PCORI: odd name, important job, potential trouble

In the past few years there has been much debate in the United States about comparative effectiveness research, defined by the Institute of Medicine as “the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care.” The institute says that the idea is “to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels.”1

Comparative effectiveness research first vaulted into the news here when Congress dedicated $1.1bn (£0.7bn; €0.75bn) to it as part of the fiscal stimulus package passed in 2009, at the height of the recession.2 The Department of Health and Human Services, through the National Institutes of Health (NIH) and the Agency for Healthcare Research and Quality, quickly disbursed those funds to sponsor research and research infrastructure development to improve medical decisions and clinical outcomes by comparing various drugs, treatments, and other interventions.

Some critics, often drug and medical device manufacturers, warned that comparative effectiveness research would disadvantage certain new and expensive treatments that were found in head to head trials to be only marginally more effective than cheaper, currently available treatments. Other critics, mainly conservatives in the US, were worried that a slavish adherence to comparative effectiveness research findings would lead to rationing, inhibiting “individual choice” and dooming individuals to receive only treatments that government bureaucrats chose for them.

Despite these concerns, the government’s role in comparative effectiveness research did not end with the stimulus package. Thus was born the Patient-Centered Outcomes Research Institute. Affectionately known as PCORI, it was initially a little noticed provision of the Affordable Care Act, the health reform legislation, which established PCORI as an independent research institute to sponsor comparative effectiveness research. The institute is funded generously, mainly from a tap on health insurance premiums. By 2013 it will have a budget of about half a billion dollars a year to spend on research and research dissemination.

This sounds great, right? A well funded, independent institute to do comparative effectiveness research to improve care and reduce costs. But like most things in government, and certainly much in the Affordable Care Act, it is not quite that straightforward.

Firstly, a search of the enabling legislation fails to find any mention of comparative effectiveness research. Instead, it has been replaced by something called “comparative clinical effectiveness research.” This is meant to restrict PCORI sponsored research to non-economic assessments. In fact, PCORI funded work is specifically proscribed from using quality adjusted life years or other such measures common in cost-effectiveness analyses. Furthermore, the health secretary cannot use the results to determine or deny coverage or reimbursement for healthcare services.

Secondly, there is PCORI’s odd name. What is “patient centred outcomes research” anyway? Why not just call it the Comparative Effectiveness Research Institute? This, it turns out, is a legacy of concerns that comparative effectiveness research will only find “on average” what works best for most people in large trials, thereby discriminating against individuals, especially individuals with disabilities or guarded prognoses. Hence “patient centred.”

Organisations such as the Partnership to Improve Patient Care, an “astroturfed” (fake) grassroots group whose steering committee includes the major lobbyists for drug and device manufacturers, show up at every board meeting to make sure that PCORI is “communicating results rather than setting centralized coverage decisions, addressing the full range of information gaps that matter to patients, addressing differences in individual patient needs, and supporting continued medical advances” (www.improvepatientcare.org). Translation? Supporting expensive new drugs that extend life for a few months at huge cost.

Thirdly, creating a new research organisation the size of a small NIH institute out of nothing is not a trivial matter. Ask the people at the National Institute for Health and Clinical Excellence. The legislation mandated only the appointment of a governing board for PCORI, and the board members have had the immense job of creating a fully fledged research institute overnight in full view of a critical public. Not an easy task.

So far the board members have worked diligently to craft bylaws, hire a staff, and begin work. They first have to figure out what patient centred outcomes research is. Then they have to decide what research to fund and how to go about it. Although the board has not actually spent a dime yet on research, this hasn’t stopped the Wall Street Journal and other conservative voices from sniping at it as part of their campaign against comparative effectiveness research: “Comparative effectiveness isn’t about informing choices. It’s really about taking away options.”3 Despite the posting of PCORI board members’ biographies on the web and the webcasting of its public meetings, the Wall Street Journal demands to know “who these people are and what they favor.”

Partly crippled by its wacky name and its inability to sponsor cost effectiveness research, PCORI is treading a treacherous road towards a noble goal. I hope that it is not distracted by the special interest clamour and conservative claptrap. Comparative effectiveness research is not a panacea, but it has real potential to improve care and cut costs. If head to head trials help us compare treatments and treatment systems for common problems, we can improve quality and safety. If just a few of the expensive boondoggles we fervently believe in are exposed and expunged, comparative effectiveness research can rationalise (not ration) care and pay for itself.4 It would be nice if everyone would just leave PCORI alone and let them get on with it.