Our guest blogger is Michael Rugnetta, a Research Assistant to Jonathan Moreno for the Progressive Bioethics Initiative.
The roadmap for comparative effectiveness research has become much clearer and detailed these past few days with the release of two new reports. One comes from the Institute of Medicine at the National Academies and the other comes from HHS’s Federal Coordinating Committee for Comparative Effectiveness Research. The IOM released a list of 100 health topics for the Obama administration to prioritize as it spends $1.1 billion in stimulus funds dedicated to CER. More importantly, the Federal Coordinating Committee itself has stated in its report to the President and the Congress that CER should “complement the trend in medicine to develop personalized medicine,” and that it will be “an important partner in helping to bring about this new level of medical effectiveness, personalization, and innovation.” This bold vision of personalized medical innovation based on “patient-centered, pragmatic, ‘real world’ research,” clearly dwarfs the feeble criticisms of CER aired by conservatives in last week’s HELP Committee markup of the “Affordable Health Choices Act.”
While the act incorporates CER as part of its health reform agenda, there is another piece of legislation that will establish a dedicated, rigorously-organized federal Institute for CER.
Formerly known as the “Patient-Centered Outcomes Research Institute,” the creation of this federal body depends on the passage of the “Patient-Centered Outcomes Research Act,” (S. 1213) sponsored by Senators Baucus and Conrad. Upon a close reading of the bill, it is evident that the Patient-Centered Outcomes Research Institute will not just be another meaningless chunk of bureaucracy as its critics claim. The Institute’s goal will not be to simply spit out generic guidelines that your doctor must follow “or else.” Rather, the Institute has been designed to ramp up medical innovation for the common good by championing a new era of personalized medicine.
Taking a close look at the bill there is plenty of language about “evaluating and comparing the clinical effectiveness, risks, and benefits” of various tests, treatments, and devices. More importantly, the bill upholds a commitment to doing the best kind of comparative effectiveness research by making it personalized and reaching out to subpopulations. The bill charges the Institute with conducting “research and evidence synthesis that considers variations in patient subpopulations.” The bill builds upon this by later explaining what “subpopulations” means specifically:
“racial and ethnic minorities, women, age, and groups of individuals with different comorbidities, genetic and molecular subtypes, or quality of life preferences.” This also means that the institute will “include members of such subpopulations as subjects in the research as feasible and appropriate.”
Even more impressively, the bill kicks biomedical innovation into high gear by explicitly infusing its research with personalized, real-world considerations. The bill stipulates that CER take into account “treatment modalities that may affect research outcomes, such as the phase of the treatment modality in the innovation cycle and the impact of the skill of the operator of the treatment modality.” This basically means that the research will look at how far along a treatment is in the product development process and the skill-level of those administering the treatment. Again, these are real-world considerations that other forms of research like clinical trials often miss.
Additionally, subpopulations will not just be part of the subject pool; they will be part of the implementation process. Their representatives will be incorporated into the advisory group that will set the agenda of the Institute’s primary research. Another advisory group will devise research methodologies by including members who are experts on personalized medicine technologies like biostatisitcs and genomics. The dissemination of research findings will also “discuss findings and other considerations specific to certain subpopulations, risk factors, and comorbidities, as appropriate.”
Finally, for those who might be concerned about the intrusiveness of such an Institute, the bill also stipulates that the dissemination of research findings discuss “limitations of research;” “shall not include practice guidelines, coverage recommendations, or policy recommendations;” and would not “violate the privacy of research participants or violate any confidentiality agreements.” Indeed, far from assuming that scientific data is the infallible final word, the Institute knows its limitations, admits them, and stays within them.
Perhaps one of the most astute and intellectually honest provisions of the bill involves the usage of research findings by the Secretary of Health and Human Services. Many have feared that CER will be used to deny coverage or to tell your doctor what to do. It won’t — this is made explicit multiple times in the bill. Of course, it would not make sense to just let CER research sit there when it comes time for HHS to make Medicare coverage decisions. Thus, there are concerns that CER will be used to justify the enforcement of one-size-fits-all treatments. Once again, it won’t, and the bill states this explicitly by requiring the Secretary of HHS to consider:
“evidence and research that demonstrates or suggests a benefit of coverage with respect to a specific subpopulation of individuals, even if the evidence and findings from the comparative effectiveness research demonstrates or suggests that, on average, with respect to the general population the benefits of coverage do not exceed the harm.”
On the whole, this bill is a prime example of robust, evidence-based, scientific policymaking. In incorporates the best attributes of the scientific method and the democratic process right into the implementation of public policy. The Institute will constantly update its research in light of new methodologies and treatments, its research will be transparent, it will seek input from the public and a broad range of stakeholders like patient advocates, it will build upon existing research efforts to avoid redundancy, and submit its research to peer review. Most importantly, the Institute will respect the rights of the individual person by taking a personalized approach to CER. As Alan Garber of Stanford and Sean Tunis of the Center for Medical Technology Policy put in a recent NEJM article, “far from impeding personalized medicine, CER offers a way to hasten the discovery of the best approaches to personalization, providing more and better information with which to craft a management strategy for each individual patient.”