MediciGlobal

In early 2012, the Food and Drug Administration (FDA) will advise sponsors on how to enroll richer and more balanced patient populations in clinical trials in a new draft guidance.
FDA is planning draft guidance on approaches to improve patient recruitment in clinical trials. The guidance will include “targeted and genomic marker-driven trials to enrich the study populations.” When the agency releases these in early 2012, the industry will have a period for comment.

According to Robert O’Neill of FDA’s Office of Biostatistics at a Drug Information Association conference on targeted therapeutics, one important area the draft will address will be “how much do you need on the marker-negative group to be able to assure yourself that there is no treatment effect in that group.” FDA has been reviewing different statistical models and a major concern with sophisticated statistical methods is avoiding false positive results, known as “Type I error.” Despite these concerns, FDA views that the new statistical methods are very important for clinical trials because they can improve recruitment and speed results.

Some of the tools are starting to get a better handle on who should be recruited into a clinical trial. To date the majority of focus has been about oncology and tumor typing.