Thursday, December 17, 2009

My MS Story Chapter 22- Clinical Trial Endgame

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I want to be clear.  I never met the people who screwed me over in this trial. I don't know their names. I don't know what they look like. I just know they screwed me over.

The people that I dealt with personally, the doctors and nurses and technicians, were all wonderful people. They just weren't holding the cards. Neither was I.

For some background on my experiences with this clinical trial, please read my previous posts:

MY MS Story #14- All in the Name of Science
My MS Story #16- The Clinical Trial Begins
My MS Story #17- There Will be Blood
My MS Story #19- Measuring the Immeasurable
My MS Story #21- A good friend turns into a coldhearted sonofabitch.

In the two extreme cases of a clinical trial, the endgame is straightforward. Often times it is clear even before the scheduled completion date of a clinical trial that the treatment is either not working or is actually doing some harm. In those cases the trial is stopped early or simply considered a failure at its conclusion. Everybody just packs up and goes home. Nice try. Good effort. We’ll get ‘em next time! This is not what happened in my trial.

On rare occasions a clinical trial may be a rousing success- high fives all around. When this happens sometimes the participants continue with the treatment in what is called an open label phase. After all, it would be cruel to just snatch a miracle drug away from long suffering patients who are finally getting relief, wouldn’t it? Plus, this gives the trial sponsors, usually the drug company, an opportunity to continue studying the long term effects of the treatment. This is how the trials worked out for several of the RRMS drugs that currently have FDA approval. I was hopeful that my trial would fall into this category, but it didn’t.

My trial endgame was in limbo. It was not an abject failure. It was not a rousing success. So there was no clear path forward.

I expressed an interest to my trial physician, Peter, about continuing with Rituxan infusions after the scheduled completion of the trial, but how? Peter indicated that he would be going to a conference of the trial doctors in a few weeks, and would let me know what he learned. We naively assumed that timely and compassionate decisions would be forthcoming.

For me the formula was simple. MS bad. Rituxan good. Must have more Rituxan.

I worked two parallel paths. I was hopeful that Peter would learn of an open label phase to the trial. But at the same time I was consulting with my local neurologist about setting up Rituxan treatment outside of the trial. Since Rituxan was not approved for MS, it seemed unlikely that my insurance company would cover it. I assumed the cost to be $5,000 per infusion, for an annual cost of $20,000.

I had my last two trial infusions of Rituxan in November of 2006. Like all the previous infusions, it went well. I felt that if I did not get another pair of infusions six months later then the effect of the Rituxan treatment would wear off, and my disease progression would return to the pre-trial rate.

The trial plan called for continued close tracking of the patients for a period of one year after their final infusion. The trusting soul in me assumed that this plan existed so that doctors could monitor for and react to any negative side effects. It was done, you know, for my safety.

The cynic in me believed otherwise. Perhaps this post-infusion period existed not for my benefit, but for the trial’s benefit, and by extension for the drug company’s benefit. Maybe the trial designers wanted to shore up their statistical case by demonstrating that two things occurred in the trial. People not only got better when they took the drug but also got worse again when they stopped taking the drug.

Had they even considered the possibility that in order to gather this information some group of trial participants, like me, would need to stop taking the only treatment that had ever worked for us?  I think it is easier for trial administrators to ignore these nuisance considerations when they have never met any of the patients face to face. Clinical trials permit nameless, faceless trial administrators to make medical decisions that would never be made by a compassionate physician treating a sick patient.

Probably it was a little of both- patient safety and data collection. I don’t mean to attack the trial administrators personally.  They are most likely decent people.  Our lovely system of approving new drugs is what elicits this heartless behavior from them.  

In April of 2007, about the time I would otherwise have received another pair of infusions, I had a routine appointment with Peter. I learned that the trial sponsor was still pouring over the data, and there was some hope of an open label study in…get this…January of 2008…maybe! That would be almost a year away, and well over a year after my last pair of infusions. I was not pleased with this information, because I expected that in the very near future my disease progression would resume with vigor (I was right). They were going to spend another year just watching and thinking and graphing and pondering! What amount of disability would I accumulate during that year? Nobody asked me.

