Guest Blogger Gwen Mayes, JD, MMSc – Cautiously Awaiting the Release of Mavacamten

            Fifteen years ago, I referred myself to Mayo Clinic in Rochester, Minnesota, to be evaluated for a septal myectomy by what was at the time, one of the country’s few expert centers for the treatment of hypertrophic cardiomyopathy.   I traveled to Mayo from my native state, Kentucky, on a brief hiatus from a well-established career in health policy in Washington, DC.

            Having lived with HCM symptoms since birth in 1956, the early years of living with HCM meant living in the dark.  There was no internet, no HCMA, no social network, there were no women’s health networks and there was no concept of “shared decision making” where patient and provider individualize a treatment plan which considers the personal wishes and values of the patient.

Despite having a graduate degree in advanced cardio-respiratory care and access to the country’s top doctors, I lived in fear that any skipped beat would be the beginning of the end. 

            Now I am 65 and I have just returned home from my 5th evaluation at Mayo Clinic with the same outcome:  let’s wait and see.  This was a welcomed result because much has changed since my last visit in 2018. I’ve had five bouts of atrial fibrillation, changed local doctors, changed drugs for the first time in thirty years, been genetically tested, retired, enrolled in Medicare, changed my fitness routine, and aged. 

            But one thing about my recent visit to Mayo Clinic was new: my doctor and I discussed whether I would qualify for mavacamten; a first-in-class drug due to be approved by FDA in April 2022. As a HCM patient, I wish I could simply jump up and down for joy, pop the champagne, and look forward to the possibility of easing into old age without the need for surgery.  But my 40+ years in health policy and government affairs has awakened me to all that can stand between me and the care I desire.

            Simply put, when it comes to the availability of new drugs and everything that can pit patients against their health payers, I know too much.

            You see, I was the Director of Government Relations and Reimbursement for ABIOMED, a cardiac device manufacturer specializing in the world’s smallest heart assist device and artificial heart. For five years, I emmeshed myself in the maze that consists of government regulations, hospital codes, preferred providers, step therapy, and pharmacy benefit managers. It was my job to obtain new ICD-9 (now ICD-10) and CPT codes, decipher Explanation of Benefits, and untangle patients’ denied claims to enable them to get a device they not only needed but could afford.

            It was as messy and tough as you can imagine. The only industry I can compare this labyrinth of health insurance coverage and reimbursement to is the airline industry. The price of airline tickets can vary depending upon frequent flier miles, senior discounts, seat selection, promotions, black-out dates, the day you travel, holidays, and available flight attendants.  No two passengers pay the same price, nor does the price stay the same.

            I’m concerned something like this will happen with the pricing of mavacamten and there will be wide variability in who actually qualifies for mavacamten and who can afford it.   Here’s why:

  • Just because FDA approves a drug does not mean that a payer (i.e., a health insurance carrier) must pay for it. The FDA requires a drug to be ‘safe and effective’. This is a different standard than that used by a payer which requires the same drug to be ‘medically necessary.’ Medicare uses yet a third standard which is that the drug be both ‘reasonable and necessary.”
  • Just because a health payer ‘covers’ a drug and adds it to their formulary (list of drugs they cover) does not mean the drug will be fully paid for by the insurer. You may have a co-pay, co-deductible, or be required to try another drug first before you are prescribed mavacamten (this is called ‘step therapy’).
  • Initially, approval of mavacamten will only be for those patients with “obstructive” HCM. The patients who qualify for the drug at first will likely resemble those patients who were include in the EXPLORER-HCM clinical trial.
  • FDA’s approval will include a Risk Evaluation Mitigation Strategy (REMS) which is designed to help reduce the occurrence or severity of a serious adverse events. A REMS program can be required for certain medications with serious safety concerns to help ensure the benefits of the medication outweigh its risks. A REMS program is designed to ensure that prescribing, dispensing, administering, and monitoring of the drug all support the safe use of that medication.
  • The initial use of mavacamten is likely to be limited to high volume centers that have specific experience treating HCM. This may necessitate additional expenses (e.g., travel, telemedicine, home monitoring equipment) that add to the expense of the drug.
  • Earlier this year, the Institute for Clinical and Economic Review (ICER) performed a review of the clinical and economic benefit of mavacamten and recommended a price of $12,000-$15,000 annually. However, ICER refrained from commenting on the long-term cost-effectiveness of the drug. A price has not yet been set by the manufacturer, Bristol Myers Squibb (BMS). HCMA has stated publicly they believe the ICER review was premature.
  • Payers tend to have a ‘herd mentality,’ Coverage and reimbursement policies set by only a few can be leveraged to convince others to do the same. Hence, if one or more payers decide not to cover the drug or to require step therapy or other pre-requisites, others are likely to follow.
  • A Patient Assistance Program established by mavacamten’s manufacturer, Bristol Myers Squibb (BMS) may assist patients in covering the cost of the drug, but even so, only some patients may be eligible, and the program is not indefinite.

            The uncertainty surrounding the release and affordability of mavacamten is understandable. Historically, HCM patients have taken drugs already FDA approved and hence they have been spared the challenges of accessing a new drug. In the case of mavacamten, not only is it a new drug, but it’s also a first-in-class drug without another drug to which its price can be compared.

            The future of mavacamten gives HCM patients a much-needed treatment option. As patients, we need to understand as much as possible about how a drug goes from clinical trial to FDA approval, how it is placed on a health payer’s formulary, and then arrives in our medicine cabinet.  It is not going to be an easy journey, but I’m cautiously optimistic that in my lifetime, and for those after me, HCM can be more easily and predictably managed. 

 

About Gwen Mayes:

Gwen Mayes, JD, MMSc, is a lawyer and healthcare consultant who has lived with symptomatic HCM since birth.  She currently serves as a consultant to HCMA and as a patient representative on various national committees including the National Quality Forum, WomenHeart, and the Society to Improve Diagnosis in Medicine.  Visit her website at:  www.gwencohealth.com or you can contact her directly at: gwen@gwencohealth.com

 

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