What should the criteria be to enter a cancer clinical trial?
When 29-year-old Carly Bastiansen was diagnosed in January 2016 with advanced pancreatic cancer, doctors told her a clinical trial was her best shot at slowing the notoriously quick-killing and hard-to-treat disease. She found one that appeared promising and went through the screening process. But the trial would not accept her.
“Participating in a clinical trial is really my only chance at living longer,” Bastiansen, a children’s librarian in Baltimore, said this fall as she was growing weaker. “To have had that option taken off the table was devastating.”
Bastiansen was ruled out of the trial because she did not meet its eligibility criteria, which include many characteristics — for example, age and the type and stage of cancer — that all trial participants must possess. Lab tests showed that her bilirubin, a compound in the blood that is predictive of liver function, fell just outside the normal range, raising the red flag that barred her enrollment.
She refused to give up and not long ago took a leave from work to devote all of her energy to finding a trial that would enroll her. “The researcher heading up the second trial I applied to said I was ‘too sick’ for his trial,” Bastiansen said.
Her unsuccessful search — six trials would eventually reject her — help illuminate a debate over the eligibility criteria that are part of every clinical trial. Many doctors and researchers argue that the criteria are too narrow and should be expanded to include more patients who have exhausted standard treatment options or who, like Bastiansen, never had any to begin with.
Clinical trials test whether a treatment is safe and effective, a prerequisite for Food and Drug Administration approval. Eligibility criteria are typically written by the trial’s sponsor — usually a pharmaceutical or biotech company — or the trial’s investigators, and they serve two purposes: They protect study participants, particularly those thought to be at greater risk, from experiencing unnecessary harm. They also ensure that participants are similar enough that any changes they experience during the trials are caused only by the drug under investigation.
“Otherwise it would be impossible to know, for example, whether a patient experienced a cardiac event due to a side effect of the drug under study or due to an underlying health condition,” said David Gerber, a lung cancer specialist at the University of Texas Southwestern’s Simmons Cancer Center.
Despite the essential role of eligibility criteria in clinical trials, many oncologists believe that such guidelines are overly restrictive, resulting in too many patients being barred from potentially life-extending treatments and helping to keep the nation’s cancer clinical trials participation rate hovering at 3 percent.
“Everyone thinks it’s time to take a hard look at eligibility criteria, especially doctors who have to look their patients in the eye, after telling them about an exciting new trial, and explain to them that they aren’t eligible to participate,” said Mikkael Sekeres, director of the leukemia program at the Cleveland Clinic’s Taussig Cancer Institute.
But that has proved easier said than done. While Sekeres advocates for “more rational eligibility criteria,” he also says that defining a study’s eligibility criteria is an “inherently complicated and high-stakes process.”
|Read Full Article: Cancer clinical trials exclude many desperate patients. Should that change? – The Washington Post|