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Will PCORI's Patient-Centered Comparative Effectiveness Research Track with Personalized Rx?

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Originally published March 2.

By Turna Ray

After the Patient-Centered Outcomes Research Institute held a meeting this week to gather public input on its comparative effectiveness research priorities, personalized medicine stakeholders are still uncertain to what degree the institute will fund studies that aim to define how well drugs work in molecularly distinct patient groups or if it will mostly fund research to gauge how interventions work in the general population.

Another unknown as PCORI further defines its CER framework is whether "patient-centered research" – a term the institute has been working to define with public input – will explicitly mention personalized medicine principles. Whether it does or not could signal whether comparisons of genomic medicine to the standard of care will be a major focus of PCORI's CER efforts.

PCORI, a non-profit organization formed by the 2010 Patient Protection and Affordable Care Act, has issued a draft document outlining the research areas in which it wants to conduct studies comparing the safety and efficacy of medical interventions, healthcare delivery models, and infrastructure. The findings from such CER, PCORI hopes, will help drive informed healthcare decision making, improve patient outcomes, and reduce unnecessary spending in healthcare.

The public was invited to discuss the preliminary research agenda with PCORI and key stakeholders at a meeting this week. PCORI is also accepting written comments on its draft research agenda until March 15.

PCORI is planning to spend $122 million for research activities in 2012, and it's possible that some of this money may go toward funding CER on molecularly targeted personalized medicine products. According to PCORI’s statutory purpose, the research the institute supports must consider how disease can be prevented, diagnosed, and treated in patient subpopulations, which could include groups defined by molecular subtypes.

Regardless, some believe that the focus areas outlined in PCORI's draft research agenda are too broad, and personalized medicine principles, which are still new and evolving, can very easily get lost in the mix.

"PCORI was designed to address specific, practical questions of national importance," Amy Miller, vice president of public policy for the advocacy organization Personalized Medicine Coalition, said at the meeting according to prepared comments provided to PGx Reporter. "However, the broad and vague drafting of the research priorities is more appropriate for traditional, investigator-driven research, which may or may not address the types of questions PCORI must answer."

In addition, "since broad drafting does not allow for an examination of individual research proposals, topics, or research questions, it is not possible to say whether PCORI’s work will support personalized medicine or not," Miller said.

Since PCORI was formed, the PMC has been trying to remind the institute's leaders that their charge isn't just to look at whether most people respond better to one drug over another, but to investigate how and why treatments work best in some people with a unique set of characteristics. "It is not enough, in the PMC’s opinion, to say that one therapy works for most people in the aggregate," Miller said. "To enable personalized medicine, research must explain why a therapy works and for what types of patients."

To better align personalized medicine principles with CER objectives, Miller told PGx Reporter that PCORI should form an expert advisory panel on personalized medicine; incorporate new information and technological innovations to improve the quality of CER performed by the institute; identify more specific research priorities than the broad focus areas currently presented in its research agenda; and expand its internal scientific and clinical expertise to evaluate and award research proposals.

Adolph Falcon, senior VP of the National Alliance for Hispanic Health and a panelist at this week's PCORI pubic meeting, told PGx Reporter that it is too early to draw any conclusion about the role of personalized medicine in PCORI's CER efforts.

NAHH is committed to diversifying genomics research, and PCORI could position itself to drive advances in this regard. "One of the biggest challenges today is that 96 percent of participants in recent genomic studies trace their ancestry to Europe. That means we have little understanding of the role of genetics in health for much of the United States with non-European ancestry," Falcon said.

"From my point of view, PCORI's success will be measured by whether or not it fosters personalized medicine," he added.

Healthcare advisory firm Avalere Health, which has been closely tracking PCORI's CER efforts, agrees that it may be too early to tell where personalized medicine will fall among the institute's research priorities. According to Jenny Gaffney, senior manager at Avalere, there is still time for stakeholders to influence the research agenda in this direction.

