Clinical Trials Are a Perplexing Issue for Many Patients
February 28, 2012 
9 Comments
Despite the prospect of receiving one of the latest new drugs that might extend survivorship, a lack of understanding and access, among other issues, leave many saying “I’ll pass.”
Clinical trials are a crucial component of a drug’s development and ultimate approval by regulatory agencies. On the upside is the chance that a participant will benefit from the therapeutic effect of a new compound that can add months if not years to their life. On the downside, a patient might delay more immediate, already-approved treatments only to find out that they were in the placebo arm of the study and are left to resume their original plan. And then there is the real bonus–finding yourself in a Phase III trial in which the drug meets the study’s pre-specified interim efficacy stopping criteria, demonstrating a clinically meaningful and statistically significant improvement in overall survival compared to the placebo. In that case, something called the Independent Data Monitoring Committee (IDMC) recommends that the trial is stopped and all participants be offered access to the drug. Such was the reality for recent Phase III trials of abiraterone (Zytiga) which was approved by the FDA in April of last year and MDV3100 which is in the pipeline and awaiting approvals.
And then, of course, there are the side effects. In earlier Phase I/II trials, the toxicity and side effects of the new compound may be largely unknown or unpredictable, turning many potential participants away. Even for Phase III trials in which potential side effects are better known, patients may be side-effect-adverse.
I’ve spoken to many fellow patients who have participated in trials. Most of those who elected to participate were glad to do so both for higher, altruistic reasons as well as the hope that they would derive some positive benefit. A good friend of mine, Terry, up in Portland Oregon, was diagnosed shortly after me and was offered the opportunity to participate in a Provenge (immunotherapy) study prior to surgery to see if using the drug earlier in the course of the disease could improve outcomes. Currently, Provege is administered to patients with advanced metastatic disease who have failed hormone and chemo therapies. One year later and declared cancer free, he was very pleased to have been offered the opportunity. (I have to admit I told Terry that I was somewhat disappointed that I missed the same opportunity, having had my prostate removed before the study was open.)
Other patients I know have been enrolled in multiple studies over the course of time and, even though some of the trials did not work for them, they remain grateful for the opportunity. As one patient and friend says, “you don’t want to lose this battle knowing that the right drug for you might have been sitting on the shelf waiting to be tried.” Even though not all of the trials worked for him, he is in his fifth remission ten years after being diagnosed with advanced disease.
I asked Dr. Tomasz Beer, Deputy Director of the OHSU Knight Cancer Institute in Portland Oregon and Professor of Medicine, Hematology & Medical Oncology, to share some of the hurdles to patient participation on clinical trials. (He is the one who ran the pre-surgical Provenge trial at OHSU.) He responded with:
“If you are asking about hurdles, the words “how do I love thee…let me count the ways” come to mind. There are many and I think it depends on where folks are. Hurdles might include access to studies for some folks, knowledge about the options and level of comfort with the idea of experimental therapy for others, types of studies available for others yet. Worries about insurance coverage and costs (whether ultimately proven justified or not) are also important. Probably the single most important thing, though is knowledge and access. Folks that are knowledgeable about their cancer and all their options including clinical trials and who have access to trials where they are receiving care are far more likely to take advantage of studies.”
These are just some of the roadblocks to patient enrollment. I have also heard some patients say that their physicians never brought up the possibility of a trial. Proof again that we need to be our own advocates and partners in treatment.
But Dr.Beer hasn’t stopped with just identifying the hurdles to patient participation. He has just co-authored a new book with Larry W. Axmaker, ED.D., a prostate cancer patient. Titled Cancer Clinical Trials: A Commonsense Guide to Experimental Cancer Therapies and Clinical Trials, the book’s discussion goes a long way to demystify the pros and cons of trials so patients can arrive at a decision that is right for them. Easy to follow, it provides a comprehensive overview of trials with patient perspectives. It’s a resource that should be in the lending library of physcians across the country.
Click here for more information on clinical trials.
For more information on the book, click here.
