PCORI Biweekly COVID-19 Scan: Treating Patients Hospitalized with COVID-19 (April 15-28, 2021)
The Briefing provides an at-a-glance view of some important developments in the information universe surrounding COVID-19. The views presented here are solely those of ECRI Horizon Scanning and have not been vetted by other stakeholders.
On April 19, everyone in the United States older than age 16 became eligible to be vaccinated against COVID-19. Although it is still early, there are signs that younger people are less likely to be vaccinated.
This is prompting incentive programs in at least one state and speculation that COVID-19 could become a young adult’s disease. Already, states like Michigan are reporting shifting demographics in hospital wards.
Across the nation, there are still more than 30 000 hospitalized patients and a need for better treatments (see Topics to Watch). The debate also continues about whether children need to be vaccinated to further suppress COVID-19.
ECRI Horizon Scanning has selected the topics below as those with potential for impact relative to COVID-19 in the United States within the next 12 months. All views presented are preliminary and based on readily available information at the time of writing.
Because these topics are rapidly developing, we cannot guarantee the accuracy of this information after the date listed on this publication. In addition, all views expressed in the commentary section are solely those of ECRI Horizon Scanning and have not been vetted by other stakeholders. Topics are listed in alphabetical order.
Fostamatinib (Tavalisse) to Treat Patients Hospitalized with COVID-19
At a Glance
- Fostamatinib (Tavalisse) is an orally administered spleen tyrosine kinase (SYK) inhibitor being investigated for treating adults hospitalized with COVID-19. The US Food and Drug Administration (FDA) approved the drug for treating chronic immune thrombocytopenia.
- By inhibiting SYK, fostamatinib inhibits mediators of proinflammatory cytokine release and hypercoagulability thought to contribute to the COVID-19 disease course.
- Preliminary data from a phase 2 trial that enrolled 59 adults hospitalized with COVID-19 and treated with standard care found that the addition of fostamatinib significantly reduced serious adverse events, the median number of days in the intensive care unit (ICU), and death rates, compared with those outcomes in patients given placebo.
- Fostamatinib is in a phase 3 trial, with primary completion expected in August 2021. The manufacturer announced plans to pursue emergency use authorization (EUA) from the FDA.
- Based on clinical-trial dosing and fostamatinib pricing information, a course of fostamatinib to treat COVID-19 is estimated to cost $5760.
Fostamatinib is an orally administered SYK inhibitor. The FDA has approved it under the brand name Tavalisse (Rigel Pharmaceuticals Inc, South San Francisco, California) for treating chronic immune thrombocytopenia, and fostamatinib is being investigated to treat adults hospitalized with COVID-19. By inhibiting SYK, fostamatinib purportedly inhibits downstream mediators of the COVID-19 disease course that promote proinflammatory cytokine release and hypercoagulability. These processes are thought to contribute to acute lung injury, acute respiratory distress syndrome, acute kidney injury, and blood clots in COVID-19.
A phase 2 trial enrolling 59 adults hospitalized with COVID-19 reported preliminary data. Investigators reported that in the trial, patients given fostamatinib compared with placebo displayed significantly reduced serious adverse events (by half), improved clinical status at day 15, and reduced mortality (0 vs 3 deaths). The median ICU stay was shorter in the fostamatinib group (3 vs 7 days; p = 0.07). A phase 3 trial enrolling 308 participants is ongoing, with primary completion expected in August 2021.
The manufacturer announced plans to discuss pursuing an EUA with the FDA for fostamatinib to treat adults hospitalized with COVID-19. Based on clinical trial dosing and fostamatinib pricing information, a course of fostamatinib to treat COVID-19 is estimated to cost $5760.
Fostamatinib is an FDA-approved drug being investigated as a repurposed treatment for patients hospitalized with COVID-19.
Early feedback from ECRI internal stakeholders suggested that fostamatinib could improve patient health outcomes by decreasing severe complications and death in patients hospitalized with COVID-19. Preliminary results from the phase 2 trial are promising because they suggest that fostamatinib led to positive improvements in several health outcomes. However, the trial size was small, and more data from larger studies are needed to confirm the drug’s efficacy and potential impact.
Fostamatinib’s impact might be limited by the number of other investigational COVID-19 treatments and the availability of vaccines. However, effective treatments for severe COVID-19 are still needed because some people will not seek vaccination.
- Category: Treatments
- Areas of potential impact: Patient outcomes, population health, health care costs
VERU-111 to Treat Patients Hospitalized with COVID-19 at High Risk of Developing Acute Respiratory Distress Syndrome
At a Glance
- VERU-111 is an investigational, orally administered, small-molecule drug. It is being developed to treat patients hospitalized with COVID-19 who have underlying health conditions that place them at high risk of developing acute respiratory distress syndrome (ARDS).
- VERU-111 is an alpha- and beta-tubulin inhibitor and microtubule disruptor with purported antiviral and anti-inflammatory properties.
