Originally published on March 22. Updated March 23.
By Turna Ray
Personalized medicine became a part of the national healthcare agenda this week as US President Barack Obama signed the historic healthcare reform bill into law.
Over the weekend, the US House of Representatives passed HR 3590 by a 219-to-212 vote. The bill, which cleared the Senate in December, includes a section creating an independent Patient-Centered Outcomes Research Institute.
The non-profit institute will be charged with conducting research that informs the public and healthcare providers about the comparative risks and benefits of marketed drugs, devices, and medical products.
Specifically, with regard to personalized medicine, the institute will also examine the utility and effectiveness of medical products and services in "various subpopulations" differentiated by race, ethnicity, sex, age, co-morbidities, as well as genetic and molecular subtypes.
"For personalized medicine, this vote is historic," Amy Miller, public policy director at the Personalized Medicine Coalition, told Pharmacogenomics Reporter. "It represents the first time that the principles of personalized medicine have been passed by both houses of Congress."
Following the President's signing of the bill, the Senate is scheduled to vote on a companion bill of proposed changes made by House Democrats to the main healthcare reform legislation. This so-called "reconciliation side car" bill passed in the House 220-211. The Senate is slated to vote on this bill by March 27.
According to a post by Miller on the PMC blog, "Age of Personalized Medicine," the reconciliation package does not mention CER, which suggests that the personalized medicine-friendly version of CER in HR 3590, as signed into law by the President, will stand.
Originally, the section on CER in the House version of the bill did not contain language specifically on genomic subpopulations. However, it seems the House listened to the urgings of personalized medicine proponents, such as NIH Director Francis Collins and the PMC , to ensure that CER initiatives in the healthcare reform bill include research into genomically defined subpopulations.
About a month before the Senate voted on its version of the healthcare reform bill, the PMC and the Lewin Group released a report outlining areas of alignment between CER and personalized medicine efforts such as product labeling, payment policies, and utilization management. Lack of such alignment would risk the acceptance and advancement of "inadequate or misleading" efficacy and safety data about medical interventions, the report stated [see PGx Reporter 10-28-2009].
It also helped that top HHS officials supported and urged for a personalized medicine-friendly CER agenda. When Human Genome Project pioneer Francis Collins warned that CER legislation without a focus on pharmacogenomics strategies would deter the advancement of personalized medicine, all stakeholders, including those on Capitol Hill, paid attention [see PGx Reporter 10-28-2009].
Similarly, Janet Woodcock, director of the US Food and Drug Administration's Center for Drug Evaluation and Research, said that CER and personalized medicine should converge, and urged for increased investment in the nation's research infrastructure to conduct studies in the real-world setting [see PGx Reporter 11-04-2009].
The PMC has also sent letters to House and Senate leaders pushing for CER policies promulgated in healthcare reform to recognize and promote genomically-guided personalized medicine.
In addition to creating the Patient-Centered Outcomes Research Institute, HR 3590 instructs for the establishment of a 15-member methodology committee that will be responsible for "developing and improving the science and methods of comparative clinical effectiveness research." Members of this committee will be experts from various fields, including genomics and biostatistics.
The institute's research will also be subject to peer review from independent experts who don't have conflicts of interests with the products or interventions being considered. Any research coming out of the institute will be publicly available.
Finally, the bill states that the HHS Secretary "may only use evidence and findings from research conducted [at the CER institute] to make a determination regarding coverage … if such use is through an iterative and transparent process which includes public comment and considers the effect on subpopulations."
However, CER data cannot solely be used to make coverage decisions. Additionally, the institute cannot use quality-adjusted life year estimates, or QALYs, to compare the relative cost-effectiveness of treatments or interventions, and make coverage decisions, according to the bill.
Although the CER provisions in HR 3590 will likely not be impacted by the reconcillation package vote, Senate Majority Leader Harry Reid (D-Nev.) has publicly stated that there are sufficient votes in the Senate to pass the side car bill. In the mean time, Republican Senators are planning to offer amendments to the bill. However, since any changes to the bill will require it to return to the House for a vote on the revisions, Senate leaders have expressed they will attempt to keep the bill "clean."
Although genomically-guided personalized medicine is now a part of the nation's healthcare reform plan in a legal sense, how readily pharmacogenomic strategies will be implemented in CER in practice still remains to be seen.
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.
The Federal Coordinating Council issued a set of priorities to HHS for CER research last year [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.
Around the same time the Federal Coordinating Council released its report, the Institute of Medicine released its 100 top CER priorities, which included only a few proposals for comparing the effectiveness of treatments or clinical management strategies using genomics.