Abstract
Time and again, the hand of government has guided, or prodded, private investment toward the greater good. The pattern is always the same, whether it is applied to roads, canals, and railroads; telephone and electricity networks; the interstate highway system; and even the Internet. Patchy infrastructure, inadequate for the nation's needs, is eventually upgraded by way of “internal improvements” or some other device. And now this pattern may unfold yet again to realize another public good—precision medicine.
Calling precision medicine “an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person,” the National Institutes of Health (NIH) has indicated that it is coordinating efforts to realize the Precision Medicine Initiative, which was launched with a $215 million investment in President Barack Obama's 2016 Budget. The Precision Medicine Initiative also figures in the regulatory deliberations of the Food and Drug Administration (FDA), which has already issued two draft guidances for laboratory-developed tests, a category that includes many genomic tests.
These developments have attracted commentary from experts in the medical community. For example, on May 25, the New England Journal of Medicine (NEJM) published six policy papers on genomic sequencing applications, policy, and challenges. And on June 3, the Journal of the American Medical Association (JAMA) published a pair of viewpoints on the Precision Medicine Initiative. By and large, the experts suggested that they would welcome the helping hand of government, provided the government demonstrated a light touch.
Official White House Photo/Pete Souza
In one of the NEJM papers, “The FDA and Genomic Tests—Getting the Regulation Right,” three leading researchers in law, bioethics, and medical genetics acknowledged that the development of genomic data systems of adequate scale would require significant investments, and that these investments would go far beyond what federal funding agencies like the NIH could or should support. Instead of advocating massive federal spending, the researchers—Barbara J. Evans, Ph.D., J.D. of the University of Houston Law Center, and Wylie Burke, M.D., Ph.D. and Gail P. Jarvik, M.D., Ph.D, both of the University of Washington School of Medicine—focused on bolstering existing regulatory oversight.
In particular, these researchers advocated an expansion of the FDA's application of postmarketing surveillance. This practice, initiated after a series of safety incidents, including those with rofecoxib (Vioxx), has already been implemented to improve the safety of approved drugs. Now it can serve, the researchers argue, to incentivize the development of the massive data systems that doctors and regulators will need to make genomic tests safe and effective for patients.
“Establishing the clinical validity of genomic tests is largely a postmarketing pursuit,” wrote Evans et al. “It requires the accrual and review of evidence throughout the entire commercial life of a test and, indeed, requires access to postmarketing data not just from that test but from all other tests that are trolling the same region or regions of the human genome. Premarket review is the wrong tool, and the traditional product-by-product regulatory focus of the FDA is myopic.”
The authors add that the Precision Medicine Initiative proposes to develop a database reflecting genetic and clinical data for one million volunteers. Even this, the authors say, will be insufficient. Adequate scale means collecting data not just from research settings, but from commercial clinical laboratories to large, diverse populations. According to Evans et al., doing so will require revamped regulations: “Statutory reforms should focus on granting the FDA a correct package of legal powers, seed funding, and legal pathways to encourage public–private partnerships to develop and sustain data resources for the right regulation of genomic testing.”
Similarly, one of the JAMA Viewpoints—“The Precision Medicine Initiative: A New National Effort”—noted that a “new era of medicine also requires new approaches to regulation.” The editorial, which was contributed by Stanford's Euan A. Ashley, MRCP, D.Phil., also emphasized that the debate over regulation “has had polarized views, and recent articles have acknowledged the delicate balance between keeping the public safe while maximizing productivity and potential.” So it is perhaps unsurprising that the editorial included an optimistic assessment of current data-sharing approaches.
“Such approaches have been successful in defining novel syndromes and allowing resources to flow toward targeted therapeutic development,” wrote Dr. Ashley. “National Institutes of Health–funded efforts such as ClinGen and the Undiagnosed Diseases Network are moving forward in scaling such goals.” He added that “scaling is an area in which industry has been particularly successful.” Finally, he cited the example of the Global Alliance for Genomics and Health, which “has, as its central mission, the establishment of a common framework of harmonized approaches for effective and responsible sharing of genomic and clinical data.”