Briefing for April 29-May 12, 2021 - PCORI
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BIWEEKLY
PCORI HEALTH CARE HORIZON SCANNING SYSTEM
COVID-19 SCAN VOLUME 1, ISSUE 26
Briefing for April 29-May 12, 2021 Remestemcel-L (Ryoncil) to Treat Moderate
This briefing provides an at-a-glance view of some important to Severe COVID-19 Acute Respiratory
developments in the information universe surrounding COVID-19. Distress Syndrome
The views presented here are solely those of ECRI Horizon
Scanning and have not been vetted by other stakeholders.
AT A GLANCE
COVID-19 has had an enormous impact on the US health
care system. As we emerge from the acute impacts of the f Remestemcel-L (Ryoncil) is an investigational cell
pandemic, some long-established norms of the health care therapy consisting of donor-derived mesenchymal
journey are being reconsidered. In particular, the delivery stems cell for treating ventilated patients with
and payment models for nursing and palliative care are being moderate to severe COVID-19 acute respiratory
reconsidered in reaction to the harm caused when nursing distress syndrome (ARDS).
home residents were separated from their families during the f Remestemcel-L purportedly modulates the body’s
peak of the pandemic and the emerging need for ongoing immune response to offset the cytokine storm by
care for patients with long COVID. Shifts toward in-home care suppressing pro-inflammatory cytokine production,
and insurance coverage without a hospice requirement will increasing anti-inflammatory cytokine production,
greatly change the experience of patients and those who care and facilitating the release of naturally occurring anti-
for them. inflammatory cells to involved tissues.
The current US administration is looking at partnering f Remestemcel-L was granted Fast Track designation
with dollar stores and fast-food restaurants to reach both by the US Food and Drug Administration (FDA) and
the vaccine-hesitant and those who live far from current is in phase 3 development in ventilator-dependent
vaccination sites. A recent study found that more than 90% of COVID-19 patients. In December 2020, a Data Safety
the US population would be within 5 miles of a vaccination site if Monitoring Board determined that the trial was unlikely
Dollar General stores were added to the network. to meet its primary end point of reducing mortality and
As global cases rise, vaccine uptake levels increase to 58.9% recommended that the trial stop enrollment.
of US adults receiving at least 1 dose, and US public health f In April 2021, remestemcel-L’s manufacturer released
constraints are relaxed, there is still a need for new testing 60-day results from the 223 patients enrolled before the
programs and better treatment options for younger patients trial was halted. In a prespecified subgroup of patients
(see Topics to Watch). younger than 65 years, remestemcel-L reduced the risk
of death by 46%, and among patients younger than 65
Topics to Watch who were also taking dexamethasone, remestemcel-L
reduced the risk of death by 75%. The number of days
ECRI Horizon Scanning has selected the topics below as those
hospitalized was reduced and the number of days off
with potential for impact relative to COVID-19 in the United
mechanical ventilation was increased.
States within the next 12 months. All views presented are
preliminary and based on readily available information at the f The manufacturer announced plans to discuss next
time of writing. Because these topics are rapidly developing, we steps with the FDA.
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 Category: Treatments
and have not been vetted by other stakeholders. Topics are listed Areas of potential impact: Patient outcomes, health care
in alphabetical order. delivery and process, health care disparities, health care costs
RESEARCH DONE DIFFERENTLY®
Description: Remestemcel-L (Ryoncil) is an investigational Commentary: Remestemcel-L might improve clinical
cell therapy consisting of donor-derived (ie, allogeneic) outcomes by helping the body curb the cytokine storm
mesenchymal stem cells. It is intended to treat moderate to that often occurs in patients with severe COVID-19 and
severe COVID-19 ARDS, a form of severe respiratory failure contributes to poor outcomes.
that is characterized by inflammatory injury and impaired Early feedback from ECRI internal stakeholders suggested
oxygen exchange. Remestemcel-L purportedly modulates the that remestemcel-L might provide a novel treatment
body’s immune response to dampen the ARDS cytokine storm for patients with COVID-19–induced ARDS, because of
by suppressing pro-inflammatory cytokines, increasing anti- its immunomodulatory and anti-inflammatory actions.
inflammatory cytokines, and facilitating the release of anti- Stakeholders expressed concerns about the failure of
inflammatory cells to involved tissues. Remestemcel-L to reach its death-risk end points in the
Remestemcel-L is in phase 3 development among ventilator- initial cohort of patients. However, the results shown in
dependent COVID-19 patients. The treatment was granted a prespecified subgroup analysis found remestemcel-L
Fast Track designation by the FDA. In December 2020, a Data in combination with standard of care (dexamethasone)
Safety Monitoring Board determined that the phase 3 trial was significantly reduced death rates in patients younger than
unlikely to meet its primary end point of reducing mortality and 65 years.
recommended that the trial stop enrollment. Stakeholders thought that, because many COVID-19 patients
However, in April 2021, remestemcel-L’s manufacturer, currently hospitalized fall into this age group, the treatment
Mesoblast (Melbourne, Australia), released 60-day results on the might have a substantial impact on patient health outcomes.
