United States: The President's Precision Medicine Initiative – The First Annual Check-Up

Watching President Obama's recent 2016 State of the Union Address reminded me that one year has passed since the President announced a new "precision" or personalized medicine initiative to advance personalized, effective therapies for the American public. It was during his 2015 State of the Union Address that the President 1

"[T]onight, I'm launching a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes, and to give all of us access to the personalized information we need to keep ourselves and our families healthier. We can do this."

Soon after the President's address, details emerged regarding the Precision Medicine Initiative ("PMI"). $215 million was committed from the President's 2016 budget to be broadly allocated to National Institutes of Health ("NIH"), the Food and Drug Administration ("FDA") and the Office of the National Coordinator for Health Information Technology ("ONC"). The money was to be invested as 2

  • $130 million to NIH for the development of a voluntary national research cohort of a million or more volunteers who will voluntarily share health, familial and genetic information to be shared among responsible researchers;
  • $70 million to the National Cancer Institute (NCI), part of NIH, to understand the genetic drivers of cancer and improve therapies;
  • $10 million to FDA to modernize the regulatory structure and process to advance precision medicine;
  • $5 million to ONC for the development of interoperability standards and requirements that support PMI.

The money was to be invested to support PMI's stated objectives 3

  • More and better treatments for cancer;
  • Creation of a voluntary national research cohort;
  • Commitment to protecting privacy;
  • Regulatory modernization; and
  • Public-private partnerships.

NIH's Voluntary Research Cohort

In March of 2015, a PMI Working Group ("Group") was organized by NIH to develop a framework for the volunteer cohort program. As an initial matter, the group was challenged to develop a framework for "creating and managing the voluntary research cohort to understand the variables that contribute to health and disease."4 The Group's report (The Precision Medicine's Initiative Cohort Program – Building a Research Program for 21st Century Medicine, "Report" available here) was published in September of 2015 with the following recommendations:

  • Cohort Assembly. The Group supported the initial goal to include one million diverse volunteers with the number of volunteers to increase over time. The volunteers should be required to share their health data, provide a biospecimen, and be available for future research. Two approaches were recommended for the recruitment of volunteers. First, any individual living in America may volunteer provided they consent to undertake a baseline health exam and provide a biospecimen. They also would be required to share available health data by either directing their electronic health record (EHR) data to the researchers and/or by undergoing an initial exam with a health care provider. Volunteers also must consent to be contacted for follow up. The second recommended approach would recruit volunteers by collaborating with healthcare provider organizations (HPOs).
  • Participant Engagement. The Group recommended that the volunteer cohort be developed and allowed to contribute in all aspects of the cohort. Participants are recommended to have significant representation on governance and oversight committees. To ensure that participant-related interactions remain consistent throughout the cohort study, the Group recommended that activities related to engagement and communications be organized and managed by a central entity. Volunteers should get their individual results as well as information from the cohort aggregate. Volunteers also should be free to set preferences to dictate how much personal information they receive, and to be able to change their preferences throughout their participation in the study.
  • Data Considerations. A combination of well-proven and innovative methods and technologies for data collection and management is noted by the Group to be necessary for successful development of the cohort program. To that end, the Working Group recommends that the initial core data set acquired from all cohort participants be collected and stored centrally and that this initial data set be aligned comparable core data sets where possible. The recommended initial core data set includes data from electronic health records, health insurance organizations, participant surveys, mHealth technologies, and biologic investigations, and would be expected to grow with time. Report at page 3. Existing data standards and common data models are recommended to be used where possible to normalize information from various platforms.
  • Security. Data access control is recommended to ensure that the collected data is used responsibly and securely. Access will depend on the sensitivity of the data, from open-access to role-based access for more sensitive information. Report at page 4. Participants should have access to their own data where appropriate, but access could be denied if there are compelling concerns about potential harm that may arise from such access.
  • Biospecimens. The collection and use of biological specimens is considered essential to the successful development of the cohort program. While there are many different types of tissue that could be collected, a new blood specimen is recommended as the initial specimen. The biological specimens should be sent to central biorepository, "which will support collection, processing, storage, retrieval and biochemical analysis and/or shipment to analytic laboratories." Report at page 5.
  • Policy considerations. The Report noted that the success and longevity of the cohort program would be influenced by the "laws, regulations, and policies surrounding research, data security and privacy, and access and interoperability" electronic health records" Report at page 4. One notable recommendation of the Group is the use of a single IRB to reduce administrative burden and associated costs of the cohort, review time, and to harmonize inconsistent or conflicting policies between different patient groups.
  • Governance. The Group recommended that a director be appointed to lead the patient group. The director should "have authority to set short and long-term goals, develop solicitations and make award recommendations, and have authority over data sharing, storage, and use. Report at page 5. Also recommended is the creation of a Steering Committee of critical stakeholders led by a smaller Executive Committee and five subcommittees that would report to and the director. An Independent Advisory Board should also be created to provide external oversight for the patient cohort group.

