What Should Mavacamten Cost?

On October 22, patients, physicians, and other interested parties will have the opportunity to provide input on the value and cost of mavacamten – the first drug specifically designed to treat hypertrophic cardiomyopathy. Last year, Bristol Myers Squibb paid $13.1 billion to acquire MyoKardia, the San Francisco biotech company that developed the drug and brought it through clinical trials.

At a virtual public meeting, The Institute for Clinical and Economic Review or ICER will listen to further testimony in order to evaluate mavacamten’s value and potential benefitsICER is a non-profit organization that evaluates the cost effectiveness of drugs and medical procedures. Many insurance companies rely on ICER’s findings when deciding how much to pay for a certain treatment or test. 

In an Effectiveness Report which was published today, ICER valued the benefit that mavacamten would bring to a patient at between $12,000 to $15,000 a year. By contrast, some analysts have suggested that mavacamten could carry a price tag as high as $75,000 per patient per year.

 If you would like to share your thoughts at the online public meeting click here to sign up.  

You can find a press release from ICER about their review of mavacamten here.

HCM Patient Competes on Television Show “The Voice”

Berritt Haynes, a 19 year old with hypertrophic cardiomyopathy, was chosen for Team Blake Shelton last night on the MGM Television/NBC show THE VOICE.  After Berritt’s mother submitted a tape, Berritt was chosen to audition on stage in front of the coaches.

Berritt had hoped to attend a taping of the show last season through Make-A -Wish Foundation which grants wishes to kids with serious health issues. However, due to COVID, he was not able to make that happen.  Instead, this year his mother helped make his dream come true by making it possible for him to actually perform.

Berritt was diagnosed with HCM when he was 8 and received an implantable defibrillator when he was 14.  Clearly, Berritt’s HCM has not interfered with his performing talents.  Watch him performing on The Voice here.

Good luck Berritt. All of the HCM world will be rooting for you to advance to the next round!

Read more at:

Yahoo Entertainment

USA Today

ET Canada

THE VOICE can be seen on NBC on Monday and Tuesday nights. Check your local schedule for times.

Women and Babies Get HCM Too

Recent HCM research has taken a much needed pivot away from a focus on adult men to the exclusion of others. Women and babies get HCM too, yet until this point, there has been far less attention and research on their issues. HCM is not just a disease of adult men, though they do make up the majority of participants in research studies which inform current treatment protocols.

Finally that may be changing.  It seems that people are finally starting to pay some attention to other folks who have HCM.  Here is a recent European article looking at HCM in infants  and here is a recent article looking at HCM in women. 

Let’s hope the trend continues since previous research has shown that HCM is often more severe in these two groups. 

This past article on HCMBeat takes a look at the more severe clinical course of HCM in women while this past article discusses the severity of HCM in children.

New MRI Technology Spots Scar Tissue Using AI

A new technology called “virtual native enhancement” (VNE) may soon eliminate the need for gadolinium as a contrast agent for patients with hypertrophic cardiomyopathy undergoing cardiac MRI. Gadolinium, a heavy metal contrast agent which is injected intravenously, has long been used in cardiac MRI scans to spot cardiac scar tissue in patients with HCM.  In 2017, the FDA issued a safety communication relating to gadolinium  because it was found that gadolinium remains in the body for months to years after the use of the drug.

The new VNE technology, recently described in the journal Circulation, uses artificial intelligence (AI) to virtually enhance the standard MRI image. The technology was developed using data taken from 1348 HCM patients and was validated in the HCM population, but the technology may have uses extending beyond HCM.

By avoiding the use of the contrast agent, this technology avoids side effects and long term consequences from the use of gadolinium. Additionally, it will make cardiac MRI available to patients who are allergic to gadolinium. VNE is also faster and cheaper that current technology used for cardiac MRI, which may make more frequent MRI monitoring of patients feasible.  

To read more about VNE, see also this article in UVA Today, this article in ACM Tech News,  this article in Engineering and Technology, and this article in Science Daily.

