“Free” med for the minority of rare disease patients was at a cost to the majority

My ultra-rare autoimmune disease, Lambert Eaton Myasthenic Syndrome (LEMS), has been all over the news in recent weeks.  The controversy arose when Catalyst Pharmaceuticals recently launched the first FDA approved medicine, Firdapse®. Catalyst has been lambasted for the high price tag for Firdapse®. I am one of the few who knows both sides of the story. I know LEMS and I know Catalyst – I was one of three patients who were invited to attend the recent Firdapse® launch in Coral Gables to share my patient story. I have had patients around the world reach out to me in anger because of my being happy to attend that launch and share my patient story. Fortunately, I have also had many patients reach out to me to thank me for helping them gain accurate information in regards to Firdapse® and how to access Catalyst Pathways.

I have a special perspective on why the approval of Firdapse® has been met with some unhappy patients. These patients were receiving a similar medicine, freebase 3, 4- diaminopyradine for “free” to some patients through an exceptionally long-run clincal trial operated by Jacobus Pharmaceuticals.  Only Jacobus knows why their trail ran the unprecedented course of roughly three decades, or if whether they did, or did not, submit their data to the FDA for approval. The end result was some lucky patients received their 3,4 DAP for “free” (US pharma companies cannot charge clinical trial patients for an unapproved drug) for so long that many of them forgot they were part of a clinical trial.  Unfortunately, over the decades, the vast majority of LEMS patients did not have access to the freebase 3,4-DAP. Many found out about it, as I did, only through Facebook groups. However, once patients found out about the possibility of accessing this miracle drug, many could not access it because to get the medication from your local physician required that physician to do the paperwork required to become an investigator in Jacobus’ clinical trial. Completing the required paperwork is no small feat for any doctor, let alone a local doctor who is not part of an academic center. Many of those who did access 3, 4-DAP had to travel to a faraway clinical trial center.

As a single person on social security disability, I could not travel to a Mayo clinic for treatment, like other patients I know, or travel on a regular basis to an out of state neurologist who participated with the Jacobus program. When I was first diagnosed, I did not know about the Catalyst trial.  Fortunately for me, my neurologist was willing to do the mountains of paperwork involved to get me onto the clinical trial with Jacobus Pharmaceuticals. However, it still took 8 months before all the signatures were given and my case made it to the internal review board for hospital approval for my neurologist to treat me with a product not yet approved by the FDA.

When it came down to the very last days awaiting approval, I finally reached out to Catalyst about possibly going onto one of their active trials for Firdapse®.  When I spoke to them, they gave me all the options. I told them how long I had been waiting for my neurologist to gain approval for me to treat me with 3,4-DAP.  I mentioned that I was going to give her through the end of the week as she had put so much time and effort into it.  Sure enough, the next day I received the call that she had received the approval needed to start treating me with the unapproved 3,4-DAP.  When I called my Catalyst contact back, his response was “Dawn, the important thing here is that you are getting the treatment that you need. We are happy for you.  Please don’t hesitate to reach out if you need anything else.”  That was my first interaction with Catalyst Pharmaceuticals, complete class and always putting the patient first.  That patient-oriented culture has been a part of every interaction I have had with Catalyst ever since.

When I started on 3,4- DAP, I was more aware of the fact that Catalyst may gain FDA approval for Firdapse®. So, what did I say to my neurologist?  I told her to “Document! Document! Document!” how I did on 3, 4-DAP because I might need insurance to approve of it soon. I never ever thought I would receive 3,4-DAP for free, forever!  I know not much in life is “free” (at least not material goods)- somebody somewhere is paying for it. I was only receiving it “free” through their compassionate distribution program that included having many protocols including timed speed tests, strength tests, and various other medical exams.  I  also have other expensive treatments when it comes to managing my LEMS, so, when it came time to add my Medicare Advantage plan, I reached out to the best insurance agent I knew, and she did a phenomenal job advising me.

