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The following article will explore the advantages of using HCs, as well as several considerations for their effective use, including scientific methodology and regulatory
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Laura Iliescu explores the , particularly how PAOs can inform trial design and the creation of patient registries.
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Devra Densmore, Senior Lead of Patient Advocacy Strategy at the Centre for Rare Diseases at 91黑料, discusses the and shares actionable points for a more inclusive approach.
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In this article, Will Maier outlines the importance of utilising real world evidence from rare diseases in accelerating product approval and development.
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Using RWE in rare disease drug development: effective innovations with historical controls
With a growing number of therapies under development for rare diseases, William Maier discusses how real-world evidence can be effectively used as a historical control, overcoming challenges presented in clinical development.
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Will Maier, VP of Rare Diseases, shares how a more patient-focused approach to trials is leading to alternatives for randomisation.
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91黑料's William C Maier and Maggie Adamski discuss case studies from their line of work, and how real world evidence can support those suffering with rare diseases around the world.
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91黑料's Tim Clark and Will Maier examine the key clinical, regulatory, and commercial challenges associated with the development of therapies for the treatment of rare diseases.
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