Adults with cancer enter clinical trials at a dismal rate of 3 percent or less, with even lower participation among minority populations. To improve this absurdly low number it is time that we look at this life-and-death decision from a patient’s perspective, a viewpoint that is rarely considered in trial design.
Cancer patients know they have a potentially terminal illness, have often failed first and second therapies, and are desperate for better treatment of their disease. Why wouldn’t they be clamoring to join trials in large numbers? Assuming they meet inclusion criteria, why wouldn’t the vast majority be anxious to participate?
Without answering those basic questions and making the process more focused on the needs of patients, clinical trials will continue to get inadequate accrual numbers.
One would think that the immediate intent of clinical trials is to help patients, but that is not correct. Trials are performed to answer specific scientific questions. It is wonderful if patients are helped, but the purpose of any given trial is to obtain a statistically valid answer to the question raised.
By the time cancer patients have failed their first or second courses of therapy, most have a good understanding of their cancer. They have searched the Internet, sought second opinions, and have definite ideas about their next potential treatment. These educated patients should be prime candidates for clinical trials.
Suppose a patient truly researched a potential new treatment and has found a trial utilizing the new agent. In discussing it with the principle investigator, imagine their profound disappointment when he tells them they have a 50/50 chance of getting the experimental treatment or the standard treatment for their second or third relapse. The principle investigator patiently explains that no one knows which arm of the trial will do better. In his heart of hearts, however, he has his opinion, and so does the patient.
Whether it is right or not, the patient has an opinion and that opinion should have standing! Give patients all known information to date regarding the experimental treatment, and let them decide which arm they want to be part of. Accrual numbers will increase dramatically with patients who finally feel empowered with some degree of control over the cancer inside them.
Yes, I know that is statistical blasphemy. Anything less than the gold standard, the randomized clinical trial, would not count as a proper trial. Bias would make the whole trial suspect or worthless. The trial would not have statistical validity. I have heard this answer dozens of times.
I have also watched dozens of good trials close early because they did not attract patients. At the 2010 American Society of Hematology meeting, there were at least 10 promising new agents for myeloma, but there are not enough patients attracted to the clinical trial process to even test these treatments.
As it is now, patients are left with the feeling that their clinician is offering them a choice of treatments based on no more than a coin flip—which is exactly what he or she is doing. When patients have strong feelings about what they want for their next course of therapy, it is no wonder that they are completely turned off by such an approach.
Cancer patients are more than experimental “subjects.” They are not lab animals who have no reasoning ability, but are humans who should be allowed to make their own value decisions—right or wrong.
Some patients will be willing to accept side effects and potential unknowns of the new experimental treatment. Others, however, will be quite content to be in the control arm and ‘stay with what is known.’
The decisions of both groups should be accepted, and their numbers should be added to the corresponding trial arms. After their choice of arm, they should be treated and followed just as if they had been randomized by computer.
Statisticians will cringe and say the whole trial is worthless, despite the fact that accrual numbers would soar.
Consider, however, the woeful ineffectiveness of our current clinical trial system. What good is a statistically perfect, well-designed trial if nobody shows up? Our current system is broken and needs new approaches to randomize patients based on their informed right to choose their own treatment. This profound decision affects their life and their cancer. Treatment should be their choice.
Having personally known myeloma for 13 years, I have definite opinions regarding what my treatment course number 5 will be when it is time to make that decision. Like so many of my fellow patients (97 percent), I would refuse to be randomized and leave this life-and-death decision to the flip of a coin.
To all who denounce such an unscientific approach, please outline an improved system based on a patient’s absolute right to choose his treatment. We can ‘think outside the box’ and use a different approach, or we can keep the same system and get the same results—3 percent participation with many drop-outs. Without a change, we will continue to observe that only a tiny minority of clinical trials open, accrue, close, and are reported in a timely manner with useful information for clinicians and cancer patients.
Statisticians can take nearly any set of numbers, apply their formulas, and make pronouncements regarding those numbers. I contend that they can also devise statistical systems that provide relevant answers to outcomes of trials that are based on the right of cancer patients to choose their own treatments.
Jim Omel MD is a retired family physician and 13+ year myeloma survivor who is active in many areas of cancer patient advocacy.
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