their patients.
“Patients who had these lesions were very uncomfortable, and that is putting things mildly,” she said. “They were calling and emailing us, sometimes telling us they were in excruciating pain. We wanted to get them potential relief as soon as possible.”
By the end of 2022, nearly 170 patients had received tecovirimat as treatment at Kaiser Permanente Southern California. This would not have been possible without the clinical trials team, Dr. Hudson noted.
“It is important for folks to know just how robust our expanded access program is, and what an important role it plays in helping us secure access for investigational treatments for our patients,” she said. “Expanded access is key to keeping our patients well and staying at the forefront of emerging infectious diseases.”
Bringing scientific rigor through a new clinical trial
As valuable as the expanded access program has been in getting patients access to a promising treatment, the medical community still needs data to better understand the safety and efficacy of the drug in treating human mpox.
“Tecovirimat was developed as an antiviral against smallpox, which is in the same family as mpox,” said William J. Towner, MD, FACP, FIDSA, physician director of the Division of Clinical Trials Research in the Department of Research & Evaluation. “We won’t get the scientific insights we need through data from expanded access. We really need the rigor of a trial.”
In late summer 2022, Dr. Towner and his team heard that the National Institute of Allergy and Infectious Diseases was sponsoring a Phase 3 clinical trial evaluating tecovirimat for treatment of human mpox. The study would be led by NIAID-funded AIDS Clinical Trials Group, also known as ACTG.
Kaiser Permanente Southern California had previously participated in several ACTG studies, including recent trials for COVID-19 treatments. At Dr. Towner’s request, Clinical Trials Operations Director Tiffany Castanon, MHA, reached out to see if there might be an opportunity for Kaiser Permanente Southern California to join as a site for the study. A little more than a month later, Kaiser Permanente Southern California had been established as a site for the Study of Tecovirimat for Human Monkeypox Virus, or STOMP.
“Our program had an opportunity to contribute to Kaiser Permanente Southern California and our local community in 2 very important ways, first by facilitating access to an important investigational drug and now by helping to add to the evidence needed to understand its safety and efficacy,” said Dr. Towner. “I am so grateful to all of the people across the organization that helped us succeed on both fronts.”
By late fall, the number of cases in Southern California began to decline. But infectious disease specialists remain vigilant, aware that cases could begin to climb again if the disease gets a new foothold in our community.
“It is reassuring to know that if we start to see more cases in our area again, our clinical trials division will be able to respond by helping our physicians and patients receive treatment through expanded access and by adding to the scientific evidence about treatment of mpox through participation in this important study,” said Dr. Hudson.