223 patients who were enrolled before the trial was halted. In The treatment might substantially reduce the burden on
a prespecified subgroup of patients younger than 65 years, hospital resources and costs if the length of hospital stays is
remestemcel-L reduced the risk of death by 46%. Additionally, reduced and fewer intensive resources are needed. However,
among patients younger than 65 years who were also taking stakeholders were also concerned that, if expensive,
dexamethasone, remestemcel-L reduced the risk of death by treatment with remestemcel-L might increase disparities for
75%. The number of days hospitalized was reduced and the individuals in need.
number of days off mechanical ventilation was increased.
But death risk was not reduced in patients aged 65 years or
older. The company announced plans to discuss next steps
with the FDA.
Say Yes! COVID Test Initiative to Reduce AT A GLANCE
the Spread of COVID-19
f The Say Yes! COVID Test Initiative is a cooperative
Categories: Systems and management effort among local, state, and federal health
Areas of potential impact: Patient outcomes, population authorities to evaluate whether frequent self-
health, clinician and/or caregiver safety, health care delivery administered testing can limit the spread of
and process, health care disparities COVID-19 in communities.
Description: The Say Yes! COVID Test Initiative is a f Federal agencies are providing about 2 million
cooperative effort among state and local health departments, rapid antigen COVID-19 tests to as many as
the National Institutes of Health (NIH), and the Centers for 160 000 residents of Pitt County, North Carolina, and
Disease Control and Prevention (CDC) to evaluate whether Hamilton County, Tennessee. Up to 2 unvaccinated
frequent self-testing can reduce COVID-19 spread in members of each participating household, those
communities. with the most public contact, will give themselves a
COVID-19 test 3 times a week for about 1 month.
For the initiative, NIH and CDC are providing about 2 million
rapid antigen COVID-19 tests (the QuickVue At-home test, f A digital assistant will guide the participants through
Quidel Corp, San Diego, California) to as many as 160 000 the testing process and give reminders.
residents in the communities of Greenville and Pitt County, f The initiative seeks to determine the effects of
North Carolina, and Chattanooga and Hamilton County, frequent self-testing on the knowledge, attitude, and
Tennessee. Up to 2 members (aged 8 years or older) of each behaviors of people regarding hygiene practices and
participating household will test themselves 3 times a week vaccination, as well as the effects of frequent testing
for about 1 month. Testing should be prioritized to household on infection and death rates.
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.
2 Biweekly COVID-19 Scan | Volume 1, Issue 26members with the most contact outside of the home, who are community transmission of SARS-CoV-2 by increasing
not vaccinated, or have not previously contracted COVID-19. awareness and mindfulness of infection risk in unvaccinated
Participants will have access to a digital assistant to guide them people living in multiperson households.
through the testing process and to set testing reminders. Early feedback from ECRI internal stakeholders suggested
The study will monitor COVID-19 infection and death rates that the Say Yes! COVID Test Initiative is a novel and welcome
in the participating geographic areas and compare them to program. It might reduce COVID-19 incidence in targeted
rates observed in similar, nonparticipating geographic areas. communities of unvaccinated people with barriers or
Participants will also have the option to voluntarily enroll reluctance to seek COVID-19 vaccination, as well as in children
in an NIH-sponsored study that will use surveys to assess who are too young to seek COVID-19 vaccination.
whether frequent self-testing has led to changes in behavior Earlier detection of symptomatic and asymptomatic
to prevent COVID-19 spread and has helped participants people with high exposure to the public who live with other
gain knowledge about preventing viral spread and receiving household members might substantially improve population
vaccination shots. health outcomes in COVID-19 hotspots. By increasing
Commentary: The Say Yes! COVID Test Initiative might awareness and education of SARS-CoV-2 infection risk among
reduce community transmission of COVID-19 and improve unvaccinated individuals, the program might also increase
population and health outcomes. Reducing the time between vaccine acceptance. However, stakeholders question the
individuals’ infection with SARS-CoV-2 and their awareness financial sustainability of such testing programs if they do not
that they are infected would allow quarantine protocols or increase vaccine uptake over time.
care to be started sooner. The program might also reduce
About Horizon Scanning strategies, and systems changes with potential for high impact
to patient outcomes—for individuals and populations—in the
Horizon scanning is a systematic process that serves as
United States in the next 12 months.
an early warning system to inform decision makers about
possible future opportunities and threats. Health care horizon The HCHSS COVID-19 supplement produced 3 main outputs:
scanning identifies technologies, innovations, and trends • Biweekly COVID-19 Scans (eg, this document) to provide
with potential to cause future shifts or disruptions—positive ECRI Horizon Scanning with a vehicle to inform PCORI and
or negative—in areas such as access to care, care delivery the public in a timely manner of important topics of interest
processes, care setting, costs of care, current treatment identified during ongoing scanning and topic identification
models or paradigms, health disparities, health care or through the ECRI stakeholder survey process.
infrastructure, public health, and patient health outcomes.
