FDA: Enrichment Strategies to Improve Efficiency of Drug Development

Conclusion  Temple noted that there are some issues with predictive enrichment.  One is that you always believe the characteristic you use to enrich predicts the good responders; it may not do this as well as you hope.  So it’s very important to characterize the test that leads you to select those patients; then see whether it’s true that patients with the characteristic always (or most of the time) respond, and that patients without the characteristic don’t respond very much.   An issue to consider in any enrichment design is how much you need to study the people who don’t have the enrichment characteristic.  This is something to be worked out over time. But thinking about the question, “Have I picked the population that is most likely to be able to show an effect?” is important.  Sometimes called individualization of therapy, this approach has shown some drugs to be dramatically effective in targeted populations.  Enrichment design studies help you reach this kind of individualization.   Oncology drugs were high on the FDA’s approval list in 2012 and will likely remain a good area for investors in 2013 as the FDA division that reviews those treatments seems more willing to clear new medicines, Ira Loss, an analyst at Washington Analysis LLC, said in a telephone interview with Bloomberg News.  The agency approved three drugs to treat a rare blood and bone marrow disease -- chronic myeloid leukemia -- including Cambridge, Massachusetts-based Ariad...
Source: Policy and Medicine - Category: Health Medicine and Bioethics Commentators Authors: Source Type: blogs