William Looney looks at the three P’s of effectiveness research: PCORI, Priorities, and Politics
If you ever wanted to know what health economists might do with a billion dollars, the Obama Administration’s Patient-Centered Outcomes Research Institute [PCORI] is about to tell all: not right down to the penny, but good enough. The congressionally funded – but independent – group has just released a document outlining priorities that will fuel a flood of research grants by mid-year. Application of that research will carry a significant impact on the evidence stream that increasingly determines Big Pharma’s access to providers and patients.
An outgrowth of the 2009 economic stimulus package, PCORI has a key role in implementing Obama’s health reform legislation with a mandate to evaluate the “comparative clinical effectiveness” of a vast array of health interventions, from drugs, diagnostics and devices to structural programs geared to prevention or wellness. Congress authorized $1.1 billion for this task, a figure that dwarfs what other countries currently spend on all forms of effectiveness research. It follows that the PCORI priorities will carry influence outside the US. And there is an even larger effect should PCORI end up enabling research that industry has traditionally eschewed, such as head-to-head drug comparisons and broad, population-based observational studies.
PCORI’s Draft National Priorities for Research, unveiled on January 23, strikes a tentative tone. It repeatedly states the document is unfinished, and the sub-head to the title refers to the proposed research agenda as “version one.” Much of the text is devoted to how PCORI intends to consult widely with a broad array of stakeholders – led by patients, care-givers and their representatives – prior to final action by the group’s governing Board, probably in April. PCORI staff will initiate the outreach through private meetings and focus groups, but there is also an all-day, open-to-the-public consultation scheduled for February 27 near the PCORI headquarters in Washington. The comment period on the draft continues to March 15.
The five priorities set forward in the document are also very broad and will likely be read differently by different stakeholders. The first, comparative assessment of prevention, diagnosis and treatment options, is arguably the most important. In fact, PCORI intends to devote 40 per cent of its time and resources to this area. The goal here is research to evaluate alternatives to designate those products and practices that produce superior patient outcomes. Although cost is not an intended path of inquiry, PCORI does have the right under law to fix priorities that take into account “the effect on national health expenditures associated with a health care treatment.” Hence there is some risk that activities under this priority, which focuses on new technology and product interventions, could range further afield than clinical aspects alone.
Ranking behind this are two priorities that will take another 40 per cent of the PCORI budget: improving health care systems; and accelerating patient-centered methodological research. In the first case, this entails examining changes in health services and infrastructure, including the impact on efficiency of changes in the way these services are delivered, and by whom. The research in methodologies priority will support work to improve the quality and usefulness of clinical data, as well as developing standards for new forms of research like patient registries, observational studies and meta-analyses. It is hoped these new standards will promote better decision-making around rare diseases – where, it should be noted, current treatments are quite costly, with drugs leading the way.
Some 20 per cent of resources will be spent around the two final priorities: investigating health disparities; and communication and dissemination. The disparities agenda is politically popular and focuses on revealing areas of implicit bias in the health system, from gender and race to age and social parameters, where practice patterns differ and outcomes can suffer because of them. Expect from this an upswing in attention to the social determinants of health. The latter priority is geared to raising patient compliance rates with treatment, improving patient understanding of options they have in treatment and promoting better patient-clinician communication, and evaluating the promise and success of electronic health records and other new information technologies.
The patient interest is the common thread running through each priority. Patient representatives will not only be formally consulted as specific projects are developed, they will serve on in-progress review committees and will be asked weigh in on results.
Word to the Wise..
While PCORI has taken pains to consult and to avoid stepping into the minefield of cost and “value,” industry has good reason to be wary as its ambitious work plan moves forward. The high level of inclusiveness given to the patient community may end up politicizing the process and interfere with the integrity and robustness of the research protocols. Raising this is prudent given the level of aggressiveness that patient advocates show in rallying support for their disease. Even with a billion dollars to spend, prioritization means that while some will be let in, others will be left out. The temptation will be for some groups to ask Congress to intercede – few scientists or researchers, of any stripe, want that.
Likewise, PCORI’s interpretative skills will be tested if it proceeds with proposals to “synthesize” existing studies to “create a more cohesive body of evidence.” There will invariably be disagreements in rendering judgments from different studies and it entails the risk that important clinical decisions will proceed without acknowledgment of methodological gaps and important disease variations in the populations under study. In fact, PCORI funding for more meta analyses in place of clinical trials raises questions of encouraging population bias, particularly as the technological capacity to expand the data set to millions of people becomes commonplace. PCORI’s Methodological Committee has not publicly weighed in on this yet, but when it does the discussion is bound to be controversial.
In a country where mere mention of the word “rationing” spawns howls of protest, PCORI is probably right in framing any document it publishes in the vaguest possible terms. But this may in time damage its effectiveness in facilitating superior health outcomes. I ask: can an organization that calls itself “patient centric” still be scientifically pure and empathy free? In the messy politics of health care, isn’t that the same as being tone deaf?
PCORI’s online survey to gauge reaction to the strategy document can be accessed here.