ESMO 2017: Patients Struggle With Key Aspects of Clinical Trial Methodology

Key Points

  • Over half of previous medical trial participants, and 73% of those who had never been on a cancer clinical trial, did not understand that in a randomized trial, the treatment given was decided by chance.
  • We also found that most patients did not understand clinical equipoise. This was more marked in previous clinical trial participants, 60% of whom believed that their doctor would know which study arm was best.

Clinical trials are fundamental to the development of new treatments for cancer, yet the annual accrual to cancer clinical trials worldwide is low, estimated at 3%–5%. A nationwide study in Ireland, the preliminary results of which were presented at the European Society for Medical Oncology (ESMO) 2017 Congress in Madrid, showed that although most oncology patients consider it important to have clinical trials available, many struggle with the central concepts that underpin trial methodology (Abstract 1465P_PR).

“As a medical oncologist, I have experienced situations where patients have declined clinical trial options because of misconceptions about them,” said study author Catherine Kelly, MD, of Mater Misericordiae University Hospital in Dublin, Ireland. “To improve participation in clinical trials, we need to understand the factors influencing patients’ decisions about taking part.”

Study Results

In the course of the study, 1,090 adult patients with a diagnosed malignancy and being treated at one of 14 participating oncology centers across Ireland filled out anonymized questionnaires in which they were asked to evaluate statements about clinical trials and research.

“Consistent with previous studies, the concepts of chance and randomization posed difficulties to a significant proportion of patients. Over half of previous medical trial participants, and 73% of those who had never been on a cancer clinical trial, did not understand that in a randomized trial, the treatment given was decided by chance,” Dr. Kelly reported.

“We also found that most patients did not understand clinical equipoise: the fact that no one knows which treatment is best. Surprisingly, this was more marked in previous clinical trial participants, 60% of whom believed that their doctor would know which study arm was best,” she said.

“To provide informed consent when participating in a trial, patients need to understand these key concepts—and doctors explaining them well is essential to alleviating any fears that might prevent patients from participating. For example, many didn’t realize that clinical trials are not just an option for when standard treatment has failed,” she observed.

“Doctors have a responsibility to properly inform their patients in this regard, because they are the ones patients trust the most,” Dr. Kelly said. “As we analyze the data further, we will be able to offer physicians a more detailed picture of the questions patients need answered, and the factors that influence their decision-making according to age group, cancer type, educational background, and other demographics.”

Commentary

Bettina Ryll, MD, PhD, Chair of the ESMO Patient Advocates Working Group (PAWG), commented, “The question of whether patients understand clinical trial methodology is a very valid one, and what makes this study so interesting is that more than a quarter of the patients questioned had actually been on clinical trials before,” she said.

“However, I was surprised at the median age of the cohort: 60 years. It would be interesting to compare the data collected here with younger patient groups, who access information in a very different way,” Dr. Ryll observed. “I would also expect to see differences across tumor groups: among breast cancer patients, for instance, who make up almost a third of the study cohort, and for most of whom there is a well-established standard of care, clinical trials are likely to be of less interest than among lung cancer patients, for whom the standard treatment is less effective.”

Dr. Ryll further cautioned, “When we talk about understanding, it is important to consider that patients and physicians approach clinical trials from different perspectives: for example, the concept of randomization is one that many patients question from a moral standpoint. Equipoise, by contrast, may be a laudable moral concept, but it is difficult to uphold if the results of earlier trials are already known: finding out whether a treatment is, say, 51% better or only 49%, may matter to a Health Technology Assessment assessor—but not to a patient. This undermines the conclusion that patients simply do not understand equipoise.”

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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