Central Nervous System (CNS) Active Drugs: Complex Considerations

As you work towards a successful New Drug Application (NDA) submission, there are many considerations that must be taken into account, specifically for CNS-active drugs. Molecules or compounds that are centrally active (the parent drug or metabolite[s]), may require additional evaluations to characterize the drug effects and unique safety characteristics. Not all centrally acting drugs require additional assessments; however, strategic direction early in a drug development program can help determine if such studies should be planned, or may be waived.

The Landscape

CNS-active drugs have certain unique attributes that necessitate additional specialized study:

• Cognitive impairing/enhancing effects
• Reinforcing effects (abuse potential)
• Physical dependency and tolerance
• Additive effects (when combined with drugs and alcohol)

Because CNS-active drugs can have unintended effects in the critical neural system that influences so many aspects of human health, the required preclinical and clinical studies are typically more detailed and rigorous than for other types of drugs. In addition, the results of such studies will have a material impact on your development program going forward, both in terms of timelines and budget, so you will want the answers for yourself as much as for the regulatory bodies.