Not all clinical trials are the same – some involve just a single blood draw, or interview, or procedure. But others, like the MPS II intrathecal trial, are very demanding, in time, energy, emotion, funds, and on and on.
The longer we continue in this process (we are now over 3 1/2 years since the beginning of qualifying in September 2010), the more I reflect on what it has required and how it has changed Case’s life and the lives of our friends and family – for the good, but also in countless ways that I would qualify as … something other than good.
Why do I mention this?
I think the reflection is helpful in two ways: (1) Families need to be realistic about the financial, emotional, and logistical toll that a clinical trial can have on your child’s and family’s life. Only by being (somewhat) prepared can we deal with some of the issues up front. (2) FDA and pharma need to better understand the demands of their trial designs so they can be more realistic in designing trials that are not too difficult for families to participate, for rare diseases, often on a long-term basis.
I’ve written a lot about the benefits of the trial, about Case’s advances, the miracle, but especially after the last 10 days, I wanted to be very candid about the toll as well.
Why mention this now?
We are in the final day of our annual 10-day visit to UNC in connection with the extension of the Phase I/II intrathecal trial – auditory testing, genetics clinic, OT/PT testing, pulmonology, Elaprase infusion, MRIs, bronchoscopy, ABR, cognitive testing, physical and neurological testing, and intrathecal dosing with 36 hours of pharmacokinetic studies (blood draws).
That may not sound like a lot each year, but that’s more vacation than many families take, that’s family taking in my two other children full-time and Case’s Nana coming with me. Add that to almost 40 3-day trips, 3 previous 10-day trips, 6 initial 9-day trips (the first 6 months of the trial, we were here for 9 days each month), and several miscellaneous trips for port surgeries over the last 3 1/2 years.
A little math equals probably around 220 days we’ve spent in NC over the last 3 years. That’s ….
– 220 days of (usually overnight) child care for my 2 other kids (for school, discipline, homework, studying, lunch-packing, activities, practices, birthdays, and other responsibilities) since Chris often travels for work,
– 220 days of Case missing his Dad and brothers, missing school, rescheduling appointments and activities, recovering from procedures, processing medical trauma,
– over 50 anesthetic events, after most of which Case suffers from severe emergence agitation,
– thousands of dollars of extra expenses for the above responsibilities, food, hospital reward/bribes for Case, supplies and modifications, parking and airport costs, loss of income, etc.
– packing and unpacking me and Case (and often my two other kids as they stay with relatives) over 50 times.
Case’s medical trauma has always been a delicately balanced proposition. After just the first year in the trial, I had to research so much to maintain some semblance of normalcy that I wrote this ebook back in 2012 – Calmer: Medical Events with Cognitively Impaired Children. But the ongoing long-term and intense nature of the clinical trial often pushes him over the edge.
Over the last 36 hours of blood draws, every waking 10-15 minutes I hear whimpering cries of “I’m done! I’m done!” And his body further contorts with his right arm stiff from guarding over his port for the entire 36 hours, screaming when he’s forced to get out of bed because he doesn’t want to move or have anything get near his port when accessed. It breaks my heart to watch and he won’t even let me comfort him. He doesn’t understand why he has to do all this, although he is asking questions now.
So when doctors say that intrathecal dosing visits need to be changed because they’re going to be out of town (as happened to several of us at different hospitals recently) or when a local Nashville hospital, instead of finding a way to bring to fruition a two-year process to allow Case to dose there (as almost all other U.S. patients dose at their local hospitals, some beginning with dose 10, while we are at dose 43) says, “Is it possible for you to continue at UNC as you have been doing? This would be best, if you can do it.” – what do you do? You do what you have to do to save your child, but it sure doesn’t make it easy, on your child, your family, or anyone else involved.
A clinical trial teaches you to be flexible. Very, very flexible. And it teaches you just what you’re willing to sacrifice, as if MPS alone hadn’t already done that.
Luckily, the next phase of the trial will not involve so many repeated extensive visits, but initially in a Phase I/II trial, these are often what FDA and pharma have required to establish the safety of a new drug. I know of families who could not fathom coordinating the logistics to participate in this first trial and thus, did not try to qualify their child. And even with shorter visits for the next phase, families will have to be prepared for the unexpected, with contingency plans, backups, and the like.
Hopefully in the future, patient groups can be more involved in clinical trial design to help craft and streamline clinical trials so more families can meaningfully participate.
In the meantime, we get discharged from the hospital in two hours and fly home tomorrow, our 11th day of this trip. I’m ready to see my husband and my kids and sleep in my own bed.