Refining Processes for the Co-Development of Genome-Based Therapeutics and Companion Diagnostic Test. Workshop Summary.

摘要

The initial fruits of the human genome project are beginning to be seen, with novel technologies based on genomic information being implemented in clinical practice. At the same time, however, the cost of developing new technologies has risen at a significant rate. With new pharmaceuticals estimated to cost more than $1 billion on average to develop and to take 10 years to bring to market (DiMasi et al., 2003), many drug developers have examined new strategies for creating efficiencies in their development processes, including the adoption of genomics-based approaches. Genomic data can identify new drug targets for both common and rare diseases, can predict which patients are likely to respond to a specific treatment, and has the potential to significantly reduce the cost of clinical trials by reducing the number of patients that must be enrolled in order to demonstrate safety and efficacy. Somatic genome information can be used to guide therapy for cancer treatment and germline information can be used to assess risk of inherited diseases and to avoid adverse reactions to drugs. Recently, the expectation of such benefits has led to the development and approval of a number of targeted therapeutics for diseases such as nonsmall- cell lung cancer, metastatic melanoma, and cystic fibrosis (Chiang and Million, 2011; Davis et al., 2012).