Drs Michos and Baum provide payer considerations regarding HoFH treatment pathways.
Erin Michos, MD, MHS: I would make it really streamlined. If a patient is being seen in an established lipid clinic and a lipid specialist documents that the patient meets the criteria for homozygous familial hypercholesterolemia [HoFH], and you have a blood test that shows their LDL [low-density lipoprotein] is high, it shouldn’t be so hard with so much back and forth. It should be streamlined so that we can start therapy as soon as possible and without delay, again, because these therapies can be lifesaving when you’re talking about a condition that can cause atherosclerotic cardiovascular disease before the age of 20 and even cause mortality before the age of 30 if not treated. So it’s important to start treatment right away without delay.
Seth Baum, MD: I wish the payers would understand…. This is going to sound like a dream, but I wish the payers would understand that those of us who treat these very sick patients are not just haphazardly throwing therapeutics at them. We understand unequivocally the relationship between LDL cholesterol and cardiovascular disease and cardiovascular events. This truly is settled science. There’s a direct relationship. The higher the LDL, the greater risk of atherosclerosis, and the greater the risk of cardiovascular events. We want to lower LDL to the best of our ability. All of these strategies are aimed at lowering LDL to levels that will reduce the risk of these patients who are suffering so much, reduce their risk of having heart attacks, strokes, peripheral arterial disease, shortened life spans, and diminished quality of life.
There are not that many [patients with HoFH]. After all, how many of these patients are there in the United States, 1300 or so; 1 in 250,000 people in the United States has HoFH. We’re not looking to treat everyone with very costly therapeutics. We’re looking only to treat those people who really need the medication and the therapeutics such as apheresis.
Transcript edited for clarity.