The Promises and Challenges of Precision Medicine

When President Obama announced the Precision Medicine Initiative in January, it was more of a broad goal than a specific action plan. In calling for a million-member cohort, he raised significant medical and technical challenges –  challenges with costs that will far surpass the initial $215 million allotted for the initiative.

Nevertheless, Obama was absolutely right in calling for a focus on precision medicine, which proposes the customization of healthcare by implementing treatment models tailored to individual patients.

I trained in surgery, a blunt tool compared to today’s targeted treatments. The future of medicine is in the specific, the less invasive, and often the more conservative approach. But less intervention requires more information.

An area of healthcare that has recently become very important to me is community transformation projects focusing on the social determinants of health: environment, economic stability, access to care, education and community resources. Only 15 percent of our health is determined by the healthcare we receive, so paying more attention to the remaining 85 percent is not only imperative – it’s just good math.

Precision medicine aims to take targeted genetic and molecular information and consider it in tandem with data about our environmental exposures and lifestyle choices. Integration of the smallest building blocks with the macro environment is exactly the direction in which medicine should be moving.

Getting there will take some new habits, a new funding model and new technologies.

This new medicine is fundamentally dependent on a team approach. When I did my medical training, doctors were not taught to work in teams. But precision medicine is by definition a multidisciplinary approach that includes physicians, researchers, computer scientists and bioinformaticians.

As we look more closely at our molecular and genetic underpinnings, we are integrating our findings with a holistic view of human and environmental interaction. The goal of medicine is no longer only a cure for what hurts, but preventative care to establish health over a lifetime. Medicine’s future lies with teams and cross-disciplinary collaboration.

As a former lawmaker, I think the president’s initiative responds to some important healthcare economics. The federal government currently funds about one third of all medical research and one third of delivered healthcare.

But the economic health challenges facing us are still vast. For instance, the NIH currently spends about $1.3 billion a year on Alzheimer’s research, though the disease is estimated to cost Medicare and Medicaid $154 billion this year, and the total cost of care for Alzheimer’s disease and dementia is expected to be $20 trillion over the next 40 years.

Preventing or more efficiently managing diseases like Alzheimer’s, cancer, heart disease and diabetes could be a huge cost saver in the long run. The hope is that precision medicine offers some answers.

But collecting, coordinating and mining data from a million volunteers will present huge technological challenges. Data are likely to come from various public repositories and perhaps even for-profit databases. In order to be as useful as possible, those datasets need to include genomes, medical histories and biological samples. They’ll also need to harness ongoing data streams from wearables and other environmental inputs. There are many smart groups working to integrate all of that information, but the difficulty of combining what is now disparate data from varied sources is staggering.

Once the data are collected, how will we use it? Clinical programs applying personalized medicine are already in place across the country. At Vanderbilt we have PREDICT (Pharmacogenomic Resource for Enhanced Decisions in Care and Treatment), which sequences patients for metabolism of clopidogrel, warfarin, tacrolimus and TPMT pathway drugs. But even these established programs haven’t fully answered whether sequencing based on risk factors results in long term cost savings and improved patient outcomes.

These are crucial questions to consider as we think about the impact of using federal dollars for scientific advancement. No one doubts that precision medicine is the future. However, use of public funds comes with huge responsibility.

Obama’s initiative and the success of precision medicine across the country will depend on the leadership of many groups, both public and private, to help steward the use of funds and steer technology development efforts.

Today there are targeted oncologic therapies, areas of micro DNA testing such as non-invasive prenatal testing and tracking tumor DNA in peripheral blood, sequencing patients for genes that affect their health risks like PCSK9 for LDL metabolism, and sequencing for genes that affect drug metabolism like CYP2C19 for clopidogrel.

All of these uses are part of the future of precision medicine, but the key thing to impact here is the clinical encounter, the patient, the person. And that requires a multidisciplinary collaborative approach with eyes to innovation and new methods. I see precision medicine as a microcosm for the medicine of the future – and from where I stand the future is exciting.


Bill Frist is a heart and lung transplant surgeon and a former U.S. senator for Tennessee.