Studies, Clinical Trials, and Being an Informed Patient

[Arlyss]

I have not seen this topic discussed - perhaps it has been. However, I wanted to share some thoughts about being approached for studies, clinical trials, and specific procedures.

Individuals approached regarding participation in "studies" or clinical trials need to ask many thoughtful questions in order to evaluate what is being offered. Even though they may be approached in a very kind manner, they really need to know as many facts as possible in order to make a good decision.

This is also true when discussing details about surgical procedures and options for valvular and aortic disease. It should always be our choice, without pressure, and it is not easy to be sure that complete, factual information is available to us in making decisions.

I am thinking of a young woman who went to have AVR and aortic aneurysm resection. She went planning on having a mechanical valve, but came home with a tissue valve. She told me that the draw backs of coumadin had been described to her, and the tissue valve would allow her to avoid coumadin. It wasn't until months later when she was contacted to come for a "free" echo, that she realized that the tissue valve she received was part of a clinical trial - it was not officially approved for use in this country at that time. This is a bright young woman who along with her family was very stressed by having an aortic aneurysm that needed removal. It is understandable that they could be confused, but it should not have happened. I checked with someone else who also had AVR and received the same valve, and this person knew that he was part of "some study" but without much detail. In both cases, I was concerned that they did not have sufficient information.

I have spoken with someone who said that they were part of a "study" and the valve that they received appears to be leaking. I did not ask, but I am wondering why they were convinced that they should be willing to help "study" something when there are alternatives with a proven track record in the market.

It is important to remember that those who want to study something have an agenda - they need someone to participate in the study! The patient has an agenda too - to get the best possible solution for their situation. These can be two different things. We should always feel free to decline to participate, or to seek other opinions in evaluating our options.

In one of the examples above, the person told me that they were told that the surgeon "had never had to take out one of these valves". "Never" sounds good, but the detail was that the time frame in question was only about two years.

For example, if someone is told, "I have only had one patient come back for another surgery" - that may not mean that all the surgeries except that one had a good outcome. It literally means that only one person has come back to that surgeon for a second surgery. Some may have died - so of course they will not be coming back - others may have gone to a different surgeon for their redo operation. It is very possible that what happens to all the patients is not known, because it is very costly and time consuming to keep track of all this information. So, one patient may have come back for a second surgery, but what did all the others do? How many of them died? How many of them went else where the next time? Has the time and effort been spent to find out?

I would be interested in others experiences and thoughts.

Best wishes,
Arlyss

 

[Magic8Ball]

I have no experiences of this but agree with your viewpoint.

I have no interest in the greater good of mankind or the fact that my help will help millions of others in the future, i want the best, most trusted, long term fix for MY valve, MY aorta and MY family.

I'd be very unhappy if someone approached me to go on a 'trial' and would certainly be letting them know about my satisfaction level.

I'ts pretty down and dirty to do the old switcheroo on someone at the last minute based on some questionable information given in a slanted perspective for the benefit of the company not for the patient.

I always associate trials with those patients who are beyond reasonable help and need the latest and greatest treatment to stand a chance of survival, not what is becomming run of the mill surgery these days.

 

[mntbiker]

Thanks for raising this issue Arlyss.
Everything below is simply my opinion not fact.

I have been wondering for a while now if the trial I am a part of was the deciding factor for the valve choice my surgeon made during the surgery. I was 95% convinced, based on conversations presurgery that I would end up with a mechanical valve and yet I walked out with a tissue valve. I did have a preference for a tissue and the specific tissue I was looking for was implanted. But I had issues with the ascending aorta as well and that was the reason I was expecting a mechanical. The surgeon found that my aorta although enlarged was thicker then normal and healthy.

This has raised the concern though of just how ethical are surgeons, in general. If he had an opportunity to get another patient into a study and just fudged the numbers a bit would they do it? Did he do it? Will they make the decision that will ultimately benefit them if they believe it will do no harm to the patient. Now keep in mind that most surgeons believe reops to be no issue so would probably not use that as a deciding factor.


This just reinforces why the patient must be informed and have clearly defined choices that the surgeon has agreed to. Unfortunately (you get the idea) the patient is asleep during the surgery and unable to enforce the choices that had been agreed upon.

So yes, I did agree to be part of a study, after the surgery, at that point the valve was already installed (he asked three weeks after the surgery) so I did not see any reason to decline. If I had been approached on this pre-surgery my questions would have been very different and the answer may have been very different.

 

[gijanet]

Good topic!

Good topic!

I agree that one has to be very inquisitive about studies. We learned the hard way firsthand. My sister had cervical cancer. She underwent surgery to remove what they could, the maximum amount of radiation that she could have, and a regimine of chemotherapy treatments, but the cancer still metastisized. WE ended up down at MD Anderson for the last six months of her life, undergoing one experimental protocol after another, but with no success.

Christie tolerated one experimental chemotherapy treatment well, so "they" asked us if we wanted to double the dosage on the next round. Since CHristie had never lost her hair with any of the other chemos, but was starting to with this one, we were hopeful that this one would finally do the trick, so we agreed. We signed the consent form. Looking back, I was so ignorant at the time. I was new to the medical world. WE didn't know to ask questions................They doubled the chemo dosage,and that alone damn near killed her. She was in the hospital for three weeks that time just trying to recover from the chemotherapy. When I inquired how others had fared when they doubled the dose, we were only then informed that no one, NO ONE had had the dose doubled before. NOw that we weren't told beforehand, and I certainly did not know enough at the time to ask about that prior to our consenting.

