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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.

As flu season approaches and the United States experiences another wave of COVID-19, the US Food and Drug Administration (FDA) approved 2 vaccines targeting the Omicron variant XBB.1.5 on September 11, 2023. Data reported earlier in September suggest the updated vaccines should generate a robust immune response to the newest circulating variant, BA.2.86.

In women’s health news, results of a large cohort study published on August 29, 2023, found that treating postpartum depression with selective serotonin reuptake inhibitors benefits both mothers and their children up to 5 years after birth. On the same day, the first over-the-counter at-home menopause stage indicator was launched to help women determine their likely menopause stage.

On August 28, 2023, the FDA approved the first generic versions of lisdexamfetamine dimesylate (Vyvanse) to treat attention-deficit/hyperactivity disorder (ADHD) and binge-eating disorder. This may potentially ease the ongoing supply shortage of ADHD drugs. Furthering improved access to care, naloxone nasal spray (Narcan) is now available over-the-counter for opioid overdose reversal, and injectable buprenorphine (Sublocade) can be administered at certain retail pharmacies for patients with opioid use disorders.

Under the Inflation Reduction Act, the Centers for Medicare and Medicaid Services selected for Medicare price negotiations 10 drugs with high prescription costs to treat common conditions, potentially lowering drug costs starting in 2026.

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.

MCO-010 to Treat Stargardt Disease

At a Glance

  • Stargardt disease is a rare, inherited genetic disorder that causes progressive retinal degeneration, vision loss, and diminished quality of life.
  • No targeted treatments are approved for patients with Stargardt disease. Currently, specialists recommend patients use low-vision optical devices (eg, magnifiers) or assistive audio technologies (eg, screen readers, digital assistants) for everyday activities.
  • MCO-010 is an experimental gene therapy intended to improve vision by delivering a gene encoding an engineered ambient light–sensitive protein, called multicharacteristic opsin, to a patient’s remaining retinal cells.
  • Results from a phase 2a, open-label trial of 6 patients with Stargardt disease found that MCO-010 injected into the eye improved vision and light sensitivity outcomes, while maintaining the patient’s vision-guided mobility and object recognition.
  • The developer announced plans to present detailed results at an upcoming medical conference.

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

RLS-0071 to Treat Hypoxic-Ischemic Encephalopathy

At a Glance

  • Hypoxic-ischemic encephalopathy (HIE) is a type of brain injury that occurs due to a lack of blood flow and oxygen to the brain at birth.
  • HIE causes significant health issues, neurodevelopmental disabilities, and neonatal death. An unmet need exists for treatments to complement standard-of-care therapeutic (induced) hypothermia.
  • RLS-0071 is an experimental anti-inflammatory and antioxidant peptide designed to limit cell damage and death due to HIE. Preclinical data and preliminary studies in healthy adult volunteers suggest RLS-0071 might minimize brain injury, which could improve long-term cognitive outcomes.
  • Recently, the first patient was dosed in an ongoing placebo-controlled phase 2 clinical trial investigating the safety and efficacy of intravenous RLS-0071, in addition to therapeutic hypothermia, to treat newborns with moderate to severe HIE.
  • Topline data are expected in the second quarter of 2024.

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: September 22, 2023

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