For weeks and weeks I had been planning my next blog update: "FINALLY we have a start date," "ANNOUNCING: The news we've all been waiting and praying and praying for"...
Instead of being able to start drafting this highly anticipated post, we have unfortunately been met again and again with stumbling blocks that have been, very, extremely, infuriatingly, frustratingly, quite largely avoidable.
I'll just say this now - I am not here on this blog to "out" anybody. I am not here to complain for the sake of complaining (believe me, I do complain when I need to vent, but generally I try to limit my hardcore venting audience to a select couple of people).
However, I also always try to keep in mind that either today or in the future, another parent may be reading this that is in the same unthinkable position and may be looking for answers or may benefit from hearing from others' experiences. So with that in mind...
Here's What Has Happened
As you know, we've raised the funds necessary (the first $500K from our campaign!!) to run a study that will allow Damian to take his first experimental treatment: a small molecule drug that will be taken orally by cracking open a capsule and pouring the powder onto a spoonful of yogurt or something before feeding it to Damian. No one in the world with ASMD is on this treatment right now. In fact, the very first hurdle we were tackling at the start of all this was trying to get a hold of the pharmaceutical company and convince them that they already owned the drug that we needed (they were using the drug to study an entirely different disease and had never even heard of ASMD/Niemann-Pick). It was a miracle that when we finally did get a hold of them, they very graciously and very quickly agreed to supply the drug to us!! SUCH a blessing!
Thinking that getting the pharmaceutical company's cooperation would be the biggest hurdle to jump through (I'm pretty sure it typically IS!!!), I assumed the rest of the beaurocratic process would go by fairly quickly. And it SHOULD!!! But it hasn't.
My understanding is that typically, if a company's drug is not yet FDA-approved, the patient's doctor (or a clinical research coordinator as requested by the doctor) basically sends a formal request for it in the form of an application and then a handful of approval reviews occur. If it all gets approved, the patient can start taking the drug. It's a lot of paperwork.
We've got some added steps in there because not only is our drug not FDA-approved yet, but it's also not part of a study program that's currently happening. There is no active clinical trial for this going on. So the process of getting FDA approval requires a little more information included in the application specific for "single-patient emergency use."
Unfortunately, some grave miscommunication occurred and instead of processing an emergency application, the FDA started down the path they would take if a doctor was looking to begin a large-scale nationwide clinical trial, which would require a ton of other patients, a ton more data, and ... obviously that's not at all what we are trying to do. We don't remotely have the number of patients or data support to start something massive like that. The most pertinent setback of this miscommunication mess, though, is that setting up a formal clinical trial takes a LONG TIME.
I have been told by multiple people (including by the FDA this week) that in a single-patient emergency use situation, the FDA typically has to respond to the doctor within days of receiving the application (it's an emergency after all!). But in our case, they took a whole month to respond, and when they did, they said we couldn't start a full-blown clinical trial with so much lacking information and patients (because they thought that's what we were trying to do). And then the FDA said they would follow-up again later.
When the FDA was contacted and told they had it wrong - that we were instead attempting to get a single-patient emergency access to a drug, they basically told us they couldn't pivot from the path they were on - they instructed us to wait until we receive the official "rejection letter" (essentially) from them before they would be willing to receive more specifics from us and start processing information for a single-patient emergency use. Infuriatingly, that rejection letter was turning out to take "a little longer to write than anticipated" and suggested it may take some time to send out.
Enter Mama Bear.
If there are any skills I've gained in the past year that have been particularly useful as a Medical Mom, it's been the ability to successfully guess what the emails are of executive-level people from just knowing their name. I've unfortunately had to use this skill a handful of times already when efforts seemed to be going in circles or assumed responsibility kept being passed around, resulting in unreasonable pauses in progress. But it's amazing how much time and attention someone can suddenly afford to spend on your terminally-ill son's case when their boss' boss is asking for updates. (By the way, I'm speaking broadly here, as this has happened not just in regards to this treatment but in MOST aspects of Damian's overall health care - appointments, insurance, the works).
When I was told that we were supposed to wait a week for the FDA to send this rejection letter, I absolutely refused. I was not going to wait another week before we would, no doubt, be told "Oh, sorry, it'll be a little longer" or "Oh, sorry, there was some confusion and we went down the wrong process," or any other unacceptable excuse. No more giving people the benefit of the doubt and assuming they have all the info they need before I get in there and ensure myself that they do. We are running out of time. Damian was slated to start this treatment MONTHS ago. So that day, I guessed (correctly) at some email addresses and made some calls to boot.
I conversed with 5 different people from the FDA throughout the week. I think they were all taken back (some seemed genuinely upset/annoyed) that they were being contacted by a lowly parent, but I was persistent and direct. Each conversation got me more and more frustrated, but ultimately, FINALLY, they complied with what I kept insisting: that there is MASSIVE confusion and unnecessary delays regarding Damian's application and it is absolutely imperative that the FDA and Damian's team get on a SIMPLE phone call immediately and iron it out.
That call is now scheduled for Monday (tomorrow). *your prayers regarding the productivity of this call are so so appreciated!!*
I am exhausted
I'm so tired of all this, y'all. Watching Damian get weaker and weaker is torture enough. On top of that - having to deal ("deal" = correct mistakes, manage communication, make do with maddening inefficiencies) with not just the FDA but 2 other government agencies in regards to Damian's health care makes it so much harder ("fun" fact: BY FAR the easiest experience of the 3 has been dealing with the DMV when we got the handicap placard for our car - I only had to ask for a manager's intervention ONE time!). And then the exhaustion of scheduling appointments and managing insurance, special needs equipment vendors and their drama, and a jam-packed weekly schedule... it's just a lot. A lot. A lot. A lot.
Seriously, thank goodness for Damian's therapies, our experience with them has been nothing short of angelic.
During the strenuous 5-day sprint, I really look forward to the weekends, but then Friday afternoon hits and I just want to keep fighting because the idea of no progress being made for two whole days is almost maddening.
How I Cope
One of the most frequent questions I've been asked over the past few weeks has been "How do you cope?" If I'm being totally honest here, my answer is "not too well so far." I'm trying to work on it though.
I have a professional background in project management and have always had an incessant need for a plan and a schedule (Brock teases me that I've always needed a clock and a calendar in every room). It disgusts me not knowing what is going to happen. Unfortunately, this week I've also caught myself scolding myself for being so upset because that might mean I'm not trusting in God's plan and timing the way I should be.
I am so thankful that God is way more understanding and empathetic than that.
I'm living a nightmare right now. God knows it. I know it. But as someone in our church's congregation mentioned today over the pulpit (and I'm paraphrasing here): "it is ok to not be ok and 'to feel all the feels:' sad, angry, down... but it's not good to live there. We need to let ourselves feel God's love." The fact is that I do feel God trying to comfort me, when I let Him. And I do feel like there is a light at the end of this tunnel. I think it's totally ok for me to get upset and frustrated, but I really want to work on reminding myself in those moments that the drama of our situation will make for an even better story when we come out of this on top. I do trust in God's plan and I do have hope. I think if I really didn't, I'd be doing nothing at all but wallowing in self pity. But I am taking action, a heck of a lot of it, so that must mean I have hope. I just need to be more conscious about acting in faith, not in fear. Determination over desperation. I will try to be really purposeful about that this week.
Keep a sharp eye, because Mama Bear isn't going away. I'm getting my baby that treatment.