Tag Archives: Off label

The Harmful Embrace of “One Size Fit All” in Drug Development

In light of the news coming out from NurOwn this morning, it’s clear that the rare disease community needs a wake up call. 


NurOwn has shown great results in ALS patients with less-advanced disease. It’s important to note that disease progression for any condition varies from patient to patient. In addition, patients with more advanced ALS were also included in the trial. So the varying disease states absolutely play a role in how the data appears at the end of the trial. But instead of looking at the overall picture, why are we not looking closely at the group that had a “statistically significant” response to NurOwn? 


The rare disease community needs to stop thinking there is a “one size fits all” approach to drug development. We always complain about Pharma silos, and how it’s so hard to work with industry. Well, it’s hard to work with patient groups and the rare disease community, too. We create silos, letting our rare brothers and sisters fight alone. 


ENOUGH. 


I can tell you from personal experience that advocacy groups who think this way end up hurting patients. A decision made in the mid-2000s on Rituxan still hurts myself and other patients like me. I do not have guaranteed access to my chemotherapy drug that stops my immune system from killing my platelets. It is an “off-label” therapy, which means my doctor, the hospital that administers it, and my insurance company all have to agree to dispense it.


All of this could have been prevented by conducting a clinical trial and working on identifying the ITP patients who would likely respond (something I did myself in 2014 when I made the decision to use the therapy). However, that did not happen. Why? Since not ALL Immune Thrombocytopenia patients responded, no one felt it was a worthy endeavor. It left patients like me to struggle and fight based on an arbitrary assessment from industry and a patient group that was suppose to represent every ITP patient. They didn’t represent all of us, people like me were left out. We are hurt by this mistaken belief that drugs to treat our rare diseases are only worthy under a “one size fits all” approach.


The “data” and “charts” looking at Rituxan use in ITP patients wouldn’t look good if you looked at EVERYONE who was given the drug. But if you looked at the patients who fit certain criteria, you would see an AMAZING response with a certain group of ITP patients. That’s where we are with ALS right now, and it’s where we will be in the months/years to come as more orphan drugs go through the FDA. You can’t just read the headlines of an article about clinical trial data to get the total picture. Unfortunately, this happens in disease groups and people quickly dismiss promising therapies. It has to stop, the rare disease community is better than this.

I sincerely hope the ALS community pushes for approval of NurOwn and applies pressure to the FDA.

I look forward to lending my voice to this fight, because all rare diseases need to support each other.

Remission Accomplished

Left: Bloodwork two weeks after IVIg. Right: Bloodwork one week after my final Rituxan infusion.

My second attempt at repurposing Rituxan was a success. The last two weeks were busy and I was tired, so I decided not to blog.

It was incredible to see my platelets return to normal half way through the infusions. I continued with the IV steroids as part of my pre-medication routine, so I did not have any reactions. However, I had to ask for it every time so I will be requesting that this becomes a standard part of infusion prep when we start a trial. While they generally just give Benadryl and Tylenol to cancer patients using Rituxan because they need to gauge their reaction, this is unnecessary for ITP.

On the left is my bloodwork taken before my third infusion, on the right was my CBC before my final infusion. Huge difference in one week!

Overall, I am relieved that I am back in remission again. However, this was quite the physical and emotional rollercoaster. I am still tired, and likely will be for the next few weeks. I am trying to take a short nap every day to keep my energy up. I have noticed that the brain fog is completely gone, and I am very “clear headed” now. The purpura is gone, but I do have some darker spots where they once were. I am hoping they will go away in a few more weeks. My taste buds are still a little off, but not nearly as bad as before.

I’m still pretty burnt out emotionally, this was a hard fight and it took a lot out of me having to constantly advocate for every little thing. I certainly don’t want to go through this again in a few years when ITP comes back, so I am pushing to get Rituxan on label.

I’ll see the doctor again next month, and it will be interesting to see what my platelets are at. From there, we will decide how often I see him. I am looking forward to a break! I will be seeing a new Rheumatologist to address my Fibromyalgia and Sjögren’s Syndrome. However, I have noticed less joint pain since I finished my infusions. Perhaps some of that was B-cell related as well.