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.

 

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An ER Specially for Cancer Patients — Created Thanks to Patient Partnered Input

A couple of days ago, I had an acute pain incident and was trying to decide if it might be heart or stomach related — either a possible but probably not likely byproduct of my medical situation and treatment.  I (correctly it turned out) did not call in because I did not want ER hassle.  But now I know that my hospital, Hopkins, has a relatively new service that would have fit the bill — and indeed one set up at the urging of a fellow member of the  Hopkins Oncology Patient and Caregiver Advisory Council.   The Cancer Urgent Case service is described in an article in the Baltimore Sun, here by Meredith Cohn.

Most cancer patients going through treatment struggle with pain, fevers and nausea, sometimes severe enough to send them to the emergency room.

But the chaos of the emergency room and related stress are “an assault on your well-being,” said Richard Dean, who took his wife many times while she was battling ovarian cancer six years ago. He told Johns Hopkins Hospital officials they needed an urgent care center especially for cancer patients.

“Anyone who gets cancer discovers it’s just a messy disease,” Dean said. “Your white blood count runs down, you get a fever, and you’re at really high risk for infection and you go sit for hours in a waiting room with a bunch of kids with the flu.”

Hopkins listened and, using some patient data crunching from Dean, who teaches engineering at Morgan State University, launched a cancer urgent care center in 2014 in space adjacent to where chemotherapy is delivered. The Hopkins center is open 12 hours a day during the week and plans to open on Saturdays starting in March.

The center now sees about 10 patients a day — about 3 percent of those Hopkins treats for cancer on an average day — and most go home after a few hours.

“We knew sending our patients to the emergency room was not in their best interests,” said Sharon Krumm, director of nursing administration at the Johns Hopkins Kimmel Cancer Center. “If they have a heart problem or a stroke, yes, it’s a good place for them, but that’s not what was happening.”

The urgent care center’s patients don’t just get better, more efficient care, Krumm said. A study released this week suggests that avoiding hospitalizations is significantly lowering costs for patients and reducing the burden on the health care system.  .  .  . Hospitalizations accounted for up to 46 percent of the patient’s bill. The study found that among a group of patients at Hopkins, the average number hospitalized was cut in half after the center opened.

The article concludes with a great explanation of the need for patient partnered care.

Dean said his wife always wanted to be at home in her own bed in Columbia. Now serving on the Hopkins Patient and Caregiver Advisory Council, Dean crunched patient data to suggest how many beds the Hopkins center would need and how it should be staffed.

His wife died before the Hopkins urgent care center opened. But his daughter Samara, who also was diagnosed with ovarian cancer, has visited the center.

Dean called it comforting just to know the center was there and looks forward to it adding weekend hours. He’d also like to see community hospitals pool resources to support a center if they can’t manage one on their own.

“Patients and caregivers really become the experts on what it’s like to live in a system,” he said. “I have great regard for the folks at Hopkins, but they don’t have the eyes that I have. When you’re in the system you can see the gaps. And now a big gap is being filled.”

As described in the article, there are now ten such cancer urgent care centers in the country. What we, as patients, need is so different from general ER services — and not just a shorter wait.  Our needs are more focused, more comprehensive, and with different likely tests upon arrival.  Interestingly, upon arrival we probably often present as much less urgent than others arriving.

The potential impact for this kind of service is graphically highlighted by a recent story in the New York Times about the emerging knowledge of the risks of immunotherapy.  One individual’s story, which would surely have been very different with a capacity like the one described above is detailed here.

Mr. Peal, an engineering technician who tests the performance of helicopter parts, started taking nivolumab and ipilimumab [new immunotherapy drugs] on July 8. Dr. Kluger [at Yale] told him he might feel drowsy or nauseated, or he could get a rash. A rash indeed struck with a vengeance on Aug. 30: red welts from his knees to his waist. On Sept. 1, a Thursday, he visited Dr. Kluger’s office, where he was given a steroid.

The next day, he had a fever, nausea and was “dying of thirst — like beyond being in the desert,” he said. He threw up everything. His girlfriend, Jo-ann Keating, called Dr. Kluger’s office, and an on-call doctor prescribed an antinausea drug. Later, Ms. Keating called back to say it was not working, and he was prescribed a second antinausea drug. By Sunday morning, Mr. Peal, unable to move, took an ambulance to the emergency room.

In his wallet, he kept an information card published by Bristol-Myers Squibb. It lists dozens of risks, including that the therapy “can cause serious side effects in many parts of your body, which can lead to death.” Mr. Peal’s family told the emergency room doctor about the treatment, Ms. Keating recalled.

“The doctor kept on saying he was on chemotherapy,” she said. “I said, ‘They’re calling it immunotherapy.’ He went on his phone and started looking for information.”

But even Dr. Kluger’s experienced team, which answered the distressed phone calls that weekend, was caught off guard and did not react immediately to the symptoms.

“It took us by surprise. He looked absolutely fine on Friday,” Dr. Kluger said. Part of the problem, she thinks, is that Mr. Peal was relatively new to the clinic, and so she and her staff members did not have the experience with him to accurately assess his symptoms. “It also happened very quickly. It spiraled within hours.”

Ultimately, Mr. Peal spent 24 days in the hospital, where trouble mounted. First his pancreas failed, then his bowels inflamed and his kidneys became dysfunctional, and “to top it off, he has a fever of 103 for which we can’t find a source,” Dr. Kluger said in an interview during the crisis. She was trying to figure it out and had emailed other experts around the country to see if they had ever had a patient with this combination of acute immune reactions. No one had seen it before.

Even without a formal new service, its clear to me that those in research trials should have not-understood side-effects responded to quickly, in case they escalate.  I would think that protocols for this should be established or rather tightened.