Studies on the comparative effectiveness of procedures and technologies will continue in wake of Supreme Court’s decision.
Comparative effectiveness research, and its cousin, technology assessment, have been around for quite some time. Using these techniques, researchers attempt to find evidence-based answers to the question, “What procedures (and related products) produce the best patient outcomes?” It’s a simple question, but answers can be tough to find. Nor is the healthcare community, or lawmakers, always receptive to the findings of such research. The Supreme Court decision this summer to uphold the Affordable Care Act kept the door open to comparative-effectiveness studies; now it’s up to the healthcare community to decide where to take them.
One survivor of the Court’s decision is the Patient-Centered Outcomes Research Institute, or PCORI. Created by the Affordable Care Act, PCORI is intended to fund research that will provide patients, caregivers and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI has sought to include patients in every step of the process. “Our legacy will include standards for research that anyone can use to address the health outcomes that matter to patients,” it says on its website.
PCORI considers its mission to include research that will help patients and caregivers communicate and make informed healthcare decisions. Its research is intended to answer questions such as:
- “Given my personal characteristics, conditions and preferences, what should I expect will happen to me?”
- “What are my options and what are the potential benefits and harms of those options?”
- “What can I do to improve the outcomes that are most important to me?”
- “How can clinicians and the care delivery systems they work in help me make the best decisions about my health and healthcare?”
“It is, in its own way, a very radical perspective, because almost all of healthcare has been created from a provider perspective,” says Jeff Lerner, PhD., president and CEO of ECRI Institute, Plymouth Meeting, Pa. “‘These are things we do to and for patients,’ and that includes the research end of it. It’s quite radical to turn that around and ask, ‘What do patients think of all this, and what are their needs?’’
To answer these questions, PCORI has pledged to consider in its research people’s preferences, autonomy and needs, including their beliefs in and preferences surrounding survival, function, symptoms, and health-related quality of life. It has also pledged to incorporate a wide variety of settings and diversity of participants.
Reflecting [this pledge], PCORI has prioritized five research areas, and is allocating funding to each:
- Assessment of prevention, diagnosis, and treatment options: Comparing the effectiveness and safety of alternative prevention, diagnosis, and treatment options to see which ones work best for different people with a particular health problem.
- Improving healthcare systems: Comparing health system-level approaches to improving access, supporting patient self-care, innovative use of health information technology, coordinating care for complex conditions, and deploying workforce effectively.
- Communication and dissemination research: Comparing approaches to providing comparative effectiveness research information, empowering people to ask for and use the information, and supporting shared decision-making between patients and their providers. Research should take into account the health literacy of individual patients.
- Addressing disparities, that is, identifying potential differences in prevention,
- diagnosis or treatment effectiveness, or preferred clinical outcomes across patient populations and the healthcare required to achieve best outcomes in each population. Such research would include strategies to overcome barriers (e.g., language, culture, transportation, homelessness, unemployment, lack of family/caregiver support) that may adversely affect patients and is relevant to their choices for preventive, diagnostic and treatment strategies or their outcomes.
- Accelerating patient-centered outcomes research and methodological research. Improving the nation’s capacity to conduct patient-centered outcomes research, by building data infrastructure, improving analytic methods, and training researchers, patients and other stakeholders to participate in this research.
True to its pledge
Thus far, PCORI has remained true to its pledge to involve patients in its decision-making processes, notes Ann-Marie Lynch, executive vice president, health care delivery and payment policy, Advanced Medical Technology Association, or AdvaMed. Its decision-making processes have been open to the public, via the web and other means.
“If you think about patient-centered research, going directly to patients and finding out what matters to them is important,” says Lynch. And what matters to them may go beyond what has currently been considered relevant from a clinical perspective.
Chandra Branham, AdvaMed’s vice president, payment and health care delivery policy, points to one hypothetical example offered by PCORI in its proposed methodology report. On the one hand, you have a treatment that can be delivered once a week by injection at the clinic, vs. one that can be delivered in a daily pill by the patient in his or her home. A patient-focused study would consider not just the clinical outcomes related to the injection and the pill, but other factors, such as the logistics of the patient getting to the doctor’s office every week.
Traditionally, comparative assessment research looks at Technology X vs. Technology Y, or one technology vs. no intervention at all, says Lynch. And PCORI will conduct such research. “But that’s a very small window to look through when you’re thinking about patient care,” she says. “You can have a great product or drug, but if the patient isn’t taking it, or if there’s no follow-up care [after discharge from a hospital], their outcome won’t be appropriate.” That’s why PCORI is devoting 20 percent of its funding toward research on improving healthcare systems.
PCORI will fund research into other non-clinical issues as well, such as optimum ways to communicate and disseminate research findings. “Many improvements to care never make it out of the research community,” points out Lynch. So, PCORI will fund research into the best ways to communicate research to patients and caretakers. In addition, the organization will fund studies on how to address disparities of care, such as certain patients’ inability to access preventive care or treatments. And PCORI will also fund research into ways to accelerate patient-centered outcomes research.
“From an analytical and overarching perspective, we see this as very positive, because it truly recognizes there are many parts of the overall patient experience [from which] patient-centered outcomes research can benefit,” says Lynch. “It’s important that PCORI recognizes this, so much so that they have built it into their planning.”
More than a bump in the road
Had the Supreme Court overturned the Affordable Care Act and with it, PCORI, the impact would have been significant, if only because of the funding that has been committed to patient-centered outcomes research, says Lerner. It is a field in which ECRI has long been invested, however, he says.
“We pushed for patient-related evidence-based medicine in the early 1990s,” he says. “So I would have to say we were an extremely early proponent of the approach.” But it hasn’t always been a smooth road.
For example, when ECRI questioned the effectiveness of bone marrow transplantation in breast cancer patients in the 1990s, it was criticized from many quarters. “People were so convinced the technology worked, there was not a single controlled study of it,” he says.
Technology assessment itself has faced a tough road for several decades, says Lerner. In fact, in the mid-1990s, the Office of Technology Assessment was one of the few agencies that lost its funding, after 23 years of work. “It’s a very interesting conundrum, overall, because you think to yourself, ‘Why is it so controversial to provide objective, evidence-based information?’ What we’re doing at ECRI ought to be considered mundane, but it’s not.”
Had the Supreme Court decision overturned the Affordable Care Act, and with it, PCORI, “you would have been left with more traditional comparative effectiveness research.” Granted, traditional research has been moving toward incorporating the patient perspective, though not on the scale of PCORI, he adds.
All that said, Lerner is cautiously optimistic about PCORI’s work. “The results of its first pilot funding look good, but you need to wait and see the actual quality of the research that comes out of it. This is a real first test of the concept. A real field test.” And he hopes ECRI will be involved. The organization is applying for funding for research on shared decision-making.
“The real challenges for PCORI lie ahead, despite the Supreme Court decision,” he adds. “What you haven’t seen yet is, ‘How do the various elements of the healthcare community or industry react when the data start to come out?’ That’s when the rubber will meet the road.”