Based on the findings of randomized clinical trials, physicians may prescribe a medication that has been shown safe and effective â€“ on average -- in large population groups.
Whether an individual actually benefits or suffers from the medication is another question. A drug that may be the acknowledged standard treatment for the many who suffer a given disease could be completely inappropriate, if not lethal, for a specific patient.
Now, a new field has emerged that is starting to revolutionize how medicine is practiced. Advances in the ability to study an individualâ€™s genetic code have enabled researchers and other health professionals to personalize treatment to that patient. Physicians will be able to select the most effective medications that are able to provide the most benefit with the fewest side effects. Moreover, such information can allow doctors to identify pathways for preventing illness long before it starts.
Health policy analysts, physicians, pharmaceutical company executives, business leaders and government officials recently met in Washington at the CMPI-Duke University Patient-Centric Leadership Forum to discuss developments in this exciting field.
A full report will be available shortly. But to whet your appetite, we will begin posting some of the comments made at this event. First off, the introductory remarks of Dr. Michael Weber, Chairman, Center for Medicine in the Public Interest, Professor of Medicine, SUNY Downstate College of Medicine Brooklyn, New York
â€œMany of us who practice medicine have been living for some time now with what some people call the â€œburdenâ€ of evidence-based medicine, which apparently is derived from the results of large clinical trials.
These trials typically show that the small group of patients who seem to get some particular benefit from one form of treatment might do somewhat better than another small group of patients who might get benefits from another form of treatment. Most patients and their needs are not well addressed by large clinical trials. Too often, these huge studies fail to give clinicians useful guidance in the management of their individual patients. But the whole idea of personalized medicine is going to change that.
In my own field of cardiovascular medicine, weâ€™re told by the National Institutes of Health (the JNC Guidelines) that we should use thiazide diuretics as universal treatment to start off hypertension management. Yet, another government agency, the Veterans Administration, has shown that these drugs are not different from placebo in terms of reducing blood pressure in Caucasians under the age of 60. Why would NIH make the global recommendation to use such a drug? Itâ€™s based on so-called evidence-based medicine, where in one large trial (ALLHAT) performed in older patients it was shown that this particular treatment was not significantly inferior to other types of treatment in terms of heart disease and strokes. Unfortunately, issues of study design, uncertainty about the diagnosis of clinical endpoints, differences in outcomes among the patient groups enrolled in the trial and controversies over the interpretation of findings have made this â€œevidenceâ€ of little value to practitioners.
It makes so much sense to talk about personalized medicine, and yet there are major groups of people who donâ€™t necessarily warm to the idea. The big pharmaceutical companies are certainly going to have re-think the blockbuster approach to medicine. Itâ€™s very convenient to think of diseases like hypertension, where there are tens of millions of potential candidates for a drug, likewise asthma or diabetes or arthritis or peptic ulcer disease.
But what if we now take these conditions and divide them into 20 or 50 or 100 different subtypes, each of which is going to require a special approach to therapy? Suddenly, Big Pharma is going to be competing with dozens or hundreds of smaller companies, each of which has identified one of these particular small subtypes and has designed a unique treatment for it. Large companies are going to have to change their way of thinking quite dramatically. So will the insurance companies have to rethink where they stand. There is no question that personalized therapies are going to be more expensive, because overall there will be much more work needed to address the multiple patient subtypes, and relatively fewer patients in each case to amortize the cost of investment.
Even practicing clinicians may resist patient-centric medicine in an environment where itâ€™s often necessary to see large numbers of patients every day because reimbursement rates for each patient are so meager. With only a few minutes to spend with a patient, whoâ€™s got time to worry about individualized medicine? â€œThiazide diuretic for you, my friend, and donâ€™t hurry back!â€ Changing our approach is not going to be easy.â€