Latest News & Updates from the Personalized Medicine Coalition: Letter from Washington.

Abstract

When the USA was debating healthcare reform and negotiating how to provide the best quality care for the lowest cost to all Americans, some looked to the UK’s NICE, and thought that developing a similar organization might be a good idea. Americans were hesitant, however, to copy the British model due to fears that comparative effect iveness research (CER) would lock the US healthcare system into a ‘one-size-fits-all’ paradigm and restrict patient access to new treatments that may benefit many patients. By design, the Patient Centered Outcomes Research Institute (PCORI) put in place by America’s Affordable Care Act, included language intended to align CER with personalized medicine. Still a young organization, PCORI has already chosen an Executive Director, seated both its Board of Governors and its Methodology Committee, hired a few key staff, and asked the public for feedback on its definition of Patient Centered Outcomes Research, Draft Translation Framework Components, and Draft National Priorities for Research and Research Agenda. While PCORI has laid a foundation, thus far, its broad and vague research priorities leave the personalized medicine community uncertain about whether that foundation will support the continued development of personalized medicine. Amy Miller, Personalized Medicine Coalition’s (PMC’s) Vice President for Public Policy, noted in a letter the PMC sent to the Institute: “Broad drafting does not allow for an examination of individual research proposals, topics or research questions; thus, it is not possible to say whether PCORI’s work will support personalized medicine or not.” The challenges ahead for PCORI, however, do not stop with the drafting of specific research priorities. The Institute must also create an infrastructure that supports its mandate to align personalized medicine and CER. Congress required procedures to assure that alignment. But thus far the Institute has not fully developed the internal structure or expertise necessary to develop calls for research proposals, evaluate them, make awards, and do so through an open, transparent and focused process. In the PMC’s letter to the Institute, the coalition offered five recommendations about how PCORI could better define its research agenda and build the infrastructure needed to execute its mission as congress intended.