ross • November 22, 2021
ICER Publishes Final Evidence Report and Policy Recommendations on Mavacamten for Hypertrophic Cardiomyopathy

ICER has released its evidence report and recommendations.

Here is a list of highlights:

  • All stakeholders have a responsibility to facilitate meaningful patient access to multidisciplinary centers of excellence for HCM in ways that do not exacerbate disparities.
  • The manufacturer of mavacamten should commit to sponsoring research that will address the lack of evidence on the comparative effectiveness of mavacamten versus disopyramide and septal reduction procedures.
  • The manufacturer should align the price of mavacamten with the explicit and transparent estimates of its treatment benefits for patients and families. Pricing should also be moderated to reflect the uncertainty about longer-term safety until such time as further outcomes data are generated.
  • Post-approval clinical registries should be established to detect rare side effects as well as assess the efficacy of mavacamten in more diverse populations.
  • Payers should use the FDA label as the guide to coverage policy and engage clinical experts and diverse patient representatives in considering how to address coverage issues for which there is limited or no evidence at the current time.

To read the entire report click here

HCMA Blog

April 30, 2025
Medical Management means using medicines and other non-invasive therapies to relieve symptoms. It's exciting that new treatments for HCM are being studied and that the FDA approved Camzyos in 2022! HCM can cause various symptoms depending on how stiff the heart is, how much scarring (fibrosis) there is, and whether there is obstruction. Some people have no noticeable symptoms. Common symptoms are chest pain/pressure, shortness of breath, lightheadedness, fatigue, palpitations, and brain fog. Syncope (fainting) is dramatic but not as common as the other symptoms. Medications are the first-line treatment for symptoms of HCM. If patients can't take one class of medication, they can try a medication from a different class. Here are some medications used to treat HCM and what they're for: Beta Blockers - make your heart beat slower and relax better. They allow the heart to fill more completely between beats. They can lower blood pressure and relieve symptoms. Calcium Channel Blockers - used to lower blood pressure and slow the heart rate. May reduce symptoms from obstruction in HCM. Myosin Inhibitors - cause the heart to beat with less force. They may reduce obstruction and septal thickness in some patients. Camzyos (mavacamten) is the first of this class of medication. For more information, click HERE . Antiarrhythmics - help to restore normal rhythm in the heart. Blood Thinners - anticoagulants and anti-platelets make it harder for blood to clot. They are prescribed to reduce the risk of stroke. This risk is higher in patients with afib, aflutter, and artificial heart valves. Diuretics , or "water pills," help the kidneys get rid of extra water and salt, reducing swelling in the body. Sodium Channel Blockers - may reduce gradient in obstructive HCM and can treat chest pain. Some also act as antiarrhythmics. Antibiotics - treat or prevent bacterial infections. In HCM, they may be given before dental work to prevent infective endocarditis. To learn more and see the side effects of these medications, click HERE .
On a green background, a line drawing of a head that has complicated arrows pointing outwards
By Gordon Fox April 25, 2025
Part 2 of a series on making decisions in HCM. A key reason is that decision making is so difficult is that most decisions involve more than one risk.
Hypertrophic Obsctructive Cardiomyopathy
By Erica Friedman April 22, 2025
Bristol Myers Squibb announces that Camzyos has been approved for Obstructive Hypertrophic Cardiomyopathy in Japan.
More Posts