Cost-effective, complete and personalized care for all: Moving Mountains Beyond Mountains

HIV, AIDS, can be eradicated. Multi-drug resistant Staphylococcus aureus, and tuberculosis, can be overcome. An astronomical obesity rates, and the diabetes, heart disease and cancer it and unhealthy lifestyles bring, can again be reduced to the levels seen in the 1970s—5% nationally. Today, 20% of children are currently obese; among African-American girls, the number is closer to 30%. Like polio, small pox, cervical cancer, or HER2 positive breast cancer, we can begin to prevail over these diseases. We can do so in the US—including the poorest areas, and globally. And, we can expect nothing less of ourselves, where, as Tracy Kidder saw while writing Mountains Beyond Mountains, “Patria Es Humanidad...the only real nation is humanity”.

In this book, protagonist Paul Farmer, and others, personified the altruism of doctoring, and daring of social entrepreneurship. Farmer and Jim Yong Kim have compelled the community to remember that disease can be defeated, even in the poorest areas of Haiti, Peru, Rwanda, Russia, and, Boston. Personalized and complete care can be delivered and new disease can be prevented, while cost-effectiveness analysis is used as a guidepost for which levers of therapy or diagnostic supply and demand need most to be pulled—not as a trap for justifying the status quo. They have compelled us to remember that in doctoring, we must first be Partners in Health (PIH), for each individual, and, community.

The “P's of medicine”-- personalization, prediction, prevention, patient participation and partnership, are all part of the current drive to reform the approach to health care, locally and globally. Over $48.8 billion was allocated in the American Recovery and Reinvestment Act (Stimulus) for health information technology. This is largely to facilitate the complete aggregation of electronic health record (EHR) data required for prediction of clinical outcomes that would enable the prescription of personalized lifestyle and therapy choices necessary to prevent illness. Further, the HIT investment will foster patient-driven self-care and doctor-patient communication through secure 'Health 2.0' technologies such as personal health records and supplemental online video virtual doctor visits. Another $10 B was allocated to the National Institutes of Health (NIH), and much is being dedicated to the development of new genomic technologies, 'theranostics,' and the analytic techniques and computational tools necessary to harness the petabytes (one quadrillion bytes) of data to be collected via EHRs. Another $1 B is being centered on the wellness and healthy lifestyles infrastructure for prevention—an area which has traditionally only accounted for 5% of health spending--in the US--and is reflected in our lower life expectancies, especially in areas where the poor live, like Washington, DC.

These approaches, while being augmented by new diagnostics, therapeutics and information technologies, have already been modeled in efforts by doctors, such as Farmer and Kim. The treatment of infectious diseases—such as tuberculosis—has already been personalized through the process of culturing an individual patient's pathogens and assessing their resistance to antimicrobials. But, this had not been readily available to poorer nations. Those that developed multi-drug resistance (MDR) to tuberculosis (TB), were essentially left to die—until Farmer and Kim dared to apply an 'expensive' diagnostic and therapeutic cocktail to save the lives of a handful of daughters, mothers, and brothers, outside Lima, Peru. While participating in World Bank negotiations for loans to Russian prisons to stem TB, Farmer ensured money for better nutrition—a.k.a. vitamins—was also included, to help prevent disease development, and recovery, among prisoners. And, in Haiti, and other communities, he has built schools, where children and parents can learn to participate in the prevention of TB. Kidder asked, “How does one person with great talents come to exert a force on the world?” He also answered: “I think in Farmer's case the answer lies somewhere in the apparent craziness, the sheer impracticality, of half of everything he does...” When tending to an individual human being, when given the opportunity to share in and help protect their life, physicians must first be impractical innovators.

Farmer and Kim have helped save an incredible number of families, by saving patients from MDR TB. But the problem is far from solved. So they, as good doctors, good people, stepped back after their individual encounters, to take an aggregate view, a data-driven view, a public health view, a world view—so that next time they meet a person—or their colleague meets a patient—they are armed with better tools to help.

