By Turna Ray
This article was posted May 28.
A personalized medicine bill ─ originally introduced three years ago by then Illinois Senator Barack Obama ─ was revived in the US House of Representatives this week by Congressman Patrick Kennedy (D-RI) and Congresswoman Anna Eshoo (D-CA) with several changes to address the three oft-cited barriers to the rapidly evolving field: regulation, reimbursement, and translational research.
The Genomics and Personalized Medicine Act of 2010 (HR 5440), introduced to House Committee on Energy and Commerce, would create an Office of Personalized Healthcare within the HHS Office of the Secretary to not only coordinate activities between government agencies, but also create a translational research agenda; ensure that coverage and reimbursement decisions about personalized medicine products at the Centers for Medicare & Medicaid services take into account "best data available;" identify evidence gaps with regard to whether genomic technologies are cost-effective and improve outcomes; and clarify regulation of personalized medicine products to reduce conflicts between interagency oversight.
To create the OPH, the Kennedy/Eshoo bill requests $5 million for fiscal year 2011. The OPH will be headed by a director appointed by the HHS Secretary.
While Obama's 2007 bill and a version introduced by Kennedy in 2008 touched on the topics of regulation of personalized medicine and interagency coordination, the latest iteration has been updated to address the needs of a field challenged by rapid technological advancement and a dearth of genomic expertise to make sense of emerging data about drug response and people's predisposition to disease.
HR 5440 includes much more toothy language about FDA regulatory muscle over companion tests, instructs the Institute of Medicine to report on the reimbursement environment for personalized medicine products, and creates several committees to address translational challenges of personalized medicine.
To standardize the collection of human biological samples and make access more equitable, HR 5440 also proposes to create a national biobank for use in personalized medicine research, as previous legislative versions have recommended. HR 5440 proposes to award grants for the development of the national biobank and requests a total of $150 million in fiscal year 2011 to fund proposals to expand and accelerate research in this field, including the national biobank.
According to a statement from Kennedy, the new bill is in response to increased public awareness and use of genomic tests.
"The legislation addresses several issues that have arisen with the increased prevalence of genetic testing, including coverage and reimbursement of personalized medicine products, and oversight of genetic tests (including direct-to-consumer marketing)," Kennedy said in a statement. "Given the recent issues surrounding direct-to-consumer marketing of some genetic and genomic tests, the legislation would also direct the FDA, FTC, and CDC to evaluate these products that circumvent both the normal regulatory environment and patient-healthcare provider relationship."
Regulatory Teeth
Compared to previous versions of the personalized medicine bill, HR 5440 is much more specific with regard to the authority the FDA has in not just "encouraging" companion diagnostic development, but requiring it under certain circumstances.
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The HHS Secretary would be able to delineate authority to the FDA commissioner to "require" a drug maker to develop a companion diagnostic in connection with an investigational new drug or biologics application; to develop such a test if post-marketing data for a drug suggests a safety or efficacy benefit in a certain group of patients; or to conduct additional studies for a drug in genomically predefined subpopulations.
The bill would also facilitate the expansion of an interagency adverse events reporting mechanism for personalized medicine products, including laboratory-developed tests. Plans are already underway to incorporate genomics into the FDA's adverse events capture capabilities.
Currently, the Adverse Events Reporting System, or AERS, is the agency's computerized system to monitor the ill effects of drugs. But in the post-market setting, ADRs may not be detected for several years after the drug has been approved. As genomics firms have begun to amass large databases of genetic risk association data, the FDA has expressed a desire to use this information to improve its ability to detect adverse events.
In March, FDA sought input from the Advisory Committee for Pharmaceutical Science and Clinical Pharmacology on the agency's preliminary efforts to develop a predictive safety system that integrates pharmacogenomics, chemical structural data, and systems biology approaches to determine drug-induced adverse reactions before they happen (PGx Reporter 03/24/10).
Kennedy's last personalized medicine bill recommended the development of a test registry to collate information on the analytical and clinical validity of laboratory-developed tests. This year, the National Institutes of Health announced plans to launch such a registry.
Regulatory officials agree that NIH's web-based registry is a much-needed tool for tracking the marketing claims and clinical features of genetic tests. However, the NIH has not announced too many details with regard to this repository, particularly how it plans to encourage participation from sponsors and whether direct-to-consumer genomics services will be listed in the web-based portal (PGx Reporter 03/24/10).
In the Kennedy/Eshoo bill it seems the Office of Personalized Healthcare will have some input in the regulation of genomic tests and personalized medicine products. The legislation instructs the OPH director to issue annual reports outlining which personalized medicine products require regulation and draw "a clear delineation" between FDA and CMS' roles in oversight of personalized medicine products, including laboratory-developed tests.
The current deficiencies in the regulation of genomic tests have recently been the topic of much public debate as DTC testing firm Pathway Genomics attempted to sell saliva-collections kits for its genomic testing service at Walgreens and CVS retail stores. Pathway's plans moved the FDA to take regulatory action against the firm, after a long period of silent vigilance over the industry, saying the company had overstepped regulatory boundaries. Simultaneously, the House Committee on Energy and Commerce launched an investigation into the DTC genomics industry, and sent letters to Pathway, 23andMe, and Navigenics requesting information about their business practices (PGx Reporter 05/26/10).
Although these firms are working with FDA and Congress to iron out regulation guiding the nascent field, industry observers have long pointed out that current regulations raise more questions than they answer about how DTC genomics firms should operate. Currently, CMS oversees most laboratory-developed tests, but FDA regulates test kits and certain high-risk laboratory assays that allow a doctor to make treatment decisions. The FDA hasn't issued formal guidelines with regard to DTC genomics but maintains that it has the power to regulate the field, and has been in discussion with industry players all along.
