People in general, and cancer research subjects in particular, are reluctant to participate in clinical trials, a trend that could undermine progress toward treatment, according to a survey of some 1,500 consumers and 600 physicians conducted on behalf of Memorial Sloan Kettering Cancer Center (MSK) in New York City. Only 35% of Americans indicated that they were “likely” to enroll in a clinical trial. Other studies have shown that only 4% percent of cancer patients enroll in clinical trials nationally each year.

That’s particularly concerning, since the vast majority of modern advances against cancer were the result of clinical trials. Clinical research is increasingly dependent upon larger numbers of cancer patients participating, says Paul Sabbatini, MD, Deputy Physician-in-Chief for Clinical Research at MSK Cancer Center. He recently agreed to an interview with IRB Advisor about this critical research issue.

IRB Advisor: It’s surprising that only 4% of cancer patients enroll in clinical trials nationally — do you have a have a reference on that?

Sabbatini: That figure has been reported with a slight variation between 3% to 5%. Recently, we’ve seen Vice President Joe Biden noting 3% to 4% percent participation. [] At Memorial Sloan Kettering, this figure is higher: Nearly one-third of our patients participate in clinical trials. Yet it is imperative that we increase understanding and participation in clinical trials to fuel the research pipeline moving forward — and to be able to offer the best treatments to our patients, now and in the future.

IRB Advisor: Can you elaborate on this critical point in the survey: “Clinical research is increasingly dependent upon larger numbers of cancer patients participating?” Is that in part to find results that can be extrapolated to larger populations?

Sabbatini: There is an urgent need to have more patients enrolling. In fact, this low participation in clinical research could very well be our Achilles heel in curing cancer as we know it. Thanks to the many advancements in precision medicine, researchers are able to sequence more and more genes and to do it at a faster rate. But we must start by having the necessary volume.

Not only does having more patient participation fuel this knowledge, it also helps us explain “anomalies” or extraordinary responders. For instance, a particular new treatment may not work for most clinical trial participants, but could have an exceptionally positive outcome for one or few. Why is this the case and what can this tell us for future patients? There is much more we can uncover on these topics. Research has the potential to help bring better treatments faster, but we need participants in clinical trials to keep the momentum going.

IRB Advisor: After reading a brief statement defining clinical trials, the number of respondents who had a positive impression of these studies jumped significantly, from 40% to 60%. Can you provide the educational statement that had this effect?

Sabbatini: It reads, “Clinical trials are research studies in which patients volunteer to help test new ways to treat, diagnose, or prevent diseases. They are used to determine if a new test or treatment works and is safe. These trials are used for thousands of medical conditions, including many types of cancer. By participating in clinical trials for cancer, you have the opportunity to:

  • Receive drugs or therapies years before they are available elsewhere.
  • Receive the newest treatment being studied (in the majority of cases) in addition to the standard current treatment available.
  • Better manage symptoms or side effects from the treatment of cancer or improve your overall well-being.
  • Receive a higher level of oversight and care.
  • Treatments studied can include new drugs, new surgical procedures, or devices or new ways to use existing treatments or improve them.
  • Typically, one group of the study receives the new treatment in addition to the standard treatment, while a comparison group receives the current standard treatment. Note that regardless of the treatment group you are in, you are free to leave a trial at any time. The costs of the new treatment are typically covered by the clinical trial, while the standard treatment is covered by the patient or his/her insurance.
  • Clinical trials are key in helping physicians develop medical breakthroughs. Nearly all cancer drugs in use today were first tested and made available to patients through clinical trials.”

IRB Advisor: Do physicians respond to education about clinical trials in a similar manner?

Sabbatini: Physicians also reviewed the statement and the majority responded in a positive way, sharing that this type of statement could be helpful/useful; specifically:

  • 68% would be likely to use the statement with patients.
  • 69% feel it would be effective in educating patients.

IRB Advisor: How can “real-world” concerns, like side effects/safety, insurance, out-of-pocket costs, and trial locations be addressed?

Sabbatini: The survey findings illustrate that there is more work to do to help improve clinical trials on several fronts: in the quality of information/education we provide, in increasing access to treatment, and, of course, in providing quality care. Education early on in the process is vital so participants know all of their options and understand what clinical trials are and are not. For instance, many people worry about costs, but the reality is that most clinical trials present no additional out-of-pocket expenses for the treatment itself. Others worry about getting a placebo, but the vast majority of clinical trials do not use a placebo. The good news is that many of these concerns are simply misperceptions that can be addressed through education.

The location concern, however, is not a misperception, but it is still valid. Memorial Sloan Kettering is pioneering a new model — in the NYC area and beyond — to expand outpatient treatment and surgical centers, as well as to establish partnerships with regional providers. These efforts will enable more people in more locations to have access to best practices in medicine and, in some cases, to the very same clinical trials that were previously only open to patients close to MSK’s main facility in Manhattan. Patients want high-quality clinical expertise, but they also want options that are convenient and close by — so we must work to bridge this gap.

IRB Advisor: The “guinea pig” misperception may be rooted in reports of unethical research in the past, but were you surprised that more physicians (53%) were concerned about this than consumers (34%)? What do you think may be the issue there with so many docs having a concern about the experimental aspect of trials?

Sabbatini: To clarify, we asked physicians what they think are the biggest concerns for their patients when considering participation in a clinical trial for cancer treatment. Their responses included side effects/safety (63%) and concern about getting a placebo (63%). Additionally, when we asked consumers about their main deterrents when considering participating in a clinical trial for cancer treatment, they pointed to side effects/safety (55%), potential costs (50%), location of treatment (48%) and worries over getting a placebo (46%).

IRB Advisor: Many physicians seem to also view clinical trials as late-stage or last resort interventions, but you’re saying that there are clinical trials being conducted on much earlier stages of cancer?

Sabbatini: Yes, the research stresses the importance of educating both patients and physicians on the benefits of clinical trial enrollment at every/any stage of the diagnosis, not just as a “last resort” option. Of the almost 600 physicians polled, more than half (56%) of physicians said they considered clinical trials late in treatment, with 28% noting them “as a treatment of last resort.” Only one-third (32%) said they discuss the topic with their patients at the beginning of treatment. However, it is important to evaluate clinical trial enrollment every time a change in treatment is considered.

With more than 900 trials underway, Memorial Sloan Kettering runs one of the largest clinical trials programs in the country, with trials available at almost every stage of treatment. To us, the survey data reflects a possible missed opportunity on behalf of patients. Clinical trials can offer our very best thinking on how to discover newer, better ways to treat cancer — especially early on.

IRB Advisor: How can the cancer community specifically address these common myths and misunderstandings around clinical trials, particularly in physicians?

Sabbatini: The burden of education is on all of us within the medical community, but it is not insurmountable. The survey results indicated that only 40% of Americans have positive impressions of clinical trials. Yet once consumers read a brief statement describing their real purpose and function, favorability jumped to 60%. This desire for education is both an opportunity and a challenge that we must embrace. We want to start a national dialogue in which other institutions and providers can take part. Speaking with a collective, collaborative voice is the best way to ensure that the message is being heard. And as we heard from Vice President Joe Biden during his recent visit to MSK [May 26], breaking down barriers to scientific collaboration and being able to share data is critical to achieve the “Cancer Moonshot.” Curing cancer as we know it is a larger scale problem that we believe we can solve, working together with other leading cancer centers, researchers, and the most important group: the brave patients who choose to participate in clinical trials. They are the true heroes.