VANISH Trial: Valsartan Shows Positive Results for Early HCM

A common cardiac drug called valsartan has shown positive results in those who carry the gene for hypertrophic cardiomyopathy but who do not have overt disease. 

As reported by Dr. Carolyn Ho of Brigham and Women’s Hospital and Harvard Medical School at the online European Society of Cardiology meeting, the Phase 2 double blind VANISH trial looked at 178 pre-symptomatic young people who carried a sarcomere gene mutation known to cause HCM.  These patients were randomly assigned to take either  valsartan, an angiotensin II receptor blocker (ARB), or a placebo.  

At the time of trial enrollment, these patients showed no or only mild signs of the disease.  At the end of the two year trial, the individuals who took valsartan had a better overall cardiac picture compared to the group taking only the placebo.

Dr. Ho had this to say in a story about the trial results in Mirage News:  

“Valsartan improved cardiac structure/function and remodeling in patients with early stage sarcomeric HCM, suggesting that this strategy may help prevent disease progression among those who have received a genetic diagnosis of HCM.”  

“Our results suggest that valsartan may not only stabilize disease progression but may also promote improvement.” 

You can also read this summary of the presentation on MedPage Today.

Artificial Intelligence Identifies HCM in Children and Adolescents

A study by researchers from Mayo Clinic recently published in the International Journal of Cardiology found that a deep learning artificial intelligence algorithm using a standard 12 lead electrocardiogram was able to detect hypertrophic cardiomyopathy in young people with impressive accuracy. This accuracy was particularly strong among adolescents aged 15 – 18.  

Mayo has been looking at artificial intelligence for its potential to screen populations for HCM for some time now.  Here is a previous HCMBeat story about Mayo’s work on artificial intelligence from February 2020. 

 

 

 

ACC Focuses on HCM with Cover Story

The July edition of the the American College of Cardiology’s magazine features a cover story about HCM. 

The article surveys the history of HCM and then moves through the evolution into contemporary treatments.  The article also contains a summary of important topics from the new 2020 AHA/ACC Guidelines and highlights EXPLORER-HCM, the recent groundbreaking clinical trial of mavacamten

Check it out when you can.

Mayo Clinic’s Dr. Steve Ommen Talks About HCM Care

Watch the video or read the transcript of Dr. Steve Ommen’s recent interview on Medscape.

In this interview, he discusses the recent AHA/ACC treatment guidelines for hypertrophic cardiomyopathy (HCM), his thoughts about the new HCM drug mavacamten, and the importance of collaboration between your local care team and the team at a HCM specialty center.

Reduced Cardiac Perfusion in HCM Gene Carriers

Researchers in the U.K. have found that 20% of HCM gene carriers who do not show overt signs of HCM do show reduced blood flow to cardiac tissue.  

Although the gene positive individuals lacked the characteristic left ventricular wall thickening of HCM, 1 of 5 patients who carried the HCM gene showed marked regional perfusion defects when compared to healthy individuals. Hence, the researchers concluded that a person who is gene positive for the disease may show reduced cardiac perfusion before they develop hypertrophy.

The study compared 50 patients who carried the HCM gene but had no signs of left ventricular hypertrophy to 28 healthy individuals. Both groups underwent Cardiac MRI testing. 

The researchers theorize that perfusion mapping may be a useful way to identify HCM gene carriers who will go on to develop the disease.

To read about more early signs of HCM click here and to read the findings of another study describing reduced cardiac volume in gene positive people, click here.

Scientists Seeking Patient Input: Gene Therapy to Treat Inherited Cardiomyopathies

A group of scientists working on gene therapy for inherited cardiomyopathies are seeking input from patients about their interest and willingness to participate in gene therapy trials. These researchers hope that treatment with gene therapy will ultimately prove to be a cure for hypertrophic cardiomyopathy as well as other genetic cardiomyopathies.

If you are interested in learning more about gene therapy and are willing to answer a few questions about your willingness to participate in this type of research, watch this video.  Then, fill out this short questionnaire.  It just takes a few minutes and you may help to find a cure for HCM.