November 4, 2010
Listening to Patients Living With Illness
By PAULINE W. CHEN, M.D.

Wiry, fair-haired and in his 60s, the patient had received a prostate cancer diagnosis a year earlier. When his doctors told him that surgery and radiation therapy were equally effective and that it was up to him to decide, he chose radiation with little hesitation.

But one afternoon a month after completing his treatment, the patient was shocked to see red urine collecting in the urinal. After his doctors performed a series of tests and bladder irrigations through a pencil-size catheter, he learned that the bleeding was a complication of the radiation treatment.

He recalled briefly hearing about this side effect three months earlier, but none of the reports he had been given or collected mentioned it, and once he had recovered from the angst of the emergency room and the doctor’s office visits and the discomfort of the clinical work-up, he didn’t give it more thought — until a few weeks later, when he started bleeding again.

By the time I met him, he was in the middle of his third visit to the hospital. “I feel like I’m tied to this place,” he said. He showed me a plastic jug partly filled with urine the color of fruit punch, and he described a post-treatment life marked by fear of going to the bathroom and discovering blood. “If I had known that my life would be like this after radiation,” he sighed, “I would have chosen the surgery.”

Whether conducted at a laboratory bench or in clinical trials, medical research has long been driven by a single overriding goal — the need to find a cure. Usually referred to more modestly as a search for “the most effective treatment,” this standard has served as both a barometer of success and a major criterion for funding. Most published studies are marked by a preponderance of data documenting even minor blips in laboratory values or changes in the size of a spot of cancer or area of heart muscle damage on specialized X-rays. Some studies bolster the apparent success of their results with additional data on societal effects like treatment costs or numbers of workdays missed.

Few studies, however, focus on the patient experience.

The result of all this emphasis on cure has been a nearly embarrassing richness of choice for patients with diabetes, heart disease, H.I.V. infections and even some cancers. The increasingly sophisticated treatment regimens that now make up medicine’s armamentarium have transformed once life-threatening diseases into chronic ones, but in the process have given rise to a group of individuals who are caught in the unenviable position of living with an illness that never quite goes away. No longer forced to make once-in-a-lifetime life-or-death treatment decisions, they instead struggle each day with the side effects of the very treatments that keep them alive.

For many of these patients, the most important question becomes not “What treatment will allow me to live the longest?” but rather “Which treatment can I live with?”

Now, according to a paper published last month in the policy journal Health Affairs, the Patient-Centered Outcomes Research Institute, a new initiative mandated by the federal health care overhaul, may offer a chance to alter the prevailing winds of medical research so the needs of this growing segment of the patient population are addressed. With a 21-member board made up of individuals from both the public and private sectors, the institute has a mandate to set the agenda for research comparing treatments for common diseases and to finance the most important studies. But according to the paper’s authors, the potential of such an enterprise will be fully realized only if the institute supports initiatives and strategies that place the patient experience not only front and center in research but also smack in the middle of the medical mainstream.

“To some extent, we’ve conquered death,” said Dr. Albert W. Wu, lead author and a general internist and professor of health policy and management at the Johns Hopkins Bloomberg School of Public Health in Baltimore. “Now we have people living with chronic illnesses, and how they experience quality of life every day has become an important part of how they are doing and whether the treatments are worthwhile.”

To effectively capture the patient perspective, Dr. Wu and his co-authors propose making patient-reported outcomes a more routine part of clinical studies and practice and administrative data collection, in some cases requiring the information for reimbursement. For several months now, they have been incorporating data about patient experiences into routine medical practice at Johns Hopkins. Using a dedicated portal called PatientViewpoint.org, patients can, at the suggestion of their physicians, fill out surveys about their energy levels, social functioning, mental health, nausea and pain. The information is then handled like more traditional clinical testing; access is reserved for the patients themselves and their doctors.

Currently, the site is open only to patients diagnosed with breast or prostate cancer, but Dr. Wu and his colleagues hope that eventually all doctors will be able to order patient surveys that measure experiences like levels of pain, physical functioning or depression, proactively identifying results that are high or low, then reviewing those results with patients. And despite initial concerns that patients might feel overburdened by the questionnaires, most have been enthusiastic.

“Patients want to have more conversations with their physicians and other providers about these kinds of issues,” Dr. Wu said. “Sometimes it’s difficult to cover all this information during the medical encounter, and these surveys may be another opportunity to do that.”

Not all clinicians or researchers, however, are eager to embrace patient-reported outcomes. Many have never received training in the research methods involved and are skeptical about the value of a discipline or drill that is unfamiliar. “This is considered to be squishy, soft stuff,” Dr. Wu said. “It’s still not something you can order on a laboratory slip.”

Nonetheless, Dr. Wu is hopeful that the new Patient-Centered Outcomes Research Institute will provide more direction to all researchers, while establishing new and more comprehensive standards that require patient-reported and quality-of-life outcomes. “I think we have a real opportunity right now, and this institute has great potential to be a positive force in understanding how treatments work and what’s effective and for whom,” he said.

But he also cautioned: “We have to remember to include the patients. We have to include their perspective if we want to come up with evidence that they can really use to make better decisions for themselves about treatment and care.”