NCI and Genetic Research

In August of 2015, the National Cancer Institute announced that it was focusing its PMI activities in for broad 5

  • Expanding Precision Medicine Clinical Trials. NCI announced that in partnership with both the public and private sectors, several precision medicine trials were initiated. An example of such is a clinical trial initiative that seeks to match patients with clinical trials, based on the genetic abnormalities in a patient's tumors, regardless of the type of cancer they have.
  • Overcoming Drug Resistance. NCI is increasing its support of research to address treatment resistance. This initiative is reported to involve:
    • Using tumor profiling to better understand treatment resistance; and
    • Establishing a repository of laboratory models derived from patient tumor samples to investigate drug resistance.
  • Developing New Laboratory Models for Research. NCI will increase its efforts to develop new laboratory models of human cancer.
  • Developing a National Cancer Knowledge System. NCI reported that a national database would be established to house and integrate genomic information from tumors with clinical response data (e.g., tumor shrinkage) and outcomes information (e.g., length of survival) as a resource for scientists, health care professionals, and patients.6

Health Information Technology

In September of 2015, the Health Information Technology Standards Committee (HITSC) provided their recommendations to support of PMI. The group's recommendations, outlined in their reporting letter, fell into four categories:

  • Readily Applicable Standards for PMI – defined as standards that have been tested and implemented in the health IT market that can be put to use to support the NIH PMI cohort and precision medicine.
  • Promising Standards for PMI – defined as standards that may require additional effort, including further pilots and testing, to support precision medicine.
  • Standards Gaps for PMI – defined as areas where considerable additional standards development work is needed.
  • Accelerators – identified as opportunities to advance and improve the standards that fell in the previous categories.

Food and Drug Administration

In December of 2015, FDA announced the launch of an online, cloud-based, portal that will allow interested stakeholders to collaborate and develop the applications of next-generation sequencing. As explained by FDA's blog,

"The precisionFDA platform is a part of this larger effort and through its use we want to help scientists work toward the most accurate and meaningful discoveries. precisionFDA users will have access to a number of important tools to help them do this. These tools include reference genomes, such as "Genome in the Bottle," a reference sample of DNA for validating human genome sequences developed by the National Institute of Standards and Technology. Users will also be able to compare their results to previously validated reference results as well as share their results with other users, track changes and obtain feedback."

One Year Later — Much Done, Much More to Accomplish

In just one year, much has been accomplished to move the President's initiative forward.

An extensive framework has been created and associated funding and other support is being provided. We are at an exciting time for personalized medicine.


1. Remarks by the President in State of the Union Address, January 20, 2015, released by the White House Office of Press Secretary, available at https://www.whitehouse.gov/the-press-office/2015/01/20/remarks-president-state-union-address-january-20-2015.

2. The Fact Sheet: President Obama's Precision Medicine Initiative, released by the White House Office of Press Secretary, January 30, 2015, available at https://www.whitehouse.gov/the-press-office/2015/01/30/fact-sheet-president-obama-s-precision-medicine-initiative.

3. Id.

4. https://www.nih.gov/precision-medicine-initiative-cohort-program/pmi-working-group.

5. NCI and the Precision Medicine Initiative, available at http://www.cancer.gov/research/key-initiatives/precision-medicine.

6. Id.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

To print this article, all you need is to be registered on Mondaq.com.

