Clinical Trials and Tribulations
Imagine! Me, in a clinical trial! I was skeptical at first, and that skepticism eventually changed to fear. Not a massive, overpowering fear like the kind that grips you in the midst of an important exam, but a small, gnawing fear that nibbles away at your determination to see the clinical trial, for example, through to its conclusion. The fear stemmed from not knowing what to expect: what was expected of me, and what to expect from this new experience ahead of me.
The whole concept of clinical trials was foreign to me. I knew what they were, in a general sort of way, but never considered participating in one. I have a spinal cord injury due to surgery more than twenty-four years ago. Too many years had passed for me to think about anything like that, or so I thought. Then, when I began working for the Paralysis Resource Center, all of that changed. Or, at least some of it did.
I learned of a clinical trial for a medication that could potentially help reduce spasticity and bring about better mobility for me. My first reaction was: Sign me up!
After reading the possible side effects that included seizures, however, I wasn’t as sure as I had been just a few minutes earlier. After some soul searching, and urging from family and my co-workers, I decided to go to the rehab center where the trial was being held, about an hour and a half away from my home, and find out more information. Maybe I could help with research that could help others, and also help me in some way, as well. That would be a win-win situation.
Entering the large rehab center, I was impressed with the obvious easy rapport the therapists had with their patients. It was April, and many therapists were working with their patients outside near the gardens that lined the sidewalk leading up to the front door. “This is a good sign,” I thought to myself. “It’s a lot different here than when I was in rehab.” It made me feel better to know that the place where the trial was being held had such a positive image surrounding it.
Inside, I met the physiatrist (rehab doctor) in charge of the trial. Dr. Fonzetti met me in the large meeting room across from his office. He spoke with me for a few minutes and asked some questions to see if I met the qualifying criteria set for the trial. Yes, I was between the ages of 18 and 65, my level of injury was within the guidelines, and it was incomplete. I made it so far!
The next test would be whether I would tolerate the medication, if I happened to end up in the group that gets the medication instead of the placebo group. When you sign up, there is never any guarantee that you’ll get the medication. But that’s the chance you have to take. But first, I would have to come back in a week for baseline tests to which later tests would be compared. As I mentioned, the trip to the rehab hospital where the trial was being held was about an hour-and-a-half from my home, which was a bit intimidating, at least at first.
Dr. Fonzetti gave me a schedule of the days as well as a schedule of tests that would be done during the next four months. I could choose the time, and know in advance when which particular sets of tests would be done. Every visit would entitle me to a blood test. Every three weeks was an electrocardiogram (EKG). A complete physical was scheduled for every sixth week. In addition, I was required to complete a questionnaire on any improvements I experienced during the previous two weeks, and I needed to rate my level of satisfaction with the medication/placebo.
I was also assigned to a physical therapist. Jody was my PT for the duration of the trial. Each visit, after checking in with Dr. Fonzetti, I met with Jody, who checked me for the level of spasticity. Jody tested my arms and legs by bending and straightening them, watching for ease of movement as well as range of motion. She assigned a numerical rating. In addition, Jody asked me to rate my spasticity from each previous visit, rating it from “Mild” to “Severe”. This information was written down, sealed in an envelope and sent to the company collecting the data for the trial.
Another of my responsibilities was a dial-in report. I was given a phone number and a nine-digit identifying number, and had to phone in on a daily basis, to report any missed doses, and any functional improvements based on prerecorded questions that I answered with numerical responses.
As time passed, I became more familiar with the trial, the tests, and my responsibilities. Despite the blood tests, I began to look forward to my trips to Burke, even when they jabbed both arms, looking for a good vein!
That is because, after just a few weeks, I noticed some improvement in my spasticity. Dr. Fonzetti couldn’t tell me for sure that I was on the medication, but I was sure that I was. It was either that, or I was experiencing a really powerful placebo effect!
The improvements continued. I remained on the medication for almost ten months. By the time of my last visit, I had a list of improvements: increased sensation in my right leg for hot and cold, reduced tingling in both hands, increased fine motor movement with my fingers, and goose bumps when I got chilled. I was even able to stand better due to my reduced level of spasticity!
