CER and Personalized Medicine: More Work to Be Done

I just got back from a meeting of the Board of Governors of the Patient-Centered Outcomes Research Institute in New York City. PCORI, as you probably know, is the independent Institute established by the health reform law to conduct comparative clinical effectiveness research. Regarding CER and personalized medicine, I’ll say now what I said late in late 2009: in the midst of the debate over health care reform — it remains to be seen whether CER will align with personalized medicine. CER that aligns with personalized medicine should be  patient-centered, because it will be structured to recognize and respect patient differences. 

We’ve made important progress toward that goal, with CER statute for the first time referencing genetics and individual patient difference.  I am hopeful we’ll  eventually get there, but we’re not there yet. Personalized medicine was, as far as I saw, mentioned only once during the PCORI Board meeting. NIH Director Francis Collins raised it during a discussion of the report from the PCORI Methodology Committee. Commenting on the Committee’s charter and the “perceived tension between CER and personalized medicine,” he said he “would hope that the Methodology Committee would take that on,” and suggested emphasizing alignment with personalized medicine and patient preferences in the document. I applaud Dr. Collins for raising this point, but we will need more voices joining him to make sure the Board and Methodology Committee to integrate CER and PM. It seems to reinforce some concerns expressed earlier by PMC about lack of an expert in genomics or personalized medicine on the Methodology Committee. The idea proposed by PMC of creating an advisory panel on personalized medicine and innovation could be one step to help ensure alignment between CER and personalized medicine.
   
One of the big news items from the PCORI Board meeting was the appointment of Dr. Joe Selby as the PCORI Executive Director. I was heartened by Dr. Selby’s comments at the meeting, in which he stressed the importance of hearing from, and focusing on, patients. It will be equally important for Dr. Selby and the additional staff he hires to understand that part of the Institute’s charge from Congress is to account for genomics and subpopulation differences. Failure to do so will prevent us from realizing the potential for personalized medicine to improve health care quality and value. 

The next meeting of the PCORI Board of Governors is in Washington, D.C. on July 18 and 19th. I hope you’ll join me there in support of comparative clinical effectiveness research that is centered on patient needs and aligned with personalized medicine.

Personalized Medicine and Health Care Reform: Looking Back, Looking Ahead

Will health care reform support personalized medicine?  In my mind, that depends on two important factors: 1) the extent to which health care reform is truly patient-centered (does it make room for patient differences, room for patient voices, and time for patient care?) and, 2) the extent to which it is innovation-friendly.

I’m focusing on the first topic in this post.  Earlier posts have rightly focused on comparative effectiveness research as one key provision.  If CER is structured correctly, it can help inform patients about optimal medical and health care options based on our differing needs.  These differences come from a number of factors, including different clinical conditions we may have, differences in our preferences and the way we view risk/benefit trade-offs, cultural differences, and certainly molecular differences.  CER structured to recognize, and respect, these differences can only accelerate the move to personalized care.  Yet it remains unclear if this is the kind of CER we’ll get.  I think only the Senate bill’s CER language gets us close to this goal, by fully including patients and providers in the process, fully embracing patient differences, and focusing the program on results communications and not national policy recommendations. 

CER is one of several aspects of health care reform that will have an impact on patient-centeredness and personalized medicine.  Just as important are provisions that will apply the scientific evidence to policy decisions.  This includes proposals to establish an independent Medicare advisory board, define physician “best practices” and performance standards, and establish standards for use of health information technology.   

These types of policies, when deployed as cost-control levers, could be one of the single biggest factors influencing the future of personalized medicine.  Payment policy measures designed to control costs and expand access—but that fail to encourage continued development and adoption of personalized medicine—could substantially delay or diminish opportunities for meaningful, measurable improvements in health care value and quality.

That’s because cost containment proposals that impose access restrictions based on average, population-wide study results risk ignoring the different needs of individual patients and discouraging adoption of personalized tests and therapies based on these differences.  For example, “pay for performance” programs focused on short-term provider efficiency could discourage physicians from using gene-based tests and targeted therapies to optimize care for the individual.  As a patient with epilepsy myself, I take a very personal interest in how these policies get developed and applied.  

This nexus of CER and policy levers was highlighted earlier this year in an NPR commentary from Anne Brewster, an internist and instructor at Harvard Medical School.  “Physicians may agree with the end goal, but many of us worry about the methods and unintended consequences.  Comparative effectiveness research sounds sensible.  Of course we need more studies to define best practices.  But I find myself afraid that the results will be used by policy makers, hospital administrators, and lawyers to further limit my autonomy by setting hard and fast rules about what is “right”.  Clinical situations are always nuanced, never black and white.  Perhaps it is semantic, but I want to feel that CER will empower rather than constrain me.” 

Enacting, and implementing, health care policies that support patient-centered care and the science of personalized medicine won’t be easy; but it is absolutely essential.  Let’s work together to make it happen.

By Tony Coelho, Chairperson, Partnership for Improved Patient Care