The Future of CNS Clinical Development

Central Nervous System Clinical Trials, Central Nervous System

A decade ago, CNS-focused therapeutics—traditionally a well-funded area of research—were facing a sharp decline in funding and their growth outlook was bleak. Today we face a different reality, one where third parties and large pharmaceutical companies like Acadia, Biogen, Novartis, and Denali are investing in neuroscience development programs. See why there is a resurgence and where we see the greatest potential in neuroscience therapeutics.

Development of investigational new drugs is an expensive investment requiring funds which historically came from the profits of commercialized products in a company’s portfolio. Companies had to be selective in the compounds they chose to advance, and CNS programs typically had more challenges than other therapeutic areas. The complexities of neuroscience endpoint selection, the often-limited understanding of disease pathophysiology, and the poor predictive validity of preclinical models meant development in neurological indications was a risky investment. Over the past 10 years, there has been a sharp increase in funding from government agencies and public-private partnerships, much of which has gone to neuroscience programs. This new funding meant companies no longer had to rely on the profits of their approved products to advance new programs.

Why the uptick? The renewed interest comes partially from the advancement in scientific understanding of many of these diseases. Medical breakthroughs have opened the door for therapeutics to be developed with biomarkers and genetic targets as surrogate endpoints which better predict clinical improvement. New therapeutic modalities have also been developed, allowing for the exploration of new treatments with better outcomes. With these advancements, researchers are better able to reduce risk and minimize chance of program failures, making neuroscience clinical development a more attractive investment.

The shift in funding can also be attributed to regulatory agency incentives. The US Food and Drug Administration (FDA) released four regulatory approaches in their Accelerated Approval Program which fast-tracked development and approval, and the European Medicines Agency has created similar program incentives. While these programs affect many therapeutic areas, the impact on neuroscience programs has been notable. The FDA even published five guidance documents for neurological conditions in 2018 and 2019, and the EMA has issued or revised guidance for seven neurological indications.

This resurgent focus on clinical development in CNS comes at a time when psychiatric disorders are socially better understood, accepted, and supported. Neurological disorders are the second leading cause of death globally, and close to a billion people have mental and addictive disorders. With the widespread effect of the diseases falling into this therapeutic area, the investment in neuroscience programs will inevitably lead to better health outcomes and treatment options for millions. We expect these factors which have led to the refocus on CNS indications have created the circumstances needed for a sustained investment in the development of treatments for these diseases.

The funding and development for neuroscience programs in recent years has primarily fallen into three areas of research. Find out what these three areas are and what programs have seen great success by reading our article in the Journal for Clinical Studies: Anticipating a Renaissance in Neuroscience Drug Development.

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