Comparative Effectiveness Studies Get Big Boost From Health Reform

The Patient-Centered Outcomes Research Institute is getting down to business, thanks to funding that can change care practices

John Carroll
Contributing Editor

Deborah Stewart, MD, the medical director for provider and client solutions at Florida Blue, spent a recent weekend with a small group of health care advocates reviewing a thick portfolio of grant requests from researchers. The investigators had proposed ways to probe the way disparities in the system — including cultural and language barriers commonly confronting Blues members in her state — block some groups from getting the care they need and ways those obstacles can be overcome.

In an office in Alexandria, Va., were patient representatives, clinicians, a handful of experts from the health insurance industry, and others. They had been selected to develop new quality standards for care practices that would improve the health of all Americans.

Deborah Stewart, MD

So far the funding for PCORI is impressive, but there is a deadline, says Deborah Stewart, MD, a member of the panel. Government funding is expected to last only through 2019. That means PCORI should show some results by 2017.

“I was amazed by the amount of funds that will be available,” says Stewart, citing a budget of $300 million this year, with another $500 million coming next year. Four panels have now gotten down to the business at hand: Screening a list of proposed projects and then narrowing it down to a few key studies that can begin to offer guidance on the best way to provide care for a list of conditions.

Clinical value

Years after the United Kingdom set up regulatory groups to offer treatment protocols and select the therapies its government-funded health care system is willing to cover, the United States is gingerly stepping forward with the Patient-Centered Outcomes Research Institute (PCORI), which will fund comparative effectiveness studies to test the clinical value of treatment options.

Unlike the United Kingdom, where regulators won’t hesitate to refuse a drug they feel is priced too high for the results it delivers, PCORI — a not-for-profit organization set up under the Affordable Care Act — is barred from using overall treatment costs as a comparison criterion. But in interviews last month, Stewart and others made it clear that the issue is likely to creep into the conversation in some fashion.

Concentrating on payers

For payers, comparative effectiveness research — even without the cost factor — gets to the heart of the medical director’s role in managed care: making sure members get the care they need without wasting money on less effective measures and preventing the high costs incurred when insurers and providers fail to manage a member’s ailment.

There aren’t many managed care players like Stewart on these panels, but there is WellPoint’s Alan Rosenberg, MD, leader of the insurer’s clinical pharmacy and medical policy programs, who is on a panel for the assessment of prevention, diagnosis, and treatment options. Getting to add some input on the front end of PCORI’s existence, he said at the time of his appointment in April, helps when it comes to “developing policies to help improve members’ health.”

There’s a lot to learn from these studies, says Rosenberg. “It’s amazing how often we don’t have good head-to-head comparisons, whether that’s drug-to-drug or drug-to-surgery. At the end of the day, for very important questions we don’t really know oftentimes what the answers are.

“American medicine tends to be pretty aggressive without being clear about the trade-offs,” he adds, citing prostate cancer treatments as an example where the side effects of therapy and questions about monitoring versus aggressive treatment have to be carefully considered. More information from these studies can be very useful in establishing treatment guidelines for a host of conditions.

Anne Beal

PCORI won’t just compare two drugs, says Anne Beal, PCORI’s deputy executive director. It will most likely compare drugs with other treatments, such as surgery, to give all the players, especially patients, a better view of the options.

Anne Beal, deputy executive director of PCORI, says it’s unlikely that organizations will finance a comparison of just two drugs. Its research will probably compare drugs with other available interventions, such as surgery, to give patients, doctors and payers a more comprehensive review of the choices at hand.

Patient input

More than a third of PCORI’s panelists are patients, caregivers, or representatives of patient groups. “We’re very proud of the way patients are represented on these panels,” says Beal.

“PCORI is really trying to provide research prioritization for patient-centeredness,” notes Stewart, whose day job entails supporting the development of patient-centered medical homes and accountable care organizations, just the kind of health care operations in search of credible information on the most effective care available.

The heavy concentration of patients and patient groups helps PCORI address its objective of maintaining singular concentration on the effects of various treatments on the patient. If researchers get grants to, say, study the effect of various therapies for pediatric epilepsy, clinicians’ main concern will probably be whether available medications control the disease. Parents, however, may be just as interested in the long-term effects of these treatments, including their influence on a child’s functioning at school. They’ll want to know whether an ideal treatment for the doctor also lines up with the best interests of the family.

But to have the maximum effect, Stewart adds, cost needs to be part of the discussion in some sense. “It’s a very real issue,” she says. “The top three concerns to my members are cost, cost, and cost.”

Because of PCORI’s focus on patients, Beal says she believes there is one conversation about cost that makes perfect sense. It should center on what people have to pay out of their own pockets for the treatments on PCORI’s radar, and whether that cost proves to be an obstacle in getting the care they need.

A $68 million chunk of PCORI’s budget has already been reserved to fund a pair of research networks that will gather a stream of standardized patient data. It’s the kind of raw information on outcomes that can be mined for insights on how patients respond to various therapies. And that statistical analysis of comparative effectiveness data can, in turn, lend significant authority to the group’s conclusions.

“Right now, we’re spending trillions on health care, and 99.9999 percent doesn’t contribute anything to medical knowledge,” National Coordinator for Health IT Farzad Mostashari tells the Washington Post’s WonkBlog. “That’s the real vision, that we actually take routine care, the health care happening in the real world, and have that contribute to knowledge.”

There is likely to be a tidal wave of reports coming out of this endeavor. Beal says that about 250 projects will be funded this year, and that more than 2,000 proposals have flooded in for review.

For them to achieve their mission, says Beal, the completed reports have to be widely disseminated in the health care field — not published in journals and then forgotten. Each grant request is expected to include a plan for spreading the news about its results. And PCORI is hammering out its own plans, to make sure it can help spread the word, as required by law.

Special focus

Each of the four panels now in operation has a special focus. In Stewart’s case, that’s addressing disparities in health care delivery. A big issue at Florida Blue, for example, is whether a diabetes prevention program is available in all relevant languages, with an ear to regional dialects. “Spanish is not Spanish is not Spanish,” says Stewart, and health care programs need to understand and address those cultural differences if they expect to have a real impact.

But Stewart also notes that the legislation only provides support for PCORI through 2019.

“We’re under the gun to have some impact no later than 2017,” says the medical director. “We will have to be as impactful as possible as quickly as possible.”


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