Every Cure转发了
Physician-Scientist @ UPenn | National Bestselling Author, Chasing My Cure | Co-Founder @ Every Cure & CDCN
The FDA approved a drug for a new disease without requiring a clinical trial! Tacrolimus, initially used off-label for lung transplant rejection, proved so effective that once the data was analyzed, the FDA approved it for this new indication. This highlights the power of real-world evidence analysis, a strategy we embrace at Every Cure. By leveraging patient-level data., we can uncover which drugs are actually working and identify opportunities to expand their use to even more patients. This approach is not only faster and less expensive than traditional clinical trials but also a game-changer in getting lifesaving treatments to people worldwide. #raredisease #pharma #drugdevelopment
More drugs should be approved this way. When a drug has a big safety dataset, the population-specific safety differences can be handled by covariate analyses through PopPK and E-R analysis. I think RFK Jr would agree. Do-no-harm and right-to-try will hopefully flourish in the future FDA
Wow! Great news! Kind of makes you wonder why they’re pushing back on the bill I helped Craft the Promising Pathways 2.0 Act? I’m gonna have to dive deeper into connecting the dots as to why. Unfortunately, there’s a lot of political games that are played. But congratulations on the new approval.
This is a significant step forward for drug development. The FDA’s approval based on real-world evidence shows how analyzing patient-level data can uncover valuable therapeutic opportunities. It’s a faster, more cost-effective approach that has great potential to expand access to life-saving treatments. Well done to Every Cure for driving this important work!
Great example and congratulations. RWE definitely has its place in clinical development, be it for smarter study designs, identfying target sub-populations or like in this case, actually substantially accelerating access to treatments. ??
Love this. Every FDA approval that relies upon RWE is a win for the whole research community and for patients awaiting better treatments!
Insightful!! It would be great to collaborate. How can we engage?
About time and good case of right use of RWE ??
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Learn more about this insight: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-use-transplant-drug-based-real-world-evidence