PHILADELPHIA – As pharmaceutical companies plan strategies for conducting comparative effectiveness research, they should pay close attention to the activities of the Patient-Centered Outcomes Research Institute, PCORI Methodology Committee member Clyde Yancy said.

Speaking at the “Integrating Comparative Effectiveness Research” conference Dec. 6, Yancy, who also is chief of the cardiology division at the Northwestern University Feinberg School of Medicine, offered the institute’s mission statement and suggested pharma and other researchers take it into consideration when planning CER activities. The conference was sponsored by Q1 Productions.

The mission statement states: The Patient-Centered Outcomes Research Institute helps people make informed health care decisions and improves health care delivery and outcomes by producing and promoting high integrity, evidence-based information that comes from research, guided by patients, caregivers and the broader health care community.

“A number of you are involved in outcomes research [and] health quality,” Yancy said. “You represent a component of your professional entity that looks at comparative effectiveness. I would submit to you that something out of this mission statement needs to be embedded at least in your thought process so that your approach to this information is in sync with this nationalized approach.”

“PCORI Has The Wherewithal”

Speaking with “The Pink Sheet” following his presentation, Yancy expanded on why those in the research community should be following PCORI’s activities.

“Right now, there are multiple iterations of CER that exist that reflect different dimensions of CER,” Yancy noted. “What might benefit the field considerably is to establish a definition, and PCORI has the wherewithal and it has the empowerment by statute to be able to do that and then to define the attributes of what constitutes comparative effectiveness research, particularly that which is patient-centered.”

The PCORI Methodology Committee has been developing a definition of “patient-centered outcomes research,” a final version of which should be available early in 2012. The committee is currently reviewing public comments on its working definition Also see "PCORI Following Intent Of Legislators, But Needs To Be Careful In Defining “Patient-Centered Outcomes Research” – Former Senate Staffer" - Pink Sheet, 3 October, 2011.. That definition will provide the foundation for all the work the committee engages in.

Once that definition is in place, it will provide a foundation to, among other things, help define the methods that will be used in conducting the research funded by PCORI. And Yancy believes that PCORI’s methods can be applied beyond the institute’s work.

“The point being is that if PCORI generates a set of standards and a set of definitions that reaches the same tier as other organizations have achieved in this space, it will inform the future investigations that are being planned,” Yancy said.

“So, when an academic, for-profit, not-for-profit entity is sitting around a worktable to define a new research direction that is ostensibly going to be comparative effective research, they will either refer to papers we publish as PCORI that are definitional, a website that in its construct has the attributes embedded, or some other litmus test – literally a litmus test – that is refined based on the work we are doing now that will make it clear to whomever is putting together something that is akin to CER will say, ‘OK, this is the standard, this is the bar,’ and if our proposal mirrors what these individuals of this entity has come forward and determined, then we are in a good space.”

How the methods ultimately take shape will be carefully tracked and scrutinized in order to determine how much credibility the resulting research will have, particularly in the physician community Also see "CER Credibility Lies In Methodology For Studies, Physician Tells BIO" - Pink Sheet, 11 July, 2011..

“All Databases Won’t Be The Same”

And if researchers do follow the methods set by PCORI, it will create data that has standardized elements, bringing more utility to the research to make it more comparable.

“I think for the way we intend to use the new research, data standardization is key, because if we have bad data, we will make bad decisions,” Yancy said. “The first step toward getting good data is to define what constitutes such and then to standardize the data acquisition process to the extent that you can. All databases won’t be the same, but all databases ought to have some attributes that define this quality.”

He continued: “Right now, today, if you wanted to do this, there are one or two guidance documents from different government entities, but there is nothing yet that has the imprimatur of any organization that says, ‘This is it. This is the way to do this kind of research.’ And it could be that where we are going with our work in PCORI, provided the community accepts it, is to basically get to that point of imprimatur on this kind of research.”

He underscored the diverse stakeholders that are a part of PCORI as a reason why he believes the methods developed by the institute could achieve a high level of acceptance across researchers.

“That’s the benefit of starting off with the stakeholders,” he said. “It’s not a backroom process that then emerges with, ‘Here it is, boys, this is the way it’s going to be done.’ That’s not fair to everyone. This is fully transparent. People see it coming. People are having an opportunity to contribute to the way it’s being formulated, so what should emerge is something that is largely acceptable to most of the people who are going to be doing this work.”

Industry’s CER Efforts Should Follow PCORI Methods, Cmte. Member Yancy Says

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