Challenges and Opportunities in Developing Neurologic Therapies - Page 4

New Approaches to Neurologic Drug Development Ongoing developments in basic science and in efficient capture and sharing of data are creating opportunities for advancing the treatment landscape. Targeted therapeutics. Increased use of new technologies such as genetics, genomics, proteomics, and imaging will play an important role in helping researchers to identify biomarkers to improve our understanding of the disease process (Gomez-Mancilla, 2005). This will be the prerequisite to tailor drugs to the particularities of the disease, serving both purposes, becoming more efficacious as well as limiting unwanted side effects by unspecific non-targeted pharmacological effects. Having a greater understanding of genetic and molecular pathologies allows for development of more targeted therapies. For example, several drugs in clinical development target individual protein kinases, which are enzymes involved in intracellular and extracellular signaling (Chico. 2009). Onset of disease. Parties in industry and academia are developing biomarkers for detecting the presence of mild disease (Pritchard, 2008). Detecting disease before symptoms appear could lead to development of therapies that block progression to severe disease. Efficacy evaluations. In measuring subjective outcomes, trained independent evaluators may be used in clinical trials to overcome concerns about inter-rater and intra-rater reliability in measuring subjective outcomes. Biomarkers are also a valuable tool in evaluating the effectiveness of a novel therapy. As more are developed, the use of subjective measures in evaluating progression of neurologic disease should decline. However, a hurdle to be overcome is the predictive value of a given biomarker with regard to its therapeutic effect to be established later in the drug development process. Increasing emphasis is being placed on finding approaches for early assessment of a drug’s potential for long-term success. Evaluations can be done at the stage of screening potential compounds that can save time in refining the composition of candidate drugs (Chico, 2009). Use of technology. Digital collection and dissemination of data allows for rapid communication of efficacy and safety data, which can be key to ensuring the success of early phase clinical studies. Technology adoption can speed and facilitate start-up of study sites, patient accrual, clinical monitoring, site management, inventory monitoring, and study close-out.