Evaluating a New Treatment for Hypertrophic Obstructive Cardiomyopathy

Patients with hypertrophic obstructive cardiomyopathy (HOCM) who received mavacamten showed meaningful improvements in patient-reported outcomes in a randomized clinical trial, according to a recently published commentary.

“Patients with HOCM are on the verge of adding another arrow in their quiver in managing their disease state that has demonstrated not only clinical benefits but also improvements in quality of life,” wrote Patty Taddei-Allen, PharmD, MBA, BCACP, BCGP, of WellDyne in Lakeland, Florida.

In the report, Dr Taddei-Allen urged managed care pharmacists against inadvertently limiting access to treatment by failing to consider the complexity of HOCM.

“Managed care pharmacists should review the ICER study and understand the difficulty in assessing not only an interheterogeneous but also intraheterogeneous disease,” she advised. “This heterogeneity should be considered when developing coverage policies not only for mavacamten but also for other treatment-related policies.”

Because no medications are specifically indicated for the treatment of HOCM, many patients are prescribed beta-blockers and calcium channel blockers as first-line therapy for symptoms. Despite a lack of high-quality evidence, disopyramide is sometimes also prescribed to patients who need added control, but supply chain issues have affected its availability.

The EXPLORER randomized controlled trial evaluated mavacamten, compared with placebo, added to first-line treatment. Patients with HOCM showed significant improvement on patient-reported outcomes as measured by 2 scales: the Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score and the Hypertrophic Cardiomyopathy Symptom Questionnaire Shortness-of-Breath subscore, according to the commentary. After a washout period, scores reverted to baseline on the Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score for patients who previously had received mavacamten.

“When developing coverage criteria, it is clinically reasonable to place mavacamten as second-line after beta-blockers and calcium channel blockers but not necessarily to also require the use of disopyramide,” Dr Taddei-Allen wrote.

Reference:
Taddei-Allen P. Considerations for managed care pharmacy in evaluating mavacamten, a novel agent for obstructive hypertrophic cardiomyopathy. J Manag Care Spec Pharm. 2022;28(3):376-378. doi:10.18553/jmcp.2022.28.3.376