Feb 29, 2012

Guest Post: Clinical Trials Are a Perplexing Issue for Many Patients

We asked a number of thoughtful people to read a pre-publication copy of our book and give us some feedback.  We are incredibly fortunate to have the counsel of Dan Zenka.  After looking at the book, Dan posted the following on his blog and allowed us to re-publish his thoughts here to share them with you.

Dan Zenka is senior vice president of the Prostate Cancer Foundation, the world’s leading private supporter of research for a cure. Two years after joining PCF, Dan was diagnosed with his own case of prostate cancer. Following a radical prostatectomy, it was determined that he had metastatic disease. He has undergone seven weeks of radiation therapy and is currently on two to three years of hormone deprivation therapy. Just days after his diagnosis, Dan started his own cancer blog, www.mynewyorkminute.org where he discusses a range of issues related to navigating prostate cancer and is read by a growing audience of men and their caretakers around the world.  What follows are Dan's words:

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.
For more information on the book, click here.


  1. Another issue/decision involved in the participation decision, is future exclusions. For instance, participating in the Ipilimumab trial, may be an exclusion for future and perhaps more effective immunotherapy based treatment trials. Likewise, one might delay a currently available treatment, ie: Provenge, which might exclude participation in a future immunotherapy based clinical trial.
    I would also add that by the time a trial is phase 3, there is already a decent amount of information available regarding efficay & side effects, to those willing to spend time searching the internet. At that point, a patient really should scrutinize how the drug's earlier trial participants faired, ie: % of responders, degree & % of side effects, effectiveness vs stage of disease, ect. - and weigh the promise of the trial drug, given one's own stage of progression vs. the risk of being in a trial with a placebo arm.

    1. A really great comment. In the clinic we are always trying to think several steps ahead to foresee the impact of a particular clinical decision on subsequent clinical trial eligibility. Not always possible to anticipate every turn of events, but this is always something we think about. We want to keep as many doors open as possible.