Originally published Jan. 30.
The Patient-Centered Outcomes Research Institute is seeking public comment on a draft document outlining priority areas for comparative effectiveness research.
PCORI, a non-profit organization formed by the 2010 Patient Protection and Affordable Care Act, is planning to use this research priorities list to guide the types of CER it funds going forward. The public can comment on the proposed research priorities until March 15.
Based on the broad research areas outlined in the draft document, it is still unknown to what degree PCORI plans to fund CER on personalized medicine strategies. Personalized medicine stakeholders are hoping that research proposals focusing on genomic medicine and PGx approaches will receive grant funding from the institute.
Last year, PCORI announced it would award as much as $26 million in grants for 40 projects that will help the institute establish methodologies for conducting comparative effectiveness research in healthcare. Letters of intent were due by Nov. 1 and the deadline for the grant application was Dec. 1, 2011 (PGx Reporter 10/5/2012).
In the draft document laying out PCORI's research agenda, the institute has identified CER priorities in five areas: assessing prevention, diagnosis and treatment options that work best in specific patient populations; improving healthcare services, particularly for patients with chronic conditions; improving research communication and dissemination so people can make more informed healthcare choices; ensuring that research addresses healthcare disparities; and promoting efficient and safe patient-centered outcomes research.
The document should help "identify the broad questions that must be addressed so that patients can make better and more personalized decisions in partnership with their clinicians across all areas of health,” said PCORI Executive Director Joe Selby in a statement, adding that the initial agenda is only "a starting point."
Over the coming months, the institute plans to "apply this framework to identify the specific questions that are most important for PCORI to address," Selby said. "We will be asking the community to think boldly about research projects across all five priority areas.”
PCORI's Research Agenda Workgroup reviewed CER research recommendations released in the past by government agencies and stakeholder groups and settled on 10 priorities, which address prevention, acute care, care coordination, chronic diseases, palliative care, patient engagement, safety, overuse of healthcare products or services, information technology infrastructure, and the impact of new technology.
"What I found most interesting, and a little disheartening, is that the priorities are drafted in vague language, making it difficult to determine how they may (or may not) support personalized medicine," Amy Miller, VP of the Personalized Meidicine Coalition said in a blog post. "I hope that as PCORI moves forward, it will give assurance to the PMC and other stakeholders that its research supports personalized medicine, as was the intent of Congress at the time of the Institute’s formation."
The priorities proposed by PCORI in the draft document may provide opportunities for funding CER to assess the benefits and risks of adopting pharmacogenomic tests and genomically targeted medicines in patient subsets. Although the proposed research agenda is not specific in this regard, personalized medicine and genomic subpopulations are mentioned in several examples for potential CER proposals.
PCORI’s statutory purpose, as it is noted in the draft document, is "to assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis that considers variations in patient subpopulations.”
Additionally, PCORI has said it invites CER proposals that consider how healthcare treatments and services impact "various subpopulations," based on race, sex, age, comorbidities, genetic and molecular subtypes, and quality-of-life considerations. Such research could explore, for instance, whether new information for a subpopulation could increase understanding of treatment strategies or inform "a personalized assessment of an individual's unique biological characteristics and/or social circumstance."
However, the extent to which personalized medicine currently features in PCORI's draft research agenda, hasn't appeased supporters of the discipline. As Miller points out in her blog, although PCORI's report notes that research conducted in the category, 'Comparative Assessment of Options for Prevention, Diagnosis, and Treatment,' will compare treatments, it fails to address at what level.
In a recent report characterizing the state of the personalized medicine, the PMC reported that the number of commercially available personalized medicine products has grown from 13 in 2006 to 72 today. "Researchers now know more about individual response to some drugs based on biomarker information. For example, when comparing a red pill to a blue pill, it is imperative that biomarker information be included in those examinations, especially when biomarker information is in the label for the drug," Miller wrote. "But, without clearly defining the details of PCORI’s research, we really can’t know whether or how the Institute will consider biomarker information in its priorities and research agenda."
PCORI is funded by the Patient-Centered Outcomes Research Trust Fund through 2019. The institute was allocated $10 million for 2010, $50 million for 2011, and is slated to receive $150 million for 2012. After 2012, the funding for the institute will come from an annual $2 fee per Medicare beneficiary transferred from the Medicare Trust Fund, and an annual $2 fee per-covered-life assessed on private health plans adjusted for health expenditure inflation.