Download this Innovation Brief


Highlights provide a timely synopsis of selected interesting developments emerging in the last two weeks from the information universe covered by the PCORI Health Care Horizon Scanning System (HCHSS). Information covers currently emerging innovations in patient-centered care that may or may not be directly related to the Topics to Watch. The views presented here are solely those of ECRI Horizon Scanning and have not been vetted by other stakeholders.

Since May 2020, the Biweekly COVID-19 Scans have highlighted key developments related to the COVID-19 pandemic. However, we continued to gather information on potentially disruptive developments across the PCORI HCHSS’s 5 other focus areas (i.e., Alzheimer’s disease and other dementias, cancer, cardiovascular diseases, mental and behavioral health conditions, rare diseases) and high-level trends cutting across multiple focus areas.

The COVID-19 pandemic has caused impacts and disruptions across the entire health care system (e.g., hospital wait times for many conditions are longer); therefore, PCORI has decided to expand the scope of this brief to include all horizon scanning focus areas, including infectious diseases and COVID-19. Below, we include a few highlights from the past 2 weeks.

Two disease–modifying drugs for Alzheimer’s disease are on the horizon for patients: aducanumab (Aduhelm, approved in June 2021), and lecanemab-irmb (Leqembi, see Topics to Watch). Aducanumab has faced scrutiny for worrying side effects and, most recently, a congressional investigation’s conclusion of an “unjustifiably high price.” The accelerated approval for lecanemab-irmb has been met with a call to convene an advisory committee in light of 3 patient deaths during clinical trials.

COVID-19 vaccines are again in the news with differing expert opinions on the value of bivalent boosters, a look at what it might take to develop next-generation vaccines, and a study that suggests cash incentives for vaccinations do not have negative consequences. Meanwhile, the need for treatment remains high with steady case rates (see Topics to Watch for a new treatment for higher-risk outpatients).

Topics to Watch

ECRI Horizon Scanning has selected the topics below as those with potential for impact within the PCORI HCHSS’s focus areas in the United States within the next 3 years. 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.

Lecanemab-irmb (Leqembi) to Treat Alzheimer’s Disease

At a Glance

  • Alzheimer’s disease (AD), the most common cause of dementia, is a progressive neurodegenerative disorder characterized by gradual, significant decline in cognitive function, and increasing difficulty performing activities of daily living (ADLs).
  • Many researchers think that abnormal accumulations of amyloid-beta (Aβ) protein in the brain are toxic to neurons and are key contributors to AD pathology. No cure exists for AD, although some therapies might temporarily slow the worsening of dementia symptoms. A serious unmet need exists for effective treatments.
  • Lecanemab-irmb (Leqembi) is an antibody designed to bind, neutralize, and eliminate a type of pathogenic Aβ aggregate, called protofibrils, which might slow AD progression.
  • In January 2023, the FDA granted lecanemab-irmb accelerated approval to treat early AD based on surrogate effectiveness measures in a phase 2 trial enrolling 856 patients with early AD. Confirmation of clinical benefit and full approval are pending.
  • Treatment is estimated to cost $26,500 per year.

For a description and commentary about this topic/issue, download this Innovation Brief.

RHB-107 (upamostat) to Treat Symptomatic COVID-19 Outpatients

At a Glance

  • An unmet need exists for treatments to prevent patients at higher-risk from progressing to severe COVID-19, in part due to the potential for new SARS-CoV-2 spike variants.
  • Studies suggest that processing of SARS-CoV-2 by certain enzymes (proteases) present in epithelial cells, such as those lining the respiratory tract, play a key role in infection.
  • RHB-107 (upamostat) is a novel, oral antiviral therapy designed to inhibit proteases thought to mediate SARS-CoV-2 entry into host cells. By targeting the host cell factors, the manufacturer suspects RHB-107 might be more likely to retain effectiveness as new variants emerge.
  • Early data from a phase 2 clinical trial (carried out before the Omicron variant emergence) enrolling 61 outpatients with symptomatic COVID-19 suggested that RHB-107 was well tolerated and might reduce the development of new symptoms, hospitalizations, and time to recovery.
  • To further evaluate benefits of RHB-107 the developer is negotiating its inclusion in a multinational platform trial, and planning an independent phase 3 trial.

For a description and commentary about this topic/issue, download this Innovation Brief.

Back to Main Page Download this Innovation Brief

We welcome your comments on this Innovation Brief at [email protected].

Posted: January 26, 2023

What's Happening at PCORI?

The Patient-Centered Outcomes Research Institute sends weekly emails about opportunities to apply for funding, newly funded research studies and engagement projects, results of our funded research, webinars, and other new information posted on our site.

Subscribe to PCORI Emails


Hand pointing to email icon