10/29/2012 01:43 pm ET Updated Dec 29, 2012

The Patient-Centered Outcomes Research Institute Should Focus on High-Impact Problems That Can Be Solved Quickly

Comparative effectiveness research (CER) has the potential to improve patient care because it focuses on enhancing the evidence for making decisions about tests and treatments. In the Affordable Care Act (ACA), Congress authorized the Patient Centered Outcomes Research Institute (PCORI) to develop a national CER program. In 2013, PCORI will have a budget of approximately $500 million, and to date, it has built an infrastructure to identify the best research proposals. The program enjoys wide support from the health care field and the public. Still, the program continues to be a political target and remains at risk in the present polarized political climate.

The ACA contains one potentially game-changing proviso that could determine PCORI's fate. It specifies that the funding for PCORI will cease as of July 1, 2019. Presumably, Congress would have to re-authorize its funding for PCORI to survive. Regardless of the political climate seven years from now, PCORI will have to show that its programs have made an impact on health care. As one measure, the Government Accountability Office (GAO) will evaluate the effect of research findings on payment policy.

Given that the pace of clinical research is measured in years, and clinical practice changes even more slowly, how should PCORI respond to its impending deadline? One approach would be to design its research portfolio to make the largest possible impact in the shortest possible time.

The language of the ACA provides PCORI with the tools to do just that. It calls on PCORI to establish national priorities for which specific research questions to study. (Congress seems to think that this strategy is important. In addition to the ACA language, it commissioned the Institute of Medicine to set national priorities in 2009). The Act also instructs PCORI to specify (at least in some cases) the study methods that researchers should use. So, the foundation exists for PCORI to influence its own destiny. And earlier this month in the latest issue of Health Affairs, I called on PCORI to move more decisively to formulate a strategy that uses these tools.

So far, PCORI hasn't drawn up a list of priority research questions. In its first national priorities and research agenda, the institute left it to researchers and stakeholders to choose their own questions and research methods. However, PCORI recently announced that it was developing a process to identify high-priority specific research questions. Hopefully, this announcement is the first signal that PCORI is implementing a strategy to deal with its pressing deadline. In such a strategy, PCORI would identify high-impact research questions, give them top priority for funding, and recommend research methods that can answer them quickly.

PCORI has the potential to provide a key element of health reform: a stronger evidence base to support decisions about health care. It has until 2019 to prove that it can make a difference in health care. Now, the principal uncertainty is whether it has the will to mobilize quickly in pursuit of this goal.