Once seen as supplemental—patient registries are now becoming central tools in shaping regulatory decisions specifically for rare disease drug development.

For many rare conditions and hard-to-study cancers, traditional clinical trials are simply not feasible—as there are just not enough patients.

Leveraging high-quality real-world data  📊 from patient registries and natural history studies is becoming increasingly useful to:

• Understand disease progression and outcomes in diverse, “real-life” populations
• Identify meaningful trial endpoints and develop external control arms
• Evaluate safety and effectiveness in rare and ultra-rare populations

Recent approvals,✅  like Dordaviprone for H3 K27M-mutant diffuse midline glioma, and breakthrough therapy for Prader-Willi Syndrome (PWS), demonstrate how registry-based evidence can directly support regulatory decisions when clinical trials are limited by small numbers. 

At Clinakos, we are proud to enable Smart Patient Registries through our Integrated Patient Data™ platform that creates rich, longitudinal Patient 360 profiles by integrating real-time data from EHRs, labs, pharmacies, genomics, and devices. Our Medically Smart AI™ dives deeper to extract vital insights from millions of unstructured clinical documents. By unifying clinical records with patient-reported outcomes and connecting with a network spanning 1,300+ disease panels, we deliver robust, high-quality, patient-centric RWD data that facilitates registry building, enables the understanding of the natural history of disease & fuels innovation in rare diseases and oncology.

Links to approval news are below🔗

Oncology approval – https://lnkd.in/gyfWeCqh 

Rare Disease approval – https://lnkd.in/ggBC7MAh

Reach out to us to learn more at [https://lnkd.in/gp3gUmXc] 

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