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NIH Director Collins Warns Against Comparative Effectiveness Research Without Genetic Strategies

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By Turna Ray

WASHINGTON, DC — The limited focus on pharmacogenomics strategies in the federal government's comparative effectiveness efforts may be a step backwards for personalized medicine, National Institutes of Health Director Francis Collins cautioned at a meeting here this week.

Studies on genomically defined subpopulations that would advance personalized medicine are "going to get lost in the wash by considering everybody equivalent, which we know they are not," Collins said at a colloquium on personalized medicine hosted by the American Association for the Advancement of Science.

Collins, the former director of the National Human Genome Research Institute and the steward of the Human Genome Project, was tapped to lead the NIH earlier this year by President Barack Obama.

At the AAAS meeting, Collins called for comparative effectiveness research to focus more on pharmacogenomic strategies to investigate the efficacy and safety of drugs in genomically defined subpopulations.

"We need to be mindful of the goal of comparative effectiveness research and not lose all that we have gained in understanding how individuals differ and how that could be factored into better diagnostics and preventive strategies," Collins said at the meeting.

Collins' comments echo the recommendations of a report released this week by the Personalized Medicine Coalition and the Lewin Group Center for Comparative Effectiveness Research on ways to align genomically guided personalized medicine strategies and comparative effectiveness research (see related article in this issue).

"While CER has been oriented largely toward evaluating treatment effects across study populations, personalized medicine focuses on using individuals' genomic information and other personal traits to inform decisions about their health care," the Lewin Group said in a statement. "It is essential that these emerging initiatives evolve to complement, not contradict, each other."

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.

In a report released in June, the Federal Coordinative Council proposed CER priorities for the HHS Secretary to consider when allocating ARRA funding. According to the council, the Secretary's primary investment should be in creating the data infrastructure for CER. The council identified as a secondary funding priority investments to disseminate and translate CER and research into priority populations.

Some personalized medicine advocates felt that genomically guided strategies did not receive adequate attention in the report [see PGx Reporter 07-08-2009].

For instance, although the council's report mentioned the need to look beyond randomized-controlled studies to advance personalized medicine, most of the priority areas identified for funding correspond to traditional clinical trials to determine the "interventions and strategies [that] work best on average" populations, Amy Miller, PMC's public policy director, told Pharmacogenomics Reporter at the time.

Another area of concern was that the council identified "priority" populations and subgroups as demographic groups that are under-represented in medical research, including ethnic minorities, children, the elderly, and the disabled. However, the report did not specifically cite any genetic subpopulations for study.

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Around the time the Federal Coordinating Council released its report on CER, the Institute of Medicine released its 100 top CER priorities, which included only three proposals for comparing the effectiveness of treatments or clinical management strategies using genomics.

The PMC is one organization that has been actively promoting heightened focus on genomics in CER. "PMC wholeheartedly supports the comments made by Dr. Collins," Miller told Pharmacogenomics Reporter this week. "We are pleased that he is at the helm of NIH and thus are assured that CER that comes out of the NIH will take a personalized approach."

What's Being Done?

There are several efforts underway to ensure that personalized medicine strategies play a bigger role in CER.

In announcing the release of the PMC/Lewin Group report, which highlights how genomic strategies can improve clinical trial design and data mining, Clifford Goodman, VP of the Lewin Group, stated that "CER must account for the different and sometimes entirely unanticipated ways in which therapies and other healthcare interventions can affect individuals."

At the same time, genomic strategies should have "to meet higher evidence hurdles that increasingly apply across many health technologies in the US and globally" Goodman said, adding that wider adoption of health information technology is also necessary before the power of CER and personalized medicine can be fully harnessed

The PMC has also pushed for the incorporation of language highlighting the use of genomics in legislation currently pending in Congress.

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.

Furthermore, Miller pointed out that the Senate Finance Committee version of the health care reform bill incorporates personalized medicine throughout CER policy aspects. "We hope it becomes the policy voted on in Congress," Miller said.

CER with NIH ARRA Funds

Despite the lack of prominence of PGx-strategies in CER currently, NIH is funding several research efforts in this area.

Earlier this month, the National Cancer Institute awarded $4 million under the federal stimulus package to the Fred Hutchinson Cancer Research Center for the development of Comparative Effectiveness Research in Cancer Genomics, or CancerGen, which will focus on assessing the predictive and prognostic value of genomic tools in treating cancer. The first study conducted at CancerGen will investigate the predictive ability of Genomic Health's Oncotype DX test to determine which node-positive breast cancer patients will benefit from a tamoxifen-based chemotherapy regimen [see PGx Reporter 10-21-2009].

With ARRA funds, the NIH has also granted researchers at the Kaiser Foundation Research Institute around $2 million to study comparative effectiveness in genomic and personalized medicine in colon cancer and nearly $2 million to the University of Pennsylvania for comparative effectiveness studies in genomic medicine.

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