Deadly hyperinflammatory disease finds curative treatment from a repurposed drug! DADA2, a rare hyperinflammatory condition, tragically forces children to endure strokes at a young age, often leading to premature death in their teenage years. Without a single clinical trial, the application of TNF inhibitors is widely used and has demonstrated profound efficacy thanks to the work of my colleague Chip Chambers, MD and the @dada2foundation Foundation. Children who once faced a bleak future are now living full, stroke-free lives. This scenario raises important discussions around the necessity of clinical trials to maximize a drug’s full potential. It highlights the potential for direct observation and real-world application to guide treatment practices for rare and severe conditions. How can we leverage similar observations in other rare diseases to accelerate access to effective treatments? #MedTwitter
@DavidFajgenbaum There needs to be a way to accelerate RCTs for FDA approved drugs. The administrative and financial burden of this process is causing these drugs to sit on the shelf.