Monday, February 9, 2015

Three Recommendations for President Obama’s Precision Medicine Initiative

Attribution:  Spencer Nam

One of the pleasantly surprising announcements President Obama made during his 2015 State of the Union address was “a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes.” 

Although the term ‘Personalized Medicine’ is also used to convey this meaning, that term is sometimes misinterpreted as implying that unique treatments can be designed for each individual.[1]

Given precision medicine’s potential to solve many outstanding health care issues and lower costs without compromising clinical quality and performance, the President’s proposal is a welcome initiative. Many of the challenges we face practicing symptom-focused intuitive medicine could be overcome by turning toward precision medicine, a process of precisely diagnosing and targeting disease.
However, announcing the initiative is one thing. As with all policy discussions, the devil is in the details – and there are three details specifically that could make the difference between political rhetoric and a policy that truly improves the health of American citizens.
1.  Focus on the entire process of the disease – starting with prevention. Because most chronic diseases show few symptoms until the disease has significantly progressed, treatments for cancer and diabetes patients are primarily at the disease management phase. However, we are acutely aware that the best way to “cure” cancer or diabetes is prevention, and prevention requires better early diagnosis. Unfortunately, we still lack convenient and accurate ways to diagnose for various cancers and diabetes. Given the high costs of treating advanced-stage chronic diseases, precision diagnosis of risk factors or disease progression will materially lower the costs of health care.
For example, currently the only place we can check hemoglobin A1c (HbA1c), or blood glucose levels, is at physicians’ offices. With nearly 30 million Americans with type-2 diabetes and another 30 million pre-diabetic, it is time to develop a more convenient and affordable way to check for HbA1c so more regular testing can be done, particularly for those with risk factors. Cancer diagnostics is also confusing and difficult, with imaging modalities (CT, PET, MRI, X-ray) as the only reliable diagnostics methods. We need to develop more reliable and accepted diagnostics to identify and monitor cancers in their early stages.
2.    Strategically target diseases. Particularly in cancer and type-2 diabetes, two of the fastest growing disease segments in the United States, there is a significant opportunity for precision medicine to improve early diagnosis and treatment, and lower the costs of care. Remember, we tackled HIV and AIDS issues over the past thirty years with a precise target (HIV) and with research focused on quickly translating basic science to clinically effective and safe drugs. Because cancer and diabetes are systemic diseases, affecting multiple aspects of a human body, focusing on translational science based on specific types of cancer or specific aspects of diabetes may in fact accelerate not only the understanding of the diseases but also improve the treatment methods at each stage.
While our understanding of cancer and diabetes has substantially grown over recent decades, we’ve also found the core issues of these diseases to be much more complex and involved than previously understood. As PresidentKennedy set a national goal of “landing a man on the moon and returning him safely to the earth,” by setting their sights on one or two of the nation’s most critical diseases, President Obama and his team of experts could provide a strategic framework for marshalling resources to make real progress toward their cure.
3.      Set standard definitions and metricsOne of the major challenges in migrating toward precision medicine is lack of a common clinical language and metrics that help us to refine our interpretations and focus our messages to physicians and patients. Because cancer and diabetes are still treated in the realm of intuitive medicine, different physicians can provide different opinions on these diseases. Although we need to appreciate individuals’ genetic and biological uniqueness in discussing chronic diseases, precision medicine cannot establish deep roots without more commonly accepted definitions and associated metrics.  
Gaining more precise understanding of cancer and diabetes and developing precise diagnostics and treatments to be administered at the right time will reduce inefficiency and waste –delivering substantial dividends. However, let’s not forget that a detailed education plan as well as appropriate reimbursement schedule will be integral for the initiative to truly go beyond the drawing board. By providing strategic focus and direction to lead the Precision Medicine Initiative forward,President Obama has an opportunity make a real impact.

Often, though not necessarily, PM involves the application of panomic analysis and systems biology to analyze the cause of an individual patient's disease at the molecular level and then to utilize targeted treatments (possibly in combination) to address that individual patient's disease process. The patient's response is then tracked as closely as possible, often using surrogate measures such as tumor load (v. true outcomes, such as 5 year survival rate), and the treatment finely adapted to the patient's response.[2] The branch of precision medicine that addresses cancer is referred to as "precision oncology".[3]
Inter-personal difference of molecular pathology is diverse, so as inter-personal difference in the exposome, which influence disease processes through the interactome within the tissue microenvironment, differentially from person to person. As the theoretical basis of precision medicine, the "unique disease principle"[4] emerged to embrace the ubiquitous phenomenon ofheterogeneity of disease etiology and pathogenesis. The unique disease principle was first described in neoplastic diseases as the unique tumor principle.[5] As the exposome is a common concept of epidemiology, precision medicine is intertwined with molecular pathological epidemiology (MPE). MPE research is capable of identifying potential biomarkers for precision medicine.[6]
Precision Medicine is and will be in a state of evolution as markers are defined and testing becomes cost-effective. Moving Precision Medicine into the clinic, from research will usher in a new age of medicine.
Spencer Nam is a senior research fellow at the Clayton Christensen Institute for Disruptive Innovation, where his work focuses on disruptive innovations in the health care industry.

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