During my last visit, Dr. Fonzetti assured me that since I had completed the trial, that I could stay on the medication (or begin the medication, if I was on the placebo), until the drug is approved by the FDA. I was all set!
Unfortunately, the plans that I thought were firmly in place, changed rather quickly. Within a week of that last visit, the company sponsoring the clinical issued a statement that the results of the trial were disappointing for the spinal cord injury portion of the study, but very promising for the multiple sclerosis study.
As a result, the MS study was going forward. The spinal cord study data was not going to be used for FDA approval of the drug! The company was not going to be able to continue to provide medications to participants such as myself due to financial considerations! My hopes were dashed!
But there was nothing left for me to do but gradually wean off the drug, which took a couple of weeks. I eventually got over my deep disappointment, but I don’t mind telling you that it took a long time, longer than I would like to admit.
I kept in touch with my contact at the company that was sponsoring the clinical trial. Periodically, I’d call just to check on the progress of the continuing trial for multiple sclerosis. I was very pleased to hear that the Phase 3 trial was going well with great results! But even so, I knew it would take a long, long time before the trial was completed, the data recorded and sent to the FDA to be reviewed and hopefully, be approved for the prescription drug market.
Fast forward to Monday, February 1, 2010. I saw a press release announcing the approval of Ampyra, the market name for fampridine, the clinical trial drug that I was on all those years ago. The drug is approved for patients with multiple sclerosis to improve their walking speed. I called my friend at Acorda and she told me my doctor can prescribe it for me because it is “open label”. The only barrier is that the monthly cost is more than $1000. I have to wait and see if my insurance will help with the cost. The patient assistance program is for MS patients, at least at this point.
I’m waiting for a call back from my doctor. She can call and get more information on insurance assistance for me. I’ll let you know what happens…
9/2/10
Looks like I've hit another snag, though! My doctor's office hasn't gotten back to me yet on whether they've actually faxed the information Aetna needs to complete the application.ARGH! Nothing's ever easy, is it??
9/27/10
All of a sudden, everything is changing--and NOT for the better! Apparently, the one form needed for Aetna was received. now I have received a letter denying Aetna's coverage of the drug for me. Because I have a spinal cord injury and not MS, I have been denied access to a medication that I KNOW will help me.
My doctor's office has appealed twice and twice denied. Now I a in full-throttle panic. I'm calling everyone I think wouldbe even mildly interested in hearing me talk/vent//cajole about it.but most times I am leaving messages that are not being returned. I gues it's more bad news. I'm going to let it go for a few days. Not mentally because I'm thinking about it all the time. I just have to plan what to do next.
10/26/10
After making some calls on Friday, I got a call back from someone at Acorda. She gave me some ideas on how to pursue different ways to possibly get Ampyra. No guarantees, of course but at least it's something to keep in mind.
I made some more calls today and am playing the waiting game again.
11/1/10
I received a voicemail from my doctor. She tried to call for a phone appeal to the insurance company. They never called back. She asked for the contact information again. She said she wants to try this way because it would mean a lot less paperwork than trying one of my other remaining options.
I called her back and left a voice mesage with the information and asking her to let me know what happens.
And the wait goes on....
11/8/10
No word from my doctor yet. I called and left another message. I have a feeling that this means the phone appeal ddin't work. Looks like a may have to go to Plan B. As soon as I develop one, that is.
11/16/10
Still no word on what happened with the insurance appeal. The only glimmer of hope that I have3 (and it is a small glimmer, I admit), but I haven't received a letter saying "NO!" rfrom the insurance company. I'm afraid to call because of what I'll hear. I still like to cling on to that itty-bitty glimmer for as long as I can.
I know I have to implement Plan B at some point but I'm afraid to stir the already-murky waters that I'm trying to traverse.
I've said it before and I'll say it again: ARGH!
This really shouldn't be this hard!!
12/1/10
I left yet another message for my doctor, asking if she would be willing to do the paperwork necessary to get the medicine.
This is my Plan B but so far no response. I think I know what THAT means...
12/9/10
In order to implement Plan B, I have to abandon Plan A. SO--no more calls to my doctor. I am tired of banging my head against the wall. It took me a while but I have learned my lesson. Continuing to make calls is useless and fruitless.
I will continue talkiing about Plan B in a new blog.,..watch for it!!
