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PGx Tools May Help Reassess Comparative Rx Effectiveness, PMC Official Tells Federal Council

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Personalized medicine methods and technologies may help re-evaluate the comparative effectiveness of treatments, according to Amy Miller, policy director of the Personalized Medicine Coalition.

Miller, who made her comments at the first of three planned meetings of the Federal Coordinating Council for Comparative Effectiveness Research in Washington, DC, this week urged the council to "outline a plan by which comparative-effectiveness findings would be reconsidered."

She suggested that pharmacogenomic strategies may be used to reconsider comparative effectiveness decisions when the US Food and Drug Administration updates labeling of a drug with genetic risk information and when a professional organization changes the standard of care for a disease.

The issue of comparative effectiveness is divisive in that results from a comparative study could alter how insurers reimburse for certain drugs and diagnostics.

The PMC is a non-profit organization focused on advancing the adoption of personalized medicine. Its membership includes representatives from industry, government, and academia.

For pharmaceutical companies, the utility of pharmacogenomics lies in the ability to market to a genetically defined subpopulation drugs that have limited efficacy in the general population. The same principles can apply to comparative effectiveness research.

At the meeting, personalized medicine came up in the context of the most divisive issue in the comparative effectiveness debate: cost.

The pharmaceutical and device industries have generally been opposed to the idea of using comparative effectiveness research to make insurance decisions because they fear it would nudge the US toward a system similar to UK's National Institute for Health Effectiveness, which bases coverage decisions on a cost-benefit calculation.

Detractors of comparative effectiveness maintain that if health insurance is expanded to more people in the US, then comparative effectiveness will only be a vehicle for payors to cover the cheapest drugs, which would not always be synonymous with the best drugs [see PGx Reporter 03-11-2009].

Although some at the meeting spoke of cost-effectiveness more favorably than others, most acknowledged that cost comparisons of various outcomes will need to be addressed going forward. In this regard, studies have shown that personalized medicine, guided by pharmacogenomic strategies, may save money.

"Comparative approaches, such as meta-analyses and systemic reviews of the literature, reward old one-size-fits-all methods that are inefficient and expensive," Miller said.

"A blood test could save Medicare about $5,000 over five years for each heart transplant patient who opts for the blood test over biopsy to indicate rejection," she added. "Although another diagnostic costs nearly $4,000, it saves on the cost of chemotherapy, which is about $14,000 per patient."

Miller, a registered federal lobbyist, issued four recommendations she said could help it design a comparative-effectiveness research agenda: Define evidence levels that demonstrate effectiveness; sequence every participant in comparative drug studies; consider the full range of relevant research questions to support the timely and appropriate adoption of high-valued personalized technologies; and continue the personalized health care initiative at HHS, and incorporate the initiative in comparative health-care research.

Other panelists also acknowledged the need to consider biomarkers and subpopulations when conducting comparative effectiveness studies.

A representative for Advamed, a trade association representing medical device manufacturers, said comparative effectiveness studies using subpopulations should be adequately powered, and their findings should be clinically meaningful.

Payors often want to see data from large prospective studies proving the clinical utility of pharmacogenomic-guided treatments before deciding to reimburse for diagnostic tests. For instance, the debate surrounding the clinical utility and cost-effectiveness of PGx-guided warfarin dosing sheds light on the difficulty of garnering reimbursement for personalized treatments [see PGx Reporter 01-21-2009].

The Federal Coordinating Council for Comparative Effectiveness Research was formed earlier this year under the America Recovery and Reinvestment Act, which granted more than $1 billion for comparative-effectiveness research.

Out of those funds, $400 million went to the National Institutes of Health, $300 million went to the Agency for Healthcare Research and Quality, and $400 million went to the Office of the HHS Secretary to develop the Federal Coordinating Council for Comparative Effectiveness Research.

The council did not issue any recommendations at the session, which was held to gather healthcare stakeholders' ideas and concerns regarding comparative effectiveness research. The council said it plans to hold two more listening sessions on the topic in coming months, and will be accepting public comments online.

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