Both the Institute of Medicine and the Federal Coordinating Council for Comparative Effectiveness Research are placing a high priority on building an infrastructure around comparative effectiveness to facilitate the research as well as the dissemination of findings.

In separate reports that were statutorily required by the American Recovery and Reinvestment Act to offer recommendations on how to spend the $1.1 billion allocated in the stimulus package, both highlighted the current disjointed comparative effectiveness research efforts and recommended actions to bring the research - both public and private - together.

In its June 30 report, IOM recommended that the CER program "should help to develop large-scale clinical and administrative data networks to facilitate better use of data and more efficient ways to collect new data to inform CER."

Federal Government At The Helm

The IOM report, "Initial National Priorities for Comparative Effectiveness Research," was released June 30 and focuses on the government's role, putting HHS at the helm of the research effort.

"The committee strongly agreed that Congress should direct the HHS secretary to implement a sustainable strategy to coordinate government CER activity," the report notes.

ARRA provides only short-term funding for CER, but a number of proposals to create a permanent CER system are before Congress. Current legislative proposals in the Senate Health, Education, Labor and Pensions Committee as well as in the House would keep the governance of CER activity within HHS. Senate Finance Committee Chairman Max Baucus, D-Mont., is working to get his vision for a joint public-private entity included in his committee's health care reform bill (¹ Also see "Baucus Comparative Effectiveness Proposal Runs Into Roadblock From Senior Republicans" - Pink Sheet, 17 June, 2009.).

IOM is not discounting the role of the private sector. In fact, the report recommends changes to privacy laws to facilitate better sharing of information with private networks within its infrastructure recommendations.

"To develop the concepts of shared data research networks to their potential for advancing health care, the CER program must address both data holders' proprietary interests and the costs that they incur in order to share data," the report states.

The report identifies a variety of data points, including patterns of medication use, rates of use of various procedures, organizational performance with respect to quality and complications of therapy as "subjects of intense proprietary interest," and private organizations "may not share their information if doing so threatens a competitive advantage."

To help offset costs, the report recommends creating financial incentives to encourage network connectivity with private databases as well as for direct participation in comparative effectiveness research, such as helping Medicare gather data under a "coverage with evidence development" determination.

"Other types of incentives to share data may also be helpful," the IOM report states. "For instance, organizations might provide data and analysis in return for periodic reports - suitably deidentified - comparing their data (e.g. on drug utilization or performance metrics) with that of other participants."

The IOM report also offered the 100 highest priority research topics as well as 10 broad CER program recommendations (see chart: " ² Broad Recommendations For Comparative Effectiveness Research ").

Coming Challenges

The Federal Coordinating Council for Comparative Effectiveness Research focused its recommendations, released June 29, on how HHS should spend the $400 million allocated to it in ARRA on infrastructure (³ Also see "Comparative Effectiveness Council Urges Infrastructure Improvements" - Pink Sheet, 29 June, 2009.).

It also outlined a number of near-term challenges facing a cohesive CER plan, such as listening to and engaging external stakeholders, including patients, providers, payers, employers and industry representatives to help guide CER or broader patient-centered outcome efforts; continued coordination to ensure there are not duplicative research efforts; training more researchers; keeping data current because "the catalog of federal activities and data infrastructure in CER needs to become a living document"; finding a way to partner with the private sector to ensure a continued funding stream; and developing methods to periodically re-evaluate data to make sure that conclusions from CER have not changed over time.

"These issues reflect both the fact that comparative effectiveness research remains in its infancy and that it must be seen as a continuous and iterative process that needs to constantly evolve based on the changing needs of the patient," the council's report says.

Reports Well Received From Observers

First impressions on the work of IOM and the Federal Coordinating Council have been favorable.

"I think they both took a thoughtful, objective view on the issue," Randy Burkholder, associate VP for policy at the Pharmaceutical Research and Manufacturers of America, said in an interview, noting that nothing specific jumped out as a negative in the first look at the reports.

"They recognize and see that there is an opportunity in CER to advance understanding of optimal treatment for people in those different groups, and if oriented in the right direction, those kinds of groups and the patient community at large can benefit from it," Burkholder added.

Both reports emphasized conducting research on patient subpopulations to help narrow disparities in outcomes.

Les Paul, VP of Clinical and Scientific Affairs at the National Pharmaceutical Council, applauded the two groups for considering feedback from stakeholders and actually incorporating it into their recommendations.

However, he noted that the role of industry, while mentioned in both reports, should be featured with greater prominence.

"Industry is mentioned in both reports but you have to search for it," Paul said. "I think people talk about, in both reports, industry being an important stakeholder and their recognition that a lot of research is conducted by industry."

Paul noted the federal council's report mentions the need to "have significant stakeholder input and coordination as much as possible around the research enterprise. ... I think there is a real interest in having industry in some fashion at the table, but we don't exactly know in the context of ARRA how that will be spelled out if there is no follow-on sustainable infrastructure created in the current legislative activity with health reform, so we will see."

But while industry's presence is welcomed, willingness from the pharmaceutical and biological industries to provide input for the IOM report appears to have been somewhat limited. PhRMA and NPC, along with the Biotechnology Industry Organization, all participated in an IOM-sponsored forum to solicit feedback, but of the 2,073 respondents to an online questionnaire, only 11 were identified as coming from drug and biologic manufacturers. This was described as a "muted response" from the pharma industry by Harold Sox, IOM Committee On Comparative Effectiveness Research Prioritization co-chair and editor of the Annals of Internal Medicine, during a June 30 press call.

Ian Spatz, principal at Rock Creek Policy Group, recently called out the biopharma industry for approaching the CER debate in a misguided fashion and urged the industry to focus on building strategies related to standards of evidence that can be used for decision support ("⁴ Fighting The Next Battle On Comparative Effectiveness: Creating Consensus Around Standards," The RPM Report, June 2009).

- Gregory Twachtman (g.twachtman@elsevier.com)

Comparative Effectiveness Infrastructure Is Priority, Federal Advisors Say

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