Costs of MPN Clinical Trials May Hinder Enrollment

Clinical trials are leading the way in developing new treatments for hematologic malignancies, but the cost associated with them may be holding many patients back from participating, according to a recent international study that was published in the European Journal of Hematology.

The observational study that was conducted in the United States, Spain and Italy examined 143 consecutive patients with myeloproliferative neoplasms (MPNs) to find out what kind of economic burdens came along with clinical trial participation.

In the group, 51 percent had myelofibrosis, 36 percent had polycythemia vera and 13 percent had essential thrombocythemia. Sixty-eight percent were from Italy, 17 percent from the U.S. and 15 percent from Spain.

“Patients are often reluctant to participate in research studies, as this can result in extra time and effort and sometimes money spent on their part,” the study’s authors explained. “In this sense, the results of the present study show significant travel and lodging and out of pocket expenses for patients with MPN being treated in trials.”

The results of the self-assessed questionnaire for the study revealed that 35 percent of patients reported spending more money during their clinical trial than in previous treatments, 21 percent missed work days and 12 percent even reported that they would not have participated in the trial if the financial consequences were known beforehand. Additionally, 10 percent reported that the trial had greatly affected their interpersonal relationships.

However, 91 percent of participants, “believed that participating in the clinical trial was worth the financial or emotional suffering.”

This finding was not different among different income groups.

As new treatments such as janus kinase (JAK) inhibitors and other novel agents continue to be explored in hematologic malignancies, clinical trials — which are increasing in number — are crucial in this field, and patients with MPN are often recommended for them.

Further, “Since once of the ways cancer centers are being rated is the number of patients recruited in trials, oncologists are often subjected to pressure for recruitment,” explained the authors.

But, as the trial found, most patients wish they knew more information beforehand.

“Of note, measures implemented to reduce the expenses have been shown to increase trial participation,” the authors stated.

In fact, 54 percent expressed that the number of visits to the doctor should be clearly expressed beforehand, 60 percent recommended travel reimbursement and 23 percent recommended hotel reimbursement. It should be noted, however, that the average cost of being treated on a clinical trial was higher for patients in the U.S. (45 percent in the U.S., 10 percent in Spain and 3 percent in Italy), where the distance to treatment center was typically farther away.

And while patients clearly expressed through the clinical trial the financial burden that trials put them through, they were not as transparent with their health care teams, as only 28 percent of participants mentioned that they expressed their issues with their doctors.

When asked about approaching drug companies to help mitigate the financial burden, only 11 percent of patients or their doctors said that they did.

Noted by the authors in the study was that more information could be made through the informed consent documents, which typically go into greater detail about potential side effects, but not so much about potential financial and emotional burden from the trial, which can have negative effects on outcomes.

“The results of this present study reinforce the needs for upfront transparency with the patients during the enrollment process in clinical trials,” the authors concluded.