Indeed, simultaneous with the push for innovations in the P's of medicine, a new era of comparative-effectiveness research (CER) has begun in the United States. Some of the $1.1 billion allocated for CER and the $10 billion allocated to the National Institutes of Health in the Reinvestment & Recovery Act (Stimulus) was already released by the NIH in the form of 69 CER high priority challenge grant topics. Cost-effectiveness analysis--a component of comparative effectiveness research--has long been used in public health, where utilitarian arguments married with data on “Quality Adjusted Life Years (QALYs)” in an attempt to discern which diagnostics, therapies or programs are giving the most bang for their buck. But, the intention of doing such analyses is not to blindly ration care based on the status quo of a fixed pool of constrained health care dollars, fixed costs or fixed effectiveness. Rather, it is to help distributors of medicines and technologies rationally choose the lowest cost item among those that are of the highest efficacy. Further, it is a tool to help set priorities on where it is most critical to invest in innovation to improve efficacy and effectiveness—perhaps through new genomics and health IT, and lower costs—perhaps through increased supply, lower manufacturing costs, or new business models.

One of the Stimulus-related priority NIH Challenge grants called on the research community: “Develop diagnostics and drugs for multiple or extensively drug-resistant tuberculosis.” New personalized diagnostics could help identify MDR TB patients earlier, and more quickly. Some, which utilize genomics and environmental data, might even predict response to therapy and prevent MDR altogether. And new drugs are a necessity, given the dangers of entirely resistant TB, and may help to increase cure rates and further lower costs.

Indeed, Jim Yong Kim and collaborators were able to stimulate new business for generic drug manufacturers that brought down the treatment of MDR TB by nearly 95%. They did not accept the notion that 'limited' economic resources define which human beings are worthy of an investment in their life. Rather, he, Farmer and others have treated people, who have TB and MDR TB, and simultaneously raced to improve effectiveness, lower cost, and shine a light on what 'limited' economic resources actually means. According to Kidder, in considering the costs to overcome TB globally, Farmer estimated $5 billion would be needed; George Soros responded, “Is that all? You only need that?” He, Bill and Melinda Gates, Warren Buffet, Bill Clinton, One.org, Jeffrey Sachs and the Global Fund, the World Bank, WHO and others have responded. And now, so have the Obama Administration and the United States Congress-- more than $140 billion in stimulus funds dedicated to health care, more than $90 billion for education, a $634 billion health care 'initial investment' toward reformed health care for all, and definitive health reform legislation in process. The bulk of funds are focused locally first, but the US has also made a renewed commitment to ending global poverty, though one that can never be fast enough when time is measured by the life of the individual patient.

Some like to draw a divide between medical ethics, focused on the individual patient, and utilitarian public health ethics focusing on delivering the best care to populations with limited resources. This divide is artificially induced by our culture's own perceptions. It is in fact, the public health and clinical research fields which are providing the tools enabling the 'new P's'--which support the call of medicine to personalize care, prevent illness and teach the individual to aid in protecting their own health. Ethical public health uses epidemiology and comparative effectiveness research to design a roadmap to allow the good work of medicine to occur; ethical public health does not design blueprints for fences.

The ethic of doctoring, of helping to lift up the destitute, is what will build the roads from that roadmap. Another movement is afoot- a rebirth of service, and social entrepreneurship, as exemplified by Farmer and the now thousands that change the world through Partners in Health. The Stimulus package will help to fund some of the infrastructure, among the poorest areas of the US.

Just over 500 years ago, the world was flat, and America the continent, 'did not exist.' Kidder quoted Farmer as saying, “I have fought for my whole life a long defeat.... Now I actually think sometimes we may win...[but,] I don't care if lose, I'm gonna try to do the right thing.” And in doing so, there will be many victories, like the discovery of America, and many new diseases (like those brought to Native Americans), we cannot immediately defeat, but will, with time. What Farmer, good doctors and good social entrepreneurs share in common is the urgency of now, and the identification with every human being. The confluence of comparative effectiveness research, new approaches for personalized and preventive health, fueled by social entrepreneurship, service and an ethic to heal and keep healthy every person, globally, is unprecedented, and will help us win. As we follow the right ethic of concern for each individual, as we give the poor the tools to lift themselves up, we will continue the long defeat, and accept Farmer's—our own—frustration at the state of illness and poverty in the world. But, we will be rewarded with his freedom from the “self-consuming varieties of psychic pain.” We will win battles every day with the lack of ambivalence of purpose that makes Farmer's life, and so many others, so beautiful.