I was grateful to be a part of the Jacobus compassionate distribution program; however, I am more than happy to be on the FDA approved Firdapse® now.  I no longer must make sure I always have refrigeration wherever I go to keep my med in.  I am confident that the production of Firdapse® is up to FDA regulation standards, which is exceedingly important.  And, being as active as I am in the rare disease community, it is paramount to me that everyone who has LEMS now can have a prescription written for the ultra-rare disease that we share.  It shouldn’t just available be for those who have the means get to a specific doctor or world renowned clinic.

I know patients who, after learning about it, brought in the words “3,4- DAP” to their doctor, who refused to do anything with the med for them because it was an unapproved drug or it took too much effort to join the clinical trial. So many patients couldn’t access the drug that allows us to walk!  Imagine reading about how life-changing a med is and not being able to have a doctor write it for you? I also know patients who couldn’t get a diagnosis of LEMS, despite having all the symptoms. Now, thanks to the studies that Catalyst invested in and submitted to the FDA for approval of Firdapse®, the diagnostic criteria is clearer for neurologists. Getting a diagnosis of a rare disease is the first needed step in getting treatment. Thanks to Catalyst’s investment in their clinical trials, patients that have been without diagnosis for so long are finally able to get a diagnosis.  I went undiagnosed for decades. I understand the relief these patients feel to finally have a medical reason to these ongoing vague and yet debilitating symptoms.

Most importantly, thanks to “Catalyst Pathways”, a program that Catalyst has put into place to assure access for all patients, as well as other reputable programs that Catalyst donates to, I have yet to hear from a patient who has told me they can’t get Firdapse due to the price tag.   I do hear from patients when they get their free month of Firdapse® while “Catalyst Pathways” reps continue to work with their insurance to see what is needed to gain approval for their treatment.

As a rare disease patient turned advocate, I am thankful for research, not just for my rare disease. I’m thankful for research for my friends with different rare diseases that treatments haven’t been able to stop the progression of yet. I don’t want that research to stop. Rare Disease Day is fast approaching, so I must share that if we were to put all the rare diseases together, there are over 7,000 of them, and they impact more people than cancer and AIDS combined! Are we trying to give cancer drugs and AIDS drugs away for free? I think not.

I believe if the profit incentive was removed from our system and everything was “free,” research would halt. Most people don’t work for free in America; and most investors want a return on their investment. Developing drugs involves risk and requires substantial resources and investment. If that investment has to be recouped over a few hundred or a few thousand patients with drug prices at the same level as, let’s say a diabetes drug, where you can get money from millions of prescriptions, then I know rare disease patients are doomed. Research in rare disease will dry up.  If research on rare diseases were to stop, it would mean that anyone who is rare isn’t important. I’m sorry, I think everyone that has a disease that affects their quality of life and that could kill them, is very important.   Free is not necessarily best.  I’m happy to be on the Firdapse®, newly approved by the FDA.  I’m willing to bet that if your son, daughter, wife, partner, husband, friend or loved one had a rare disease that was causing them lose the ability to enjoy life or perhaps live at all, you would want a pharma company to invest in research to find a way for them to enjoy life again, no matter how rare their disease was.

Rare Disease Day is Feb 28, 2019

Please take a moment to see how you can help all those impacted by Rare Disease on February 28, 2019. 

Sen. Bernie Sanders letter to Catalyst Pharmaceuticals after some patients complained to him about the cost of Firdapse®.

Catalyst Pharmaceutical’s response.

Disclaimer: Prior to becoming disabled due to Multiple Autoimmune Syndrome (MAS),  This post, like all of my blog posts, is written from my own personal experience as a LEMS patient and is not in any way commissioned or sponsored by any entity.




Dawn DeBois

About Dawn DeBois

Florida born and Maine grown, my life has been atypical. My childhood was full of loss and severe physical pain. Both emotional and physical stress during childhood has been found to contribute to autoimmune disease. My first autoimmune diagnosis was at the age of 28, which has led to juggling multiple autoimmune diseases (Hashimoto’s, Fibromyalgia, Ankylosing Spondylitis, Psoriatic Arthritis and most recently LEMS- Lambert Eaton Myasthenic Syndrome). I am officially now classified as having “Multiple Autoimmune Syndrome.” You know what they say, go big or go home! You can now follow my LEMS jouney on my weekly column, LEMme Tell Ya, on LambertEatonNews.com.