Patient-Centered Comparative Effectiveness Research – Where this will lead us in terms of better patient outcomes and more effective financial investments

- Kathleen Heuer, Executive Consultant, McConnell International

The American Recovery and Reinvestment Act of 2009 provided $1.1 billion for Comparative Effectiveness Research (CER).  The Federal Coordinating Council for Comparative Effectiveness Research (the Council) has been established to foster optimum coordination of CER conducted or sponsored by Federal departments and agencies.   The Council’s report to the President and Congress was published on June 29, 2009.  The new tag line for CER is Patient-Centered Research, which is a derivation from positions expressed by the Director of OMB Peter Orszag, who refers to patient-centered health research, and Senator Baucus, who refers to patient-centered outcomes research. 

Both gentlemen spoke at the Brookings Institution’s Engelberg Center for Health Care Reform conference on Implementing Comparative Effectiveness on June 9 (See report Implementing Comparative Effectiveness Research: Priorities, Methods, and Impact).  I attended the conference, and am heartened by the direction of the dialogue of distinguished panelists as well as the content of the report from the Council.  Stakeholders are definitely focusing on quality outcomes for individual patients as a priority which should result in downward pressure on escalating healthcare expenditures.  If a provider has better information to make an appropriate diagnosis and select a treatment option optimal for their patient early on, much unnecessary expense can be avoided and the quality of life for the patient should be enhanced.  That is where we need to be.

Unfortunately today medical providers are stuck in a situation of trial and error for their patient’s treatment options based on their own experience as practitioners and limited evidenced-based medicine.  Because the scientific community has demanded research based on randomized clinical trials to determine recommended therapeutic treatment options, the trial populations are not broad enough to allow results to be driven down to a sub-population level.  So, although a drug may be safe for the average population, it may pose risks for children, or post-menopausal women.  Discussion at the Brookings event and reiterated in the Council’s report affirmed the need to develop other methods for evidenced-based research findings, including observational panel studies.  This is a step forward, but not the complete path.  The ultimate goal is towards personalized medicine.  To me, the most important text of the Council’s report (pages 21-22) follows:

Personalized Medicine and Patient Sub-groups
The need to identify and address the needs of emerging patient sub-groups, and indeed the very concept of sub-categories of conditions to which medical products are applied, is expected to change and grow as our understanding of genomics and molecular medicine increases and becomes an integral part of health care. Better understanding of an individual’s genomic and other individual biological characteristics will enable us to recognize and respond to human variability with a new degree of specificity. Understanding biological differences at the molecular level promises a significant leap in our ability to use and develop medical technologies more effectively, targeting interventions at more defined groups of individuals with greater precision. This potential, sometimes referred to as personalized medicine, has strong bearing on comparative effectiveness research.24 Many drugs prescribed in the United States today are effective in fewer than 60 percent of treated patients. This is not a fault of the drugs, but reflects the variability of metabolism or other factors from person to person.25

Unfortunately, it remains common medical practice to follow a trial-and-error approach in selecting medical interventions for patients to achieve a satisfactory therapeutic outcome. In the case of breast cancer, for example, while chemotherapy can be an important positive treatment for some patients, we have few tools today to successfully predict which patients will benefit– and the result is that many women who are treated with chemotherapy today are receiving treatments that may not be effective for their condition.

Personalized medicine aims to make medical care more precise and effective. Increased understanding of our individual genomic profiles and other individual biological characteristics will enable us both to use more effectively the therapies we have now and to identify significant areas where research and development of new products may be needed. Pharmacogenomics, the use of genetic information or other biomarkers to assist in accurate medical therapy decision-making, is expected to be a hallmark of this approach.

CER can be an important partner in helping to bring about this new level of medical effectiveness, personalization, and innovation. At the same time that CER is being used to identify which interventions and strategies work best on average, it can also help to identify different responses by different groups of patients. In some cases, different existing therapies may be identified as most effective for specific sub-groups. In other cases, CER may help to identify significant sub-groups for whom effective therapies do not yet exist. CER may also help steer research efforts toward the development of products and strategies for areas of significant need.

Let’s hope we can get to this vision.  Patients shouldn’t have to suffer through multiple treatment options before an effective one is found.

More Information:

Brookings: http://www.brookings.edu/events/2009/0609_health_care_cer.aspx

The New Yorker: http://www.newyorker.com/reporting/2009/06/01/090601fa_fact_gawande