🚀 Noteworthy update for the world of rare disease innovation! The FDA’s head of CBER, Vinay Prasad, stated at the recent National Organization for Rare Disorders conference that the agency is committed to making rare disease drugs available at the very first sign of promise. Here’s what you need to know:

🔹 Faster Access: The FDA is prioritizing speed, promising to act quickly when new treatments show early signs of benefit for rare disease patients.
🔹 Surrogate Endpoints & Conditional Approvals: Instead of waiting for lengthy randomized trials, the agency will use surrogate endpoints and real-world evidence to grant conditional approvals—giving hope to patients who can’t afford to wait.
🔹 Real-World Data (RWD) Front and Center: RWD will be leveraged “in every way possible” to track patient outcomes and ensure ongoing safety and efficacy.
🔹 Patient-First Approach: The FDA’s new direction puts patients at the heart of decision-making, offering earlier access and more therapeutic options.

At Clinakos, we’re passionate about accelerating progress for rare disease. We work with rare disease patients, work in >1500 rare diseases, and curate Integrated Patient Data™, which helps researchers develop and make treatments available.

As the FDA opens new doors for rare disease therapies, Clinakos stands ready to deliver the robust RWD needed to turn hope into reality. Together, we’re making every patient story count. ✨

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👇 Read the full news update here: https://lnkd.in/gsJt8PTv