Is Personalized Medicine on the Way?
From the earliest days of "medicine" to the days of chemists, druggists, and apothecaries, we have used "drugs" — natural extracts, tinctures, mixtures, lotions, and ointments — to treat our patients' diseases and discomforts. As physicians, our role remained somewhat consistent as the source of our treatment materials moved from the local forest to the chemist and then to the pharmaceutical industry. We continued to make treatment decisions based upon observed symptoms as we improved our diagnostic methods and tools. Our strategic approach to diagnosing and treating patients isn't going to change — our treatment tools, i.e., our drugs, however, are.
Soon there will be a dramatic change in the therapeutic tools available to us as a result of advances in science with respect to new "genomic level" diagnostic tools and therapeutic modalities. The driver of this change is our increasing ability to identify and describe an individual patient's problem not only from symptoms but at a molecular level, and to understand and appreciate the great magnitude of differences in the causative factors — and appropriately different interventions — for different patients who present with essentially the same set of symptoms.
The pharmaceutical industry is beginning to think of the development of drugs in terms of individual groups of patients, rather than maintaining its "blockbuster drug" mentality. The age of molecular-targeted cancer therapeutics has already arrived. Recent regulatory communications from the FDA confirm that oncology drug developers will now need to develop and deliver, for all new oncology drugs, a "biomarker" for use with the drug to select and subset oncology patients before prescribing the new drug. We are narrowing the target population of patients to that group already identified to be relevant for that drug.
What we have observed in recent years — with emerging biotechnologies and genomics, proteomics, metabalomics, transcriptomics, and other "-omics" — is the gathering of more and more data confirming that one drug does not fit all patients with a given diagnosis or symptom set. We are now seeing the leading edge of truly personalized medicine, not individually tailored therapies, but rather improved strategies to identify smaller groups of patients with common gross symptoms to permit us to get the right drug to the right patient at the right dose level.
Pharmaceutical companies are already beginning to change and will conduct more specific clinical trials with smaller numbers of more adroitly identified patients. The predictable end result is increased efficacy. Soon we will identify only patients within the broader "symptom" category who share the same underlying molecular structure — maybe a similar molecular abnormality — and treat only those patients with the relevant therapy.
We are on the threshold of a new era in medicine, an era in which we will have both the intent and the tools to treat patients on a "personalized medicine" basis. The pharmaceutical industry's focus is moving away from the "blockbuster drug" mentality and recognizing the value of "-omics" in identifying, selecting, and treating patients as much smaller groups rather than merely identifying them by broad symptomatic categories. Tomorrow — and it is coming soon — we will match molecularly targeted drugs with molecularly defined patients.