The Tribulations about Trials

The Tribulations about Trials

Don S. Dizon, MD, FACP

@drdonsdizon
Mar 13, 2015

I happened to be standing in her office when Tina, our research nurse, received an email from one of our patients. This patient had recurrent ovarian cancer and was on her third-line of treatment. She was seen at our center for clinical trials, and Tina and I had spent some time discussing one particular trial, the hypothesis around why it was being done, and then the specifics of trial enrollment and the treatment/follow-up schedules. “I am very interested in signing up for this,” she said when we had finished talking.

“I’m glad to hear, and I do think it’s an exciting option,” I said. “However, we are just one of several centers participating on this study. As a result, the availability for this trial is limited, and right now, we are working off of a wait list.”

After much discussion, she opted to join that wait list. I felt she was doing rather well on her current treatment and had no symptoms due to her cancer, and suggested that she continue on the same course for the time being. As an alternative, I mentioned she could take a treatment holiday to regain some strength and experience life without constant therapy. However, she was not comfortable “not” doing anything and hence continued on her current regimen.

Every week following her visit, I became aware that she would email Tina and would inquire about the trial, how it was going, and most importantly, whether there was a spot on it for her yet. In the meantime, one of the drugs made its way from “investigational” to “immediately available” with an FDA approval for recurrent ovarian cancer. I had emailed her that one of these “on-study” drugs was now available, mentioning that—to me,—it was a major milestone for patients and a much-welcomed addition to our medicine cabinet for this disease. While she was overjoyed it was now available, she preferred not to receive it, because she would then be “ineligible for the trial.”

While I welcomed her enthusiasm in wanting to participate in our research study, I was a little taken aback that she preferred a clinical trial rather than starting a drug that was so recently made available. After all, that is the goal of our research efforts—and by “our” I mean the investigators, patients, and yes, the drug companies that invest in newer and hopefully, more effective ways to treat cancer. Not all trials succeed—indeed, a commonly cited statistic is that only 1 in 5,000 drugs make it to market. So, having a drug actually get approved is a big deal—especially when it is against cancer.

Ultimately, it occurred to me that perhaps, I had “oversold” the trial—making it seem so much more promising than anything else available as “standard of care” that she may have felt that her life would depend on participation in a trial. This had alarmed me because the odds being what they are, clinical trials should be treated as that—trials, because they are testing hypotheses, not affirming “proven” treatments.

This explains why trial eligibility often requires that patients receive prior standard treatments and many “cap” prior treatments to a certain limit, too. It also explains why there is a hierarchy or prioritization of clinical trials with phase III trials more important to complete than phase II, which are more important than phase I trials.

To me, this makes sense. A drug that makes it to phase III has been shown to be so promising that a positive clinical trial has the potential to change our standards of care. But, our early clinical trials are more hypothesis-generating or trying to define the activity in specific population (for phase I and II trials, respectively).

I suppose the bottom line is while I am committed to clinical research and believe, as most of my colleagues do, that often the best treatment for a patient with cancer is on a clinical trial, I have a responsibility not to “oversell” it as an option, especially when standard treatments are available.

Likewise, I must work with my patients so that they can better understand that clinical trials are aiming to improve on treatments we have available now. They represent are our attempts to improve approaches to cancer now and for the future. Trials represent hopes for better cancer- not assurance that every investigational approach represents the next best thing. And for me, as for many of my own patients, therein lies the most important point.

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