Dan,
When I balked last year at the Lupron schedule, I did so after radiation treatments had begun. My oncologist told me that had I balked before radiation began, I might have been eligible for a new study regarding Lupron injections, a study that covered the exact situation over which I had balked–length of duration of injections under certain circumstances based on a combination Gleason score and PSA number.
If eligible, I might very well have chosen to join the study.
Tom,
Unfortunately, timing is everything… As I wrote, I would have welcomed a spot in the Provenge pre-surgery study, but alas, my surgery was just a few months too early! As patients, it’s up to us to stay as informed as we can and hope that timing is also on our sides.
Best regards,
Dan
Like Jim I too was diagnosed with Cancer of ptotsare in my 70s and was put onto `Watchful Waiting` and told that I would not `die of it` but that I would `die with it.` I had to retire from nursing after completing 58yrs. but I refused to accept the `watchful waiting verdict` and, as Jim did, I became involved in research advances made in the treatment of cancer of the ptotsare and found that a breakthrough had been made by a brilliant Urological Surgeon in Basingstoke and I contacted him and he offered to take me onto his trial despite my age . It had been long, inconvenient and uncomfortable for me as well as the expense of traveling to Basingstoke and staying overnight but after a year I am free of the curse of Cancer and have only one more review in the New Year and will have no need to attend further if I am still clear. Do not accept your GPs opinion on your condition. Search elsewhere and try to find where there has been an advance or breakthrough made and ask to be involved. GET A LIFE?? Cliff Charlesworth RGN. RMN. RNMH
CANCERcare has a free, hour-long webinar scheduled for Friday, May 18 from 1.30-2.30 Eastern titled” Understanding the Importance of Clinical Trials: What You Need to Know”. To register, go to http://www.cancercare.org/connect.
If you have never listened to a CANCERCare webinar, they are fairly elementary but useful and helpful – more so the less you know. The Q & A can be very informative.
Rick,
Thank you for the webinar infor. It sounds like it could be useful for many.
Best,
Dan
Dan,
Thanks again for your thoughtful comments. Your post, which you were kind enough to let us re-post on our blog, cancer-clinical-trials.com is already the third most popular post in our blog’s history! We are building a community of folks who are interested in learning more about clinical trials and would welcome comments or questions on our blog site.
All the best and thanks for all the good work you do.
Tom
Tom, you are very welcome. I am always ready to take a stand on important issues for fellow patients.
Best regards,
Dan
I am a physician going on two years now with Stage 4, Gleason 8/9, all 12 cores were positive, lots of metastatic lymph nodes. In short, bad disease. Besides being a physician, I am also an ordained Orthodox Rabbi and I can definitely see the hand of G-d on my shoulder as I am currently going through a clinical trial with ippilumimab (Yervoy). This drug was FDA approved for melanoma this past April and now is in Stage III clinical trials for prostate cancer.
My PSA stayed down for a year with Lupron and casodex but then my PSA began to steadily rise again, going higher than when I was first diagnosed. I entered the clinical trial for ippilumimab at the University of Maryland Medical Center and was blessed by G-d in three direct ways:
#1- I did get the real drug and not the placebo,
#2, I know I got the real drug as evidenced by the fact that my PSA has plummetted to barely detectable and on my CT scan large areas of metastatic lymph nodes have shrunken to barely detectable
#3-my side effects have been minimal and tolerable. Someone else in the same study at the same hospital also had his cancer shrink dramatically but he almost died from horrendous side effects
So, clinical trials are important and I feel privileged to be part of one because not only is it helping me, but I feel it will help others as well. But a lot of prayer is definitely needed in hopes of getting the real drug, not a placebo, and in hopes of getting good response without horrible side effects.
I do not know how long this ippilumimab will keep by cancer down but I pray to G-d it is for a long, long time.
If a person is able, I encourage participating in clinical trials. Todd Meister, MD
Thank you, Dr. Meister. Ipilimumab shows a lot of promise for patients. I hope it works well for you for a very long time.