IoM to Research Key Topics
The Kennedy/Eshoo bill asks the Institute of Medicine to issue a report tackling problems in the field, such as reimbursement of genetic tests, reimbursement for genetic counseling services, and the link between family history of disease and pre-existing conditions.
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Obama had introduced an amendment to his 2007 personalized medicine bill for an Institute of Medicine study on the safety of genetic tests, which was criticized by some industry observers as superfluous since several government and independent investigations on the topic had already been conducted (PGx Reporter 06/20/07).
In the new version of the bill, the IoM will study and report on the current billing, coverage, and reimbursement methods of personalized medicine products. Currently, there is no systemized method for payors to see which tests doctors are billing for due to the use of stacked CPT codes. Simultaneously, test developers feel they are not receiving payments commensurate with the value their genomic tests are adding to the personalized care of patients.
The American Medical Association is working on this problem. The AMA, which issues CPT codes, has formed a molecular pathology workgroup to issue recommendations for modernizing the reimbursement codes for molecular diagnostic and genetic tests, and is planning to implement a new coding structure in the next two years (PGx Reporter 03/17/10).
The proposed IoM report would also address reimbursement of genetic counselors, who are currently not recognized by Medicare as healthcare providers. Under current CPT rules, licensed doctors providing genetic counseling can seek reimbursement through CMS for genetic counseling services provided to Medicare beneficiaries, but genetic counselors who are not MDs cannot.
Last year, the National Society of Genetic Counselors was planning to introduce new legislation in Congress proposing that the CMS reimburse for genetic counseling services for Medicare beneficiaries (PGx Reporter 09/16/09). The NSG has not yet introduced its bill, but the Kennedy/Eshoo legislation takes a step toward addressing some of the groups' concerns.
While Kennedy's previous version of the bill included a tax incentive for researchers in the field of genomics, that proposal has been removed from the new version of the bill, likely in light of the $45 million the National Institutes of Health doled out this year for at least 80 pharmacogenomics projects under the American Recovery and Reinvestment Act.
The Kennedy/Eshoo bill asks IoM to recommend "appropriate mechanisms to promote research and development to advance personalized medicine (which may include tax credits, grant programs, or extensions of patent or exclusivity) to include costs and benefits to society."
Addressing concerns of healthcare insurers regarding the implementation of the Genetic Information Nondiscrimination Act, which restricts insurers from asking about or using family history information for underwriting purposes, the proposed bill asks the IoM to develop "criteria for defining when a family history should be considered a personal history of disease for reimbursement purposes under title XVIII of the Social Security Act," Which addresses health insurance for the aged and disabled.
Representatives from the insurance industry have complained that GINA's restriction on family history data undercuts their risk models and restricts payors' ability to offer incentives for participating in health risk assessment questionnaires (PGx Reporter 03/31/10).
Translation GAPP
Although the Kennedy/Eshoo bill includes much more specific language with regard to regulation and reimbursement than previous legislative efforts, it is currently unclear to what degree the proposals in the new version will be divisive or consensus building.
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Obama's bill languished in Congress as a result of being too broad. In Kennedy's version of the bill introduced in 2008, the proposal to give tax credits to the players in the personalized medicine field was a controversial item.
Although this new bill gives OPH the power to make regulatory recommendations and address reimbursement of personalized medicine products, many government advisory bodies have already issued numerous proposals for advancing the personalized medicine industry, many interagency workgroups and public-private partnerships are already mulling how to work together to translate genomic discoveries to patients' bedsides, and countless reports have concluded that the regulation of genetic test needs more clarity.
Very few of these efforts have helped to untangle the web of regulatory, reimbursement, and translational research challenges barring broad incorporation of personalized medicine strategies into the healthcare system. With this in mind, the jury is out as to how the more collaborative, research-driven proposals in HR 5440 will be received in Congress and by stakeholders.
The HHS Secretary's Advisory Committee on Genomic, Health, and Society has broadly proposed solutions to many of the challenges taken up in the Kennedy/Eshoo bill. The advisory body was the first to formally recommend the development of a test registry, and the group has also called for aligning CMS/FDA regulation of laboratory-developed tests, and extending FDA's oversight power over all tests. While some of SACGHS' recommendations regarding test regulation have materialized, such as the testing registry, a majority of them have fallen by the wayside.
Muin Khoury, director of CDC's Office of Public Health Genomics, recently bemoaned the paucity of genomics-related prevention and education objectives proposed as part of the federal government's Healthy People 2020 Project, an HHS-led effort that works to improve certain health outcomes in areas of great need as identified by a diverse consortium of stakeholders.
Under the "genomics" category of the project, the 55-member interagency consortium has proposed two goals involving genomics: Increase the proportion of newly diagnosed colorectal cancer patients who receive genetic testing to identify Lynch syndrome or familial colorectal cancer syndromes; and increase the proportion of women with a family history of breast and ovarian cancer who receive genetic counseling (PGx Reporter 02/17/10).
Khoury is also the head of GAPPNet, a new CDC and National Cancer Institute-led effort to drive the adoption of validated and clinically useful genomic technologies (PGx Reporter 07-15-09). In its effort to increase the use of genomic technologies at a population level, GAPPNet will use, among other resources, evidence-based reviews of genomic technologies by CDC's Evaluation of Genomic Applications in Practice and Prevention to guide its work.
HR 5440 would establish several committees to help drive adoption of personalized medicine, including one entity under the CDC to conduct comparative analysis of laboratory review requirements under CLIA; another group to engage the private sector on issues impacting the industry; and an advisory body that would allow CDC to continue conducting evidence-based reviews into the utility and validity of genomic technologies through the creation of a new advisory committee called the Committee on Evaluation of Genomic Application in Practice and Prevention. However, it is currently unclear how all the "GAPPs" at CDC will play together.