Advocates Speak for the Severely Ill

The peril is great. The promise, initially, is small. And the fears can be overwhelming.

But desperate patients sign up for medical experiments anyway, willing to be human guinea pigs, clinging to the thinnest sliver of hope that the latest discovery might save their lives.

It’s such eagerness that puts vulnerable patients into the hands of doctors and scientists keen to test new ideas and treatments. Patients are needed for these tests, but do they know what they’re getting into? Not always, said ethicist Haavi Morreim, which is why she has helped create a system that paired a ‘patient advocate’ with the first volunteer to receive an implanted mechanical heart.

Patient advocates have been used before, but usually as part of a hospital or research team’s staff, not as wholly independent outsiders. In a sense, this new, independent advocacy system resembles the use of public defenders in court. The patient advocates are paid expenses and fees from an irrevocable trust fund, and they are available to help sick people understand a clinical trial and decide whether to participate and when to leave.

The goal, Morreim said, is to make sure patients understand the hazards, the potential benefits, the alternatives, and what it will mean to be the subject in a daring and dangerous experiment. Patients in experiments such as the heart trial risk extreme discomfort. Because they are already so sick, death is likely.

The advocate’s role includes ensuring that a patient “is not subjected to inappropriate pressure” from doctors or scientists, Morreim said, and advocates can advise patients and their families to opt out of the experiment.

The danger that ethicists see for patients is their emotional vulnerability. In the desperate quest for help, patients tend to minimize or dismiss the hazards, while magnifying the hope, focusing on even tiny hints of benefit mentioned by doctors. “The mind is not prepared to perceive the problem” when it’s presented in such enormity, Morreim said.

And because researchers have invested years of work and hope in a new idea and are enthusiastic about the possibilities, they may unconsciously oversell what they’ve got.

Indeed, the first testing of the artificial heart is daring. Only patients who are very sick will be selected, and they will have to submit to having their heart removed and an electrically powered device stitched onto the small part of the heart that remains. The mechanical heart, made by Massachusetts-based Abiomed Inc., has worked quite well in dozens of young cows, but only now has the first test in a human volunteer begun. The FDA has approved the device for four more human tests.

Morreim was recruited a year ago by Abiomed, executives said, specifically to make sure there were no ethical barriers to running the experiments. An Independent Patient Advocacy Council was established for the first five heart implant experiments. Morreim was in Louisville, Ky., to make sure the first patient and the first advocate got together before the July 2 surgery.

“We want to be sure there is an advocate available for each patient” in the critical heart tests, Morreim said. Deeply involved in ethics issues, she teaches at the University of Tennessee Health Science Center in Memphis.

To make sure the patient advocates are wholly independent, Abiomed established the irrevocable trust fund, though company officers would not specify the amount of funding. Eight advocates have been recruited and are ready to assist patients as they enter trials.

Morreim, as leader of the advocacy project, has done much of the recruiting. The advocates hold occasional conferences to compare notes, she said, but they emphasize the idea of independence. They have no formal ties to the company or medical centers, but they have experience working with patients, doctors and ethical issues.

“That sounds like the way to go,” said Robert Cook-Deegan, an ethicist working at the Kennedy Institute of Ethics at Georgetown University in Washington. Cook-Deegan, who is not involved in the project, added, however, that he has not studied the Abiomed advocacy idea in detail.

Abiomed’s first test was on an unidentified man in his mid-to late 50s. He was reported doing better than expected in the first week after surgery.

Doctors said the patient was very sick when he arrived at Jewish Hospital, not expected to live beyond 30 days because of congestive heart failure. He underwent surgery twice more in the week after the implant, to stop bleeding from stitches and to remove fluid accumulating near his new heart.

More fluid was drained in a third surgery last week.

If he manages to survive 60 days or even longer, this first test of the implant heart will be considered a success, opening the door for even more patients to try it. In time, the people at Abiomed hope the heart will be widely used to save lives. The heart and its battery-pack power system are designed for use at home, so it can readily be plugged into an electric outlet to recharge when the batteries run down. Patients would carry spare charged batteries when away from home.

This pioneering test of the device is also the first test of the Independent Patient Advocacy Council. “It has gone wonderfully in terms of receptiveness, not only of the institution [Jewish Hospital], but also the patient and his family,” said Dr. Douglas McNair, who came up with the idea for the patients’ advocates, arranged the trust and keeps track of the program. “What I’ve been told by the advocate is that he felt very good” with how the interaction progressed.

It was McNair, vice president for clinical research at Abiomed, who insisted three years ago that truly independent advocates be recruited to help patients who were in line to get artificial hearts.

McNair said that when the first patient and his family were offered the advocate’s help, “they gladly asked for a discussion with the advocate. They had a series of discussions throughout several days that were detailed and extensive. It seems to be a useful addition to the normal informed-consent process.” In fact, Morreim said, this first human test of the mechanical heart “sets an excellent precedent” for the advocacy system.

Morreim and McNair would not identify the advocate in Louisville or the advocates they’ve recruited at other medical centers, because of the need to ensure patient privacy. They worry that if hospital staff members learn who the advocates are, their visits to hospitals would perhaps reveal some patients’ identities against the families’ wishes. Morreim and McNair said it has taken time to find and recruit the advocates, because these people need a combination of talents, must have enough time for the tasks at hand, and have to be extraordinarily compassionate and understanding. They must also have deep experience within the medical system, either as doctors, nurses or in allied occupations.

“It depends on our people having a deep personal devotion to how people in these life-threatening situations are cared for,” McNair said. “And each advocate has a history of commitment to care decision-making. Some of them are retired and are volunteering, but others are 30 or 40 years old, individuals whose professional interests have straddled the boundary between curative and palliative care.” Morreim said most of those recruited “jumped at the chance to participate, because advocacy could be such an important thing to introduce to research.” But the advocate’s job is not clear-cut. An independent approach funded by private money hasn’t been tried before. So, Morreim said, “we’re kind of making it up as we go along.” Federal rules already require research institutions using government money to set up “institutional review boards” to oversee human experimentation, but the members typically include hospital staff and a few outside “public” representatives.

In contrast, Abiomed’s patient advocates are intended to be completely unfettered by the company or the institutions involved in the experiments.