Access to New(er) Treatment Might Not Be As Cut-and-dry As You Might Expect

Disclosure: I just want to say, as usual: I do not recommend any of the drugs mentioned in this blog post. These are solely my own experiences, research, and conclusion at this time. Mine, and no one else's. No drug companies or medical facilities contributed to or are to benefit from the opinions listed here.

Also, I would like to mention that I am aware of the privilege I have to have options and choices for treatments. Millions others (without exaggeration) are not as fortunate as this.

As you might remember, back in May (http://livingwithfh.blogspot.com/2023/05/treating-heart-disease-haggling-style.html), my LDL cholesterol was 125 mg/dl. This is hardly “acceptable” for someone with my load of risk factors (previous heart surgery, heart attack, family history of strokes and heart attacks, and almost 20 years of coronary artery disease). My target LDL is 70 mg/dl at this time - lower than a person with no risk factors. 

But with FH, especially with Homozygous FH, it’s what you have to settle for, most often. Maybe you cannot ever get to your target levels, but you can reduce the untreated levels massively - as it happens in my case and just call it your best effort. Last time my cholesterol levels were measured without any medication, my LDL was 475 mg/dl. Coming down to the current 125 is clearly an achievement not to be contested, I think. 

But could we do better than this?! I have heard of first-hand reports from HoFH individuals that have tried the new drug Evkeeza (https://evkeeza.com/s/) that they managed to reduce their LDL down to as low as 50 mg/dl. Evkeeza is in the class of drugs called “ANGPTL3 inhibitors” and it reduces the levels of all three major cholesterol-related fractions (LDL, HDL, and triglycerides). There is still no proof that it does reduce the advancement of atherosclerosis or cardio-vascular disease, but from what we know about high lipids, reducing their levels normally triggers the reduction of atherosclerosis. But this is just a hypothesis at this time in the history of FH drugs. 

The drug is approved only for Homozygous FH, at this time, but given my diagnosis (https://livingwithfh.blogspot.com/2017/08/the-long-journey-to-hofh.html) of HoFH, this seems to fit the bill for my case. 

As you might also remember from the post back in May (linked above), I usually have to haggle with my cardiologist for the right treatment.  Evkeeza is a relatively new drug. It was approved by the FDA in February of 2021. I asked my cardiologist about it in July 2021. At that time, he did not know anything about it. I asked again last year and he came back with the same answer and only evasively agreed to look into it. He suggested I might be eligible for Leqvio (Inclisiran - https://www.leqvio.com/) which is a PCSK9 inhibitor (similar to Praluent or Repatha) but given in a different dosage and with a different frequency. If I were on Leqvio, I would need to stop the Praluent that I am on now. If I were on Evkeeza, from what I know from my own research, I would be able to add it to my current drug regimen (http://livingwithfh.blogspot.com/2016/07/my-current-drug-regimen-and-diet.html). This way, I would receive the lowering benefits of all the other medications I am on, since they have different mechanisms of action. 

I tried asking my doctor again this May (2023) if I could qualify for Evkeeza. At that time, he begrudgingly said “well, I guess I can look into it, but I have no idea how hard it would be to get your approved, and I don’t even know if you would get approved.” 

I asked him to explain: I have a genetic test that shows clearly I am a Homozygous FH patient; I have lots of risk factors proven by a long history of atherosclerotic events, I have the family history, I have a worsening heart (according to the stress test that he did last year there is increased damage to the heart muscle, possible from completely closed capillaries - he speculates), and I am on four different therapies for lowering cholesterol and my LDL is still not at target. 

I asked him how he make it that I would not get approved? 

He said something like (or asked, rather):  “do you have a written test result that shows that a medical institution confirmed the HoFH diagnosis?”. It took all of my might not to scream when I heard this question and not to literally slap him. 

But I like to give people the benefit of the doubt and acknowledge that I am not their only patient and they have a busy schedule and no time for research, but still. The genetic test was one of the first documents I showed him back in 2018 when we first met. Plus, he had asked me for the “written genetic test result” several times when his office got me approved for Praluent and Nexletol (these are not medicines approved only for HoFH but the accurate diagnosis was needed to get the approval in both cases). Both times, his office confirmed that they would scan this test result into my records so it would always be on file. Either that didn’t happen, or he doesn’t read my files? It’s anyone’s guess. 

So, I sent him a new copy of my genetic test. This was in May. He said he would “look into it” and I never heard anything since (three months later). I asked for an update a couple of weeks ago and I finally got a note from him a week later: “Looking into it.” 

So, as of right now, I don’t know whether I qualify for Evkeeza (I can only guess with a strong file built by someone who understands my disease I might), nor if it’s even recommended for me. He never so much as said that it might not be good for me, for one reason or another. All I know about this drug is what I have found in my own research. 

But it irks me to no end that a treatment option might have been out there for me, for 2 years now, and I am not taking advantage of it. Especially with my heart condition still worsening ... 

In this disease, time is truly of the essence and the older we get (as we all are), the harder it will be to stave off the advancement of artery disease when LDL cholesterol levels are outside the limits. That we know. 

But switching to another doctor is not that easy - not with a complex disease as mine (and I am sure many of you can relate). There are things “set” with the doctor that takes care of both your heart and your lipids. 

To name a few of the things that would need to be restarted with another physician if I were to walk away from this one (I think you’re tired of me by now reading here how dissatisfied I am with him and how many times I thought of changing him out with another doctor): 

• He manages my yearly heart tests (echo and nuclear stress test, mainly but he is also on the vascular team that manages my carotids and abdominal aorta too). I would have to start fresh with another doctor to explain the need for all the tests, the history, etc ... 

• He approves my specialty-pharmacy drugs every year (Praluent and Nexletol) and his office does a flawless job with this. They have this down to a science (and I hope I am not jinxing myself for the future!)

• He manages my INR levels. Well, that is not fully true: I manage my own INR levels, but I cannot get measuring strips for my INR machine anywhere else unless I am enrolled in a “test at home” program that is setup through an INR clinic, like his practice has. 

• As an aside - I have met a lipidologist who would love to get Evkeeza approved for me, but he does not work with any insurance company, so I would have to pay for an office visit several times a year, and for the lab tests on my own and then ask for reimbursement. This is something that the current doctor (given that he is with a major healthcare system) does as a service right now. A minor inconvenience, I know, but I am seriously thinking at least of this switch to the lipidologist, if nothing else. I would still need the current cardiologist for all the heart-related issues. 

Our healthcare system is already complex and unyielding. Add the complexity of FH and heart disease to it and you’ve gotten yourself into quite the spiderweb. But some hair will have to come off when I pull that bandaid, eventually. Like I said: I am not getting any younger. I have less and less the desire to also get any sicker, too (although this might be unavoidable) - especially when there might be other options out there for better treatment. 

Maybe Evkeeza is not for me. Maybe there are some contraindications that I don’t know about from my own research. I am not a doctor, obviously, and I don’t know how to apply a drug to my unique disease profile. This is what I am looking at doctors to know and explain to me. But I need to know something - anything, about whether this is worth trying or not. Something more than “they’ll look into it.” They have been doing this for close to 2 years now (the first year they “never heard of it”). 

At this time, I feel like I have reached a crossroads but not sure what the direction will be from here ...