Since then, companies have advanced the science, and have even brought a few tailor-made therpies to market. NitroMed, for example, figured out that BiDil (isosorbide/hydralazine) works best in African Americans. But while pharma has pushed this and other personalized medicines through clinical development, much of the data that proves these drugs' utility in real-world settings can't be obtained through the traditional route of experimental medicine. Instead, payers are demanding clinical and cost-effectiveness information for these very expensive medicines, as well as long-term safety and efficacy information. So great is the need for coverage of these pricey, but often life-saving, therapies, that it is changing the traditional research paradigm.
The very nature of personalized medicine challenges the way scientists normally handle drug development. There are some unique issues that characterize research for tailor-made therapies.
The right patients are hard to find Even large disease categories look like a collection of rare diseases when patients are grouped based on common polymorphisms and biomarkers. For example, although lung cancer is the leading cancer killer of both US men and women, Iressa (gefitinib) and Tarceva (erlotinib) are most successful when given to a subset of just 10 percent of lung cancer patients with a particular set of mutations in their epidermal growth-factor receptors.
Although advances in technology for examining DNA, proteins, and other biomarkers allow companies to understand and describe these patient subgroups, genetic testing is not yet a widespread or standard part of most physicians' armatorium. As such, many patients who can be helped by personalized medicines are simply not recognized.
Informed consent is more challenging The Office of Human Research Protections (OHRP) and the Office of Civil Rights (OCR) offer confusing and, at times, conflicting information to guide research practices with respect to informed consent and privacy. However, researchers of genomic medicines have the added burden of requirements outlined by the Centers for Disease Control, which don't necessarily conform to those required by OHRP and OCR.