The need for CNS therapeutics is on the rise.  Neurological diseases are expected to increase steadily in the coming years.  Previously, we addressed how advances in neuroscience are identifying more appropriate targets and establishing clear biological readouts to support drug development. However, due to the difficult nature of developing drugs for neurological applications, the pharmaceutical industry is creating new models to change their strategy.1   Here we summarize their approaches:

Shared risk [pullquote]The average cost for development is $1 – 5 billion dollars, a striking 2.5 fold more than development costs in other therapeutic areas.[/pullquote]

CNS therapeutics are typically in phase II and III clinical trials for an average of 8 years.  The average cost for development is $1 – 5 billion dollars, a striking 2.5 fold more than development costs in other therapeutic areas.  Spreading the costs and potential losses/gains through strategic partnerships is necessary.  An interesting shift supporting this is the use of contract research organizations (CROs).  CROs minimize risk by allowing companies to use experts in a particular technology without developing the infrastructure required to perform the experiments.  Pharmaceutical companies are outsourcing or partnering most of their drug discovery efforts.

Predictive planning in early stages to prevent late stage failure

Drug discovery and development is expensive and can take decades.  Rethinking the process and actively trying to predict when and where a therapeutic candidate will encounter problems is a greater concern.  Improved proof-of-concept and discovery models are being implemented to ensure that the best candidate for the right target gets developed, rather than a brute force approach.  Predictive models include in silico modeling, improved cell culture models that use human cells and even organoids to predict drug response, and more advanced animal models.  Preventing costly late stage failures could improve productivity and enable research to make better progress on finding the right treatments.

Patient driven efforts

New models to develop CNS therapeutics

New models are needed to finance development of CNS therapeutics.

There is a significant public interest in moving research for neurological diseases forward, as well.  Patient driven advocacy groups have proved to be extremely effective in advancing basic and clinical research, organizing patient recruitment for clinical trials, and fundraising for their efforts.  The Michael J. Fox Foundation has pushed Parkinson’s disease research forward in dramatic ways.  Without their efforts, many candidates and companies would not have the capital to continue developing new CNS therapeutics.

Development of CNS therapeutics is a rising public health concern with the potential to revitalize the pharmaceutical industry.   Neuroscience has reached a place where the science can deliver clear targets with measurable outcomes.  This is necessary in order to overcome the challenges the industry faces.  But the pharmaceutical industry recognizes that it must use progressive new approaches to fund and advance therapeutics to treat neurological disorders.


1. Skripka-Serry, J. The great neuro-pipeline ‘brain drain’ (and why Big Pharma hasn’t given up on CNS disorders). Drug Discovery World. Fall 2013