The President recently announced the creation of a new “Precision Medicine Initiative” to “revolutionize how we improve health and treat disease” through better prevention, diagnostics and treatments. The Administration has emphasized that public-private partnerships will be critical to the success of this Initiative, and specifically calls on academic medical centers, researchers, foundations, privacy experts, medical ethicists and medical product innovators to participate. These entities should proactively take advantage of this opportunity to help shape law and policy as it develops to ensure that their interests in the future of health care are protected in the short and long term. Stakeholders will have the opportunity to do the following:

  • Participate in public meetings sponsored by the White House and the federal health agencies, including the National Institutes of Health (NIH), Food and Drug Administration (FDA), Centers for Medicare and Medicaid Services (CMS) and the Office of the National Coordinator for Health Information Technology (ONC);
  • Submit comments in response to solicitations by these agencies, especially during notice and comment rule-making; and
  • Enter into partnerships or agreements with NIH to support research or efforts to create the new national research cohort, as described more fully below.

Notably, the recently released draft 21st Century Cures Act includes a “placeholder” for the Precision Medicine Initiative, which signals at least interest, if not support, by the Congress. Accordingly, in addition to engaging NIH, FDA, CMS and ONC, the door is clearly open for stakeholders to appeal to policymakers on Capitol Hill for substantive changes and higher levels of funding in this area.

What is Precision Medicine?

“Precision Medicine” relies on an analysis of an individual’s unique genetic profile, environment and lifestyle to help prevent onset of disease, predict which treatments may be most effective and monitor response to therapy. It includes advanced diagnostic laboratory tests, including genetic and genomic tests and companion diagnostics; targeted medications that reflect individual genetic makeup and lifestyle needs; clinical decision-making software and other forms of health IT that support individualized care; consumer health and wellness tools that help individuals manage their own health; and personalized, integrated care provided by physicians, hospitals and other providers.

This Initiative is the latest, and perhaps least controversial, action in a series of Administration efforts in the molecular diagnostic arena. It follows upon the heels of the FDA’s proposed regulation of laboratory developed tests (LDTs) and CMS’ implementation of both the Molecular Diagnostic (MolDx) Services Program, which establishes Medicare coverage for certain molecular diagnostic tests, and the Protecting Access to Medicare Act of 2014 (PAMA), which will revamp the Medicare clinical laboratory payment system beginning in 2017, including providing special payment terms for certain advanced diagnostic tests.

What activities will the Precision Medicine Initiative support?

The Administration has requested $215 million to support the Precision Medicine Initiative, most of which will be devoted to research. Specifically, of this total, $130 million is allocated to the National Institutes of Health for the development of a national research cohort of one million volunteers, who will contribute comprehensive health data for research purposes. An additional $70 million is requested for the National Cancer Institute, which will help to “[expand] genetically based clinical cancer trials” and establish a “national cancer knowledge network.”

Of the remaining $15 million, $10 million will go to the FDA for regulatory science and infrastructure support, which includes a specific focus on developing a new approach for evaluating next generation sequencing (NGS) technologies. Finally, $5 million is directed to the ONC to assist with the development of necessary interoperability data standards and privacy requirements for this Initiative.

Importantly, patients and patient advocates will be included throughout the Initiative, with a major objective being the empowerment of individuals and families to “invest in and manage their health.” However, it remains to be seen whether and how the products and knowledge gained from these research initiatives will be covered and reimbursed by Medicare, Medicaid and other public and private programs.

What are some of the known stakeholder opportunities to participate?

NIH has stated its interest in identifying and leveraging existing research and clinical networks as part of the process of establishing the voluntary national research cohort. For researchers and innovators, the program provides an opportunity to work with NIH, and possibly expand NIH’s focus to include populations of interest to them. NIH has already sponsored one public workshop with commissioned white papers to discuss planning for the cohort, and by every indication, will likely move quickly to implement this part of the Initiative. Potential uses or restrictions on use of the resulting research data should also be a topic of concern for stakeholders hoping to benefit from this investment.

The White House will launch a “multi-stakeholder process” to identify and resolve data privacy and security concerns. ONC will undoubtedly play a major role in this process, particularly with respect to supporting interoperability of data systems and ensuring privacy of genetic and genomic information. Any restrictions or requirements coming from this process will likely have implications for Precision Medicine and beyond, and all stakeholders should monitor, if not participate directly, in this process.

Finally, for companies with Precision Medicine related products in the pipeline, any regulatory decision-making by FDA could have a significant impact. In particular, as part of the new Initiative, FDA will focus greater attention on next generation sequencing (NGS), which is revolutionizing understanding of genetics and genomics, and greatly accelerating progress in Precision Medicine. FDA’s potential regulation of laboratory developed tests (LDTs), if enforced, will affect such issues as the availability and cost of products critical to the success of Precision Medicine. Innovators, researchers and providers should prepare to share expertise in advance and respond to any draft regulation, including through participation in public meetings and the provision of public comment pursuant to public comment solicitations.