How Real-World Data Can Help Expedite Approval for Diseases with Few Treatments

August 10, 2022

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For patients with currently untreatable diseases, new treatment options represent a glimmer of hope. However, the classic drug approval process is slow and can delay new therapies from reaching patients before it is too late. In a new Stat News article, Elenee Argentinis explains how real-world data (RWD) can facilitate accelerated approval, as well as potential limitations that still need to be addressed.

According to Argentinis, “The 21st Century Cures Act, signed into law in December 2016, was passed in part to accelerate getting new therapies to patients. It required the FDA to create pathways and tools to help speed drug development.Under the Drug Development Tool Qualification Programs, companies can leverage clinical outcome assessments, such as a validated patient survey, and qualify biomarkers as surrogate endpoints. Surrogate endpoints are markers of drug effectiveness such as a blood test results in place of clinical outcomes such as death, stroke, or infection. Changes in these markers can be observed faster than waiting to assess traditional clinical outcomes.”

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