Click to Login as an existing user or Register so you can print this article.

Similar Articles
Relevancy Powered by MondaqAI
In association with
Related Topics
Similar Articles
Relevancy Powered by MondaqAI
Related Articles
Related Video
Up-coming Events Search
Font Size:
Mondaq on Twitter
Register for Access and our Free Biweekly Alert for
This service is completely free. Access 250,000 archived articles from 100+ countries and get a personalised email twice a week covering developments (and yes, our lawyers like to think you’ve read our Disclaimer).
Email Address
Company Name
Confirm Password
Mondaq Topics -- Select your Interests
 Law Performance
 Law Practice
 Media & IT
 Real Estate
 Wealth Mgt
Asia Pacific
European Union
Latin America
Middle East
United States
Worldwide Updates
Registration (you must scroll down to set your data preferences)

Mondaq Ltd requires you to register and provide information that personally identifies you, including your content preferences, for three primary purposes (full details of Mondaq’s use of your personal data can be found in our Privacy and Cookies Notice):

  • To allow you to personalize the Mondaq websites you are visiting to show content ("Content") relevant to your interests.
  • To enable features such as password reminder, news alerts, email a colleague, and linking from Mondaq (and its affiliate sites) to your website.
  • To produce demographic feedback for our content providers ("Contributors") who contribute Content for free for your use.

Mondaq hopes that our registered users will support us in maintaining our free to view business model by consenting to our use of your personal data as described below.

Mondaq has a "free to view" business model. Our services are paid for by Contributors in exchange for Mondaq providing them with access to information about who accesses their content. Once personal data is transferred to our Contributors they become a data controller of this personal data. They use it to measure the response that their articles are receiving, as a form of market research. They may also use it to provide Mondaq users with information about their products and services.

Details of each Contributor to which your personal data will be transferred is clearly stated within the Content that you access. For full details of how this Contributor will use your personal data, you should review the Contributor’s own Privacy Notice.

Please indicate your preference below:

Yes, I am happy to support Mondaq in maintaining its free to view business model by agreeing to allow Mondaq to share my personal data with Contributors whose Content I access
No, I do not want Mondaq to share my personal data with Contributors

Also please let us know whether you are happy to receive communications promoting products and services offered by Mondaq:

Yes, I am happy to received promotional communications from Mondaq
No, please do not send me promotional communications from Mondaq
Terms & Conditions

Mondaq.com (the Website) is owned and managed by Mondaq Ltd (Mondaq). Mondaq grants you a non-exclusive, revocable licence to access the Website and associated services, such as the Mondaq News Alerts (Services), subject to and in consideration of your compliance with the following terms and conditions of use (Terms). Your use of the Website and/or Services constitutes your agreement to the Terms. Mondaq may terminate your use of the Website and Services if you are in breach of these Terms or if Mondaq decides to terminate the licence granted hereunder for any reason whatsoever.

Use of www.mondaq.com

To Use Mondaq.com you must be: eighteen (18) years old or over; legally capable of entering into binding contracts; and not in any way prohibited by the applicable law to enter into these Terms in the jurisdiction which you are currently located.

You may use the Website as an unregistered user, however, you are required to register as a user if you wish to read the full text of the Content or to receive the Services.

You may not modify, publish, transmit, transfer or sell, reproduce, create derivative works from, distribute, perform, link, display, or in any way exploit any of the Content, in whole or in part, except as expressly permitted in these Terms or with the prior written consent of Mondaq. You may not use electronic or other means to extract details or information from the Content. Nor shall you extract information about users or Contributors in order to offer them any services or products.

In your use of the Website and/or Services you shall: comply with all applicable laws, regulations, directives and legislations which apply to your Use of the Website and/or Services in whatever country you are physically located including without limitation any and all consumer law, export control laws and regulations; provide to us true, correct and accurate information and promptly inform us in the event that any information that you have provided to us changes or becomes inaccurate; notify Mondaq immediately of any circumstances where you have reason to believe that any Intellectual Property Rights or any other rights of any third party may have been infringed; co-operate with reasonable security or other checks or requests for information made by Mondaq from time to time; and at all times be fully liable for the breach of any of these Terms by a third party using your login details to access the Website and/or Services

however, you shall not: do anything likely to impair, interfere with or damage or cause harm or distress to any persons, or the network; do anything that will infringe any Intellectual Property Rights or other rights of Mondaq or any third party; or use the Website, Services and/or Content otherwise than in accordance with these Terms; use any trade marks or service marks of Mondaq or the Contributors, or do anything which may be seen to take unfair advantage of the reputation and goodwill of Mondaq or the Contributors, or the Website, Services and/or Content.

