A custom collaboration with: Quintiles
More Than New Drugs
The tools that will help pharmaceutical companies move ahead to personalized medicine will go beyond new drugs. For example, Mehmet Toner, a biomedical engineer at Harvard Medical School, understands that advanced compounds fine-tune the cancer treatment for a specific patient, but he says, “there is no doubt that the most profound impact on decreasing cancer mortality would be derived from accurate approaches to early detection—the ‘holy grail’ in cancer biology.” Toner works on one of the leading candidates for such detection—circulating tumor cells (CTCs). First documented in 1869 by Australian physician Thomas Ashworth, CTCs are cells from solid tumors that break off into the bloodstream. The trouble with turning CTCs into accurate biomarkers arises from their scarcity, about one CTC in every billion blood cells, which makes quantification of them extremely difficult. Toner and his colleagues, however, developed a CTC chip that, as he explains, “can now isolate viable CTCs from solid tumors carried into the bloodstream.” Toner continues, “The ability to monitor cancer noninvasively using a highly specific and sensitive blood test could potentially change how doctors manage cancer care, or better yet, be a revolutionary tool for early detection.” Such a tool could also be used by pharmaceutical researchers to see if a new compound attacks cancer, potentially measured as a reduction in CTCs in the peripheral blood.
Clinical trials are another area ripe for improvement. As Peter K. Honig, head of global regulatory affairs at AstraZeneca, notes, “Randomized clinical trials have long been the gold standard for demonstrating comparative efficacy; however, the real value of a product may not necessarily be demonstrable in such studies. Medical innovation is defined by its ability to address an unmet medical need and that must be looked at more broadly.” He cites captopril as an example, which was the first angiotensin- converting enzyme (ACE) inhibitor approved as a treatment for high blood pressure. “Follow-on drugs in the same class did not necessarily lower blood pressure better than captopril,” Honig explains, “but they required less frequent dosing and had improved safety profiles. This is valued innovation in that it improves patient adherence and blood pressure control in the real world.”
Others note that clinical trials could do much more to simulate real-world scenarios for, say, chronic diseases. “Almost half of all people with a chronic condition have two or more chronic conditions,” says Gerard F. Anderson, professor at the Bloomberg School Public Health at Johns Hopkins University in Baltimore, Md. “The challenge is to evaluate the comparative effectiveness of different technologies in real-world populations, including those with multiple chronic conditions. Most clinical trials exclude people with multiple chronic conditions because it is difficult for clinicians to measure efficacy in a complex patient. One possibility is to expand the scope of clinical trials to include a more representative sample of the population. In that way, doctors would know if the technology has added value for their patients.” The best scientific approach, however, might not be the most economical. “While this is the preferred scientific option, it could add significantly to the cost of conducting clinical trials,” Anderson explains. “A less expensive option is needed.”
Anderson sees one option that’s already available and economical. “Claims data have information on a representative sample of insured individuals and can be used to see which technologies are most effective in treating different types of patients in the ‘real world’ and not in the world of clinical trials,” he says. Nevertheless, he notes that “there are some methodological issues to resolve to make sure that similar patients are being compared.”
The process of developing therapeutics has multiple expense points, which will undoubtedly change as new business models replace old ones. Here is a snapshot of the current major expenditures related to the bench-to-bedside enterprise.
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