By Angela Woodall, Healthcare and Environmental Reporter.
Despite decades of focused research, treatments for neurology-related disorders are limited. However, recent work promises to widen the window on options. Several avenues of investigation have raised hopes that research will finally begin to cross the chasm between bench and bedside by developing treatments for the millions of people afflicted by debilitating disorders like Parkinson’s, Huntington’s, ALS and stroke. In the offing in 2017 is a multiplicity of breakthroughs, including the first Food and Drug Administration approved drug to treat spinal muscular atrophy, a rare hereditary disease that impairs movement and is the most common genetic cause of infant death. At least seven other studies will be producing data in the next 18 months and two pharma powerhouses, Biogen and Celgene, are competing over multiple sclerosis treatments. Meanwhile, researchers are questioning the very theories behind how neurological disorders originate and progress.
Innovations are also happening in the technology used for research. The same is true for neurology clinical trials, which are moving toward analyzing neurological disorders according to specific symptoms and individualized molecular features rather than to take a “one size fits all” approach toward all patients with a phenotypically similar disease. This emphasis is driven in part by programs like the Precision Medicine Initiative. Likewise, an emphasis on interdisciplinary collaborations will be driven by implementation of the 21st Century Cures Act, which will begin to be felt in 2017. The $6.3 billion bill encourages research into regenerative medicine therapies for otherwise incurable conditions, including brain disorders. The goal is to get them to market more quickly, without sacrificing patient safety.
How these projections play out in neurology clinical trials over the course of 2017 remain to be seen. Much depends on the Trump administration, the 115th Congress and new leadership in key agencies. One thing is certain, though: The coming months promise to bring an impetus for change, collaboration and innovation.
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This document covers the following topics:
- Pipeline: new therapies are now being marketed or are in late-stage trials
- Technology: The gene-editing technique CRISPR is breaking through to neurology trials
- Tele-Trials: The FDA is exploring novel approaches to clinical trial monitoring
- Public-Private: The government will increasingly invite industry collaborations
- Transparency: Data is driving pressure to show rigor and reproducibility in studies
- Regulatory: The industry is watching new leadership in key agencies, user fees and stem cell regulations
- Next Steps: Innovative trial design and a strong push for interdisciplinary collaboration joins traditional trial designs
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