"PCORI left the agenda broad so that stakeholders would be the ones initiating specific research projects," Gaffney told PGx Reporter via e-mail. "This creates an important opportunity for patients, providers, researchers, etc., to emphasize the importance of personalized medicine in their research proposals, which PCORI will be soliciting later this spring."

There is plenty of high-level support for applying CER as a vehicle to spur adoption of personalized medicine, since a big barrier to market entry for such treatments has been the dearth of clinical evidence proving that they can prevent adverse reactions or save healthcare dollars. Janet Woodcock, director of the US Food and Drug Administration's Center for Drug Evaluation and Research; Carolyn Clancy, AHRQ director; and National Institutes of Health Director Francis Collins have all spoken publicly on the importance of including studies on genomically defined subpopulations as part of CER (PGx Reporter 11/4/2009).

However, PCORI will likely need to step out of conventional CER thinking if personalized medicines are to feature prominently in its work. "While ultimately synergistic in their relationship, the rise of personalized medicine, in which genetic tests play a seminal role, and CER have the potential to interact disruptively in the short term," Gaffney observed.

CER has historically focused on comparing medical interventions across broad, traditionally defined disease populations. By injecting a personalized medicine focus into the CER conducted by PCORI, researchers would need to drill down to smaller patient subsets with the help of genetic tests.

"CER techniques are less well developed to make equitable comparisons between treatment regimens with more than a single component or components that differ significantly from one another," Gaffney said. "The evaluation of molecular diagnostics, as standalone tests and companions to therapeutics, will necessitate more methodologically sophisticated CER."

Patient-Centeredness vs. Personalized Medicine

While PCORI's research agenda includes some discussion of personalized medicine, the institute is placing more importance on so-called "patient centered" research principles, a term that is part of PCORI's name.

PCORI put out a call to the public last year to help it develop a working definition for "patient-centered research." According to a preliminary definition, "patient centered outcomes research helps people make informed healthcare decisions and allows their voice to be heard in assessing the value of healthcare options."

Specifically, this type of research will assess the benefits and harms of medical interventions to inform decision making, focusing on the "comparisons and outcomes that matter to people," according to the working definition. Additionally, patient-centered research will also focus on a variety of settings and populations to "address individual differences and barriers to implementation and dissemination," the definition notes.

At the public meeting this week, personalized medicine topics were raised by some stakeholders such as PMC's Miller and Falcon, but PCORI officials and meeting attendees spent more time talking about "patient-centered research" and what the term means.

In a statement issued after the meeting, PCORI said that it will consider for approval the definition of "patient-centered outcomes research" that the institute has developed with public input at a March 5 meeting of PCORI's board of governors.

"Our methodology committee and board members have given thoughtful consideration to the input we’ve received and we look forward to discussing it in public," PCORI Executive Director Joe Selby said in a statement this week. "This is a living definition and we will revisit it in the future, as needed, as this field of research grows and the needs of patients and those who care for them evolve."

For the time being, it's unclear how closely the definition of "patient-centered research" will match personalized medicine principles, an aspect that could be a critical to advancing CER around molecularly targeted interventions.

Falcon was encouraged that the first question PCORI is trying to answer in defining "patient centered outcomes" is, "Given my personal characteristics … what should I expect will happen to me?"

However, the PMC is not so sure that research that is "patient centered" will necessarily facilitate studies that focus on personalized medicine strategies. The two terms may sound synonymous but when applied to real-world practice the words may lead down different CER paths, Miller suggested.

"Some in PCORI seem to think that patient-centered medicine is synonymous with personalized medicine. We see 'patient-centered' approaches as potentially enabling personalized medicine but see personalized medicine as a clearer approach to understanding patient needs on a molecular level," Miller said via e-mail. "Medicine can be patient-centered – taking into account patients' beliefs, values, and experience – without being truly personalized and using what we know about how molecularly defined subpopulations will respond to a given treatment option."


Have topics you'd like to see covered in Pharmacogenomics Reporter? Contact the editor at tray [at] genomeweb [.] com.

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