PCORI: Together Moving Comparative Effectiveness Research and Personalized Medicine Forward

Today, I had the privilege of addressing the Patient-Centered Outcomes Research Institute (PCORI) Board of Governors on behalf of the Personalized Medicine Coalition (PMC).  As noted in my previous blog on its member selection, the PCORI Board of Governors was established by the Patient Protection and Affordable Care Act and is tasked with assisting patients, clinicians, purchasers, and policymakers in making informed, evidence-based, health decisions. 

As a science-driven organization, PMC supports health decisions made on sound science.  When working with legislative drafters and the community to develop the legislation creating PCORI, we recognized the need to ensure that the research supported by this group must be personalized and continually updated.  The resulting law creating PCORI aligned the principles of personalized medicine with those of comparative effectiveness research.  It underscores the importance of eliminating an “average effects” approach to CER, which obscures subgroup differences and skews treatment decision-making away from individualized care.

To make sure this opportunity is realized, we requested that the Board designate an ad hoc committee on personalized medicine to work with both the Board and the Methodology Committee.  We believe that such an ad hoc committee will help the Board and methodology committee comply with the Congressional intent that the research overseen and conducted by PCORI be personalized when appropriate.  High levels of communication and collaboration have been essential to the advancement of personalized medicine, and the same will hold true for advancing CER that is aligned with personalized medicine.

Achieving alignment between CER and personalized medicine holds substantial opportunities, but we realize it will be challenging. These challenges cut across a range of issues, from study designs, to operating procedures, to research priorities.  An ad hoc committee will provide a venue for bringing a diversity of expertise and perspectives together to focus on this common objective.  Such a group will help in coordinating research across a range of public and private sector activities and ensure that the most relevant evidence gaps and research questions are identified related to personalized medicine, and will help with matching study designs to questions.

The science of personalized medicine is fast moving and sometimes highly specialized. Legislation and regulation takes time to catch up. The personalized medicine community is committed to seeing that PCORI’s research keeps pace with scientific advancement and welcomes the opportunity to achieve this end.

We are at a crossroads of research, regulation and legislation. It is an exciting time!