It is hard to make any judgments about this subject without thinking about the people who have and are participating in the investigation drug programs. With this in mind I thought it would be good to interview someone who had participated in a drug program and get a first hand look at this subject. I interviewed Sue Guinn who had been involved with several drug programs to treat her rheumatoid arthritis.
Stacie: I understand that you were a participant of an investigational drug program.
Sue: That is right. Right after the birth of my second child my arthritis really flared up. It seemed that all my joints were hurting and it was kinda scary. I had been treated with “Gold injection” before and had terrible reactions to it. It is kind funny because that was a drug that WAS approved by the FDA and yet the side effect from that drug is more severe than any I have ever experience. I was somewhat afraid even to try any other medication because of that experience, but I was going to a doctor that I trusted and who had been working with me to find something that would give me relief without causing me such pain as the Gold injections had.
This doctor approached me about going on a study for oral medication that was being investigated. The preliminary studies had been promising and I was a good candidate for this study.
Stacie: What do mean when you say you were a good candidate?
Sue: Well, I was young, OK relatively young. I was 32. I didn’t have any other medical problems. I was not obese. I was a nonsmoker, nondrinker and my symptoms were not so severe as to skew the study and I had the time to come into the office for thefollow-up studies.
Stacie: What do you mean by follow-up studies?
Sue: You know, blood work, EKG’s, chest x-rays… that kinda stuff.
Stacie: Tell me about the study.
Sue: OK. It was a double blind study, which means, that neither the doctor nor patient knew whether it was actual medication or a placebo.
Stacie: Could you take other medication while you were on investigation drugs?
Sue: I couldn’t be on any medication for my arthritis. I had to keep a record of anything I took.
Stacie: Why did you decide to participate?
Sue: I thought if they could find safer medication that would help other people with arthritis it would be worth the inconvenience. I didn’t get paid to participate. The only things I got out of it were free lab work, x-rays and EKG’s. I also would be allowed to stay on the medication, if it helped, while it was being approved.
Stacie: So how did it go?
Sue: Well, at first it went really well. I could tell right away that I was on “real medication”. I felt better. My symptoms started improving about the second week. By the forth or fifth week I was pretty much symptom free. All my labs were coming back good and I was looking forward to staying on the medication.
Stacie: So what happened?
Sue: I had been on the medication for several months and I was working in my yard. I woke-up the next day and I had a rash all over my face, hands, arms and some on my legs. I called the doctor and told him that I thought it was poison ivy. He asked my to come in. He examined me and said he couldn’t be sure but I would have to be taken off the study. I was really upset because I was doing so well and hated to start on any other medication. I had no choice. He started me on Methotrexate, which is a medication that lowers your immune system and in fact is used to treat cancer in higher doses. I did well on it but had more side effects from it than I had experienced when on the investigational drug.
Stacie: Was the investigational drug you had been on ever approved?
Sue: No it never was. While it was in the final stages of approval they found that it caused a life threatening blood disorder. I kinda looked at it as a “God thing” that I got into the poison ivy.
Stacie: Would you ever do another study again?
Sue: Yes. I still believe it is worthwhile to find treatments for illness. If I could help find a drug that might treat an illness that you or your children might have. It would definitely be worth it.
Stacie: Is there anything else you would like to say about investigational drug programs or the way that the FDA goes about approving drugs?
Sue: Yes. I think the FDA takes too long to approve some drugs, especially the ones that treat catastrophic illnesses. If people would be willing to go through the risks of taking the medication and it improved their quality of life while they are alive I think they should be allowed to take them even if it might shorten their lives. That should be their decision.
By Stacie Guinn
Sue continued to see her rheumatologist who was also involved in the study. They may have checked her progress with her doctor, but not directly with her.