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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.

The Third International Summit on Human Genome Editing, held March 6-8, 2023, concluded with a statement highlighting the potential of somatic human genome editing while calling out the need for investment in equitable access. The meeting showcased Victoria Gray’s remarkable recovery from sickle cell disease resulting from treatment with exagamglogene autotemcel (exa-cel, formerly CTX001), the CRISPR-enabled gene-edited stem cell therapy (see the PCORI Horizon Scanning Database for another gene therapy in development for sickle cell disease).

In COVID-19 developments, a recent study of metformin is raising hopes for possible prevention of post-COVID conditions (PCC or long COVID). The trial results (released as a preprint by The Lancet) show that metformin started within four days of COVID-19 symptoms and taken over two weeks reduced the relative risk of long COVID by 42 percent compared with placebo. Ivermectin and fluvoxamine, also tested in this quadruple-blind, parallel-group randomized trial, showed no statistical difference.

Prevention of long COVID is vital to both individuals and the health system. Studies continue to document the long-term troubles that arise from PCC—most recently, gastrointestinal and cardiovascular (see Topics to Watch for a potential new treatment of PCC fatigue, heart palpitations, light-headedness, headache and nausea caused by a dysregulated heart rate).

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.

Apremilast (Otelzla) to Treat Alcohol Use Disorder

At a Glance

  • Alcohol use disorder (AUD) is a chronic condition characterized by excessive and compulsive alcohol consumption. Current AUD treatment options are of limited effectiveness and novel medications are needed to improve patient outcomes.
  • Phosphodiesterase-4 (PDE4) is a crucial enzymatic regulator of cyclic adenosine monophosphate signaling, which is believed to be heavily involved in the rewarding properties of alcohol. Some genetic variants of PDE4 are linked to predisposition for developing AUD.
  • Apremilast (Otezla) is an inhibitor of PDE4 already approved by the Food and Drug Administration to treat certain types of psoriasis and Behçet’s disease and is now being investigated for AUD.
  • In January 2023, data from a phase 2, randomized placebo-controlled trial reported that patients with AUD who received apremilast reduced their number of drinks per day by roughly half from baseline.
  • Apremilast to treat AUD might cost about $6,900 for a 30-day supply. However, if approved by a patient's health plan, apremilast's direct cost to patients may be mitigated by the manufacturer’s copayment assistance program.

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

Efgartigimod (Vyvgart) to Treat Post-COVID Postural Orthostatic Tachycardia Syndrome

At a Glance

  • Postural orthostatic tachycardia syndrome (POTS) is a disorder of the autonomic nervous system characterized by a sustained increase in heart rate upon standing, difficulty remaining upright, fatigue and cognitive impairment (e.g., brain fog).
  • Research suggests POTS may be autoimmune in origin and COVID-19 might trigger production of autoantibodies that persist after acute infection, leading to inflammation and tissue damage. No treatments are approved for POTS. Symptoms are managed with lifestyle modifications and medications used off label.
  • Efgartigimod (Vyvgart) is an immunoglobin G antibody fragment designed to inhibit the function of neonatal Fc receptor, which normally protects circulating antibodies from degradation.
  • An ongoing phase 2 randomized placebo-controlled trial is investigating efgartigimod to treat adults who have post-COVID POTS. Interim data are anticipated in the fourth quarter of 2023.
  • Based on the cost of efgartigimod to treat myasthenia gravis, treatment of post-COVID POTS could cost from $12,400 to $14,496 per week.

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: March 24, 2023

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