People living with chronic conditions benefit from many medical advances developed over the past 20 years. Many diseases that were once fatal diagnoses are now managed utilizing various drug therapies. However, far too often, treatments may work well for some people, while providing marginal benefits or causing significant harm to others.

One example is antidepressants. According to a study at the Tel Aviv University (TAU) Sackler School of Medicine, the most popular antidepressants only work for approximately 60% of the patients with depression. For the other 40% of the patients, it takes a trial-and-error approach to find the right treatment strategy. As David Gurwitz, PhD, at TAU stated, patients need a “best-fit, personalized regimen.” For people with autoimmune diseases, it is even more difficult to create the right treatment plan.

Today, billions of dollars are spent annually on research, development, and clinical trials to create new healthcare products. How can we improve on this process so that patients can reap the full return on investment?

Molecular diagnostics can be an important element in the development of personalized medicine by determining the course of a disease and evaluating the patient’s response to a given therapy based on the individual’s genetic makeup. Such processes are already being tested as a prognostic indicator of congestive heart failure to better identify patients in need of more intensive forms of therapy. Molecular diagnostics have also been applied in the detection and treatment of other disease categories.

The National Health Council (NHC) has engaged in conversations with various patient advocacy groups and other stakeholders to examine new methods to more accurately identify the benefits of therapies at the point of care, taking into consideration a patient’s individual genetic makeup.

Creating and utilizing molecular diagnostic tests could allow us to better target treatments to those who will benefit and reduce costs by more accurately determining safety and efficacy of related medicines. Janet Woodcock, MD, director of the Center for Drug Evaluation and Research at the US Food and Drug Administration, has pointed out that targeting therapies to the likely-to-benefit patient groups—or to exclude those individuals with a low probability of a positive response—is a powerful method of increasing value in healthcare delivery. Such advances in science also hold promise for better health outcomes for patients with chronic diseases.

PricewaterhouseCooper states in a 2009 report on diagnostics that there is “a growing imperative, both clinical and budgetary, to accompany therapies with diagnostic tools of increasing sensitivity and specificity to better enable the identification of those patients in the relevant disease subtype and most likely to benefit from the therapy.” The company predicts that the marketing model foreseen for most specialist therapies in 2020 will include a companion diagnostic as a key component.

However, should we wait 10 years while the needs of patients are left unmet? We need a system that ensures that advances in science are more quickly married to the needs of individual patients. The NHC is proposing a public–private approach that would remove the barriers that impede progress in the development and use of molecular diagnostics.

Currently, there is ambiguity in the evidentiary standards for molecular diagnostic tests and the regulatory approval pathway. This lack of certainty discourages investment in the development of the new therapies utilizing such tests. What we need is better communication between government and industry and a goal-oriented approach to revising the regulatory process.

What we also need to do is a better job of explaining to the public that molecular diagnostic tests could ensure that individuals with different but related conditions receive targeted drug therapies that work for their specific disease subtype.

How we go about clarifying and creating a process to support greater use of molecular diagnostic testing is currently under debate. As Dr Woodcock has pointed out, “Given the huge stakes involved in reimbursement decisions and the variety of methodological approaches that could be used to compare interventions, an epistemological ‘food fight’ over evidentiary standards is likely in this emerging field.”

If we can get the appropriate stakeholders from the public and private sectors working together, we can come up with workable menu of action steps that will greatly benefit the patient community.

OUR PARENTS told us life isn’t fair. People with chronic diseases and disabilities know this more intimately than most people.

A woman with rheumatoid arthritis had a successful career in public relations and advertising and now relies on Social Security disability payments. A husband copes as best he can to care for his twin sons and a wife diagnosed with Alzheimer’s before she turned 50. A young man about to enter college worries about his course load and his treatment schedule for Crohn’s disease, a condition for which there is no cure.

The more than 133 million people with chronic conditions in America can teach us all lessons about life’s inequities. They can also teach us about being pragmatic. For them, health-care reform is not just about expanding access to cures. Health-care reform is about changing our overall delivery system so that it meets a patient’s broad and complex agenda to live longer and feel better.

The Agency for Healthcare Research and Quality says that 20 percent of the population incurs 80 percent of total health-care expenses. We also know that this segment is made up of people with chronic conditions. Developing a delivery system that addresses the needs of people with high-cost, chronic diseases and those who are at greatest risk for hospitalization will have a major impact on managing the health-care budget.

Better outcomes while curbing costs could be achieved through better care coordination for patients with chronic conditions. Care coordination focuses on the individual patient’s unique situation and ensures that medical knowledge and health care information are appropriately used to let the patient make educated decisions regarding his or her treatment.

While not necessary for everyone, care-coordination delivery models can apply a laser approach at improving the delivery of health care to people with complex chronic conditions — people whose health-care needs dramatically impact our total health costs. Help them, and you help the entire health-care delivery system.

An effective care-coordination model incorporates three equally important elements: health and medical research; recognition of the patient’s unique personal circumstances (including the individual’s genetic, ethnic, religious and socioeconomic status at the point of care); and appropriate incentives for both the provider and the patient.

A three-legged stool, all three elements are needed to support the delivery model. Ignore one and the stool collapses.

This model reimburses a health team to assess a patient’s unique health history and lifestyle and to coordinate the care. The team would work with the patient to develop an individualized care plan (ICP) that is based on quality research and the person’s life situation. In exchange for an ICP, the patient would have explicit responsibilities: be a willing and engaged participant in the treatment plan and reap the benefits of lower out-of-pocket costs, or go back to the current uncoordinated system and pay his or her out-of-pocket costs.

Too often the health-care reform debate is unbalanced — focusing on program cost and ignoring individual patient expense, or touting the advantages of research but missing the human element and “real world” application of medical advances. New delivery models that are founded on principles of evidence-based medicine, respectful of the individual patient’s unique situation at the point of care, and committed to reducing out-of-pocket expenses while reimbursing for care coordination are already proving to be the most cost-effective health-reform strategies available.

The National Health Council has undertaken a study of about a dozen health-plan initiatives in different states and different community sizes. Preliminary results reported by the programs have been encouraging. Take, for example, the Geisinger Health System, in Pennsylvania. It implemented new programs in 2005 to improve patient care, including the use of electronic health records and a personal health navigator to better manage chronic diseases. In 2007, it reported that among the participants there was a 12 percent decrease in acute-care hospital admissions and an 11.7 percent decrease in hospital readmissions. Care sites that offered the program reported an 8 percent lower differential in medical costs, compared to care sites that did not participate in the program.

Patient-focused delivery models aren’t a panacea, and patients with chronic diseases and disabilities understand that. That said, creating new plan initiatives is crucial for achieving the health-care reform patients seek.

If we can create a system that provides for and appropriately addresses the unique needs of the 20 percent of the population who are driving the health-care dollars spent in America, we’re 80 percent of the way toward a health-care solution for all.

This piece by Myrl Weinberg, president, National Health Council, ran in the Providence Journal on July 27, 2009.