Mondaq reserves the right, in its sole discretion, to take any action that it deems necessary and appropriate in the event it considers that there is a breach or threatened breach of the Terms.

Mondaq’s Rights and Obligations

Unless otherwise expressly set out to the contrary, nothing in these Terms shall serve to transfer from Mondaq to you, any Intellectual Property Rights owned by and/or licensed to Mondaq and all rights, title and interest in and to such Intellectual Property Rights will remain exclusively with Mondaq and/or its licensors.

Mondaq shall use its reasonable endeavours to make the Website and Services available to you at all times, but we cannot guarantee an uninterrupted and fault free service.

Mondaq reserves the right to make changes to the services and/or the Website or part thereof, from time to time, and we may add, remove, modify and/or vary any elements of features and functionalities of the Website or the services.

Mondaq also reserves the right from time to time to monitor your Use of the Website and/or services.


The Content is general information only. It is not intended to constitute legal advice or seek to be the complete and comprehensive statement of the law, nor is it intended to address your specific requirements or provide advice on which reliance should be placed. Mondaq and/or its Contributors and other suppliers make no representations about the suitability of the information contained in the Content for any purpose. All Content provided "as is" without warranty of any kind. Mondaq and/or its Contributors and other suppliers hereby exclude and disclaim all representations, warranties or guarantees with regard to the Content, including all implied warranties and conditions of merchantability, fitness for a particular purpose, title and non-infringement. To the maximum extent permitted by law, Mondaq expressly excludes all representations, warranties, obligations, and liabilities arising out of or in connection with all Content. In no event shall Mondaq and/or its respective suppliers be liable for any special, indirect or consequential damages or any damages whatsoever resulting from loss of use, data or profits, whether in an action of contract, negligence or other tortious action, arising out of or in connection with the use of the Content or performance of Mondaq’s Services.


Mondaq may alter or amend these Terms by amending them on the Website. By continuing to Use the Services and/or the Website after such amendment, you will be deemed to have accepted any amendment to these Terms.

These Terms shall be governed by and construed in accordance with the laws of England and Wales and you irrevocably submit to the exclusive jurisdiction of the courts of England and Wales to settle any dispute which may arise out of or in connection with these Terms. If you live outside the United Kingdom, English law shall apply only to the extent that English law shall not deprive you of any legal protection accorded in accordance with the law of the place where you are habitually resident ("Local Law"). In the event English law deprives you of any legal protection which is accorded to you under Local Law, then these terms shall be governed by Local Law and any dispute or claim arising out of or in connection with these Terms shall be subject to the non-exclusive jurisdiction of the courts where you are habitually resident.

You may print and keep a copy of these Terms, which form the entire agreement between you and Mondaq and supersede any other communications or advertising in respect of the Service and/or the Website.

No delay in exercising or non-exercise by you and/or Mondaq of any of its rights under or in connection with these Terms shall operate as a waiver or release of each of your or Mondaq’s right. Rather, any such waiver or release must be specifically granted in writing signed by the party granting it.

If any part of these Terms is held unenforceable, that part shall be enforced to the maximum extent permissible so as to give effect to the intent of the parties, and the Terms shall continue in full force and effect.

Mondaq shall not incur any liability to you on account of any loss or damage resulting from any delay or failure to perform all or any part of these Terms if such delay or failure is caused, in whole or in part, by events, occurrences, or causes beyond the control of Mondaq. Such events, occurrences or causes will include, without limitation, acts of God, strikes, lockouts, server and network failure, riots, acts of war, earthquakes, fire and explosions.

By clicking Register you state you have read and agree to our Terms and Conditions