Only yesterday, we were reading about the death of CNS drug development. The reasons were simple: first, several Big Pharma companies were abandoning or cutting their CNS programs; and second, there seemed to be a consensus that odds were against translating CNS-related innovations in basic science into successful clinical programs. That was in December 2011. A string of failed Alzheimer’s trials did nothing to brighten the outlook for CNS development.
Fast forward to August 2013. A story by John Carroll at Fierce Biotech highlights an interesting nugget in a recent report from Silicon Valley Bank. In 2012, the disease focus that led the way in Series A funding rounds for biotechs was – you guessed it – CNS. Enterprising CNS researchers deserve our congratulations for securing funding to pursue opportunities while Big Pharma looked the other way. Perhaps Churchill was onto something when he said, “A pessimist sees the difficulty in every opportunity. An opportunist sees the opportunity in every difficulty.”
As CNS startups progress, they are sure to hear discouraging stories about the operational challenges of CNS trials. These include everything from inconsistency in administration of subjective assessments and interpretation of VAS data to meeting enrollment goals while obtaining appropriate informed consent from representatives of patients with mental impairments. Newly funded CNS companies should rest assured that proactive trial management based on timely information and performance metrics can address such issues and produce high-quality data. With the right development partner, this is just one more case of finding opportunity where others see difficulties.
There are techniques that can help CNS sponsors optimize allocation of clinical development funds and accelerate programs. For example, Health Decisions:
- Enabled early termination of a recent phase II pain-management study for futility and refinement of development strategy based on a responsive subpopulation.
- Reduced timelines by almost one-third and saved $32 M in development costs for the sponsor of a phase III Alzheimer’s study.
We reduced timelines and costs in the Alzheimer’s study through close management of all aspects based on timely information. This included:
- Tracking and managing existing sites to meet aggressive enrollment milestones rather than driving up costs by adding more sites
- Quickly identifying and correcting a handful of sites that were recruiting subjects that failed to meet all inclusion criteria
- Reducing monitoring costs through a combination of remote monitoring, needs-based allocation of site visits and intense focus on sites with performance issues
- Rapidly detecting a spike in ADAS-cog results reported by one site and identifying and correcting the issue – assignment of an inadequately trained member of the site’s staff to administer the subjective assessment following personnel turnover.
We can’t improve the efficacy of novel CNS therapeutics. We will leave that to the new wave of CNS startups. When the time comes for clinical testing, we will be happy to help these young companies make optimal use of development resources and maximize chances of demonstrating efficacy.