Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Possible cardiac amyloidosis

Treatment: Vyndamax 61 mg OR CAPS

The insurer denied coverage for Vyndamax 61 mg OR CAPS

The denial is upheld
 

This is an adult with a past medical history which includes hypertension, hyperlipidemia, and transaminitis.

Recent testing, suggestive of cardiac amyloidosis, was noted. This included cardiac magnetic resonance imaging (MRI) which included findings of reduced left ventricle / right ventricle (LV/RV) systolic function, global hypokinesis, and global hypertrophy, with abnormal "T1, T2 and LGE (late gadolinium enhancement) in a classic pattern for amyloidosis. An echocardiogram supported those findings, with left ventricular ejection fraction of 43%, left ventricular hypertrophy, grade 3 LV diastolic dysfunction, and moderate mitral regurgitation. A nuclear study was "strongly suggestive of transthyretin cardiac amyloidosis,".

Vyndamax (tafamidis) is used in the treatment of hereditary transthyretin amyloidosis. Amyloidosis is a condition that involves the misfolding of proteins throughout the body, which become loaded with fibrils of this protein, interfering with the normal function of various organs. When the heart is involved, cardiac amyloidosis is diagnosed.

There are many variations of amyloidosis; those involving transthyretin (TTR) are given the designation ATTR. The hereditary form is prefixed with the letter h, as hATTR.

If hATTR amyloidosis is confirmed, then Vyndamax (tafamidis) is the appropriate treatment, and can reduce mortality, hospitalizations, and delay clinical decline.

However, in this case, while the diagnosis is strongly suspected, it has not been confirmed. There are multiple factors which clearly suggest that the diagnosis will be made - the echocardiogram, the nuclear test, and the MRI are all fairly "classic" or at least "strongly suggestive" of the condition, and the genetic testing confirms that there is a higher-than-average likelihood of the patient developing hATTR in his lifetime. 

At this time, Vyndamax is not clinical necessary for treating the patient. Based on the preponderance of the available evidence, it is reasonable to expect that this treatment is likely be proven to be clinically necessary for him in the near future. However, with the information currently available, the diagnosis has not been confirmed. Some clinicians might conclude that pre-test probability is high enough to warrant initiation of treatment at this point, but this needs to be balanced against the possibility of clinical harm, particularly if the diagnosis ultimately proves to be incorrect. The manufacturer's website indicates there are no untoward side effects, although other data indicate that diarrhea, urinary tract infection (UTI), and influenza were common.

In summary, treatment with Vyndamax was not yet necessary for the patient, and further evaluation is, clinically appropriate in this circumstance prior to initiating therapy.

Based on the above, the insurer's denial must be upheld. The health care plan did act reasonably and with sound medical judgment and in the best interest of the patient. 

The medical necessity for Vyndamax 61 mg OR CAPS services is not substantiated.