Sure enough, almost on schedule, by July of 2007 I noticed that the disease progression had returned to the same rate it was at before the trial. I needed to get me some Rituxan.

I had another appointment in July with Peter. I shared with him my observations about the resumed disease progression. His testing confirmed it. For the first time ever, he measured a significant decline in my function, specifically my leg function. There was still no word from the drug company about an open label study. I told Peter that I might have to start Rituxan on my own, which would mean leaving the trial before its official conclusion. He understood.

I asked my local neurologist to set up a pair of infusions for me. I would pay the $10,000 cost out of pocket for these first two infusions. This wasn't an expense that I could easily support long term, but I just needed to bridge the gap until Rituxan got approved for treatment of MS, at which time my insurance company would take over the expenses.

Remember, with PPMS I'm not talking about symptoms that come and go. I'm talking about irreversible, permanent damage that was occurring slowly and steadily, like a Chinese water torture…drip, drip, drip. Every smidgen of disability that I have ever accumulated has remained with me. Given this reality, time was of the essence.

A one year wait for an open label study that may or may not materialize simply was not an option.

For the next installment in the series, click here.

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  1. Mitch,
    I have sat here speechless for awhile wondering how to respond. I went back and read your trial posts from the beginning. Maybe your struggle touches me more deeply because I have seen both sides of the clinical trial experience. Before RRMS kicked me I was an RN with a promising career. I worked in an outpatient center giving chemotherapy (Rituxan was one) treatments and I was also the clinical trial coordinator. I enrolled and managed all the trials in our clinic. I had no control over the randomization and who received the real drug or placebo though. Working with some of the most endearing patients in the world, I often felt their frustration in the trial process, but you are the first to describe it so eloquently. Just when positive results are occurring the rug is pulled out. It is heartless and wrong. There needs to be a patient bill of protection law enacted that prevents the withdrawal of a trial, when the trial is over, if it is yielding positive results and stopping would cause a relapse. I am so sorry that this is not the case for you. You have really touched me with this post. I know my words offer little comfort ending your frustration, but I do understand. You are a remarkable individual. I am so pleased to have met you.

  2. Ouch Mitch. That sucks. I'm on Tysabri, a drug that has been on and off in FDA approval. Games are played with a drug that helps that serve to seriously interfere with a logical or consistent plan for a healing drug regimen. Some tricks are for our safety yet some seem to serve insurance or financial goals first. It is frustrating.

    On the positive side there are more types of drugs available for MS now. We have a large enough pool of patients in the MS community that there is a financial incentive to keep exploring new drugs. I'm glad for that because one other option is a rare disease with too few patients to make it worth a drug company's time.

  3. I too sat here wondering what I was going to say about this heart-wrenching story. What I know about you from previous posts is that you are a level-headed evaluator of any given situation. So when I can hear a deep emotional cry coming from your post, I know this is something really awful. I'm so very sorry for you and others in your situation.

  4. Rae,

    You have a unique perspective on the clinical trial world. I want to be careful to not paint all clinical trials with too broad of a brush. But you are right, something needs to be done to make the process more humane and less like a laboratory.


    Anyone on Tysabri has been through a lot of approval issues, that's for sure. You are right. As frustrating as our situations seem sometimes, at least there is some level of activity on the research front. Ask ALS patients about their situation, for example.


    I'll wrap up my final thoughts about the trial probably in my next post. It was a very satisfying experience until the ugly end game. Mine was just one person's experience though. I can't imagine all the stories that must be out there which were even worse than mine. People have been harmed or even killed in trials, and my story can't compare with that.

    Like I said, under the right circumstances I'll do it again, but I'll understand the potential pitfalls much better up front.