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Regulatory Focus™ > News Articles > Regulating Precision Medicine: Capacity vs. Overreach

Regulating Precision Medicine: Capacity vs. Overreach

Posted 05 May 2016 | By Zachary Brennan 

Regulating Precision Medicine: Capacity vs. Overreach

As the boundaries between the clinic, the research lab and industry continue to shift, researchers are calling on the US Food and Drug Administration and other similar regulators worldwide to tread carefully in how they effectively facilitate the practice of precision medicine.

In an article published online this week in the Journal of Law and the Biosciences, titled “Precision medicine: drowning in a regulatory soup?” a group of mostly Australian researchers note that the “key regulatory challenge” is how to effectively maximize the potential benefits of precision medicine, while avoiding excessive and inappropriate utilization that could harm patients, drive up healthcare costs and draw funding away from other needs.

The article focuses on President Barack Obama’s $215 million precision medicine initiative and his call to modernize the regulatory landscape to make way for the “rapid technological change.” Part of that task will be to ensure that these cutting-edge technologies are safe and that people receive the best-indicated treatment, but the researchers also call for the use of what they say are “softer regulatory and educative tools.”

“Whether regulation—either existing or tailor-made for precision medicine—can be effective remains to be seen,” the researchers say, noting five key recurring elements that must be taken into account in the development of any regulatory framework for precision medicine:

  • Appropriate consideration of safety, efficacy and patient need;
  • Cost effectiveness;
  • Consistency/equivalency across geographical, technological, and institutional borders;
  • Respect for cultural differences; and
  • Engagement with all relevant stakeholders.

‘Regulatory Soup’

The researchers point to the quagmire at hand for FDA and others, noting that on the one hand, regulators need to ensure the capacity of existing regulatory structures is adequate to respond to emerging genomic sequencing, gene editing and other technologies, but on the other hand, “regulatory overlap and confusion,” is a risk, as is “a veritable ‘regulatory soup’ that could drown the promised advances in precision medicine.”

And as the cost of genome sequencing comes down, a whole host of new direct-to-consumer offerings are hitting the market, though questions remain over the clinical utility and proof of analytic and clinical validity of some of these products. And FDA has already begun to crack its whip.

In 2013, 23andMe had to stop offering direct-to-consumer (DTC) genomic analysis because FDA said in a warning letter that the company’s flagship product was misbranded. However, in October 2015, 23andMe launched a redesigned service with FDA’s blessing.

In September, November and December 2015, FDA sent letters to six genetic testing companies, including three for marketing unapproved DTC tests, one for marketing an unapproved cancer screening tool and two others for marketing DTC pharmacogenetics tests.

One of the companies’ CEO told us that his test is intended to detect tumor DNA in undiagnosed patients, is being offered in compliance with CLIA and should be treated as a lab-developed test (LDT), which currently does not fall under FDA’s purview.

But FDA and Congress are both in the process of developing somewhat different frameworks for how FDA would oversee LDTs, and as the researchers in this article note, that framework, once finalized, may face legal challenges.

Another fight is beginning to brew over Sen. Mark Kirk’s (R-Ill.) REGROW Act, part of which would allow companies to sell safe stem-cell therapies that have not been proven effective. Similarly, aspects of the House-passed Cures legislation and the related package of bills in the Senate are being described by some experts as regressive attempts to diminish FDA’s control of the pharmaceutical and device industries, and allow questionable types of data to support marketing applications.

Right Mix

The challenges are arising as some commentators “argue that current uncertainties in regulatory requirements are deterring investment in this field” of precision medicine, according to the article. In particular, the authors warn against FDA entering the research space, which “has the potential to slow the progress of genomic discovery, interfere with scientific inquiry and freedom of speech, and upset the primacy of the states to regulate medical practice.

“In many ways, the regulatory challenges presented by precision medicine are reformulations of old tensions—community welfare versus individual liberty, risk versus benefit, autonomy versus paternalism. The potential for precision medicine to exacerbate existing disparities in health care, both within countries and internationally, should also be at the forefront of our concerns,” the authors write.

They also call for regulation be “as future-proofed as possible,” and “need not—and probably should not—result in new, highly targeted laws, which are liable to be outpaced by scientific change.” They also advocate for consumer protections, particularly with regard to the accuracy of the predictions made by DTC testing companies.

Precision medicine: drowning in a regulatory soup?

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