NPR Piece on Value of Patient Input Into Research Design

Jill Harrison of Planetree pointed out this nice piece on NPR on the value of bringing patients into the research design process.  It reports on a study testing the approach of classifying and treating malignancies by genetic type rather than initial site.  The report says that the study is having great success in participant recruitment in part because its design incorporated the patient voice.

.  .  .  [T]he researchers who designed the study stopped to ask what would appeal to potential participants. Nancy Roach, a longtime patient’s advocate who lives in rural Oregon, got involved early on, and helped advise the scientists planning this study.  .  .  .

“This is going to sound goofy, but my dad was in advertising,” she tells Shots. “Remember the scrubbing bubbles — Dow scrubbing bubbles? That was my dad. So I grew up watching commercials and thinking about what consumers wanted.”

Roach brought that sensibility to the conferences where the NCI-MATCH trial was being designed. The original plan would have split the study participants who seem to be doing well on the test treatment into two groups. One group would continue the treatment; the other would take a break, called a drug holiday.

I’m not a scientist. I’m not a clinician. I’m there on behalf of patients.” Roach remembers her immediate reaction to that design: “Taking a patient who’s responding to treatment and taking them off treatment? That is not going to fly.”

She correctly anticipated how patients like Nancy Nahmias would have reacted, as they deliberated whether to sign up for the trial.

“I would not have liked that,” Nahmias says. “If it seems to be working, let’s face it, I don’t want to do anything to sabotage myself.”

Dr. Peter O’Dwyer, a University of Pennsylvania oncologist who was involved in the study design, readily admits that “the design had certain attractions, but it clearly had certain flaws.”

As the patient advocate Nancy Roach, points out:

From Nancy Roach’s perspective, it takes a bit of nerve to speak up in a room of doctors and scientists and ask, “Will the results of this study actually help anybody?”

But it’s Roach’s responsibility to ask those basic questions. “I’m not a scientist,” she says. “I’m not a clinician. I’m there on behalf of patients.”

In order to ensure clarity about the extent and stage at which the patient input occurred, I reached out directly to the researchers for some additional information.  This was their response:

Patient input was part of the process from the outset, when the NCI-MATCH trial was still only a concept. A group of cancer research advocates and patients, including Nancy Roach the person quoted by NPR and several others, discussed the pros and cons of the scientific rationale for the “drug holiday” in detail with study leaders, among  various approaches. It became clear that the patients and their advocates were not in favor of this approach. As a result, the idea was abandoned and did not become a part of the trial design, which proceeded to formal and final review by federal regulatory authorities, including the NCI Central Institutional Review Board.