Pharmacogenetics to Spur New Drug Development?

Drugs are expensive. Making them is even more so. A recent article by Avik Roy points out that, over the last three decades, the average cost of drug research has ballooned from the equivalent of roughly $100 million per drug in 1975 to more than $5 billion today. And Roy blames clinical trials.

Phase III trials in particular increased in average length, procedures per trial, and required manpower while also suffering from fewer volunteers and more dropouts. As Roy puts it: “Overall, Phase III trials now represent about 40 percent of pharmaceutical companies’ R&D expenditures. But this… understates the gravity of the burden… When we confined our analysis to those drugs that actually get approved, we found that Phase III clinical trials typically represent 90 percent or more of the cost of developing an individual drug all the way from laboratory to pharmacy.” But Phase III trials, where the drug is tested on a large scale against both placebos and existing therapies are critical to FDA approval. What can be done?

This is, perhaps, where pharmacogenetics can step in. By genotyping the potential volunteers the drug companies can not only weed out individuals that may have adverse drug reactions, but also optimize the trial itself. Subpopulations can be analyzed for specific reactions and drug dosing information could be more easily fine-tuned. This could not just help explain unexpected or contrary trial results, but also lead to safer and more precise use in the general population.

Furthermore, by using pharmacogenetics, drug companies may be able to develop drugs targeted at specific genotypes. Trastuzumab (Herceptin) was developed this way, only targeting breast cancers that overexpressed the HER2 gene. By using a smaller specific population development they avoided nonreceptive test subjects and reduced overall study cost. Hopefully as the availability of pharmacogenetics increases this sleeker targeted approach will become a major new avenue of drug development. We may soon see drugs costs drop hand-in-hand with the cost of genotype testing.