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Comparative Effectiveness Research Begins with $1B from Stimulus

NEW YORK (GenomeWeb News) – When President Barack Obama included over $1 billion in the federal stimulus spending bill in February to study which healthcare treatments are most beneficial for most patients and most cost-effective, he signaled that comparative effectiveness research likely will be part of his administration's effort to change the healthcare system.

Obama said in his campaign that he wants to start an independent research institute to study comparative effectiveness. Because of that, and because he introduced the Genomics and Personalized Medicine Act of 2007, advocates for personalized medicine are excited about the prospects of gene-based diagnostics and pharmacogenomics being involved in the future of healthcare.

The $1.1 billion funding in the American Recovery and Reinvestment Act for comparative effectiveness research is spread among three agencies, including $400 million for the National Institutes of Health, $400 million for the Secretary of Health and Human Services, and $300 million for the Agency for Healthcare Research and Quality.

HHS a week ago completed forming its 15 member council which will help to coordinate comparative effectiveness research and related health services research. This group will not make recommendations for payment, coverage, or treatment, but will consider needs of populations served by federal programs and will provide input on how some of the stimulus funding will be spent for research. That group plans its first public listening session on April 14.

The funding for comparative effectiveness research, and the creation of the council to guide and monitor it, do not necessarily mean that personalized medicine is on the agenda. But Felix Frueh, who is VP of personalized medicine at the pharmaceutical benefits management company Medco, thinks it should be.

"If we want to get comparative effectiveness right, it is imperative that we don't simply compare one [therapeutic] solution versus another, but ask ourselves which of the two [or more] solutions work for them," Frueh said in an e-mail to GenomeWeb Daily News. "This is where personalized medicine comes into play and where we need to make sure that the right questions are being asked and answered."

For example, CER should consider therapies "in settings that are as close to everyday clinical practice as possible," he said. "It is in these settings where I believe we will see most of the benefit of personalized medicine, and the outcomes of such studies conducted in such settings I am sure will impact health care advisories and decisions."

Two common critiques of using comparative effectiveness to inform healthcare decisions is that it will eventually be used solely as a means of lowering costs by denying care, and that it would lead to a one-size-fits-all approach to drugs and therapies.

Frueh, who also is the former associate director for genomics at the US Food and Drug Administration's Office of Clinical Pharmacology in the Center for Drug Evaluation and Research, believes that personalized medicine offers an answer to such criticisms. He said that "there is absolutely no reason that I can think of why, if we demonstrate its clinical use, genotyping for warfarin should be done only in some but not all patients who will need to be put on this drug.

"If we can demonstrate equal or better effectiveness of a generic drug by using genotyping compared to a brand name drug, we should do genotyping and save millions of health care dollars," he added.

Frueh said, "While I certainly don't want the government to tell me what medications I should have access to, I do believe in protocol-driven medicine if these protocols are developed with good, solid science."

The stimulus program moves ahead with some of the efforts that were included in the Comparative Effectiveness Research Act of 2008, which was introduced last year by Senator Max Baucus (D – Mont.), but went nowhere in the 110th Congress. The main thrust of that bill also called for the creation of the Health Care Comparative Effectiveness Research Institute, which would be a non-profit, non-governmental, corporation to conduct comparative effectiveness research, collect data, identify national priorities, and to disseminate its findings.

That bill did not include any specific references to incorporating personalized medicine techniques, but the bill may be redrafted for introduction again this year, according to Amy Miller, who is public policy director at the Personalized Medicine Coalition.

For those serving on committees that will develop the CER initiative, PMC has a list of things to consider, and there are a number of changes the group would make to the Baucus bill that would strategically inject genetics and personalized medicine approaches into the bill.

Miller told a committee meeting at the Institute of Medicine last week that it would be ideal if the genomes of "every research participant in comparative drug studies" could be sequenced in the not-too-distant future. While that may be too much to expect from the CER bill, Miller told GenomeWeb Daily News yesterday that she believes that the lowering of the cost of DNA sequencing will enable this to happen sometime soon.

Miller also calls for drafting more targeted pilot studies (such as those for warfarin, breast cancer, and the KRAS mutation for colorectal cancer), definitions of the levels and types of evidence that are needed to "deem an intervention effective," and she asked the institute to consider creating "a permanent, personalized healthcare standing committee."

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