Letter from the Executive Director
When America‘s new president assumes the responsibilities of the American presidency on January 20th, he will confront enormous challenges, including a troubled economy, global warming and the developed world‘s dependence on unstable supplies of oil. Not least among those challenges is the rising cost of healthcare, which has been a major focus of the just-completed campaign. Unless these issues are resolved, the people of the USA, as well as other nations, will find our world will become poorer, meaner and less healthy.
Fortunately, we are in an era where old answers have been found wanting. There is receptivity to trying big, bold new approaches based on paradigm-changing advances in science and technology. One of those approaches is what we call personalized medicine – the idea that linking drug discovery, development and delivery to our emerging understanding of molecular diagnostics, mediated by powerful health information technologies, can improve health and lower overall costs.
When it comes to realizing the promise of personalized medicine, the new president will not be starting with a blank slate. Substantial bipartisan support for this vision has developed over the past few years, and observers outside the federal government have suggested ways the government can expedite development and adoption of personalized medicine. Now, many of those ideas have been endorsed, and others proposed, by a presidential advisory group.
In September 2008, the President‘s Council of Advisors on Science and Technology (PCAST), a group of leading figures in the private sector who offer counsel on science policy in the USA, issued a little-heralded but important report, Priorities for Personalized Medicine. According to PCAST co-chairs John H Marburger III, director of President Bush‘s Office of Science and Technology Policy, and E Floyd Kvamme, a partner in venture capitalist firm Kleiner Perkins Caufield & Byers in Menlo Park (CA, USA), the promise of better health outcomes at lower systemic costs requires “the Federal Government [to] develop a strategic, long-term plan that coordinates public and private sector efforts to advance research and development relevant to personalized medicine.”
Under the direction of PCAST member M Kathleen Behrens, a general partner in RS & Co. Venture Partners IV, a venture capital fund in San Francisco (CA, USA), Priorities for Personalized Medicine outlines a strategy to realize the vision of personalized medicine. Building on the foundation that US Health and Human Services (HHS) Secretary Michael Leavitt put in place with his personalized healthcare initiative, the report focuses on three priorities: technology and tools, regulation and reimbursement.
Technology & tools
The report states that among the significant scientific roadblocks to widespread clinical adoption of genomics-based molecular diagnostics is the lack of follow-up research to validate genetic markers that have been identified by early research. As the validation of genomic correlations with disease is a new, expensive and high-risk research area, the government will need to work with the private sector to make sure the research gets carried out, the report concludes.
In addition, the government needs to develop a long-term plan for public and private sector research and development, working with the private sector to create a public/private ‘Personalized Medicine R&D Roadmap‘ for coordinating discovery and translational research.
The report also recommends that government and industry join forces to create new tools that will facilitate further research: building a collection of high-quality biological specimens accompanied by comprehensive disease annotation; using standardized biomarkers and incorporating sophisticated statistical methods that can demonstrate the clinical validity of genomic profiles in studies; and assembling a large population cohort for longitudinal health and disease studies.
Regulation
While the US FDA has made progress in developing regulations for genomics-based molecular diagnostics, the report says its guidance remains ambiguous or incomplete in several important areas. Among them:
| ▪ | Developing criteria that define risk for products, including diagnostic tests, where information is the key result; | ||||
| ▪ | Developing standards for study design and product performance with regard to regulatory review of new diagnostic products; | ||||
| ▪ | Coordinating with the Centers for Medicare and Medicaid Services (CMS) to ensure that the two federal agencies do not have redundant regulations for personalized medicine products; | ||||
| ▪ | Setting standards for labeling therapeutic products that use diagnostics; | ||||
| ▪ | Deciding how to regulate computerized clinical decision systems. | ||||
The Critical Path Initiative launched by the US FDA in 2004, which is designed to help smooth development of drugs and devices, has been hampered by inadequate funding, a situation that needs to be remedied, the report notes.
Finally, the private sector, the report recommends, needs to work proactively with the FDA on regulatory policy. Industry should respond “in a substantive and positive way” to requests for information and draft guidance released by the FDA, by submitting alternatives “rather than primarily registering objections,” the report states.
Reimbursement
The authors identify three key challenges to containing healthcare costs without obstructing the adoption of genomics-based molecular diagnostics. First, genomics-based molecular diagnostic tests are currently reimbursed at the same rate as other laboratory tests – in other words, as low-margin commodity items. Industry is unlikely to develop new products if it is unable to recover its development costs, according to the report. Second, standards are lacking for the evidence that CMS and other payers will require to validate the benefits of these tests in practice. Third, the procedural hurdles associated with coding systems, bundled payments and complex billing procedures make it particularly difficult for patients to receive reimbursement for innovative molecular diagnostics, and, therefore, to benefit from the promise personalized medicine represents.
For personalized medicine to develop, public and private payers should set reimbursement rates for genomics-based molecular diagnostics based on their overall impact on patient care, the report concludes. Payers should also help develop standards for clinical trial designs that they would accept as providing sufficient evidence to cover a particular diagnostic test.
In addition, payers should collaborate with test developers to establish more flexible coding approaches for reimbursement. They should expand “coverage with evidence development” programs that extend coverage and reimbursement while a product is being investigated, as a way of encouraging new product development, the report says.
The Personalized Medicine Coalition supports most, if not all, of the recommendations. We look forward to working with the new administration to put them in place, beginning with the report‘s final proposal, that the personalized medicine office that Secretary Leavitt created at HHS be made permanent.
A link to the report is available online at www.ostp.gov/cs/pcast.