Report: Federally funded institute avoids comparing drugs, other treatments

WASHINGTON — An institute that pays researchers to compare medical treatments has spent only half of its more than $1.4 billion in available federal money on what is called comparative effectiveness research and has largely ignored prescription drugs, despite their role in driving up health care costs, according to a study released Tuesday by a Washington-based policy group.

The research into the comparative effectiveness of treatments and pharmaceuticals is meant to determine if lower-cost options provide the same benefit as more expensive procedures and drugs.

The Patient-Centered Outcomes Research Institute (PCORI) has paid for a higher percentage of comparative effectiveness research in the last two years, from 37% before 2014 to 58% now, according to the new study by the Center for American Progress, a Democratic-leaning research group. Since PCORI's creation in the 2010 Affordable Care Act, it has now spent 51% on comparative effectiveness research, the study showed.

The institute hasn't focused on what the law intended, says study co-author Topher Spiro, CAP's vice president of health policy and a former Democratic Senate health committee aide who worked on the legislation. The study was led by Ezekiel Emanuel, an oncologist and former White House health policy adviser who also helped write the law.

Research gaps "involve high-cost treatments, such as certain drugs, medical devices, and surgical procedures," the report said. That's in part because some experts believe PCORI has been afraid to conduct research that could antagonize "powerful industries."

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For example, only 4% of the research compares two or more drugs, although drugs account for 17% of overall health care spending, the report said.

PCORI executive director Joe Selby, a physician, said he disagreed with how the report defined comparative effectiveness research (CER) as strictly head-to-head comparisons, but acknowledged the institute had a "bit of a lull" in funding drug research at first. There also weren't nearly as many major drug introductions when PCORI started, and it's now supporting research that compares drugs to treat Hepatitis C, depression and multiple sclerosis, he said.

PCORI has been criticized since it first appeared in the legislation that became the ACA. Although CER had bipartisan support, the institute got caught in the politics surrounding what Republican former vice presidential candidate Sarah Palin called "death panels" that could decide who received medical treatment.

Now despite widespread concerns about rising drug costs, CER is being attacked by patient groups supported by the pharmaceutical industry that claim the research could limit access to some life-saving drugs.

Congress limited the ability of PCORI to consider costs when comparing health care, drugs or prioritizing research studies. The institute also has a 21-person board appointed by the Government Accountability Office, Congress' investigative arm. Drug and medical device makers have board positions along with medical and benefits experts from academia, employers and government, including Francis Collins, director of the National Institutes of Health.

Since 2010, former Rep. Tony Coelho, D-Calif., has led the Partnership to Improve Patient Care (PIPC), which has been a leading critic of CER. Its work is also paid for by the pharmaceutical industry and other patient groups that are in turn supported financially by drug companies.

"I’m their big advocate and also a critic," Coelho said of PCORI in an interview.

Coelho, who has epilepsy, said the late Sen. Ted Kennedy, D-Mass., asked him to chair the group because of his political background, his disability and because he wouldn't let anyone "unduly influence" him in efforts to get patients' voices heard. Coelho resigned from Congress in 1989 as he faced an expected House Ethics Committee probe and a Justice Department criminal inquiry into his failure to report junk bond purchases with loans from a friend who was a savings-and-loan official.

PCORI does not want to be targeted by patient groups, said Leah Binder, the CEO of the Leapfrog Group, which conducts safety ratings of hospitals, and a former member of an institute advisory committee.

Still, Binder and others say PCORI made a large and lasting contribution to research when it required patients to be involved in the studies it supports, a practice now emulated through the medical research community.

If PCORI paid for more head-to-head research, it would be easy for insurers and other business to pay for the cost analyses, said institute board member Harlan Krumholz, a cardiologist.

"I would like to see us sprint towards doing as much comparative research as possible," says Krumholz, also a Yale University professor and health care researcher. "We don't need to look at cost; we need to look at effectiveness."

Patient groups, which are nearly always backed by pharmaceutical companies, are major players in what PCORI decides to research. That can limit the focus on finding lower-cost options, which could hurt the profitability of the companies that pay for the patient groups.

The institute tries to solicit ideas from patient groups that receive money from a "diversity of sources," said Jean Slutsky, the institute's chief engagement and dissemination officer. "That's always our concern — that any one sector would be responsible for bias," she said. "Some people are more successful than others in getting public comment in."

Patient groups funded by drugmakers are largely mum on high drug prices

Comparative effectiveness research may threaten "personalized medicine" and limit access to certain higher-priced drugs, some of its critics say. PIPC's website notes that the research can help, "but it also can be misapplied in ways that unintentionally undermine patient access to care and physician-patient decision-making."

Indeed, most patients and their families have different views of cost, says Michael Rea, a pharmacist who is CEO of Rx Savings Solutions and has two young boys with autism.

"I'd pay anything I could for something that would help my sons," says Rea, whose company represents health insurance plans. "But there's a breaking point from a societal standpoint on what is realistic."

The pharmaceutical industry, patient groups and PCORI have several connections, including:

• Coelho's group, which serves as a PCORI watchdog, counts among its members the Pharmaceutical Research and Manufacturers of America trade group and a long list of patient groups, many of which get considerable funding from the pharmaceutical industry. One member, the Alliance for Patient Access, acknowledged that most of its money comes from the drug industry but would not provide a percentage. Coelho also won't say what percent of his group's budget comes from drug companies, "I don't answer that question," he said. "It is immaterial to me."

• National Health Council CEO Marc Boutin, who represents patients on one of PCORI's advisory committees, says "we work quite hard to coordinate" with PIPC. A 2011 article in the Journal of Public Health cited NHC for "principles that did not encourage transparency,"  but NHC said in response that it "requires its member companies to disclose funding from corporations and to present the information in an easily accessible manner."  NHC spokeswoman Nancy Hughes said that 70% of its membership is made up of non-profits and patient groups. These "generate the majority of our dues dollars," but she did not know the percentage of funding from drugmakers. Many of its patient group members, like PIPC, depend on drug industry money.

• Eugene Washington, a physician, was the founding chairman of the PCORI board until September 2013. During part of the time, he was also a board member of medical device and drug maker Johnson & Johnson. Washington, who is now CEO of the Duke University Health System, was also dean of the University of California, Los Angeles medical school while on the J&J board. Although Washington was not named, UCLA's medical school was one of six academic medical centers with officials on J&J's board cited in a 2014 Journal of the American Medical Association article questioning the health systems' ties to drug industry boards. UCLA's medical school's corporate relationships were also the subject of a whistleblower lawsuit that led to a $10 million settlement in 2014. Duke's press office declined to comment.

Asked to single out the most significant research findings to date, Selby points to studies including those that found oral antibiotics can work as well as those delivered intravenously to children with serious bone infections and on the benefits of giving stroke victims blood thinners when they were discharged from hospitals.

During the institute's board meeting week and in interviews, there was weariness and excitement about its future.

"Let's get this engine moving," said PCORI board member Lawrence Becker, Xerox's director of strategic partnerships and alliances. "It's been a long road the last six years."