DefinedHealth

Jeffrey M. Bockman, PhD

“’ …prospective use of any patient characteristic – demographic, pathophysiological, historical, or genetic.’”
–Dr. Robert Temple, Director of the Office of Medical Policy, FDA CDER

Oncology is often viewed as the oddball category, set apart from other therapeutic areas (TAs). When it comes to the concepts of personalized medicine and pharmacogenomics, there is no question that Oncology is at the forefront of what some envision as a sea change in the practice of medicine. And while we may all debate the timeline for this change, not many would disagree that, in theory, a time will come when medical decisions are guided by a more refined battery of diagnostic, prognostic and theranostic tests. Whether these are pharmacogenetic, clinical or other types of assays, these tests will guide, if not dictate, which drug or combination of drugs to use, at which dosages, and for how long for that particular form of disease within the background of that patient’s particular metabolism. It would therefore behoove those wishing to strategize for the future of any TA to take note of what is happening in Oncology to see what lessons might be learned.

That being said, why has segmentation and targeting of patient subsets taken root in Oncology first? (And while Oncology is leading the way, parsing of patient popoulations is by no means universally accepted or adopted yet as a clinical development and commercial strategy.) Are there other diseases where conditions are ripe for such personalized approaches? What is it that has made targeting patient subsets attractive in Oncology? Is it the near-term mortality and the low response rates, or the high rate of clinical trial failures? If so, does this make CNS a likely next TA for such segmentation? Or is it the high price of therapies, whereby treating a patient who is not responding well, or at all, is fiscally improvident and clinically imprudent (especially if
there are other products to which the same patient might respond)? Could such thinking apply to diseases that are chronic in nature and treated over decades, often for long term risk mitigation and not even for immediate symptom control, where one can adjust drugs and dosages along the way? And is it only about efficacy? Given the occurrence of known, let alone unanticipated side effects, what about guidance for safety?

How many Bio-Pharma executives, unless it is a drug within their TA space or own company, know that some 10% of drug labels right now contain pharmacogenomic
information (for example, where the label makes note of extensive metabolizers (EMs) and poor metabolizers (PMs), such as Abilify)? This Insight Briefing will compare and contrast oncology with other TAs to elucidate the lessons that can be leveraged and those that can be learned as personalized medicine moves beyond cancer to such settings as heart disease, metabolic conditions, psychiatric and neurologic disorders.