Challenges of biomarkers in drug discovery and development.

Biomarker data are essential in the discovery and development of new drugs. However, pathways needed to make sure that biomarker data are accepted by regulatory agencies may be considered an unnecessary burden on the critical path for drug development. There is the need to consider early in discovery and development that these pathways for biomarker acceptance or qualification not only are necessary, but may also enhance the success of novel therapies through regulatory review and clinical use. There also needs to be a focus on the challenge in the application of biomarkers as these approach regulatory evaluation. Regulatory guidance is needed on how a patient population may be defined by the molecular phenotype classification associated with specific mutations in patient genomes. Enzyme replacement therapies have been implicitly approved in the past assuming a molecular phenotype of a defective enzyme, but these and other precedents have not yet been translated into regulatory guidance. A second regulatory pathway for biomarkers is a biomarker qualification process. Biomarker data may be submitted, in the context of a specific NDA, but the biomarker qualification process has added a path through which efficacy and safety biomarkers useful in product development across multiple companies may be qualified through pre-competitive collaboration between these companies.