PCORI’s Selby Defines Research Agenda; Tells PMC that Institute Will Solicit Continued Feedback to Ensure CER Supports Personalized Medicine

The Patient-Centered Outcomes Research Institute’s (PCORI) Board of Governors held a conference call meeting Wednesday afternoon.  During the call, they announced the finalization of their research agenda and discussed changes made from the initial draft in response to stakeholder comments.

Given the many criticisms PCORI received for being too broad and vague in its draft, I was surprised by how quickly the research agenda was finalized.  I was gratified that the Institute publicly detailed how stakeholder comments shaped the final agenda, as urged by the Personalized Medicine Coalition (PMC) in our letter to the Institute last month.

Last week, PCORI Executive Director Joe Selby also addressed PMC’s public policy committee, discussed the Institute’s rationale for its agenda – which was short on specifics – and answered questions from PMC members.

During his talk, Selby shared that PCORI’s goal for its research agenda was to be a foundation for future work.  Through funding announcements, the Institute hopes to solicit a broad range of research proposals that will offer many options on approaches to implement and conditions to study.

Selby suggested that PCORI aims to keep the door open to provide funding for comparative effectiveness research (CER) studies that may benefit small subpopulations or rare disease research, and not to fund only research addressing common conditions such as heart disease, depression or diabetes.  However, several PMC members pointed out that ultimately PCORI’s funding decisions will pick winners and losers.

Selby encouraged the submission of proposals to the Institute for studies designed to demonstrate what types of treatments will work for different subgroups of patients (as PMC believes all CER studies should aim to do).  PMC was disappointed that he did not commit the Institute to such an approach as we believe the statute requires, although he noted that research announcements will solicit thematic proposals in addition to disease-specific ones that may address some personalized medicine topics.

PMC Members asked Dr. Selby about the development of PCORI’s infrastructure and questioned whether the Institute has what it needs to align CER with personalized medicine.  As I noted previously, PMC does not believe that the Institute has developed the internal structure necessary to carry out its mission at this time.  Dr. Selby informed the PMC policy committee that PCORI welcomes its feedback about how to assure that funded CER-studies align with personalized medicine through its open meetings, public comments, and advisory committees.

To facilitate personalized medicine, CER must explain not only what works best – but also for whom.  The future of medicine depends on a careful and critical answer to this critical question.  We are not yet sure that PCORI has embraced this idea.  For example, when the Board met to discuss altering the research agenda based on public comment, it tried to more clearly define the need to study personalized medicine. It failed to cite the most common definition of personalized medicine, and instead, discussed mostly demographic characteristics. The PMC will respond to the altered language with the expectation that the definition of personalized medicine will be accepted into the research agenda language. This is the most basic, first step to aligning CER with personalized medicine.

We valued Dr. Selby’s willingness to dialog with the PMC community and look forward to continued engagement with PCORI to ensure that CER can be supportive of quality health information, tailored to patients’ values and individual biology.