Commissioner Enhances FDA’s Commitment to Personalized Medicine
Incorporating personalized medicine into the U.S. health care system is a process rife with complexities, but last week, FDA Commissioner Margaret Hamburg outlined some initiatives aimed at surmounting a few of the primary challenges. At the Sixth Annual Keynote Luncheon Address on the State of Personalized Medicine for the Personalized Medicine Coalition, she explained plans that address three cardinal challenges, including a more flexible regulatory path for personalized therapies with a product approval process that adapts to targeted genomic and clinical data, a collaboration between government research and regulatory agencies, and transparency efforts among industry, the FDA, and the patient community in order to maximize the safety and effectiveness of personalized therapies as they are developed and move to the marketplace. The strategies align with many recommendations for collaboration in our report from last year, “Paving the Way for Personalized Medicine.” Notably:
1) The FDA will hone its regulatory approach to adapt to the emerging science of personalized medicine.
Hamburg described the FDA’s plans to build on previous successes by issuing new draft guidance this month on biomarker identification, which will give therapy developers a better idea of how to submit data on genes and proteins to the FDA so that therapies can be tailored to work on patients with specific biomarker profiles. She also encouraged the development of new clinical trial designs through university Centers of Excellence for regulatory science.
Hamburg touted the personalized medicine success stories of the past few years, such as genetic tests that can help calibrate dosing for the widely used blood thinner warfarin, and the HIV drug abacavir, which requires a genetic test to determine if a patient has a form of the virus that will respond to the drug. She also described the fruitfulness of the FDA’s Voluntary Genomic Data Submission Program. Since its inception in 2005, industry has warmed up surprisingly well to the program by submitting substantial amounts of data on the relationship between drugs and genes.
Hamburg also emphasized that in order to ensure the safety and effectiveness of these new personalized technologies, the FDA must adopt an approach to monitoring the entire “life-cycle” of a product, which necessitates post-market follow-up research. She noted that the FDA plans to devise post-market research protocols and that once they are established, it will also make regulators and businesses more confident about the pre-approval process.
2) The FDA is forming crucial interagency collaborations.
The central argument of our report—the need for interagency collaboration—was another focus of her address. The day before her address to the Personalized Medicine Coalition, Hamburg joined National Institutes of Health Director Francis Collins and Department of Health and Human Services Secretary Kathleen Sebelius to announce a new collaborative effort between the NIH and FDA designed to advance regulatory science.
Hamburg explained as well that there have been discussions between FDA and Agency for Healthcare Research and Quality, on some research topics, although she did not mention any specifically. More controversially, an audience member also questioned Hamburg about the possibility of collaboration between the Centers for Medicare and Medicaid Studies, which determines reimbursement, and the FDA. We discuss this in our report as an important way for the FDA to gather information about the real-world usage of drugs and devices and for companies to have a better idea of the economic viability of their products, as has been done with the genetic tests surrounding the drug warfarin, which are only reimbursed if the patient is part of a clinical trial.
3) FDA is making its processes more transparent
Hamburg acknowledged that coordination between CMS and FDA may raise many concerns because the reimbursement rates determined by CMS heavily influence the profitability of drugs and diagnostics, but she made clear that the FDA will need to clearly explain to the public the scientific evidence and administrative rationale behind the decisions that these agencies make. Along with greater flexibility and collaboration, Hamburg considers the transparent sharing of evidence and explanation of policy rationales as one of the major components of FDA modernization.
One of the more logistically complex issues for the future of personalized medicine is the need for coordination between the genetic test manufacturers, the drug manufacturers, and the health care providers. For instance, Hamburg described the FDA’s “scenario-based” approach to “companion technologies” such as a genetic test that is coupled to a drug whose effectiveness on a patient can be determined by the results of that test. While some drugs and diagnostics will be developed in tandem, others will follow separate paths through different companies. Hamburg acknowledged that this will require some process by which different companies can be made aware of all the data coming into the FDA from different sources that may be relevant to each specific product.
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