Imagine this: You've been diagnosed with a rare genetic heart rhythm disorder that, left untreated, can cause serious symptoms such as syncope, seizures, and even sudden death. Luckily, early diagnosis by your cardiologist has led to the effective management of your condition with a simple once-a-day dose of medication. You've been assured that your risk of sudden death is extremely low due to this life-saving medication. But, standing at the pharmacy counter, you are told that this prescription is no longer covered by your insurance. What do you do?
Unfortunately, for patients with congenital long QT syndrome (LQTS) and catecholaminergic polymorphic ventricular tachycardia (CPVT), this scenario is becoming more and more common. Beta-blocker medication represents the therapeutic mainstay for the prevention of CPVT- and LQTS-triggered arrhythmias which can lead to sudden cardiac death. In the Windland Smith Rice Genetic Heart Rhythm Clinic and most of the largest LQTS/CPVT specialty centers throughout the world, the specific beta-blocker nadolol has been preferred and used for the past 27 years.1 However, this highly preferred and effective medication is being increasingly denied by insurance companies in favor of other beta-blockers such as metoprolol, atenolol, or propranolol.
This therapeutic paradigm is under threat in several countries, despite being recognized by channelopathy experts around the world, and the drug is at risk for disappearing from the market. The impact of this development could be profound.1
Michaela Saunders, the nurse in the Windland Smith Rice Genetic Heart Rhythm Clinic has experienced this first hand. "I recently spent two hours on the phone with an insurance company appealing a nadolol denial for a patient," she explained. "It's frustrating. This is a life-saving medication and there is not a better alternative medication for our patients to take."
Insurance companies often base their denials on the lack of concrete data supporting the use of nadolol versus other beta-blockers. Currently, there are no randomized clinical trials (RCTs) that compare the efficacy of different beta-blockers for the treatment of LQTS or CPVT. It is also unlikely that these studies will ever be performed because there is no driving incentive for a randomized trial for this class of medication to treat these uncommon disorders.1 Yet, this is a common problem among rare diseases. This explains how practice guideline development may accept expert opinion, small single-site studies, and registry data.1 It also shows the importance of looking to and relying on expert opinion and the experiences of LQTS/CPVT specialists for guidance.
Thus, there is substantial consensus among experts around the world that nadolol is the preferred effective drug therapy in LQTS and CPVT and should be administered as a first-choice therapy.1
The implications of not having access to nadolol for LQTS and CPVT patients would be substantial. The Heart Rhythm Society (HRS) is concerned that if nadolol becomes difficult to obtain or unavailable, patients with LQTS or CPVT will be at greater risk for sudden death, and that clinicians will be forced to consider otherwise unnecessary and more aggressive treatment options such as an implantable cardioverter-defibrillator (ICD).1
When pressed on what is advised if nadolol is simply not available, Dr. Michael J. Ackerman, director of the Mayo Clinic Windland Smith Rice Genetic Heart Rhythm Clinic, says he likes and prescribes propranolol and its extended release preparation (propranolol ER) a lot. However Dr. Ackerman highly urges physicians against other beta-blockers. In his own words, “please DO NOT prescribe either atenolol or metoprolol for patients with either CPVT or LQTS!”
Dr. Ackerman and the Windland Smith Rice Genetic Heart Rhythm Clinic believe this issue needs to be heard. Nadolol needs to continue to remain easily available for patients with LQTS and CPVT. It can literally be the difference between life and death.
Ackerman, M., Priori, S., Dubin, A., Kowey, P., Linker, N., Slotwiner, D., Triedman, J., Van Hare, G. and Gold, M. (2017). Beta-blocker therapy for long QT syndrome and catecholaminergic polymorphic ventricular tachycardia: Are all beta-blockers equivalent?. Heart Rhythm, 14(1), pp.e41-e44.