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Highlights

Highlights provide a timely synopsis of selected interesting developments emerging in the past 2 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.

The health care world reached several important milestones in the past 2 weeks.

First, the World Health Organization (WHO) stated that COVID-19 is now an established ongoing health issue and called an end to the COVID-19 PHEIC (public health emergency of international concern) on May 5. In the United States, May 11 brought the end of the federal COVID-19 PHE (public health emergency) declaration. COVID-19 fell from the third leading cause of death in 2021 to the fourth leading cause of death in the United States in 2022.

Second, the US Food and Drug Administration (FDA) made two milestone approvals in fields long followed by PCORI's Health Care Horizon Scanning System.

One, Seres Therapeutics’s Vowst (fecal microbiota spores, live-brpk; formerly SER-109), is the first fecal microbiota treatment in a pill. Vowst, now approved to prevent recurrence of the bacterial infection Clostridioides difficile, and the field of fecal microbiota treatment has had a slow and controversial emergence.

The other approval, for GlaxoSmithKline’s Arexvy (respiratory syncytial virus [RSV]vaccine, adjuvanted; formerly RSVPreF3 OA), is the first ever vaccine for RSV. Vaccines for RSV have also had a troubled emergence, but on an even longer timescale.

This Brief’s Topics to Watch focus on treating early Alzheimer’s disease, another area of health care that is experiencing milestone approvals after many years of failure.

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 three 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.

Donanemab (LY3002813) to Treat Early Alzheimer's Disease


At a Glance

  • Alzheimer’s disease (AD), the most common cause of dementia, causes declining cognitive function and ability to perform activities of daily living.
  • Abnormal, toxic accumulations of amyloid-beta (Aβ) protein in the brain, often called plaques, are thought to be key contributors to AD pathology.
  • Donanemab is a humanized monoclonal antibody designed to bind Aβ plaques and promote their clearance. It is intended to slow cognitive decline in patients with early AD.
  • Earlier this year, the US Food and Drug Administration rejected an application, supported by phase 2 clinical data, for accelerated approval for donanemab to treat early AD, citing insufficient safety data.
  • The developer expects definitive safety and efficacy data from an ongoing phase 3 clinical trial to support an application for traditional approval by mid-2023.

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

Valiltramiprosate (ALZ-801) to Treat Early Alzheimer's Disease


At a Glance

  • Alzheimer’s disease (AD), the most common cause of dementia, causes declining cognitive function and ability to perform activities of daily living.
  • No cure is available for AD, although some US Food and Drug Administration-approved therapies might temporarily slow the worsening of dementia symptoms. Safer, more effective, and more easily administrated therapies are needed.
  • Valiltramiprosate (ALZ-801) is an oral prodrug metabolized into tramiprosate, a compound that reportedly inhibits amyloid-beta (Aβ) aggregation, thought to contribute to AD pathology.
  • Recent clinical data suggest valiltramiprosate might safely reduce Aβ aggregation in patients with early AD who have certain genetic risk factors and are at higher risk of treatment-related complications (amyloid related imaging abnormalities).
  • If approved, valiltramiprosate would be the first oral, disease-modifying treatment for early AD.

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

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


Posted: May 19, 2023

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