• Status Reports (quarterly) briefly listed and described
The PCORI Health Care Horizon Scanning System (HCHSS) all COVID-19–related topics identified, monitored, and
conducts horizon scanning to better inform research recently archived.
investments at the Patient-Centered Outcomes Research
Institute (PCORI). Initially, PCORI defined the HCHSS project • High Impact Reports (every 4 months) highlighted those
scope to focus on interventions with high potential for topics that ECRI internal stakeholders (eg, physicians,
disruption in the United States in 5 priority areas: Alzheimer’s nurses, allied health professionals, public health
disease and other dementias, cancer, cardiovascular diseases, professionals, first responders, health systems experts,
mental and behavioral health conditions, and rare diseases. clinical engineers, researchers, business and finance
In addition, the system captures high-level disruptive trends professionals, and information technology professionals)
across all clinical areas, which may lead PCORI to expand the had identified as having potential for high impact relative
project scope to include other priority areas in the future. to COVID-19 in the United States.
In early 2020, the COVID-19 pandemic created a fast-moving, In May 2021, the COVID-19 supplement was modified and
widespread public health crisis. In May 2020, PCORI expanded merged with the HCHSS. The Biweekly COVID-19 scans
its HCHSS to elucidate the landscape of potentially impactful continue, while the Status Reports and High Impact Reports
applications for COVID-19. Under a 1-year contract, the HCHSS will be folded into HCHSS Status Reports and High Potential
COVID-19 supplement scanned for, identified, monitored, Disruption Reports.
and reported on emerging and available COVID-19–related We welcome comments on this document. Send comments
treatments, diagnostics, preventive measures, management by email to horizonscan@pcori.org.
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.
3 Biweekly COVID-19 Scan | Volume 1, Issue 26About This Report Financial Disclosure Statement
The PCORI Health Care Horizon Scanning System is operated None of the individuals compiling this information has any
by ECRI under contract to PCORI, Washington, DC (Contract affiliations or financial involvement that conflict with the
No. MSA-HORIZSCAN-ECRI-ENG-2018.7.12). The findings and material presented in this report.
conclusions in this document are those of the authors, who All statements, findings, and conclusions in this publication
are responsible for its content. No statement in this report are solely those of the authors and do not necessarily
should be construed as an official position of PCORI. represent the views of PCORI or its Board of Governors. This
A representative from PCORI served as a contracting officer’s publication was developed through a contract to support
technical representative and provided input during the PCORI’s work. Questions or comments may be sent to
implementation of the horizon scanning system. PCORI does PCORI by email at info@pcori.org or by mail to Suite 900,
not directly participate in horizon scanning or assessing leads 1828 L Street, NW, Washington, DC 20036. ©2021 Patient-
or topics and did not provide opinions regarding potential Centered Outcomes Research Institute. For more information,
impact of interventions. see www.pcori.org.
Public Domain Notice
This document is in the public domain and may be used and
reprinted without special permission. Citation of the source is
appreciated.
Suggested citation: Lynch M, De Lurio J, Cuevas C, et al. PCORI
Health Care Horizon Scanning System: Biweekly COVID-19
Scan. Washington, DC: Patient-Centered Outcomes Research
Institute; May 14, 2021. Prepared by ECRI under Contract No.
MSA-HORIZSCAN-ECRI-ENG-2018.7.12.
Prepared for:
Patient-Centered Outcomes Research Institute
1828 L St., NW, Suite 900, Washington, DC 20036
Phone: 202-827-7700 | Fax: 202-355-9558 | www.pcori.org
Contract No. MSA-HORIZSCAN-ECRI-ENG-2018.7.12
Prepared by:
ECRI
5200 Butler Pike, Plymouth Meeting, PA 19462
Phone: 610-825-6000 | Fax: 610-834-1275 | www.ecri.org
Investigators
Marcus Lynch, PhD, MBA Christian Cuevas, PhD Randy Hulshizer, MA, MS
Jennifer De Lurio, MS Prital Patel, MPH Andrew Furman, MD, MMM, FACEP
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.
WWW.PCORI.ORG | INFO@PCORI.ORG | FOLLOW US @PCORI © 2021 Patient-Centered Outcomes Research Institute. All Rights Reserved.You can also read