Granted, Christie was terminal. I know that now, although we refused to accept it at the time............she was only 29. But she never really recovered from that round of chemo; it was really the beginning of the end, and it certainly hastened her death. Maybe that was a blessing as she was suffering so, but maybe not. Maybe that would have been the chemo to do the trick, just not at such a high dosage. I don't know. It just kind of gnaws on my mind sometimes at night. Maybe, that incident, at my dear sister's expense, was just prepping me for Katie's medical adventures.

Anyway, we have been approached numerous times wanting Katie's participation in various research studies, and we have agreed to participate in three of them thus far. I have to say that we were very well informed each time (in these three cases) about what it would entail on our end and what would be gained from it. However, I was very inquisitive about who was actually sponsoring the study, how it would affect our treatment plan, if at all, etc. (I wouldn't have known to do this, except from our experience with Christie. ) For example, our last participation was in a study using argatroban in children. It is not FDA approved for pediatric use. However, Katie developed HIT. Thus, she could not have heparin. She had just started coumadin the day before. I did, through my asking, find out that it was sponsored by the company, which sent up a red flag, but it was FDA approved in adults, and we had no other real options, and argatroban would have been our only choice whether we decided to participate or not, so why not? She was number 11 in that national study. NOt comforting, but I did consent knowing all the facts, and she did well with the argatroban.

Another study we declined because when I inquired about what it would entail, the resident said that when Katie had blood draws in the hospital, they would just draw an extra vial for the study. When I inquired if that was it then, she reluctantly revealed that it would also involve monthly blood draws for six months post-op. She kept pushing the "at no cost to you" aspect, but she was talking dollars, not emotional trauma for a three year old to undergo six additional blood draws that were otherwise unwarranted. We were not on coumadin at the time. Now it would not be a big thing, but back then I was not about to subject Katie to an additional six blood draws just to be someone's guinea pig.

Okay, I'm rambling, but this is a very valid point that you bring up.

Hugs. J.

 

[MikeHeim]

I have somewhat mixed feelings on the subject and posts above. First of all, no one should EVER be subjected to a medical trial that is not explained to them compltely and they consent to. I don't think anyone could seriously argue with that.

I do, however, take exception to the assertion that doctors subject patients to new treatments "willy-nilly". Unless I am given examples otherwise, I have to work under the assumption that medical professionals are doing their best to help us and move forward with new treatments only when they believe them to be effective and appropriate. As a worker in the medical device manufacturing industry, I have a pretty solid understanding of just how difficult it is to get a new product approved by the FDA for a full-scale clinical trial. Human clinical trials are only used as a final confirmation for a new product prior to launch - the manufacturer already needs to be able to demonstrate 100% effectiveness in benchtop trials and (typically) animal testing before getting approval to go ahead with the trial. Typically this does not happen until several years after the concept and prototype phases have been completed and the design is at a point where it is already well-established and proven. This certainly does not mean that these new products never fail and that there is absolutely no risk. However, I believe the FDA does a very good job of screening these products and mitigating the risk for the patient as much as is humanly possible.

Remember, someone had to be the first person to get implanted with a St. Jude, On-X, or Medtronics Freestyle Porcine Valve.

Just my $0.02.

 

[Nancy]

I agree with your point of view on the subject, Arlyss. As a matter of fact, I just posted another thread about medical ethics of a different kind.

In this day and age where there are specialties in medical ethics, one would assume that all medical things would be done in an ethical way.

Unfortunately, this is not always the case. And we all know the old saying about assuming

 

[Arlyss]

Thanks to everyone for sharing your thoughts and experiences on this topic. Patients and their family members are vulnerable, and those who care for them are in a position of great trust. The topic of ethics is an important one for us to consider.

I understand that there is a system in place that is responsible for ensuring that studies and trials involving human beings are carefully controlled. The mechanisms are in place, but I am concerned that in the examples I mentioned, patients and families would have needed to ask many questions to fully understand everything. It is a difficult thing to do, to ask these questions, when one enters a rather foreign world for perhaps the first time. Especially if you have an aneurysm in your chest. From what I was told, the information offered to these people presented the most positive view, favoring their participation in the study. We should have balanced information, pros and cons. If it is not offered, we must pursue it. We can educate ourselves ahead of time, so we are prepared to ask questions. We certainly do that for other aspects of our lives.

Janet raises an important point about asking who is funding a study. Physicians do disclose any financial relationships that they have with vendors and companies to each other when presenting papers at conferences. (They are not prohibited from having these relationships.) However, we as patients will typically not be aware of this information. I would also wish for more transparency regarding financial arrangements that physicians and hospitals have that might influence what is offered to me as their patient. For example, if a physician is a stock holder in a particular company, I would like to know, especially if he/she is recommending this company's product(s) for me.

Yes, someone has had to be first with all of the advancements in treating heart valve and aortic disease. Recently I visited the heart valve museum at Edwards Lifesciences. It was a wonderful, deeply moving experience, and it gave me many thoughts walking through the exhibits and thinking of what it has meant to come this far. My husband, like many others, would not be alive without the progress that was made over those years. My hope is that the progress still to come will be done with the best interests of patients always as the first consideration. And we can do our part to make sure that is the case, as so many on this forum do, by researching, asking, and actively engaging in the process along with the physicians who we entrust with our care. When the patient is first, our questions will always be welcomed.

Best wishes,
Arlyss