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|Comparative Effectiveness Research (CER)|
The American Brain Coalition believes it is important that our member organizations be informed about legislative and policy issues that affect them. Included among the many provisions in the current health care reform legislation is the issue of comparative effectiveness research (CER). This issue was mentioned at the spring 2009 ABC Meeting and generated a robust discussion. A working group constituted by ABC member organizations was established to contemplate CER. The group felt that creating an informational paper to provide American Brain Coalition members with strong, clear background information on the subject matter would be important to educate them and, subsequently, gauge interest in defining an official ABC policy stance on CER. This informational paper defines comparative effectiveness research (CER), the current status of CER programs, how it is integrated in proposed health care reform bills in Congress, ABC Advocacy Committee Chair’s thoughts on health care reform, opportunities or cautions about CER, and how information is generated. Thus, it serves to address the issue in a factual manner, as well as to highlight the legislative and policy issues that currently surround the conduct of comparative effectiveness research.
Definition of Comparative Effectiveness Research
Comparative effective research is the branch of health services research that attempts to ascertain which medications or treatments will be most effective for which patients under which circumstances. It may involve comparing two drugs; drugs versus procedures; procedure vs. procedure; procedure vs. watchful waiting; or drugs vs. watchful waiting. Research can be conducted as part of a clinical trial or as a review of data from already conducted trials, as well as from such approaches as chart reviews.
CER was first established as a discreet program in the Medicare Modernization Act of 2003 (MMA). That legislation created a CER program within the Agency for Healthcare Research and Quality (AHRQ) and authorized it to be funded at a level of $30 million. The program operated at that level for several years until the FY 2009 Omnibus Appropriation bill increased the funding level to $50 million for the current fiscal year. In addition, the American Recovery and Reinvestment Act (ARRA), or economic stimulus bill, elevated the field significantly by providing $1.1 billion for CER. The statute directed that money be divided among the Office of the Secretary of HHS ($400 million); the National Institutes of Health ($400 million) and AHRQ ($300 million). In addition, it created a Federal Coordinating Committee – CER (FCC-CER) to provide guidance on distributing the funding allocated to the Secretary’s Office and directed that the Institute of Medicine (IOM) conduct a study on the best uses of CER funding.
Pending Healthcare Reform Proposals
Three major health care reform proposals are pending (or will be later this month) before the Congress. They are the so-called Tri-Committee Proposal from the House Ways and Means, Energy and Commerce, and Education and Labor Committees; the Senate HELP Committee version, and the Senate Finance Committee version.
The House bill closely tracks what was included in Section 904 of the CHAMP Act of 2007, which passed the House. The provision was later dropped in the Senate to keep full attention on the SCHIP reauthorization provision that was part of the CHAMP Act. Among the significant provisions of the House version are the placement of the program within AHRQ and the funding to come from a tap, first on the Medicare trust fund, and later on private insurers. Priorities would be set by the Agency based on recommendations from an advisory committee and grants would be awarded based on peer review. The provision is largely the product of the House Ways and Means Committee.
The Senate HELP version is similar to the House version, although it is far less detailed, due to jurisdictional issues between HELP and Finance. The Senate Finance version is expected to track closely with legislation introduced earlier this spring by Chairman Max Baucus. That legislation, S.1213, would create a Patient Centered Outcomes Research Trust Fund to finance CER through a similar tap. A free-standing institute that would not be part of any government agency would be governed by a stakeholder board that would establish priorities. The legislation also has an elaborate public comment period, similar to that used for regulatory matters, for things like study design and research outcomes analysis.
Opportunities and Caution on Comparative Effectiveness Research (CER)
There are several issues of concern or controversy surrounding CER. Among them are:
Governance of CER – Many proponents of CER suggest that by placing it in a government agency (AHRQ) and by limiting stakeholder involvement to an advisory capacity, you protect the scientific integrity of the process and do not allow it to become influenced by those with profit motives. Others suggest that stakeholders, particularly industry, have a lot to contribute and that they should be involved in deciding what the research priorities are and, perhaps, even what grants are funded.
Placement Within Agency – Many proponents of CER argue that it should be located in AHRQ, which is experienced at running such a program and is a science agency with the infrastructure in place to get programs up and running quickly. Opponents argue that AHRQ, as a government agency within the Department of Health and Human Services, will accelerate the shift from clinical effectiveness to cost effectiveness, leading to rationing of care. NIH is also active in CER currently and that is expected to continue under its broad legislative mandate to conduct health-related research.
Cost Effectiveness – Advocates of CER have been vigorous in arguing that this issue is about clinical effectiveness – assuring that doctors and patients have the best possible information to know which treatment is likely to be most effective for which patients. Opponents caution that CER will inevitably slide into cost effectiveness, leading to the denial of coverage for more expensive treatments – regardless of effectiveness.
Personalized Medicine – One of the criticisms levied at CER is that it is “one size fits all” research. As the bills move through the process, we expect that an effort will be made to incorporate the concept of personalized medicine into CER, essentially utilizing an individual’s genetic code to determine variations in response to treatment.
Limitations in Usefulness – The information gained by this process can be very useful but is very limited in its availability. There is very little ‘evidence based’ information available to answer the myriad of questions surrounding the long list of 100 recommended primary research areas to be covered. Recommended procedures and treatments may not be modifiable to account for individuals with differing demographics, co morbidities (other diseases or conditions), complications and persisting chronic care issues. This would lead to a tendency to use inappropriate treatments or deny appropriate treatments as ‘not proven by evidence based studies’. In addition, these studies often are effective for a limited percentage of those studied. There would remain a group of individuals who would predictably fail. Would the practitioner be allowed to use accumulated knowledge and experience instead (e.g. experience based medicine)?
How Information is Generated
As alluded to above, there are several different ways to generate the information that is needed to conduct a CER-type study. Some of the research is done retrospectively, reanalyzing data from multiple research studies to compare the effectiveness of, for example, two different pharmaceutical products. This is the form of CER that is most likely to be conducted through AHRQ. Research can also be done prospectively, utilizing a clinical trial methodlology to make direct comparisons. This is the form of CER that is most likely to be conducted through the National Institutes of Health and, more specifically, utilizing the Clinical and Translational Science Award (CTSA) recipients. The CER portion of the study could even be a part of a larger research project through this method.
CER is not a “brain-specific” issue. Rather, it is a broad subsection of health services outcomes research that is currently a hot topic within the much larger sphere of health care reform legislation currently pending on Capitol Hill. It appears at this point that there will be significant differences between the House bill and the bill that ultimately comes out of the Senate, and that those differences will have to be resolved in a later conference committee between the two houses. It is impossible at this stage to identify how this issue will be resolved, or if it will be resolved. And, of course, even if it is resolved, the entire question could be rendered moot by the failure of health care reform, if that were to happen. Rigorous debate over health care reform, as demonstrated in numerous town hall meetings over August recess, leave many questioning what will transpire when Congress works on the varying pieces of legislation in the fall.
Issued by the American Brain Coalition on 11/24/09