FDA declares personalized medicine “has clearly arrived.”
Personalized medicine, or the ability for the medical profession to tailor therapy to particular individuals’ genetic characteristics, has been a long desired but ever elusive goal for the life sciences. However, the prospects for personalized medicine appear to be improving in recent years. These changes come in the wake of a variety of medical advances, including human genomic testing and cancer drugs targeted for individuals with specific genetic profiles.
As public attention to understanding the human genome has increased, the topic has garnered substantial controversy and regulation in this sector is poised to increase. The Food and Drug Administration (FDA) has already indicated its intent to regulate—most recently in a report clarifying its future role in personalized medicine and in warning letters to direct-to-consumer genetic testing companies.
One area of particular regulatory concern is the provision of personal genetic health services, or health risk probabilities based on genetic profiles, especially in the direct-to-consumer space. From paternity tests to DNA ancestry kits, direct-to-consumer testing has been widely available for some time. Yet, only recently the FDA indicated in a warning letter to 23andMe (one of the last operating genetics testing companies which, until last December, provided whole genome sequencing reports for $99) its intent to regulate the provision of personal genetic health services. The FDA identified concerns with commercial marketing and with the interpretation of consumers’ genetic data unless the company validated health risk assessments and information.
Parties disagree on the appropriate role of genetic regulation, or whether personal data should be regulated at all. The question raises a number of legal issues, including consumer autonomy, access to personal genomic information, and the appropriate response to incidental findings or information unrelated to the primary health concern.
The FDA maintains it has the authority to regulate personal genetic data because it defines that data as a medical device under Section 201(h) of the Food Drug & Cosmetics Act. The agency also points to its role as the federal body charged with providing guidance on medical device claims and protecting consumers.
Some health scholars and consumers have weighed in on the propriety of regulation. In a study of consumer attitudes toward regulating direct-to-consumer genetic testing, researchers found many consumers wanted unfettered access to genetics testing services without government regulation, but favored oversight to ensure that the information provided was high quality.
In recent class action litigation against genetics testing companies, plaintiffs allege false advertising because they relied on marketing claims that they could learn more about personal health risks and conditions, but health assessments are no longer available.
Aside from the debate over whether direct-to-consumer sales of personal genetic data should be regulated, scholars disagree about genetic testing’s clinical utility. Health experts who are optimistic about genetic testing and personalized medicine’s future argue the services will better identify and treat high-risk patients. Eric Topol of the Scripps Research Institute, reportedly reasoned that a failure to target higher risk patients with particular genetic variants “leave[s] them like sitting ducks.”
Other health experts caution against misplaced enthusiasm. Ezekiel Emanuel, Chairman of the Department Medical Ethics and Health Policy at the University of Pennsylvania, reportedly stated, “personalized medicine is a myth, it’s hyperbolic.”
While medical experts continue to debate, the FDA appears to be laying the groundwork for future regulation. In a 2013 report, the FDA stated that “the era of personalized medicine has clearly arrived,” because nearly one-third of newly approved drugs in 2011 had some genetic or other biomarker data to characterize their efficacy.
In its report, the FDA disclosed its plans for addressing evolving medical technology, especially in a world of viable personalized medicine. The FDA highlighted its unique role in protecting and promoting Americans’ health. In particular, the report revealed new organizational efforts, such as the creation of program “directorates” and the agency’s development of additional support structures. But, the agency also acknowledged a need to continue to develop regulatory processes, policies, and infrastructure to maintain oversight of future technologies and medical products.
In 2010, the FDA announced a “regulatory science initiative” to modernize its evaluation and approval processes. The FDA’s report cited a number of actions the agency adopted to achieve these benchmarks, including the development of regulatory standards, research methods, and tools.
Ultimately, the FDA report highlights the agency’s ongoing support to this emerging area of medicine. According to FDA Commissioner Margaret Hamburg, the report is intended to “serve as a useful resource for those looking toward a future where all stages of patient care—from prevention to diagnosis to treatment to follow-up—are truly personalized.”
