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Comparative Effectiveness, Personalized Medicine Efforts Need Alignment, PMC Report Recommends

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By Turna Ray

Comparative effectiveness research and genomically guided personalized medicine efforts should be aligned to inform which medical interventions work best, according to a report released this week by the Personalized Medicine Coalition and the Lewin Group.

Comparative effectiveness research and personalized medicine "offer complementary advantages of great potential. In a stressed healthcare system poised for reform, a continued, concerted effort is necessary to ensure that this potential is realized, "the report notes. "Whether CER and PM will be aligned or opposed is now unfolding." The report was produced by the Lewin Group Center for Comparative Effectiveness Research for the PMC.

As part of the American Recovery and Reinvestment Act, the US Congress granted a total of $1.1 billion for comparative effectiveness research: $400 million to the NIH, $300 million to Agency for Healthcare Research & Quality, and $400 million to the Office of the HHS Secretary to create the Federal Coordinating Council for Comparative Effectiveness Research.

Since the Federal Coordinating Council issued its priorities for CER research earlier this year, the PMC has been urging for greater focus on genomic strategies in CER, and this report is part of that effort [see PGx Reporter 07-08-2009]. In its report, the federal council mentioned the need to look beyond randomized-controlled studies to advance personalized medicine, but most of the priority areas for funding described traditional clinical trials for interventions for the average population.

The PMC/Lewin Group report also follows comments made this week by National Institutes of Health Director Francis Collins cautioning that personalized medicine not get lost in CER proposals that focus on traditional comparative studies in non-genomically defined populations (see related story, in this issue).

The report by PMC and the Lewin Group suggest as much, pointing out a need to synthesize the aims of CER and personalized medicine efforts.

"While the purpose of CER is to determine which healthcare intervention works best for a given healthcare problem, the purpose of PM is to ensure that healthcare delivers 'the right treatment to the right patient at the right time,'" the report notes. "Both are intended to support high-quality, evidence-based decisions for optimal patient care.”

The report points out several areas where CER and personalized medicine efforts should converge.

Additionally, product labeling, payment policies, and utilization management should be guided by whether CER findings inform the effects on the average population or account for the "heterogeneity of treatment effects."

"If CER does not investigate important differences in patient response to interventions ― for example, whether patient response to a cancer drug varies by certain genetic characteristics ― its findings may be inadequate or misleading for patient care,"the report states. "This could have extended consequences if these findings are incorporated into product labeling, practice guidelines, reimbursement policies, or utilization management that could curtail PM.”

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In turn, for personalized medicine to be useful for CER, genomically guided technologies and strategies used in this research must be based on high standards of evidence. CER that analyzes the efficacy and safety of a particular treatment in a genomic subgroup "should address strengths and limitations of this evidence, how specific it is for patient subgroups, and evidence gaps that are priorities for further CER," the report states.

As more genetic tests and Rx/Dx combination products are introduced into the market, payors are increasingly requiring higher standards of evidence showing that these interventions are cost-effective and clinically useful.

At a personalized medicine conference hosted by the American Association for the Advancement of Science this week in Washington, DC, representatives from academia and industry agreed that one of the main barriers to the adoption of genetic tests was a dearth of prospective, randomized controlled trials validating that the technologies improved outcomes and saved healthcare dollars.

"For genetic and genomic testing, health professional groups, guideline panels, and payers are calling not only for rigorous evidence of test accuracy but for evidence of clinical utility, that is, impact of test results on clinical decisions and, ultimately, patient outcomes," the PMC/Lewin Group report notes. "This applies, for example, to gene expression profile testing to predict breast cancer outcomes, pharmacogenomic testing for guiding treatment for depression, and selecting treatments for colorectal cancer."

Finally, implementation of health IT and electronic health records is critical to the ability of CER to incorporate personalized medicine strategies, the PMC/Lewin Group report notes.

ARRA provides approximately $19 billion for Medicare and Medicaid Health IT incentives over five years and provides incentives to healthcare providers for moving to an EHR system.

"In conducting CER, EHRs can capture patients' genetic and other individual health information in the course of routine healthcare, clinical trials, and other studies," the report notes. "In translating CER to practice, clinical decision support systems, EHRs, and other HIT can ensure that evidence pertaining to PM is present and actionable at the point of healthcare decisions."

At the AAAS conference several presenters discussed ongoing academic and commercial efforts to recruit patients for pharmacogenomics research using electronic medical records.

Ultimately, the PMC sees positive indicators for the convergence of personalized medicine and CER.

"Current promising signs for CER and PM alignment include explicit attention to PM in recommended national priorities for CER, pending legislation to sustain the national investment in CER, and development of CER methods and research infrastructure," the report notes.

There are several genomics CER projects being supported by NIH with stimulus funding. Furthermore, the PMC has worked on Capitol Hill to incorporate language on use of genomics in pending CER legislation.

The Patient-Centered Outcomes Research Act of 2009, sponsored by Senators Max Baucus (D – Mont.) and Kent Conrad (D – ND), would create an independent institute on CER, and includes language specifying research approaches such as "molecularly informed trials" and "genetic and molecular sub-typing." The bill also emphasizes involvement with the diagnostics community and calls for an expert in genomics to serve on a methodology committee.

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