While 2018 wasn't the last denial I've had for Case's Elaprase, it was the most difficult. Well we're here again in 2022 with a denial again and two, not one, but TWO doctors who have called the PBM (pharmacy benefits manager, read: gatekeeper trying to save money), Magellan RX, for the insurer, BCBS of Tennessee, trying to keep Case's access to his medication. Mind you, he's been on this medication for 12 years and it has kept him physically stable, pretty miraculously actually, considering the dire physical symptoms that typically accompany MPS II.

As most of you know, doctors don't have hours away from their live, need-to-be-rounded critical patients to respond to these requests over and over. And so when my son's prescriber asked his colleague, who had also seen Case as his previous geneticist in prior years, to call them, they refused to listen to him or take in his perspective since he wasn't the current, immediate prescriber (who had already submitted documentation).

So, I called them. At first, they said they wouldn't allow me to speak to a reviewer since I was "just" the mom. Then I pointed out:

- I have served on multiple international consensus conferences regarding this group of conditions;

- I have multiple peer-reviewed publications regarding my son's condition;

- I have led patient outcomes research in his condition as a co-Principal Investigator;

- I have designed patient outcome measures and presented data to the FDA on his condition;

- I have personally met and interacted with more MPS II patients and caregivers than my son's medication prescriber times about 100;

- I founded and led for many years one of the leading research organizations in his condition.

So finally, someone from the PBM is supposed to call me tomorrow so we can hopefully review their criteria and their poor understanding of the disease and the impact on children trying to survive. AGAIN.

As I said several years ago, to my knowledge, my son is the ONLY patient on Elaprase in Blue Cross Blue Shield of Tennessee. So all of this policy and effort is to save the money FROM HIM. Our community is so small, it's pretty easy to know whether or not you have the only child on a plan.

So, in other words, Magellan RX and BCBS of TN want to deny the prescribed and recommended and standard of care therapy for my son, because it will save them hundreds of thousands of dollars per year, no matter that it will result with him in the hospital, in pain, and deteriorating toward a more rapid and painful death. I could almost get it if they were just honest about the balance of cost versus accepting that this health care is the only care for him, as they are in other countries like say, Canada and the UK. Doesn't make it fair or right, but please... don't act like you're just trying to enforce a facially neutral policy when we go through this every year and it's ONLY MY SON.

The system is B R O K E N.