Biomarkers Used as Outcomes: FDA Wants to Hear More on Examples

Regulatory News

The US Food and Drug Administration (FDA) is seeking comment from interested stakeholders to determine the utility of a list of examples of biomarkers used as outcomes in development of FDA-approved new molecular entities and new biological therapeutics.

The comments, which should be submitted by 18 November, may assist FDA in developing databases on biomarkers for drug development in the future, the agency said.

Background

Biomarkers are frequently used in support of drug development, particularly as an outcome in a clinical trial.

FDA offers a look at biomarkers used in 12 therapeutic areas – including cardiology, oncology and infectious disease – that were accepted and used as outcomes in clinical trials as the basis for drug and biologic approvals from October 2007 to December 2015.  

• *T1: 1st twitch height
• *T4/T1: Ratio of the 4th twitch height for train of four (four stimuli given at a frequency of 2 hertz eliciting four twitches) divided by the first twitch height
• *RNA: Ribonucleic acid
• *Hemoglobin A1C: Glycosylated hemoglobin
• *DEXA: Dual-energy X-ray absorptiometry (or bone densitometry)
• *Bcr-Abl: A fusion oncogene between Break point cluster (Bcr) gene at chromosome 22 and Abelson (Abl) tyrosine kinase gene at chromosome 9
• *RECIST: Response Evaluation Criteria in Solid Tumors
• *EBMT: European Society for Blood and Marrow Transplantation response criteria

However, FDA notes that the appropriateness of the use of any particular biomarker as an outcome in one drug program should not be assumed to be appropriate for use in a different context or for a different drug development program. Therefore, drug sponsors are encouraged to consult with the relevant FDA review division as early as possible in drug development regarding the use of a particular biomarker in their planned clinical trials.

Call for Comments

FDA is now soliciting suggestions and comments to determine the utility of the biomarker outcomes list and to identify any areas where more information can be disseminated on biomarkers that have been used to support the approval of drugs or biologics. Specifically, FDA wants to hear about:

• Areas of improvement for communicating and disseminating information about biomarkers and their utility as drug development tools
• The best approach for updating the biomarkers outcomes list, including any modifications moving forward

Federal Register