Clinical Trial Designs for Rare and Ultra-Rare Disease

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Summary

In Med Ad News, Mariia Saldova shares innovative ways to overcome the challenges of planning and executing clinical trials in small patient populations

When designing clinical trials for rare and ultra-rare diseases, manufacturers must uncover ways to overcome the geographical and ethical challenges that do not plague chronic disease studies. The article by Mariia Saldova, “Innovating Clinical Trial Designs in Rare and Ultra-Rare Disease,” explores the importance of decentralized clinical trials in a patient population that is difficult to find and explains the benefits to patients seeking access to new therapies. The author describes how one gene therapy company was able to recruit and retain patients using innovative recruitment methods. She also discusses the importance of collaborating with advocacy groups and minority communities to identify and reach new patients.

To read the complete article, visit Med Ad News’ blog, PharmaLive.com.

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