President Obama recently announced the creation of the new Precision Medicine Initiative to "revolutionize how we improve health and treat disease" through better prevention, diagnostics and treatment. The administration has emphasised that public-private partnerships will be critical to the success of the initiative, and specifically calls on academic medical centres, researchers, foundations, privacy experts, medical ethicists and medical product innovators to participate. Such entities should take advantage of this opportunity to help to shape law and policy as it develops to ensure that their interests in the future of healthcare are protected in the short and long term. Stakeholders will have the opportunity to:
- participate in public meetings sponsored by the White House and the federal health agencies, including the National Institutes of Health (NIH), the Food and Drug Administration (FDA), the Centres 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 the NIH to support research or efforts to create the new national research cohort.
The recently released draft 21st Century Cures Act includes a 'placeholder' for the Precision Medicine Initiative, which signals at least interest – if not support – from Congress. Accordingly, in addition to engaging the NIH, FDA, CMS and ONC, the door is open for stakeholders to appeal to government policymakers for substantive changes and higher levels of funding in this area.
Precision medicine relies on an analysis of an individual's unique genetic profile, environment and lifestyle to help to prevent the 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 make-up and lifestyle needs;
- clinical decision-making software and other forms of health information technology that support individualised care;
- consumer health and wellness tools that help individuals to manage their own health; and
- personalised, integrated care provided by physicians, hospitals and other providers.
The initiative is the latest – and perhaps least controversial – action in a series of government efforts in the molecular diagnostic arena. It follows on the heels of the FDA's proposed regulation of laboratory developed tests and CMS's implementation of:
- the Molecular Diagnostic Services Programme, which establishes Medicare coverage for certain molecular diagnostic tests; and
- the Protecting Access to Medicare Act 2014, which will revamp the Medicare clinical laboratory payment system beginning in 2017, including providing special payment terms for certain advanced diagnostic tests.
Activities supported by the initiative
The government has requested $215 million to support the Precision Medicine Initiative, most of which will be devoted to research. Of this total, $130 million will be allocated to the NIH for the development of a national research cohort of 1 million volunteers, who will contribute comprehensive health data for research purposes. An additional $70 million will go to 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 technologies. Finally, $5 million will be directed to the ONC to assist with the development of necessary interoperability data standards and privacy requirements for this initiative.
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 programmes.
Stakeholder opportunities to participate
The 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 programme provides an opportunity to work with the NIH, and possibly to expand the NIH's focus to include populations of interest to them. The NIH has already sponsored one public workshop with commissioned white papers to discuss planning for the cohort, and appears likely to 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 government is to launch a "multi-stakeholder process" to identify and resolve data privacy and security concerns. The ONC will undoubtedly play a major role in this process, particularly with respect to supporting the 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 the FDA could have a significant impact. In particular, as part of the new initiative the FDA will focus greater attention on next-generation sequencing, which is revolutionising understanding of genetics and genomics, and greatly accelerating progress in precision medicine. The potential FDA regulation of laboratory-developed tests, 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 comment pursuant to public solicitations.
For further information on this topic please contact Dora Hughes or Barbara Cammarata at Sidley Austin LLP by telephone (+1 202 736 8000) or email (firstname.lastname@example.org or email@example.com). The Sidley Austin website can be accessed at www.sidley.com.
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