- Data reported from a phase 2 clinical trial enrolling 38 adults hospitalized with COVID-19 who were at high risk of developing ARDS found that by day 29 after treatment, VERU-111 reduced the risk of death or respiratory failure by 81%.
- Participants who received VERU-111 also demonstrated significant reductions in time spent in the intensive care unit (ICU; 3.00 vs 9.55 days in the placebo group) and on mechanical ventilation (1.6 vs 5.4 days).
VERU-111 (Veru Inc, Miami, Florida) is an investigational, orally administered, small-molecule drug. It is being developed to treat patients hospitalized with COVID-19 who have underlying health conditions that place them at high risk of developing ARDS. These conditions include diabetes, chronic kidney disease, and severe obesity.
VERU-111 is an alpha- and beta-tubulin inhibitor and microtubule disruptor with purported antiviral and anti-inflammatory properties. It is thought to disrupt the transport of SARS-CoV-2 within the cell, decreasing its ability to cause infection. It also moderates the severe inflammation associated with severe COVID-19 and reduces poor outcomes, including respiratory failure and death.
Data reported from a phase 2 clinical trial that enrolled 38 adults hospitalized with COVID-19 who were at high risk of developing ARDS demonstrated that by day 29 after treatment, 30% of participants in the placebo group had either died or progressed to respiratory failure, compared with only 5.6% of participants who received VERU-111. This suggests that VERU-111 reduced the risk of death or respiratory failure by 81%. Participants who received VERU-111 also demonstrated significant reductions in time spent in the ICU (3.00 vs 9.55 days in the placebo group) and on mechanical ventilation (1.6 vs 5.4 days).
VERU-111 is an investigational oral drug intended to treat hospitalized patients at high risk of developing ARDS due to COVID-19.
Early feedback from ECRI internal stakeholders suggested that VERU-111 could improve patient health outcomes for those at high risk of developing ARDS due to COVID-19. Improved outcomes might include increased survival, fewer days spent in the ICU or on a ventilator, and faster recovery. The initial phase 2 data are positive, although more efficacy data are needed in larger phase 3 clinical trials and should be compared with other COVID-19 treatments.
The increasing uptake of vaccines might lower the number of patients this treatment might benefit. However, effective treatment might help decrease disparities in health outcomes for some patients who are at high risk of developing ARDS. VERU-111 might be used in addition to standard-of-care treatments, and the treatment might be costly, limiting its use in some patients.
- Category: Treatments
- Areas of potential impact: Patient outcomes, population health, health care disparities, health care costs
Horizon scanning is a systematic process that serves as an early warning system to inform decision makers about possible future opportunities and threats. Health care horizon scanning identifies technologies, innovations, and trends with potential to cause future shifts or disruptions—positive or negative—in areas such as access to care, care delivery processes, care setting, costs of care, current treatment models or paradigms, health disparities, health care infrastructure, public health, and patient health outcomes.
The PCORI Health Care Horizon Scanning System (HCHSS) conducts horizon scanning to better inform its patient-centered outcomes research investments. Initially, PCORI defined the HCHSS project scope to focus on interventions with high potential for disruption in the United States in 5 priority areas: Alzheimer’s disease and other dementias, cancer, cardiovascular diseases, mental and behavioral health conditions, and rare diseases. In addition, the system captures high-level disruptive trends across all clinical areas, which may lead PCORI to expand the project scope to include other priority areas in the future.
In early 2020, the COVID-19 pandemic created a fast-moving, widespread public health crisis. In May 2020, PCORI expanded its HCHSS to elucidate the landscape of potentially impactful applications for COVID-19. The HCHSS COVID-19 supplement scans for, identifies, monitors, and reports on emerging and available COVID-19-related treatments, diagnostics, preventive measures, management strategies, and systems changes with potential for high impact to patient outcomes—for individuals and populations—in the United States in the next 12 months.
The HCHSS COVID-19 supplement produces 3 main outputs:
- Biweekly COVID-19 Scans (eg, this document) provide ECRI Horizon Scanning with a vehicle to inform PCORI and the public in a timely manner of important topics of interest identified during ongoing scanning and topic identification or through the ECRI stakeholder survey process.
- Status Reports (quarterly) briefly list and describe all COVID-19-related topics identified, monitored, and recently archived.
- High Impact Reports (every 4 months) highlight those topics that ECRI internal stakeholders (eg, physicians, nurses, allied health professionals, public health professionals, first responders, health systems experts, clinical engineers, researchers, business and finance professionals, and information technology professionals) have identified as having potential for high impact relative to COVID-19 in the United States.
Commentary in this COVID-19 Scan reflects preliminary views of ECRI Horizon Scanning and internal ECRI stakeholders.
The information contained in this document has not been vetted by other stakeholders.
We welcome your comments on this Scan. Send them by email to [email protected].
Posted: May 5, 2021
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