During his January 2015 State of the Union address, U.S. President Barack Obama announced the launch of a national Precision Medicine Initiative.¹ The Mission Statement of the Precision Medicine Initiative reads: “To enable a new era of medicine through research, technology, and policies that empower patients, researchers, and providers to work together toward development of individualized treatments.”

What is precision medicine?
The core approach to healthcare customized to individual patient characteristics, which precision medicine by definition represents, is definitely not a new one. Typing of blood ABO and Rh antigens for the purpose of transfusions, and typing of histocompatibility antigens prior to organ transplantation, are two common examples of treatment based upon an individual’s genetics.

However, development of precision medicine has increasingly become a focal point across a range of specific disease areas with the goal of improving patient outcomes. Traditional “one size fits all” approach, generally comprising most of today’s treatment and prevention regimens, in reality achieves only a “one size fits most” status and can leave considerable gaps that many individuals fall through. Genetic makeup, health-related behavior, environment, and lifestyle factors are all considerations for identifying subpopulations of patients for targeted treatment that broader precision medicine development hinges upon to support improved patient health and development and delivery of improved healthcare modalities. To this end, the integrated field of molecular pathological epidemiology (MPE) can be applied to ascertain disease origins, and by extension methods of prevention or control, by identifying biomolecular characteristics associated with a given disease.²

The Precision Medicine Initiative calls for significant financial investments to allow the National Institutes of Health (NIH), Food and Drug Administration (FDA), and Office of the National Coordinator for Health Information Technology (ONC) to develop and support a volunteer base, to expand their efforts in identifying genomic markers, and develop greater expertise and robust databases alongside strengthened information security.

Why should we care?
In the immediate future, the Precision Medicine Initiative intends to accelerate advances by the National Cancer Institute (NCI) and networked clinician scientists in cancer treatment and prevention via expansion of team- and biomarker-driven clinical cancer trials focused more on cancer genomics than on tumor site. According to the latest available statistics from the America Cancer Society, in 2016, there will be an estimated 1,685,210 new cancer cases diagnosed and 595,690 cancer deaths in the U.S.

Challenges to the future of cancer research regarding accelerating scientific innovation were outlined previously in the President’s Cancer Panel Annual Report 2010-2011.³ This report emphasizes the need to streamline and strengthen the process by which basic and clinical research in cancer therapies is conducted to accelerate the pace at which patients in dire need of effective treatment may optimally benefit. The need to transform oncology research and therapy development via team science-driven clinical trials initiatives employing logical approaches to selecting patients based on biomarkers, and with fortified research infrastructure in place to support data sharing and biorepositories, is key to expediting drug approval and subsequently commercial access to favorable therapies. A further impetus to the push for innovating cancer research was provided on January 28, 2016, when President Barack Obama announced the White House Cancer Moonshot Task Force to support expediting efforts in the prevention, treatment, and cure of cancer.⁴

In the longer term, the Precision Medicine Initiative will establish, via the NIH and other parties, a national research cohort of at least one million American volunteer participants from whom a range of MPE data and biological samples are obtained and then shared among cross-disciplinary clinical scientists for the large-scale development of new insights into health and disease at individual, community, and population levels.

The potential benefits of the Precision Medicine Initiative to medical researchers, clinicians, and consumers are as far-reaching as the comprehensive long- and short-term goals of the initiative outlined above. Partnerships forged between public, private, and academic scientists, clinicians, and engineers should result in innovative tools for disease diagnosis and treatment, as well as the collection and analysis of large repositories of biological specimens and data. As the fruits of these Precision Medicine Initiative partnerships mature, benefits ripe for harvest potentially include transformation of the current practice of “one size fits all” healthcare into a broad, integrated paradigm which provides molecularly-optimized treatment to patients on the basis of genetics and other factors that influence underlying disease mechanisms.

Gaining support for success
Given the massive scale of these proposed efforts, infrastructure requirements to effectively pursue the Precision Medicine Initiative’s goals must accommodate seamless communication and integration demands inherent to cross-sector partnerships as well as the legal requirements for protection of patient privacy and regulatory oversight. In February 2016, President Obama announced new infrastructure-boosting federal investments and actions to accelerate the Precision Medicine Initiative, and more than 40 private entities have joined with commitments to partner in the acceleration in supporting the key principles of the initiative.⁵

Key principles of the Precision Medicine Initiative include:

• Making it easier for patients to access, understand, and share their own digital health data, including donating it for research;
• Engaging participants as partners in research, including returning results to them in dynamic, user-centered ways;
• Bring the promise of precision medicine to everyone;
• Opening up data and technology tools to invite citizen participation, unleash new discoveries, and bring together diverse collaborators to share their unique skills;
• Adhering to strong privacy and data security principles; and
• Advancing and scale precision medicine approaches in clinical practice.

Support requirements to successfully realize the end goal of precision medicine development to the point of standard practice will require parties involved in the initiative’s work, from public to private and academic organizations, engage with seasoned internal or external service providers who can facilitate meeting demands including, but not limited to:

• Big data acquisition and handling capabilities—e.g., to store, manage, and analyze data from full genome sequencing of hundreds of thousands to millions of individuals);
• Optimization and implementation of unified systems to facilitate patient recruitment and enrollment;
• Biorepository management and dispensation capable of tracking and sharing specimens among multiple parties; and
• Streamlining of often cumbersome and time-intensive regulatory processes to expedite clinical trial initiation, track progress milestones, and ensure patient safety.

Precision medicine of the scale envisioned in the aforementioned initiative is in its early stages, and there remains much to be learned as it grows and matures. The development and standard practice of precision medicine will not be an easy path. In the second part of this two-part article, we will discuss some of the hurdles that face the Precision Medicine Initiative. 

References

1. The White House Office of the Press Secretary. (2015). FACT SHEET: President Obama’s Precision Medicine Initiative. Retrieved August 31, 2016, from https://www.whitehouse.gov/the-press-office/2015/01/30/fact-sheet-president-obama-s-precision-medicine-initiative
2. Ogino, S., P. Lochhead, A.T. Chan, et al. (2013). Molecular pathological epidemiology of epigenetics: emerging integrative science to analyze environment, host, and disease. Mod. Pathol. 26:465-484.
3. President’s Cancer Panel Annual Report 2010-2011. (2012). The Future of Cancer Research: Accelerating Scientific Innovation. Retrieved August 31, 2016, from http://deainfo.nci.nih.gov/advisory/pcp/annualReports/pcp10-11rpt/FullReport.pdf
4. The White House Office of the Press Secretary. (2016). Memorandum — White House Cancer Moonshot Task Force. Retrieved August 31, 2016, from https://www.whitehouse.gov/the-press-office/2016/01/28/memorandum-white-house-cancer-moonshot-task-force
5. The White House Office of the Press Secretary. (2016). FACT SHEET: Obama Administration Announced Key Actions to Accelerate Precision Medicine Initiative. Retrieved August 31, 2016, from https://www.whitehouse.gov/the-press-office/2016/02/25/fact-sheet-obama-administration-announces-key-actions-accelerate

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Dr. Kiley R. Prillman has more than 20 years of experience in biomedical and clinical research. She holds a Ph.D. in Microbiology and Immunology from the University of Oklahoma Health Sciences Center. Visit www.tech-res.com.